august 2007 a thomson centerwatch publication

36
U.S. Disease foundations will invest about $75 million this year in biopharmaceutical companies to fund discovery and develop- ment of new drugs and therapies for their corresponding diseases. This year’s figure represents a 10-fold increase since 2000, and the figure should continue to rise. This new avenue of industry funding has been dubbed “ven- ture philanthropy.” The amount of money that disease foundations contribute- to industry research would not pay the development costs of even one drug today. But, the strategic timing of the funding as well as the additional resources and expertise that the founda- tions provide serve to enable drug discovery and development that wouldn’t be undertaken otherwise and perhaps to speed that development as well. V enture philanthropy is a term coined in the past few years to describe the funding that disease founda- tions provide to biopharmaceuti- cal companies to research poten- tial drugs and therapies in their respective diseases. U.S. disease foundations’ investment in the biopharmaceutical industry this year will be about $75 million— 10 times as much as its investment in 2000. The figure should contin- ue to rise. These foundations believe in metrics, milestones and account- ability. They don’t just want to fund drug development; they want to change it. In the biotech sector, they may have met their perfect match. But why are non-profits giv- ing money to for-profit biotechs in the first place? On its face, it’s counterintuitive, but a number of factors have created the perfect environment for venture philan- thropy. Disappointed by acade- mia’s inability and lack of incen- tive to translate discoveries into the clinic as well as a reduced National Institutes of Health (NIH) budget, foundations real- ized they had to bring industry into the equation or let promising research languish in the lab. Biotechs have been staring for years into the so-called “Valley of Death”—the preclini- cal stage of development that presents a too high-risk invest- ment to attract venture capital- ists anymore. These companies couldn’t conduct translational research in rare diseases without external funding sources. Foun- dations are the only organiza- tions willing and able to fill that see Venture Philanthropy on page 7 FDA’s Guidance on Supervisory Responsibilities of Investigators The FDA’s new guidance document on investigators’ supervisory responsibilities is useful for both experienced investigators and novices who want to understand FDA’s latest thoughts about how they con- duct clinical research. This article explains the draft guidance and provides step-by-step insight for investi- gators who need to be in com- pliance with the FDA. T he U.S. Food and Drug Administration (FDA) released an important draft guidance document in May called “Protecting the Rights, Safety, and Welfare of Study Subjects—Supervisory Responsibilities of Investigators.” The guidance should be very useful to investigators whether those investigators are experi- enced with or new to FDA-regu- lated clinical trials. The intent of this document is to help investi- gators meet their responsibili- see FDA Guidance on page 14 Month in Review . . . . . . . . .3 CentreStage Europe . . . . . . 5 EMEA’s Bold Appointment Reflects Agency’s New Broader Role Grant Opportunities . . . . . .20 Eye On: Melanoma In the Pipeline Opportunities Underway TrialWatch U.S. Disease Foundation Investment in Biopharmaceutical Industry Source: CenterWatch estimates, 2007 2007 2000 $7 million $75 million © Copyright 2007. Thomson CenterWatch. Duplication of this publication is prohibited. Inside this issue... Venture Philanthropy on the Rise August 2007 A Thomson CenterWatch Publication Volume 14, Issue 08

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Page 1: August 2007 A Thomson CenterWatch Publication

U.S. Disease foundations will

invest about $75 million this year

in biopharmaceutical companies

to fund discovery and develop-

ment of new drugs and therapies

for their corresponding diseases.

This year’s figure represents a

10-fold increase since 2000, and

the figure should continue to

rise.This new avenue of industry

funding has been dubbed “ven-

ture philanthropy.”

The amount of money that

disease foundations contribute-

to industry research would not

pay the development costs of

even one drug today. But, the

strategic timing of the funding as

well as the additional resources

and expertise that the founda-

tions provide serve to enable

drug discovery and development

that wouldn’t be undertaken

otherwise and perhaps to speed

that development as well.

Venture philanthropy is a

term coined in the past

few years to describe

the funding that disease founda-

tions provide to biopharmaceuti-

cal companies to research poten-

tial drugs and therapies in their

respective diseases. U.S. disease

foundations’ investment in the

biopharmaceutical industry this

year will be about $75 million—

10 times as much as its investment

in 2000. The figure should contin-

ue to rise.

These foundations believe in

metrics, milestones and account-

ability. They don’t just want to

fund drug development; they

want to change it. In the biotech

sector, they may have met their

perfect match.

But why are non-profits giv-

ing money to for-profit biotechs

in the first place? On its face,

it’s counterintuitive,but a number

of factors have created the perfect

environment for venture philan-

thropy. Disappointed by acade-

mia’s inability and lack of incen-

tive to translate discoveries into

the clinic as well as a reduced

National Institutes of Health

(NIH) budget, foundations real-

ized they had to bring industry

into the equation or let promising

research languish in the lab.

Biotechs have been staring

for years into the so-called

“Valley of Death”—the preclini-

cal stage of development that

presents a too high-risk invest-

ment to attract venture capital-

ists anymore. These companies

couldn’t conduct translational

research in rare diseases without

external funding sources. Foun-

dations are the only organiza-

tions willing and able to fill that

see Venture Philanthropy on page 7

FDA’s Guidance on SupervisoryResponsibilities of Investigators

The FDA’s new guidance

document on investigators’

supervisory responsibilities is

useful for both experienced

investigators and novices who

want to understand FDA’s latest

thoughts about how they con-

duct clinical research.

This article explains the

draft guidance and provides

step-by-step insight for investi-

gators who need to be in com-

pliance with the FDA.

The U.S. Food and Drug

Administration (FDA)

released an important

draft guidance document in

May called “Protecting the

Rights, Safety, and Welfare of

Study Subjects—Supervisory

Responsibilities of Investigators.”

The guidance should be very

useful to investigators whether

those investigators are experi-

enced with or new to FDA-regu-

lated clinical trials. The intent of

this document is to help investi-

gators meet their responsibili-

see FDA Guidance on page 14

Month in Review . . . . . . . . .3 CentreStage Europe . . . . . . 5

EMEA’s Bold Appointment Reflects Agency’s New Broader Role

Grant Opportunities . . . . . .20

Eye On: MelanomaIn the PipelineOpportunities UnderwayTrialWatch

U.S. Disease Foundation Investment

in Biopharmaceutical Industry

Source: CenterWatch estimates, 2007

20072000

$7 million

$75 million

© Copyright 2007. Thomson CenterWatch. Duplication of this publication is prohibited.

Inside this issue...

Venture Philanthropy on the Rise

August 2007 A Thomson CenterWatch Publication Volume 14, Issue 08

Page 2: August 2007 A Thomson CenterWatch Publication

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Page 3: August 2007 A Thomson CenterWatch Publication

3August 2007CenterWatch

CWWeeklyA bulletin that reports on breaking news in the clinical trials industry. Available every Monday as afax or PDF, an annual subscription is $245 forCenterWatch subscribers.

JobWatchA web-based service and publication listing clinicalresearch jobs, career resources and a searchable resumedatabase. Call Sally Teebagy, (617) 856-1593, or visitwww.centerwatch.com.

CenterWatch ClinicalTrials Listing Service™

www.centerwatch.com

This service provides an international listing ofclinical trials currently enrolling patients, and directories of research centers and industryproviders. Call David Heck, (513) 754-1705.

CW PublicationsState of the Clinical Trials IndustryThe Emerging Markets of Clinical ResearcheDirectory of the Clinical Trials Industry Directory of Drugs in Clinical TrialsBecoming a Successful Clinical Research InvestigatorProtecting Study Volunteers in ResearchA Guide to Patient Recruitment and RetentionThe CRA’s Guide to Monitoring Clinical ResearchThe CRC’s Guide to Coordinating Clinical ResearchVolunteering for a Clinical Trial

Other Information Services

Month in Review

Editor’s Note: Full text articles of these

stories appeared last month in CWWeekly. For

more information about these articles, please

refer to the following CWWeekly issues:

Volume 11, Numbers 27–30.

CROs

Amsterdam-based CRO Clinquest Group has

acquired a stake in Enceladus Pharma-

ceutical, the Netherlands-based developer of

the inflammatory disorder treatment

Nanocort—the product is in phase II trials.

Clinquest also gathered a group of additional

investment groups to back Enceladus as it

brings more products to market. “The partic-

ipation of Clinquest in our activities allows us

to complete the phase IIa clinical proof-of-

concept trial with Nanocort that is currently

ongoing in The Netherlands. Therefore this

means a major boost to our clinical develop-

ment track,” said Bart Metselaar, founder and

chief executive officer of Enceladus. Clinquest

said it believed in Enceladus’ development

pipeline and in the future of Nanocort.

“By entering in this risk-sharing arrangement,

we are able to support the further develop-

ment of Nanocort and the entire liposomal

development platform most efficiently,” said

Cees Wortel, chief executive officer of

Clinquest Group.

Princeton, N.J.-based CRO PharmaNet

Development Group has moved its early-

phase subsidiary, Anapharm, to a new

150,000-square-foot facility in Quebec City.

The site includes laboratories, clinics and

open-plan work stations. PharmaNet said it

hopes to expand the facility by adding 200

beds in the future. The company expects to

open another 150-bed, early-phase unit in

Toronto in July. Anapharm is PharmaNet’s

only business that conducts phase I and bioe-

quivalence studies now that the company has

sold its entire Miami operation, which was

run by the former SFBC. “The investments in

Quebec City and Toronto reflect our confi-

dence in our Canadian operations and pro-

vide the room for future expansion as

Anapharm continues to grow,” said Mark

Ianni, president, early stage development at

PharmaNet Development Group.

Maryland-based CRO United BioSource

Corporation (UBC), in conjunction with

eClinical firm Medidata Solutions, and the

adaptive trials tools and modeling company

Tessella, have joined together to educate the

industry on the use of adaptive clinical trials in

drug development. The companies are pro-

ducing a series of free webinars—six in total—

that will feature expertise from industry pro-

fessionals and regulatory authorities on how to

overcome some of the challenges of these stud-

ies. The first webinar, entitled “Operational

Challenges and Strategic Planning,” will be

held on July 11th and can be viewed online at

www.enablingadaptivetrials.com. “Even with

the right regulatory environment and the right

technology, sponsors who are looking to exe-

cute adaptive clinical trials need to devote time

to planning for this change to the clinical

process,” said Michael Borkowski, general

manager of clinical technologies at UBC.

Wayne, Pa.-based CRO Encorium Group has

inked a $1.7 million deal to manage a phase II

trial in major depressive disorder for an

unnamed biotech company based in Japan.

Encorium will provide site management and

monitoring, data management, biostatistical

support and medical writing for the study.

Encorium stated it intends to pursue further

opportunities in the Japanese market and has

made that market one of its key long-term

business strategies. Recently, the company

signed a $3.8 million contract to conduct

The CenterWatch MonthlyISSN 1556-3367

Editorial Steve ZissonSara Gambrill

Stephen DeSantis

Research Mary Jo Lamberti, Ph.D.Paul Dewberry

Drug Intelligence Tracy Trundle

Trial Watch Tamar Skowronski

Design Paul Gualdoni

For inquiries regarding subscriptions:CenterWatch customer service lineTel: (800) 765-9647 Fax: (800) 284-3291P.O. Box 105109; Atlanta, GA 30348-9891

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CenterWatch is published monthly by CenterWatch,22 Thomson Place, 47F1; Boston, MA 02210.Annual subscription price is $425.

POSTMASTER: Send address changes to:CenterWatch Subscriber Services, P.O. Box 740056;Atlanta, GA 30353-0161. Tel: (800) 765-9647

Month in Review

see Month in Review on page 4

Page 4: August 2007 A Thomson CenterWatch Publication

Month in Review

Thomson4 August 2007

phase IIb clinical trials with a new antiviral

agent for the treatment of shingles (herpes

zoster).

North Carolina-based CRO Quintiles is con-

solidating its U.S. global central laboratory

facilities and clinical development services

offices in Smyrna and Atlanta, Georgia, into a

200,000-square-foot facility based in Marietta.

The expansion will nearly double Quintiles’

research capabilities in the state. The move is

expected to cost about $19 million. The CRO

received an $11 million incentive package and

collaborative support from the Georgia

Department of Economic Development, the

Georgia Research Alliance, the Georgia Cancer

coalition and Cobb County, Georgia. The

company plans to add an additional 400 jobs

in the next four or five years.

Mumbai, India-based contract research

organization (CRO) Vedic Lifesciences has

established a partnership with Amarex, a

CRO based in Maryland. The deal calls for the

two companies to collaborate on clinical

development and regulatory affairs services

for botanical products in the United States

and India. Amarex said its focus on phase I

through IV, combined with Vedic’s expertise

with pre-clinical services and botanical com-

pounds will help both companies to tap into

the U.S. botanical studies market. Vedic

Lifesciences has completed 24 studies, with a

specialty in herbal medicine in India.

Technology

Ft. Lauderdale, Fl.-based eClinical company

OmniComm Systems has completed the

second stage of its Oracle Clinical integration

project. Stage one of the project allowed

OmniComm System’s clients to use the

company’s TrialMaster to export EDC data

into a standard format for importing into

Oracle Clinical. Stage II of the integration

projects gives clients access to Oracle’s global

library of trial referencing to design and build

custom trials using OmniComm’s TrialMaster

product.

DSG, a Malvern, Pa.-based eClinical company,

has established a partnership with Solomon

Group Ltd. (SGL), a CRO based in Taiwan,

with major operations in Shanghai and

Guangdong, China. DSG will provide SGL

with data management and electronic data

capture services to clinical trials in China. SGL

will sell, support and host DSG’s software

solutions eCaseLink, with Chinese language

capabilities. “Our new Chinese language EDC

capability will save our clients both time and

money when conducting trials in China

because users can work in their native lan-

guage without requiring translations of clini-

cal data,” stated DSG’s chief executive officer

Tony Varano.

Patient Recruitment

The Cystic Fibrosis Foundation (CFF) hopes

to increase the number of patients enrolling in

CF clinical trials by 200% through a new

awareness initiative being rolled out across the

organization’s network of 115 affiliated cen-

ters in the United States. Part of the initiative

calls for the creation of a toll-free information

hotline, online trial awareness information

and a series of patient educational materials.

The CFF is a non-profit organization whose

mission is to find treatments and improve care

for patients afflicted with the life-threatening

disease. CF affects 30,000 adults and children

in the U.S. alone. Finding enough subjects to

conduct robust clinical trials in CF is difficult

because of its rarity, yet results of a survey con-

ducted by the organization found that most

CF patients were not aware of the shortage of

trial participants. Nearly all of the drug devel-

opment research in CF is supported by the

foundation.

Accreditation

The Association for the Accreditation of

Human Research Protection Programs

(AAHRPP) has accredited its largest group of

institutions at one time in its six-year history.

In total there were 16 organizations that

gained AAHRPP approval. The certification is

valid for three years. “This is the largest num-

ber of accreditations we have awarded at one

time and we expect the numbers to continue

to grow as we see an ever expanding list of

applicants,” said Marjorie Speers, Ph.D., exec-

utive director, AAHRPP. The list of hospitals

or health systems that received full accredita-

tion includes Cincinnati Children’s Hospital,

MetroHealth System in Cleveland, Ohio, and

the University Hospitals Case Medical Center

in Cleveland. Among the list of universities

were: University of Cincinnati, University of

Kentucky, University of Utah, University of

Rochester, University of Southern California,

and Virginia Commonwealth University in

Richmond. Three Veterans Administration

facilities were granted full accreditation,

including the Cincinnati Department of

Veterans Affairs Medical Center, Department

of Veterans Affairs Salt Lake City Health Care

System, Veterans Affairs Pacific Islands Health

Care System in Honolulu,VA Palo Alto Health

Care System, and Veterans Affairs Healthcare

Network Upstate New York at Albany. Austin,

Texas-based IntegReview Ethical Review

Board also received full certification. The

Lexington VA Medical Center received quali-

fied accreditation, which means it met the

standards with only a few minor deficiencies

in the administrative processes, not involving

patient care. It will still be accredited for three

years and if these deficiencies are corrected

within that time, the institution will receive

full accreditation status.

Month in Reviewcontinued from page 3

Page 5: August 2007 A Thomson CenterWatch Publication

CentreStage Europe

5August 2007CenterWatch

Professor Hans-Georg Eichler, a

clinical pharmacologist from the

University of Vienna, took on the

newly created post of senior medical officer at

the European Medicines Evaluation Agency

in February. In an exclusive interview with

CenterWatch, he describes the agency’s

broadening scope and how this will affect the

process of obtaining marketing authoriza-

tions for new drugs in Europe.

There wasn’t universal rejoicing when

the EMEA was born in 1995. Many politi-

cians within the then-15 member countries

of the European Union were skeptical

about the need for a pan-European agency,

believing it could undermine the status of

their own national authorities. Meanwhile,

some working in the pharmaceutical

industry were concerned that the new

organization might add a layer of costly

bureaucracy without necessarily diminish-

ing the difficulties in getting products

approved through the various national

procedures.

But in the first 12 years of its existence,

EMEA has shown that it can assist the

industry in finding a simpler and more

readily navigable route to bring medicines

to the market simultaneously throughout

the EU. Since then, the community has

expanded to its current 27 member states,

and the questions being asked of all phar-

maceutical regulators have become more

difficult to answer. So the creation of the

new post of senior medical officer was one

response to the agency’s evolving role.

“When the EMEA was set up, there were

a lot of people who thought it would never

fly, but it has taken off and it is doing its

duty. But it has been realized that we now

have to take its work to the next level,”

Eichler explained. “Legislation passed by

the European Parliament two years ago

gave new tasks to the agency’s secretariat. In

the past, its job was just to give administra-

tive and technical support to the commit-

tees [made up of experts representing each

member state], now it has a role to also

offer scientific support.”

The agency’s broadening role is reflect-

ed in the changes to its expert committee

system. At first, there were only the com-

mittee for proprietary medicinal products

(CPMP, now renamed the CHMP, or the

committee for medicinal products for

human use) and its veterinary products

equivalent, the CVMP. Then new commit-

tees were formed to deal with orphan

drugs, herbal products and, as of last

month, a committee for pediatric products.

Within the next year another committee

will be set up to look at so-called advanced

therapies.

As the number of committees grows, so

does the risk that they may reach conflict-

ing decisions on matters where they have

adjacent or overlapping interests. Rather

than risk sending out contradictory mes-

sages to applicants, Eichler has been

appointed with responsibility for identify-

ing and resolving any such conflicts.

But as well as monitoring its own per-

formance, the agency—together with the

national agencies in each member state—

wants to change the way medicinal prod-

ucts are assessed in Europe.

“It is no longer enough for the agency

to have a passive role in just accepting

applications as they come in. We also have

a responsibility to explore how we can

improve our work, to identify strengths and

weaknesses and to help drive forward a reg-

ulatory process that is more transparent,

more predictable and more consistent,” he

said. “Part of my remit is to coordinate this

research agenda. I will be looking for spe-

cific projects that will help the agency to

answer some vital questions. How can the

process for assessing risks and benefits be

improved? Are there new technologies out

there that can be applied? Also, communi-

cating to the wider world about the risks of

drug treatments is an important issue – so

how can we communicate the fact that

sometimes there is no ideal solution to the

problems of balancing risks and benefits.”

Eichler arrived at the agency’s head-

quarters in the redeveloped former docks

area in the East End of London, having held

the post of head of clinical pharmacology

at the Medical University of Vienna. After

training as a physician, his career has

included stints in research, industry and

public policy as a member of advisory

boards to the Austrian Ministry of Health

and a national delegate on EMEA’s orphan

products committee. He claims to have had

little difficulty adjusting to living and

working in a new country, and his flawless

English gives an indication why. His lan-

guage skills have been polished by his expe-

rience as a postgraduate student at the

University of Surrey, just south of London,

and as a visiting scientist at Ciba Geigy’s

former UK laboratory in Horsham and

Merck’s headquarters in New Jersey.

In his new post, Eichler reports directly

to the agency’s executive director, Thomas

Lönngren. His responsibilities lie outside

the organization’s existing departmental

structure, and he has no role in its day-to-

day operational activities of processing

license applications.

So how has he occupied his time in his

first months as senior medical officer? His

immediate task, he said, was to get proper-

ly oriented.

“When you join an organization with

this level of complexity, your first priority is

to figure out your way around it. Yes, I had

been involved already with the agency as a

member of its committees, but that only

gives you a superficial perspective on the

workings of an organization that forms

see Eichler on page 6

EMEA’s Bold Appointment Reflects Agency’s New Broader Role

Page 6: August 2007 A Thomson CenterWatch Publication

CentreStage Europe

Thomson6 August 2007

part of a network with a number of con-

stituent parts—the Commission, the

national competent bodies [i.e., national

licensing authorities], industry, patients’

organizations and so on,” he said.

More recently, his focus has been on

efforts to identify and prioritize the issues

that will be tackled in this new research

agenda. He will also be seeking suitable

partners with which to carry out these

studies. He has no staff reporting directly to

him and, as he points out, within the busy

agency “there aren’t a lot of people sitting

around waiting for you to give them work

to do.” So he hopes to set up collaborative

projects with the various national agencies

and also establish much stronger links with

the academic world. Finding the resources

needed to carry out these studies promises

to be his biggest challenge over the next few

months. The agency has no guaranteed

budget for its research.

If Eichler succeeds in his mission to

strengthen the scientific foundations on

which the agency arrives at its scientific

opinions, what effects will this have on the

clinical trials process?

“You have to look at this research in a

broader context. What does industry want

from a regulator in developing new drugs?

The main thing is predictability. So by

refining our methodologies to ensure that

we produce more consistent results, then

our work will enhance that predictability,”

he noted.

Although the direct costs to industry of

license applications won’t change, he is

hopeful that improving the efficiency of the

approval process will improve. “With all the

regulatory agencies, EMEA, FDA and oth-

ers, there is a high rejection rate for license

applications, so a company must go

through the whole drug development

process only to fall at the last hurdle. I am

sure that many of those failed applications

could be avoidable. One thing that we will

need to look into is how to get companies

to ask us for scientific advice earlier in the

process. Making our actions more pre-

dictable will also help because it could

mean that the developers may know what

the outcome is going to be before they com-

plete the work. If they know where we put

the goalposts it will be a lot easier for them

to score.”

However closely companies and regula-

tors work together, there is always the pos-

sibility of unexpected events such as the

TeGenero incident in March 2006. The

health problems suffered by volunteers in a

phase I trial of the company’s product

TGN1412 prompted several European

states to introduce unilateral measures to

protect the safety of trial subjects and gave

new impetus to plans for revising the EU

Clinical Trials Directive.

EMEA will be hosting a meeting later

this year in which EU experts will assess

measures to minimize the risks to partici-

pants in phase I studies. It has also pro-

duced guidance for companies on strategies

for dealing with potentially high-risk prod-

ucts.

“The bottom line is that there is a need

for stronger supervision of these products.

Everyone is in agreement with that idea, but

the problem lies in how you define a high-

risk product. We are well aware of indus-

try’s concerns about scope creep – that risk-

averse regulators will label everything as

high risk and insist on the strictest controls.

Our guidelines provide criteria for classify-

ing new products, and I think this offers a

well-balanced view on the issue.”

Within the agency there is an apprecia-

tion that Europe is competing with other

regions in an increasingly global clinical tri-

als industry. But there are matters in which

cooperation between rival regulatory

authorities can benefit both sides.

“We are in discussions with the FDA in

an attempt to achieve a harmonized

approach. There have been occasions when

we have told companies preparing for a piv-

otal phase III study – ‘You must do it in this

way,’ while the FDA has been saying some-

thing completely different. We both recog-

nize that this doesn’t make the drug devel-

opment process any easier and so we are

trying to develop a common approach, par-

ticularly in the areas of orphan and pedi-

atric products,” Eichler said.

Another indication of this shared

approach to drug regulatory issues on both

sides of the Atlantic came with the FDA’s

appointment of Dr. Janet Woodcock as its

chief medical officer just a few weeks before

Eichler arrived in London. In her new post,

Woodcock takes responsibility for the

Critical Path Initiative designed to improve

the scientific basis of medical product

development and her responsibilities cover

much the same ground. “Given that both

sides recognize that we have an important

role in fostering drug development for the

benefit of public health, we expect to be

working closely together in the near

future,” he said.

—John Bonner

Eichlercontinued from page 5

Page 7: August 2007 A Thomson CenterWatch Publication

Industry Reports

7August 2007CenterWatch

funding gap, but not without careful consid-

eration and a firm understanding of what

they want from biotechs in return.

But, disease foundations do not just give

money to industry. Money doesn’t solve

problems, such as protocol development

and patient recruitment, on its own. In addi-

tion to money, disease foundations provide

valuable resources and expertise to further

de-risk the joint venture between them and

biotech.

This article will focus on five of the

leading foundations that have a venture phi-

lanthropy component. Their programs

promise to be an increasingly important

part of the drug development landscape in

the decade to come. They are: The Cystic

Fibrosis Foundation, Multiple Myeloma

Research Foundation, Muscular Dystrophy

Association, Juvenile Diabetes Research

Foundation, and The Michael J. Fox

Foundation for Parkinson’s Research. Their

work and money will have positive implica-

tions not only for patients who will benefit

from the therapies that result, but also for

the industry as a whole.

Lost in TranslationThese five foundations have all created pro-

grams targeted to industry because, after

years of funding academic research, they

learned that it is very difficult to translate

promising academic discoveries into the

clinical setting, or they were aware of the

venture capital funding gap that had created

the “Valley of Death,” or they knew that NIH

wasn’t going to step in with funding, or

some combination of these factors. Potential

drugs and therapies getting lost in transla-

tion—or not even getting that far—drove

some foundations to re-evaluate their grant-

ing mechanisms and start new programs to

ensure industry could get into the mix.

While most foundations will continue to

award the larger portion of their research

funds to academia—where important dis-

coveries are made—the percentage of foun-

dation research funds targeted to industry

will rise.

Sharon Hesterlee, Ph.D., vice president

translational research, Muscular Dystrophy

Association, said, “We’d get progress reports

every year, and I noticed some grantees all

started to sound familiar. We had one per-

son in particular who was developing a gene

therapy approach for a disorder using all

kinds of techniques—vectors and parti-

cles—and had had reasonable success using

animal models using various techniques,

and every year we got a new grant [applica-

tion] for a new technique. I pulled a lot of

these files out and started calling the investi-

gators to find out what are the next steps,

and if you’re not planning to move forward

with therapy development, why not?

“It very quickly became clear that the

investigators were interested in doing some-

thing with this therapy but didn’t know

what, so what they were doing was just send-

ing new grants that incrementally improved

on the technology. They were very much

driven by the technology and whatever is the

chief hypothesis in the lab and keeping the

lab funded. They didn’t really know how to

take the next step forward and develop a

new therapy, especially with a new indica-

tion, a drug that hasn’t been in humans

before,” said Hesterlee.

Peter Lomedico, Ph.D., director of

industry partnerships at the Juvenile

Diabetes Research Foundation, also realized

that various forces were working against

successful translation. “A few years ago, we

looked at the research portfolio and realized

that there was considerable effort on the

academic side, the discovery research side,

and good progress was being made there,

but in terms of translating the discoveries,

getting more things into the clinic and driv-

ing toward development with the eventual

goal of reaching the marketplace, there was-

n’t a whole lot there in terms of trying to

deal with that. Certainly the changing

investor climate for biotech, changes in the

venture capital community and also within

big pharma really made clear that there is

this gap, whether you refer to it as ‘The

Valley of Death’ or some other phrase.

Clearly there is a challenge for an organiza-

tion like ours to see the fruits of the discov-

ery research mostly coming from the aca-

demic side and how to get that translated

over to companies so they can take on pro-

grams and drive through clinical develop-

ment and through the approval process,”

Lomedico said.

Kathy Giusti, founder and chief execu-

tive officer, Multiple Myeloma Research

Foundation, has a unique vantage point: She

has worked in the pharmaceutical industry,

worked for government, worked with acade-

mia, and is a multiple myeloma patient.“I’ve

seen all elements of drug development from

NIH to industry to academia to being a

patient myself, and I think it’s that that has

given me this ability to understand nobody

has bad intent, it’s just an old and broken

system that really needs to be updated. I

hope that we can play a major role in that,”

said Giusti.

It’s Not Just About the MoneyWhile each foundation provides funding to

biotechs to give them the ability and the

incentive to conduct research that could lead

to a cure of their disease, just throwing

money at the problem of translation isn’t

going to fix it. These foundations are well

aware of the fact that they bring a lot more

value to the biotechs than simple dollars and

cents can provide. CFF and MMRF have

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Venture Philanthropycontinued from page 1

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patient databases and work to match

biotechs with sites where they will find the

right patients to study in. All of them have

access to deep expertise in their disease as

well as the knowledge of where their money

will potentially do the most good toward

reaching the goal of getting drugs and ther-

apies approved.

Deborah Brooks, president and chief

executive officer at The Michael J. Fox

Foundation for Parkinson’s Research,

described the role of funding as a part of the

value of what her foundation contributes to

industry.

Brooks said, “Funding is an aspect of

how we get defined, but it’s not the only way

we engage and provide value to industry. We

use our capital, in particular with industry,

through the lens of how we can keep PD

[Parkinson’s Disease] prioritized in their

portfolio of activity—that’s everything from

supporting specific therapeutic ideas as well

as investing in tools that de-risk broadly. We

look at the drug development pipeline, and

we try to understand the landscape as it

relates to Parkinson’s in as much detail as we

can. We probably have among the best cat-

bird seats in the world because it’s our daily

focus. We think about what’s missing, what

are we learning, what do we know, what

action can be taken, and how can we use our

resources and expertise and connect the

dots? And that translates into a portfolio of

activity. We bring the money, but we’re also

bringing the expertise in that access to addi-

tional information and resources and prob-

lem solving.”

At the BIO2007 conference held in

Boston, Robert Gallotto, vice president,

strategic planning and alliance manage-

ment, Altus Pharmaceuticals, said during

the “Venture Philanthropy and Foundation

Deals” panel presentation, “Capital is only

one part of the equation. It’s much more

the intellectual capital that was important

for us.”

Daniel Grau, chief operating officer of

CombinatoRx, a Cambridge, Mass.-based

biotechnology company, said at BIO2007,

“We also look for access that most venture

philanthropists can provide in spades in their

various indication fields, to various tools and

to leading advisors and expertise. This is a

very important benefit.” CombinatoRx is a

biotechnology company focused on develop-

ing new medicines built from synergistic

combinations of approved drugs. In less than

six years, the company has discovered and

advanced into clinical trials a portfolio of six

product candidates targeting multiple

immuno-inflammatory diseases and cancer,

at a total investment, including development

of its proprietary screening technology, of

less than $50 million. The company has four

venture philanthropy deals ongoing, one

each with Accelerate Brain Cancer Cures,

Cystic Fibrosis Foundation, CHDI

[Huntington’s Disease], and the Spinal

Muscular Atrophy Foundation.

Added Grau, “What’s been beneficial to

us is that in some cases with these deals

we’re moving deeper into a therapeutic area

that we already have a presence in.

Oncology would be an example of that. We

were not active in glioblastoma multiforme,

brain cancer, but we had an existing set of

products in the cancer field, so this allowed

us to go deeper into that area. In other cases,

we’re diversifying and building out new

R&D verticals.”

In addition to bringing money, resources

and expertise, foundations make sure that

the companies they give funds to are meet-

ing their obligations. The foundations have

built in the power to withhold payment for

performance that doesn’t measure up, and

they aren’t afraid to use it. Grants are mile-

stone-driven, and each foundation has met-

rics they use to judge a company’s perform-

ance on a grant-by-grant basis.

“We set the milestones in a logical

sequence. The point was that if you can’t

accomplish this step, there’s no sense mov-

ing forward or providing further money.

That gives us tighter control. With an aca-

demic grant, we get yearly progress reports.

There’s some merit to that maybe, but with

a for-profit company, we’re taking donor

money and giving it to a for-profit company

and we feel we have a fiduciary obligation to

make sure we have a lot of oversight over

how it’s being spent. It’s worked out well. We

have a very good idea of where the company

is. Often, milestones are delayed. That just

happens, and we have the option of with-

holding payments when milestones aren’t

accomplished. For a very small startup com-

pany, that can mean that people don’t get

paid. I’ve found that if you want something

done in record time, you can use that carrot-

stick approach,” said MDA’s Hesterlee.

Biotech AdvantageBiotechs often find themselves in a Catch-22:

If they do not have proof-of-concept data,

they cannot attract venture capital, but if

they do not have capital, they cannot supply

those data. To compound the difficulty of the

situation, when biotechs work toward getting

these data with big pharma, they have to

choose between getting their research costs

funded or dilutive activities such as giving up

their product rights, but venture philanthro-

py money does not require that biotechs

make that choice. Sometimes foundations’

money is even the difference between a

biotech’s existing or not.

“Venture philanthropy has actually

played a very important role for us in build-

ing our busines,” said Grau. “Funding from

the bench through patient proof-of-concept,

plus commercial rights. That’s one of the

crystal clear virtues of working with venture

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9August 2007CenterWatch

philanthropists. If you were to work with a

specialty pharma or large pharma organiza-

tion, it’s very unusual that you would have

their commitment to cover all your research

costs and at the same time they would pro-

vide you with all the product rights. Those

things typically don’t go together, but they

can go together in the venture philanthropy

space, and that’s a very interesting opportu-

nity for biotech companies.”

Altus Pharmaceuticals’ Gallotto also

described the importance of his company’s

venture philanthropy deal with CFF. Altus

Pharmaceuticals is focused on developing

and commercializing novel protein thera-

peutics for patients with chronic gastroin-

testinal and metabolic diseases. The compa-

ny is developing a portfolio of products

based on its novel protein crystallization

technology and has the potential to redefine

the use of biopharmaceutical proteins for

gastrointestinal or metabolic diseases by

allowing the oral and parenteral delivery of

high value protein replacement products.

When Altus started working with CFF in

2000, it was a pre-IND company and didn’t

have the infrastructure to develop an effec-

tive product. It was in the typical biotech

catch-22. CFF committed funding in the

early stages. “They were helpful for us to

bridge the gap from an idea to animal proof-

of-principle data with a $1 million grant in

2000. From there, in 2001 we were able to

develop a broad collaboration and alliance

agreement with the CF Foundation. It allows

the CFF Foundation to fund up to $25 mil-

lion in milestone-based grants. This was

important for us because most of that fund-

ing was done prior to phase II . . . This cash

was non-dilutive. The ability to retain rights

to your molecules is uniquely important to

any biotech company,” said Gallotto.

Although this kind of money is a very

large investment for a foundation, it is rela-

tively small in the investment world. But,

aside from making translational research

possible, venture philanthropy and founda-

tion resources can attract venture capital far-

ther down the road.

“Venture capital organizations like the

fact that you’re working with these groups

that have unique perspective of the disease

that otherwise companies themselves would

not have. You have unique access to physi-

cians, nurses, patients who have a complete

understanding of these diseases and under-

stand how to design protocols. This was

very important from our perspective when

we went later on to raise outside capital from

the venture community. We took this collab-

oration with the CF Foundation with the

animal data that we had and our plan for

our IND, and we were able to raise $51 mil-

lion at the end of 2001 through a Series B

round. Without the CF funding prior to

IND, to phase I being completed, to the end

of phase II being completed, we may never

have gotten as far as we did in terms of this

product or even as a company with respect

to even existing,” said Gallotto.

Altus finished its initial public offering

(IPO) last year and has initiated phase III

clinical trials of its enzyme replacement

therapy in people with cystic fibrosis and

pancreatic insufficiency.

Foundation ModelsAlthough venture philanthropy is a collec-

tive term to describe disease foundation

investment in biotechnology for the devel-

opment of drugs and therapies in their

respective indications, it actually takes many

forms. Following is a description of the dif-

ferent foundation models.

Cystic Fibrosis Foundation (CFF).

Year founded: 1955

Money invested in research since

founding: $735 million (through 2006)

Industry program: Cystic Fibrosis

Foundation Therapeutics

Development Program (1998)

Money invested in industry drug

discovery & development since 1998:

$230 million

Money earmarked for industry drug

discovery & development in 2007:

$43 million

Biotech collaborators: 24+

Drugs in CF pipeline: 30+

CF patients in database: 23,000

Sites in clinical trial network: 64

The gene that causes cystic fibrosis was dis-

covered in 1989, and researchers know much

about the basic defect. This is the good news.

The difficulty getting pharmaceutical compa-

nies or biotechnology companies interested

in researching therapies for the disease is that,

even though it is the most common fatal

genetic disease in the U.S., it affects only

30,000 in this country and 70,000 worldwide.

Beall of The Cystic Fibrosis Foundation knew

that the foundation would have to establish a

new business model for his non-profit if it

wanted to interest pharma and biotech to

enter the field of cystic fibrosis.

“We felt that the best thing we could do

was reduce these companies’ financial risk by

supporting early stage drug discovery. Our

business model is to effectively translate the

understanding of the basic defect in the gene

into new therapies for our patients,”said Beall.

He began in 1998 by investing in a thera-

peutic development program comprising a

network of seven clinical research centers,

designated as the Cystic Fibrosis Foundation’s

Therapeutics Development Network that

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specialize in conducting phase I and II stud-

ies for treatment of cystic fibrosis. The net-

work also had a coordinating center. In

1999, CFF received a $20 million grant from

the Bill and Melinda Gates Foundation, and

in 2000, the foundation launched Cystic

Fibrosis Foundation Therapeutics (CFFT),

its non-profit drug discovery and develop-

ment affiliate that administers contracts

with biotech companies. TDP is a program

of CFFT.

Initiation of the collaborations with

industry, level of investment and deal struc-

ture vary. CFFT agrees to fund, on a match-

ing basis, the development of products/com-

pounds for the purpose of identifying new

drugs for cystic fibrosis. CFFT may approach

a biotech studying a compound being stud-

ied in another indication and give that com-

pany anywhere between $50,000 and $25

million to test it in cystic fibrosis. Some deals

with biotech are royalty-based—if the drug

is approved, the foundation gets a multiple

back. Sometimes the deals are a combination

of royalties and a multiple. The CFF also

requires additional compensation for

extraordinary sales results. If there is a sus-

pension in development activities, the CFF

obtains worldwide rights to develop the

product with agreement to negotiate royal-

ties to the original partner after CFF’s invest-

ment is returned.

Negotiated portions of the monetary

awards from CFFT are dependent on the

accomplishment of predetermined, success-

driven milestones. CFFT may withdraw

from the project under certain circum-

stances, including failure to achieve mile-

stones. CFFT requires the establishment of

a Scientific Advisory Council made up of

CFFT, sponsor and joint representatives.

The foundation has not taken any equi-

ty in any of the companies to this point. The

foundation re-invests all the money it

makes into more research. When a drug was

approved by the U.S. Food and Drug

Administration [FDA] a few years ago, the

foundation flipped the royalties and sold

the future rights to it for about $18 million

and used that money for the initial capital-

ization for its high throughput screening

program.

CFF has also created a new funding

vehicle, called the Technology Access

Program, for emerging drug discovery tech-

nology. The foundation awards up to $1

million per year for three years to focus on

new or complementary approaches to tar-

gets that may promote maturation of F508

CFTR. The cystic fibrosis transmembrane

conductance regulator (CFTR) is mutated

in patients with CF. The most common CF-

associated mutation is deletion of pheny-

lalanine at residue 508, CFTR delta F508.

Two awards were made last year.

A biotech or pharma that has a product

or idea it wants to test in cystic fibrosis can

use the network run by the Therapeutic

Development Program. CFF helps compa-

nies conduct clinical trials and does the

early phase drug discovery programs.

In February, CFF expanded its investiga-

tive site network, run by its Therapeutic

Development Program (TDP), with a $3

million award distributed among 45 new

research centers in 20 states nationwide.

The money is being used to build each site’s

infrastructure, expand its staff, or help with

patient recruitment operations. It is a two-

year program, which means sites will have

to perform studies and follow Good

Clinical Practice (GCP) procedures in order

to stay in the network the following year.

CFFT has established an operational center

in Seattle to coordinate with the network,

which is staffed with more than 40 person-

nel and can assist in protocol development

and monitoring. The investment is meant

to prepare the new sites to become part of

CFF’s existing network of 18 sites. By 2009,

CFFT hopes to have all 45 sites, plus addi-

tional sites added in the coming years.

“People like our business model because

we hold ourselves accountable. We are the

only voluntary health organization that

measures success in terms of our pipeline.

The pipeline itself is our metric for future

success. Our medical programs have con-

tinued to grow at a rate of nearly 19% for

each of the last three years,” said Beall.

Multiple Myeloma Research

Foundation

Year founded: 1998

Industry program: Lead grant

Research entity: Multiple Myeloma

Research Consortium (MMRC,

2004)

Academic centers in MMRC: 13

Money earmarked for drug develop-

ment in 2007: $11 million (indus-

try share=$2 to $3 million)

Biotech collaborators: 30+

Compounds: 37 single agents/

combinations

Patients in database: 30,000

Tissue bank: 1,000 bone marrows

and matching peripheral blood

In 1998, when Kathy Giusti founded the

Multiple Myeloma Research Foundation,

fewer than five drugs were being evaluated

in multiple myeloma. Affecting 45,000 peo-

ple in the U.S., it is a heterogeneic disease.

For this reason, Giusti believes that “myelo-

ma is probably going to be cured by a cock-

tail of therapies. It may not be exactly the

same for every patient. As a result, we need a

significant repertoire of really good, targeted

therapies from biotech and pharmaceutical

companies. And unfortunately with mini-

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mal funding from NIH or reduced funding

from NIH and the difficulty in raising [ven-

ture capital] funds, we thought we needed to

step in to build the bridge between the vali-

dation of drugs and clinical trials.”

In 2004, Giusti founded a non-profit

affiliated with MMRF called the Multiple

Myeloma Research Consortium comprising

13 academic centers that have signed mem-

bership agreements. MMRF conducted a

request for applications for centers. Those

that had the highest number of patients,

clinical expertise, in the right geographic

area and had a good blend of translational

research were the ones selected. MMRF

developed the model and all policies and

procedures for the consortium, and the

foundation raises the money to fund it.

Each center is held accountable to specific

milestones and timelines. Metrics include:

the time that a trial comes to MMRF as a

concept to the point which a protocol is

written to the time of IRB approval to first

patient enrolled to study close. Each center

is given a scorecard rating its performance

MMRF employs five people dedicated to

facilitating clinical research conducted at

the consortium’s academic centers and is

able to call on the skills of multiple myelo-

ma experts as well. Giusti has identified

problem areas in the clinical research

process, including protocol development

and contracting. For both areas, Giusti

offers solutions to speed the drug develop-

ment process.

“One of the major obstacles in drug

development is developing a protocol. A

new biotech may not have the expertise to

develop a protocol in myeloma and to

understand which sites would be best for

them to work with. We immediately offer

them that expertise. We work with the gen-

tlemen who have done all the leading

myeloma trials who can provide all the pro-

tocol expertise. We also know exactly what

trials are going on at all of our sites, and if

competing trials are happening. We can tell

our pharma partners who we think can

move quickly and would have capacity to

get this trial done.

“Contracting is one of the major obsta-

cles in getting trials done quickly, so we

developed master templates here so that we

could speed the contracting process. I hired

my own attorney and I pay outside counsel

to get all the contracting done,” said Giusti.

Communication is key for the centers,

which are required to be on monthly tele-

conferences and at quarterly face-to-face

meetings. On calls and at meetings, there

are usually also companies presenting their

compounds or discussing the protocol or

patient enrollment. Ongoing trials require a

weekly teleconference.

A unique feature of what MMRF offers

its consortium is access to its tissue bank,

patient database and its patient navigator

program.

Much of what MMRF does entails

matching biotechs with the right academic

center. The foundation will allocate $11

million for research investment this year.

Two to three million dollars of that amount

will be allocated to MMRF’s lead grant pro-

gram, which funds go directly to biotechs

that MMRF selects from those that apply.

“Speed to market is everything we’re

about because no company is going to

come to myeloma because it’s a big market.

But, they would come here because they

have a foundation like ours that can facili-

tate the process so much that you say, ‘We

could get our drug to market faster because

this group will help us from concept to

enrollment to the education of the drug,

and even with FDA relations because we do

FDA roundtables as well. We take great

pride in being an end-to-end solution for

biotech and pharmaceutical companies,”

said Giusti.

Muscular Dystrophy Association

Year founded: 1950

Money spent on research since found-

ing: $670 million

Industry Program: Translational

Research Program (TRP)/corporate

grant (2004)

Money invested in industry since start

of TRP program: $5.5 million+

Projected industry funding in 2007:

$3-$4 million

Biotech collaborators: 2

Patient database: No, but working

toward it

Industry projects: 1 active, 2 completed

The Muscular Dystrophy Association

(MDA) is an umbrella organization for

about 40 neuromuscular diseases that in the

aggregate affect close to 500,000 people in

the U.S. but, individually, they are all orphan

diseases. There have been no drugs

approved by the FDA specifically for mus-

cular dystrophy ever.

“We tailored a program to address spe-

cific categories of this gap we saw in the

translational aspects of drug development,”

said Hesterlee.

The MDA created the Translational

Research Program (TRP), which has four

categories of funding: IND Planning Grant,

Clinical Research Training Grant,

Infrastructure Grant and Corporate Grant.

The first two grant categories are designed

for sites, the third is to fund a national

patient database and the fourth category of

grant money is dedicated to industry. “We

designed a mechanism to fund companies

directly that are involved in early stage drug

development—all the steps leading up to

an IND (investigational new drug applica-

tion) and then a phase I or phase II clinical

trial. We saw it as affirmative action for rare

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11August 2007CenterWatch

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disease. If we can put some money into this

high-risk early stage, the idea is to move

things along until there’s some efficacy data

in humans, and then [companies] can start

getting venture capital money.” All four

programs are designed to increase the

number of biotechs working in muscular

dystrophy.

Two biotechs have completed TRP cor-

porate grants and one of them and a new

organization are working on a current

grant. MDA does not pre-allocate funds

every year, except for the research training

grants. MDA requires matching funds from

the biotechs. Companies need to match

MDA funds for a project with at least 50%

of their own money going into that same

budget.

Both MDA and funded organizations

choose a group of outside experts in the

field to form a steering committee that will

oversee the grant milestones. “We find that

the steering committee meetings function

not just to make a judgment on the mile-

stone, but they’re very good advisory com-

mittees. The companies find them to be a

good resource,” said Hesterlee.

In addition to what it offers through its

corporate grant program, MDA has discov-

ered some creative ways to enable and

speed drug development through strategic

funding. MDA has found a way to help

both Genzyme and Wyeth conduct clinical

trials in muscular dystrophy patients. In

each case, MDA approached the companies

and asked what it could do to help them

conduct clinical research in muscular dys-

trophy. Patients enrolled in the companies’

studies were often very ill and needed med-

ical care outside the clinical trial while in

the hospital. Because insurance companies

would not pay for this medical care, and the

companies were not allowed to, MDA

offered to give grants out of its translation-

al research budget to each of the sites

enrolling patients for the companies’

respective studies. Two of Wyeth’s sites

accepted funding that totaled more than

$55,000 last year. The money in this case

could also be used for travel in addition to

clinical trial subjects’ hospital costs. Three

of Genzyme’s sites were each given a grant

of $50,000 in 2003 to cover clinical trial

subjects’ hospital costs.

“Maybe there’s more than one way to

get from point A to point B, but just fund-

ing research through a standard NIH

model was never going to get us the drugs.

That was clear. I think that we had to do

this. I don’t think it was a choice. It’s our

mission to develop therapies and cures for

these diseases, not just find promising

research. It’s to see them all the way

through the clinics. If we don’t do this type

of program, then we’re letting people

down,” said Hesterlee.

MDA is planning a formal evaluation of

the program this year. Because much of the

funding has been focused on Duchenne’s

muscular dystrophy, MDA is going to look

at whether it’s increased the number of

phase I/II clinical trials in that disease.

Secondly, MDA is going to look at whether

the number of biotechnology and pharma-

ceutical companies working in this area has

increased. “Having drugs approved is the

long-term goal. It’s something we might

use in 10 years to see how the program’s

progressing,” said Hesterlee.

Juvenile Diabetes Research

Foundation

Year founded: 1970

Investment in research since founding:

$1 billion

Industry program: Industry Discovery

and Development Partnership

Program (2004)

Projected industry funding in 2007:

$14 million

Industry collaborations: 15

Product candidates: 5 in clinical trials,

10 in preclinical discovery and

evaluation

Patient database: No

The Juvenile Diabetes Research Foundation

(JDRF) has awarded more than $1 billion to

research of type 1 diabetes, most of it to aca-

demia. In that time, it has come to under-

stand some of the basic biology of the dis-

ease and some of the aspects of its patho-

genesis. In 2004, it launched its Industry

Discovery and Development Partnership

Program (IDDPP) specifically to fund

proof-of-concept in animal models and

proof-of-concept in man studies in type 1

diabetes sponsored by industry.

IDDPP’s grants are milestone-driven,

and the foundation asks the companies to

match their funding with an equal or greater

amount. JDRF has an industry application

process. The foundation accepts inquiries

and applications on a rolling basis. JDRF

also approaches companies.

“If your goal is to impact disease pro-

gression and patient quality of life and effect

a cure for a particular disease, the best way

that’s going to happen is for companies to

get involved and take therapeutics or diag-

nostics or medical devices through develop-

ment, through the approval process and out

into the marketplace . . . The academic inves-

tigators who are necessary to start the

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13August 2007CenterWatch

process can’t do it by themselves. We really

need to work together and engage industry to

take over some of these opportunities and

drive the marketplace ... We want to see more

clinical research programs and more product

candidates moving toward development, so

one metric of success is as we shift more and

more into supporting clinical development

programs, that means there are more product

candidates moving toward the marketplace,”

said Lomedico.

Michael J. Fox Foundation for

Parkinson’s Research (MJFF)

Year founded: 2000

Investment in research since founding:

$94 million+

Industry program: Therapeutics

Development Initiative (2006)

Money awarded to industry (2006):

$4.6 million

Money committed to industry (2007/8):

$5 million

Patient database: No

Parkinson’s disease affects approximately

500,000 people in the U.S. Its target is

unknown. The foundation has funded more

than $94 million in research directly or

through partnerships since it was founded

in 2000.

“We don’t understand a single cause of

PD, we don’t see in the natural history of the

disease the same experience for every patient.

We can’t tell you that there are subtypes of the

disease, but we suspect it. We are trying to do

some investigating to understand that better,

which means an improved therapy might

work for a small group of patients or it might

work for a broad group of patients. It might

help treat the underlying cause of the disease,

or it might manage symptoms better. It’s such

a mix of potential positive outcomes that we

maintain a portfolio that is advancing as

many of those things that we think are sensi-

ble and we can afford to do,” said Brooks.

The foundation launched its Thera-

peutics Development Initiative (TDI) last

year to expand its industry investment. This

is the foundation’s only program that is

specifically targeted to industry, though

other funding commitments often have an

industry component. Last year 10 industry

research teams were awarded $4.6 million.

These research teams are focused primarily

on the development of neuroprotective

treatments and cell replacement approach-

es. In 2007/08 the foundation plans to

award $5 million through TDI. The pro-

gram has two grant cycles—autumn, 2007

and spring 2008.

Grantees meet in-person for mid-point

assessments of their projects. MJFF’s metrics

focus on the accountability of individual

grantees, making sure that they do what they

promised to in their applications. Mid-point

meetings bring the grantees together in one

place and also offer an opportunity to solve

problems together.

“There’s a real unmet need to capture the

kernels of these ideas at very early stages that

could be transformative and powerful and

really innovative solutions and shepherd

those along by identifying them early, provid-

ing capital in places where there’s no capital

to be had because it’s just too risky and see if

we can breathe some life into these opportu-

nities with the patients in mind,” said Brooks.

Future OutlookDisease foundations have been learning from

each other, sharing best practices and are

committed to spreading the word about ven-

ture philanthropy. While they don’t have

financial relationships with each other, their

common purpose—to discover and develop

therapies for incurable disease—binds them

together and has created a strong community.

Robert J. Beall, president and CEO of the

Cystic Fibrosis Foundation, is considered the

catalyst for investing strategically in industry

research, and he has been generous with his

time and ideas. He was the first to launch an

industry program and has helped others nav-

igate the shoals of the venture philanthropy

model.

“Bob Beall from Cystic Fibrosis sits on

our board of directors, and we’ve learned so

much from him. If I hadn’t had Bob helping

me, I might have wasted time, energy and

money. I’m a big believer in foundations

learning from each other because it makes

certain that the money invested has a broad-

er impact,” said MMRF’s Kathy Giusti.

MJFF’s Brooks has also benefited from

the experiences of other foundation heads

and been able to share some innovative ideas

of her own. She said,“We learn so much from

hearing about the strategies that other groups

have crafted as problem solvers. There are a

handful of these like-minded groups that are

aggressively trying to solve a problem that is

stunningly complex and has essentially been

delegated. The natural market doesn’t solve it

for small diseases or complex diseases. These

groups are comparing notes and not every-

thing that every group does is relevant to

what every group does. It has a lot to do with

the state of your science, what your budget is

and that might have to do with how big your

community is and how wealthy they are. It

has been advantageous to have other people

working aggressively in this field that we can

learn from and share with. We’ve been both a

great beneficiary of other people’s wisdom,

and we’ve figured out some interesting things

that other people are excited about hearing

from us on.”

Foundations’ shift from basic research

funding that went to academia alone to fund-

ing drug discovery and development by

industry as well has brought about changes in

see Venture Philanthropy on page 14

Page 14: August 2007 A Thomson CenterWatch Publication

ties for protecting human subjects and

ensuring the integrity of the data from clin-

ical trials. This guidance also clarifies FDA’s

expectations concerning the investigator’s

responsibility for supervising a clinical trial

in which some tasks are delegated to

employees or colleagues of the investigator

or other third parties.

In FDA regulations, the investigator is

the person who conducts a clinical investi-

gation of a drug, biologic, or medical

device. The FDA definition for “investiga-

tor,” which is a specific regulatory term,

may be found at 21 CFR 312.3(b) for

drugs/biologicals and at 21 CFR 812.3(i)

for medical devices. Because in a clinical

trial there may be multiple investigators

(the principal investigator, one or more

sub-investigators and co-investigators), it is

important to recognize that only one of

these investigators has the ultimate respon-

sibility for the clinical trial. Throughout

this article, that investigator will be referred

to as the principal investigator (PI). The PI

is the intended audience for this draft guid-

ance document.

Under the regulations at 21 CFR 312

(Investigational New Drug Application or

IND) and 21 CFR 812 (Investigational

Device Exemptions or IDE), a PI is respon-

sible for:

ensuring that a clinical investigation is

conducted according to the signed

investigator statement for investigations

of drugs and biological products or a

signed investigator agreement for inves-

tigations of medical devices, and the

investigational plan and applicable reg-

ulations;

protecting the rights, safety, and welfare

of subjects under the PI’s care; and

for controlling the drugs, biological

products, and medical devices under

investigation.

This draft guidance document clarifies

the PI’s responsibilities in the conduct of

clinical investigations subject to these regu-

lations, particularly the responsibilities to

supervise the conduct of the clinical investi-

gation, and to protect the rights, safety, and

welfare of study subjects in drug, biologic,

and medical device clinical trials. The draft

guidance document also provides recom-

mendations on how the PI should supervise

the study-related actions of persons not in

the direct employ of the PI, including cer-

tain study staff and parties conducting asso-

ciated testing and assessments.

Draft guidance is not generally intend-

ed for widespread use; it is intended to

solicit public comment. Once FDA has

received and reviewed all comments from

the public, the agency will release the final

Industry Reports

Thomson14 August 2007

FDA Guidance continued from page 1

U.S. Clinical Investigators Submitting INDsNumber of investigators

Source: CenterWatch analysis, 2005; FDA.

2004E200320022001

27,928 27,97723,865

22,145

the way stakeholders view the drug discovery

and development process. “This was a real

change in culture. One of the things we do is

get the basic scientists at the table with [bio-

pharmaceutical companies] and it really gives

them a sense of engagement in the process.

They’re not threatened by us moving from

largely a basic research organization to one

where we’re very involved in translation. It’s

been the nucleus of a major gifts program.

You tell businessmen about our model, how

we hold ourselves accountable through that

pipeline, and people are willing to invest in

that process,” said Beall at BIO2007.

In the future, these five foundations will

be called upon to share their expertise with

other foundations not yet participating in the

venture philanthropy model.

“The message is getting around to other

foundations, and other patient groups are see-

ing the important role that industry plays.

And they’re looking to learn from their col-

leagues, including us and the other founda-

tions, so they can learn how to engage indus-

try more effectively,” said Lomedico.

While the dollar amount invested in

industry research is quite small, foundations

have found the strategic places to make a

small amount of money work the hardest.

Without it, in most if not all cases, the chance

of developing a new therapy would not exist.

An investment by a foundation is capable of

attracting tens of millions of dollars of ven-

ture capital afterward.

Hesterlee concluded,“Venture philanthro-

py is a small amount of money in the overall

sea of money floating around out there, but I

think because all the groups are really trying to

apply it so strategically, it’s going to make a big

difference—sort of like the lever Archimedes

said could move the world.”

—Sara Gambrill

Venture Philanthropycontinued from page 13

Page 15: August 2007 A Thomson CenterWatch Publication

Industry Reports

15August 2007CenterWatch

version of the guidance document. This

process may take months or even years to

complete. Nevertheless, a draft guidance

document can provide helpful clues about

what FDA officials are thinking on a partic-

ular topic. The discussion in this draft doc-

ument is useful to any PI wishing to under-

stand FDA’s thinking when it addresses the

PI’s supervisory role.

Many of the PI’s responsibilities when

conducting clinical trials of drugs or bio-

logics under the IND regulations are

included in the required investigator’s

signed statement, the Form FDA 1572

(henceforth referred to as “the 1572”), in

which the PI makes several key commit-

ments. Those commitments are:

To conduct the studies in accordance

with the relevant, current protocol(s)

and to only make changes in a protocol

after notifying the sponsor, except when

necessary to protect the safety, rights, or

welfare of subjects,

To personally conduct or supervise the

described investigation(s),

To inform any patients, or any persons

used as controls, that the drugs (biolog-

ics) are being used for investigational

purposes and to ensure that the require-

ments relating to obtaining informed

consent in 21 CFR 50 and institutional

review board (IRB) review and

approval in 21 CFR 56 are met,

To report to the sponsor adverse experi-

ences that occur in the course of the

investigations(s) in accordance with 21

CFR 312.64,

That he/she has read and understands

the information in the investigator’s

brochure, including the potential risks

and side effects of the drug (biologic),

To ensure that all associates, colleagues,

and employees assisting in the conduct

of the studies are informed about their

obligations in meeting the above com-

mitments,

To maintain adequate and accurate

records in accordance with 21 CFR

312.62 and to make those records avail-

able to FDA for inspection in accor-

dance with 21 CFR 312.68,

That an IRB that complies with the

requirements of 21 CFR 56 will be

responsible for the initial and continu-

ing review and approval of the clinical

investigation; to promptly report to the

IRB all changes in the research activity

and all unanticipated problems involv-

ing risks to human subjects or others; to

not make any changes in the research

without IRB approval, except where

necessary to eliminate apparent imme-

diate hazards to human subjects, and

To comply with all other requirements

regarding the obligations of clinical

investigators and all other pertinent

requirements in 21 CFR 312.

The PI should note that his/her signa-

ture under these commitments is followed

by a warning that a willfully false statement

is a criminal offense under section 1001 of

Title 18 of the United States Code (U.S.C.

Title 18, Sec. 1001). In essence, signing the

1572 and then willfully or intentionally not

meeting these commitments is making a

false statement under the law.

A copy of the 1572 is included in the

guidance document as Attachment A.

Interestingly, FDA has attached an expired

version of the document (it expired on

January 31, 2006). The current version

available on FDA’s website expires on May

31, 2009. PIs should check the expiration

date of the form before signing it and ask

the sponsor to provide a valid version if the

form is expired. In addition, the 1572 does

not list all the PI’s requirements. The PI

should refer to the regulations (21 CFR

Parts 50, 56 and 312) to ensure familiarity

with all of FDA’s requirements for the con-

duct of drug and biologics clinical trials.

Clarification of CertainInvestigator ResponsibilitiesThis section of the guidance document is

intended to clarify the PI’s responsibility to

supervise the conduct of the clinical inves-

tigation and to protect the rights, safety and

welfare of study participants in drug and

medical device clinical trials.

Supervising

The PI who conducts clinical investigations

of drugs or biological products under an

IND commits to personally conduct or

supervise the investigation. The PI who

conducts clinical investigations of medical

devices under an IDE commits to supervise

all testing of the device involving human

subjects. It is common practice for the PI to

delegate certain study-related tasks to

see FDA Guidance on page 16

Proportion of New Investigators to New Trials

Source: FDA, June 2005.

1996–20001991–19951986–19901980–1985

1.72

2.633.20

3.87

2001–2004

4.28

Average trials per investigator, 3.30

Page 16: August 2007 A Thomson CenterWatch Publication

employees, colleagues or other third parties

(individuals or entities not under the direct

supervision of the PI). When tasks are del-

egated by the PI, the PI is responsible for

providing adequate supervision of those to

whom tasks are delegated and the PI is

accountable for regulatory violations

resulting from failure to adequately super-

vise the conduct of the clinical study.

It is useful to read FDA’s own words in

these matters. As noted succinctly by FDA

in a 2002 “Notice of Initiation of

Disqualification Proceedings and

Opportunity to Explain” (NIDPOE) letter,

“We remind you that it is your responsibil-

ity, as the…Principal Investigator, to ensure

that the investigation is conducted accord-

ing to the…investigational plan, and appli-

cable regulations….you are responsible for

personally conducting or supervising the

clinical investigation. While in a superviso-

ry role, you may delegate…such delegation

requires careful supervision….”

FDA further wrote “As the [PI], you

remain responsible for overseeing and

reviewing their work…and must make cer-

tain that they are following the…plan.

Regardless of what…you may delegate, you

remain ultimately responsible for the prop-

er conduct of clinical studies in which you

are the investigator of record.”

This inelegant title represents a more

severe enforcement action than an FDA

Warning Letter. The NIDPOE letter is

issued to the PI who has submitted false

information or committed fraud or mis-

conduct, or who has repeatedly or deliber-

ately failed to comply with FDA regula-

tions. While the letter may be five years old,

FDA’s words seldom have an expiration

date. FDA’s overall expectations then and

now vis-à-vis the PI’s supervision of clini-

cal trial activities are quite clear in these

words.

In assessing the adequacy of supervi-

sion by a PI, FDA focuses on four major

issues: 1) whether delegated individuals

were qualified to perform such tasks, 2)

whether study staff received adequate

training on how to conduct the delegated

tasks and were provided with an adequate

understanding of the study, 3) whether

there was adequate supervision and

involvement in the ongoing conduct of the

study, and 4) whether there was adequate

supervision or oversight of any third par-

ties involved in the conduct of a study to

the extent such supervision or oversight

was reasonably possible. FDA makes the

following recommendations on these top-

ics.

1. What is appropriate delegation of

study-related tasks?

According to FDA, the PI should ensure

that any individual to whom a task is dele-

gated is qualified by education, training,

and experience to perform that delegated

task. Appropriate delegation is primarily an

issue for tasks that would be considered to

be clinical or medical in nature, such as

evaluating study subjects to assess clinical

response to an investigational therapy (e.g.,

global assessment scales, vital signs) or pro-

viding part of the medical care provided to

subjects during the course of the study.

Most clinical/medical tasks require formal

medical training and also may have licens-

ing or certification requirements. Such

licensing requirements will vary from state

to state. The PI should take such qualifica-

tions/licensing requirements into account

when considering to whom it would be

appropriate to delegate specific tasks.

During inspections, FDA has identified

instances in which study tasks have been

delegated to individuals lacking appropri-

ate qualifications. Examples of inappropri-

ate delegation include:

Screening evaluations, including obtain-

ing medical histories and assessment of

inclusion/exclusion criteria, conducted

by individuals with inadequate medical

training (e.g., a medical assistant),

Physical examinations performed by

unqualified personnel,

Evaluation of adverse events by individ-

uals lacking appropriate medical train-

ing, knowledge of the clinical protocol,

and knowledge of the investigational

product,

Industry Reports

Thomson16 August 2007

FDA Guidance continued from page 15

More Investigators, Lots More Studies

Source: FDA, June 2005.

20,000

40,000New studies

New investigators

2001-20041996-20001991-19951986-19901981-1985

6,07910,701

16,435

25,791

36,839

8,6166,6695,1414,0673,540

10,000

30,000

First Time InvestigatorsAverage number of first time investigators

Source: FDA, June 2005.

1996–20001991–19951986–19901980–1985

3,5404,067

5,1416,669

2001–2004

8,616

Page 17: August 2007 A Thomson CenterWatch Publication

Industry Reports

17August 2007CenterWatch

Assessments of primary study end-

points (e.g., tumor response, global

assessment scales) by individuals lack-

ing appropriate medical training and

knowledge of the protocol, and

Informed consent obtained by individ-

uals who lack the medical training,

knowledge of the clinical protocol, or

familiarity of the investigational prod-

uct needed to be able to discuss the risks

and benefits of a clinical trial with

prospective subjects.

According to FDA regulations, the PI is

responsible for conducting studies in

accordance with the protocol. Some proto-

cols specify the qualifications of the indi-

viduals who are to perform certain proto-

col-required tasks, and these protocols

must be followed even if state law permits

differently qualified people to perform the

task. For example, even if the state in which

the study site is located permits nurse prac-

titioners to perform physical examinations

under the supervision of a physician, if the

protocol specifies that physical examina-

tions must be done by a physician, a physi-

cian must perform such exams. There have

been similar situations where the protocol

only permits the PI to conduct the

informed consent process and to sign the

consent form. This unnecessary restriction

often has resulted in adverse audit findings

when the PI is personally unable to sign the

form. The imposition of this restriction

should be carefully thought through in

light of real-world events that occur during

a clinical trial, and which may result in

noncompliance.

PIs should also be cautioned that decid-

ing who is qualified to perform a particular

protocol-related task should not be

reduced to “completing a checklist” or con-

ducting a superficial review of a curricu-

lum vitae. People without formal medical

degrees may ably perform various med-

ical/clinical tasks, provided they have had

the proper training and experience. On the

other hand, a medical background is no

guarantee that study staff will not commit

serious errors in study-related tasks of a

medical/clinical nature. The determination

of who is qualified by education, training,

and experience to perform a study-related

task should be made thoughtfully and the

criteria that are used to make the determi-

nation should be more robust than simply

the letters after a person’s name.

FDA notes in the guidance document

that the PI should maintain a list of the

appropriately qualified persons to whom

significant trial-related duties have been del-

egated. This list should describe the delegat-

ed tasks, identify the training that individu-

als have received that qualifies them to per-

form delegated tasks, and identify the dates

of involvement in the study. A PI should

maintain separate lists for each study con-

ducted by the PI. Most sponsors create study

delegation logs (often combined with a staff

signature log) but these logs may not have

space for adding each staff person’s training.

Rather, it makes more sense to also provide

each PI with a staff training log where study-

specific training can be documented.

Combined or separated, written documen-

tation of delegated tasks and staff training is

very important.

2. What is adequate training?

The PI should ensure that there is adequate

training for all staff participating in the

conduct of the study. The PI should antici-

pate the possibility of staff turnover during

the conduct of the study (particularly if the

study is of long duration) and plan to

ensure that there is adequate training of

any replacement staff. The PI should ensure

that staff:

Have a general familiarity with the

study and the protocol,

Have a specific understanding of the

details of the protocol and the investi-

gational product, relevant to the tasks

they will be performing,

Are aware of regulatory requirements

and acceptable standards for the con-

duct of clinical trials, both in respect to

conduct of the clinical trial and human

subject protection,

Are competent to perform the tasks that

they are delegated, and

Are informed of any pertinent changes

during the conduct of the trial and edu-

cated or given additional training as

appropriate.

If the sponsor provides training materi-

als for the PI on the conduct of the study,

the PI should ensure that staff members

see FDA Guidance on page 18

New Trials GrowingAverage new trials per year

Source: FDA, June 2005.

1996–20001991–19951986–19901980–1985

6,07910,701

16,435

25,791

2001–2004

36,839

Page 18: August 2007 A Thomson CenterWatch Publication

Industry Reports

Thomson18 August 2007

receive the sponsor’s training or informa-

tion from the training that is pertinent to

their roles in the study.

3. What is adequate supervision of the

conduct of an ongoing clinical trial?

The PI should have a detailed plan for the

supervision and oversight of a clinical trial.

Supervision and oversight should be pro-

vided even for individuals who are highly

qualified and experienced. A plan might

include the following elements, to the

extent they apply to a particular trial:

Routine meetings with staff to review

trial progress and update staff on any

changes to the protocol or other proce-

dures,

Routine meetings with the sponsor’s

monitors,

A procedure for correcting problems

identified by study personnel, outside

monitors or auditors, or other parties

involved in the conduct of a study,

A procedure for documenting the per-

formance of delegated tasks in a satis-

factory manner and, where appropriate,

verifying findings (e.g., observation of

the performance of selected assess-

ments or independent verification by

repeating selected assessments),

A procedure for ensuring that the con-

sent process is being conducted in

accordance with 21 CFR 50 and that

study subjects understand the nature of

their participation, risks, etc.,

A procedure for ensuring that informa-

tion in source documents is accurately

captured on the Case Report Forms,

A procedure for dealing with data

queries and discrepancies identified by

the study monitor, and

Procedures for ensuring study staff

comply with the protocol, adverse event

assessment and reporting, and other

medical issues that arise during the

course of the study.

The PI should have sufficient time to

properly conduct and supervise the clinical

trial. The intensity of the supervision

should be appropriate to the staff, the

nature of the trial, and the subject popula-

tion. In FDA’s experience, the following fac-

tors may compromise the ability of an indi-

vidual PI to provide adequate supervision

of the conduct of an ongoing clinical trial:

Inexperienced study staff,

Overburdened study staff,

Complex clinical trials (e.g., many

observations, large amounts of data col-

lected),

Large number of subjects enrolled at a

site,

A patient population that is quite sick,

Conducting a large number of studies

concurrently,

Conducting a study from a remote (i.e.,

off-site) location, and

under the oversight of a single PI, par-

ticularly where those sites are not near

each other (e.g., sites that are geograph-

ically distant, in another city, county,

state, or country).

FDA indicates in this guidance docu-

ment that the agency prefers for any site

with substantial enrollment to have an

identified PI with clear responsibilities, but

if that is not arranged, FDA believes there

should ordinarily be an individual respon-

sible for the conduct of the clinical trial at

each trial site, identified as a sub-investiga-

tor. Sub-investigators should report direct-

ly to the PI (i.e., the PI should have clear

responsibility for evaluating the sub-inves-

tigator’s performance and should have the

authority to hire/fire the sub-investigator).

4. What are an investigator’s responsi-

bilities for oversight of other parties

involved in the conduct of a clinical trial?

Study staff not in the direct employ of the

PI—The staff involved directly in the

conduct of a clinical investigation may

include individuals who are not in the

direct employ of the PI. For example, a

site management organization (SMO)

may hire a PI to conduct a study and

provide the PI with a study coordinator

or nursing staff employed by the SMO.

In this situation, the PI should take

steps to ensure that the staff not under

his/her direct employ are qualified to

perform delegated tasks (as discussed

above) and that the staff have received

adequate training on carrying out the

delegated tasks and on the nature of the

study, or the PI should provide such

training.

The PI is responsible for supervision

of the study tasks performed by this

staff, even though they are not in

his/her direct employ during the con-

duct of the study and this responsibility

exists no matter how qualified and

experienced these staff members are. In

the event that the staff ’s performance of

study-related tasks is not adequate and

cannot be made satisfactory by the PI,

the PI should document the observed

deficiencies in writing to the staff ’s

supervisor(s). Depending on the severi-

ty of the deficiencies, the clinical trial

may need to be voluntarily suspended

until personnel can be replaced.

Parties other than study staff—There are

often critical aspects of a study per-

formed by parties not involved directly

in patient care or contact, and not

FDA Guidancecontinued from page 17

Page 19: August 2007 A Thomson CenterWatch Publication

Industry Reports

19August 2007CenterWatch

under the direct control of the PI. For

example, clinical chemistry testing,

radiologic assessments and electrocar-

diograms are commonly done by a cen-

tral independent laboratory retained by

the sponsor or the PI. Under these

arrangements, the laboratory or diag-

nostic procedure provider usually pro-

vides the test results directly to the

sponsor and to the PI. Because the

activities of these parties are critical to

the outcome of the study, and because

the sponsor retains the services of the

laboratory, the sponsor is responsible

for seeing that these parties are fulfilling

their responsibilities for the study.

Less frequently, a study may require

that the PI arranges to obtain informa-

tion critical to the study that cannot be

obtained at the PI’s facility. For example,

if the study protocol requires testing

with special equipment or expertise not

available at the PI’s facility, the PI might

make arrangements for someone out-

side the facility to perform the test. In

this case, the results are provided direct-

ly to the PI, who then submits the infor-

mation to the sponsor. Where such

assessments are retained by the PI, the

PI should take steps to ensure that the

facility is adequate (e.g., has the required

certifications or licenses). The PI may

also institute procedures to ensure the

integrity of data and records obtained

from the party providing the informa-

tion (e.g., a process to ensure that

records identified as coming from the

party are authentic). Procedures are par-

ticularly important when assessments

are crucial to the evaluation of the effi-

cacy or safety of an intervention or to

the decision to exclude subjects who

would be exposed to unreasonable risk.

The PI should carefully review the

reports from these external sources for

results that are inconsistent with clinical

presentation. To the extent feasible, and

considering the specifics of study

design, the PI should evaluate whether

results appear reasonable, individually

and in aggregate. If the PI detects possi-

ble errors or suspects that results from a

central laboratory might be question-

able, the PI should contact the sponsor

immediately.

Exception for certain device studies—In

some cases, specialized expertise from a

device sponsor is needed to perform

certain tasks. For example, when there

is no one at the clinical site who can

program an investigational pacemaker,

the expertise may be provided by the

sponsor’s personnel, such as a field clin-

ical engineer. The field clinical engineer

should be supervised by the sponsor

and not by the PI. When a field clinical

engineer is designated by the sponsor to

perform a specific activity within the

investigational plan, this activity should

be described in the protocol. The PI

retains responsibility for ensuring that

the protocol is followed.

Protecting study subjects

The PI is responsible for protecting the

rights, safety, and welfare of subjects under

his/her care during a clinical trial. The PI

should provide a reasonable standard of

medical care for study subjects for medical

problems arising during participation in

the trial that are, or could be related, to the

study intervention. The PI should be readi-

ly available to provide such care during the

study or should ensure that other identi-

fied, qualified individual(s) are available to

provide such care. Failure to adhere to the

protocol can expose subjects to unreason-

able risks.

1. Reasonable medical care necessitated

by participation in a clinical trial

During and following a subject’s participa-

tion in a trial, the PI should ensure that

adequate medical care is provided to a sub-

ject for any adverse events, including clini-

cally significant laboratory values, related

to the trial. The PI should inform a subject

when medical care is needed for an inter-

current illness. The PI should inform the

subject’s primary physician about the sub-

ject’s participation in the trial if the subject

has a primary physician and if the subject

agrees to the primary physician being

informed.

If the PI does not possess the necessary

skills to provide adequate medical care for

the subject, the PI should make every effort

to obtain appropriate care. For example, if

a carotid stent is placed in a subject by an

see FDA Guidance on page 20

Profile of Investigators Filing FDA 1572Percent of respondents

Source: FDA, 2005.

MD

MB, CHB, PharmD,PhD, Other

DO

91%

3%6%

Page 20: August 2007 A Thomson CenterWatch Publication

interventional neuroradiologist and the

subject suffers a cerebral stroke, the neuro-

radiologist should assess the clinical status

of the subject and transfer the subject to a

neurology service. Subjects should receive

appropriate medical evaluation and treat-

ment until resolution of any condition

related to the study intervention that devel-

ops during the course of their participation

in a study, even if the follow-up period

extends beyond the end of the study at the

investigative site.

2. Reasonable access to medical care

To protect subjects from unnecessary risks,

the PI should be available to subjects dur-

ing the conduct of the trial at his/her site.

Availability is particularly important where

subjects are receiving a drug that has signif-

icant toxicity or abuse potential. For exam-

ple, if a study drug has potentially fatal tox-

icity, the PI should be readily available by

phone or other electronic communication,

and in reasonably close proximity to study

subjects (e.g., not in another state or on

prolonged travel). Study subjects should be

clearly educated on the possible need for

such contact and on precisely how to

obtain it, generally by providing pertinent

phone numbers, web sites, etc., in writing.

Prior to undertaking the conduct of a

study, a prospective PI should consider

whether he/she can be available to the

extent needed given the nature of the trial.

If the PI is not going to be available for

some period during the study, clinical

responsibility for study subjects should be

delegated to a specific qualified physician

who will be readily available to subjects.

This delegation should be documented in a

1572 or investigator agreement (the physi-

cian should be listed as a sub-investigator)

and also submitted to the IRB for review

(as a change in the research activity requir-

ing IRB review). If the PI is a non-physi-

cian, the PI should make adequate provi-

sion for any necessary medical care that the

PI is not qualified to provide.

3. Protocol violations that present

unreasonable risks

There are occasions when a failure to

adhere to the protocol may be considered a

failure to protect the rights, safety, and wel-

fare of subjects. For example, failure to

adhere to inclusion/exclusion criteria that

are specifically intended to exclude subjects

for whom the study drug or device poses

unreasonable risks (e.g., enrolling a subject

with decreased renal function in a trial in

which decreased function is exclusionary

because the drug may be nephrotoxic) may

be considered failure to protect the rights,

safety, and welfare of the enrolled subject.

Similarly, failure to perform safety assess-

ments intended to detect drug toxicity

within protocol-specified time frames (e.g.,

CBC for an oncology therapy that causes

neutropenia) may be considered failure to

protect the rights, safety, and welfare of the

enrolled subject. The PI should seek to

minimize such risks by adhering closely to

the study protocol.

There are many PIs who are lacking in

their understanding of the scope of FDA’s

regulatory requirement that the PI careful-

ly supervise any clinical trial under his/her

responsibility. While sponsors routinely

provide study training (including general

Good Clinical Practice topics) to the PI and

staff before the start of a clinical trial, that

training often is inadequate for the inexpe-

rienced PI or staff member. FDA has made

a substantial contribution to the training of

study staff by issuing this draft guidance

document.

—Anna J. DeMarinis, MA, MT, CQA

Industry Reports

Thomson20 August 2007

FDA Guidancecontinued from page 19 C

utaneous melanoma is a malignan-

cy of skin cells known as

melanocytes that contain pigment

and color the skin. Less frequently, melanoma

may also develop within the eye (intraocular

or ocular melanoma). According to the

American Cancer Society, there were 62,000

cases of melanoma in the United States in

2006, which caused 8,000 deaths.

Adults over the age of 20 are more often

affected by melanoma than are children and

adolescents. Risk factors include unusual

moles (nevi), exposure to natural sunlight or

to artificial ultraviolet light, family or person-

al history of melanoma, white or pale skin,

red or blond hair, freckles and blue eyes.

Warning signs for melanoma include spe-

cific changes in the appearance of a mole or

pigmented skin region. Diagnosis can be

confirmed by biopsy, and further evaluation

should confirm the stage and extent of

metastasis. Prognosis and treatment are

affected by the location, size and stage of

melanoma.

Although melanoma accounts for about

5% of all skin cancers, it is responsible for

approximately 75% of all skin cancer-related

deaths. If melanoma is removed when it is

confined to the outermost skin layer, it is

potentially curable, but deeper lesions or

metastatic disease have a poor prognosis and

expected survival of only six to nine months.

Five-year survival in Stage IV ranges from

only 6.7% to 18.8%, depending on the site of

metastasis.

Existing treatment options for melanoma

include surgery for local or wide local exci-

sion with or without lymph node removal,

chemotherapy, radiotherapy and biologic

therapy. However, there has been little

improvement in outcomes during the past 30

years. In advanced stage melanoma, response

rates to available treatments are no greater

than 20%, and most of the available drugs are

highly toxic.

Page 21: August 2007 A Thomson CenterWatch Publication

Grant Opportunities

21August 2007CenterWatch

CenterWatch has identified a pipeline of

17 drugs in various stages of development for

melanoma treatment, many of which are bio-

logical agents or vaccines.

Antigenics is in phase III development of

Oncophage (vitespen; formerly HSPPC-96),

a personalized vaccine based on antigens

extracted from each specific patient’s tumor

combined with endoplasmin, or heat shock

protein. Because the vaccine targets only

tumor cells, it should theoretically have

improved efficacy with reduced adverse

effects. The U.S. Food and Drug

Administration (FDA) has granted

Oncophage fast track and orphan drug desig-

nations for metastatic melanoma as well as

for renal cancer.

Once Oncophage is prepared from the

patient’s tumor, it is usually administered on

an outpatient basis beginning within four to

eight weeks after surgery, with one injection

once weekly for four weeks, then one injec-

tion every other week. In a phase III trial in

metastatic melanoma, median survival with

at least 10 injections of Oncophage was 29%

greater than with the physician’s choice of

regimen.

To date, more than 12 clinical trials of

Oncophage have enrolled more than 750 can-

cer patients, many of whom have had

advanced stage cancer refractory to tradition-

al cancer treatments. Adverse effects may

include injection-site reactions, generalized

and joint pain, constipation, fatigue, fever,

diarrhea, edema, loss of appetite and weight,

nasopharyngitis, arthralgia, dizziness, anxi-

ety, depression, cough, breathing problems,

headache, vomiting, anemia, sleep distur-

bances and weakness.

Another autologous cell vaccine is M-

Vax, in phase III testing by Avax and already

approved in Switzerland for the treatment of

stage 3 and 4 melanoma. This vaccine is made

by adding the hapten dinitrophenyl (DNP) to

the patient’s melanoma cells, which are then

injected within the dermis in combination

with the immunological adjuvant bacilli

Calmette-Guerin. Most patients experience a

delayed-type hypersensitivity (DTH) to the

DNP-modified melanoma cells, and approxi-

mately half also develop the same immune

response to unmodified tumor cells.

In a trial of 214 patients with stage 3

melanoma, M-Vax therapy after lymph node

removal was associated with 44% five-year

survival, compared with 20% to 25% for sur-

gery alone. Survival was directly related to

induction of DTH response to unmodified

autologous melanoma cells. Another study

showed antitumor responses in 11 of 83

patients with stage 4 melanoma and measur-

able metastases treated with M-Vax. The

most frequent side effects include mild nau-

sea, vomiting and injection-site reactions.

IDM Pharma is in phase II testing of

Uvidem, a vaccine made from the patient’s

own dendritophages, or specialized immune

cells derived from the patient’s own white

blood cells, that are exposed in the laboratory

to specific tumor antigens derived from the

patient’s cancer cell extracts.

Oxford Biomedica’s candidate for a

melanoma vaccine is Hi8, now in phase II

development. This vaccine is composed of a

DNA plasmid (DNA.Mel3) and a non-repli-

cating Modified Vaccinia Ankara (MVA) viral

vector (MVA.Mel3) containing 7 human

HLA-A2 or HLA-A1-restricted CTL epitopes

which are designed to stimulate immune sys-

tem recognition and destruction of cancer

cells by CD8+ T-cells.

In early trials, Hi-8 MEL was well tolerat-

ed and produced immunological melanoma-

specific CD8+ T-cell responses. Nearly 20%

of patients had tumor responses; median sur-

vival was 100 weeks for immune responders

versus 37 weeks for non-responders

(p<0.001) and 42 weeks for a group of con-

trol non-HLA A2 patients who received stan-

dard of care.

Genasense, in phase III testing by Genta,

is an antisense oligonucleotide targeting Bcl-

2, a protein over-expressed in many types of

cancer. An ongoing randomized, open-label,

cross-over pharmacokinetic study is evaluat-

ing combination therapy with Genasense and

dacarbazine (DTIC) in patients with hepatic

impairment and advanced malignant

melanoma.

Medarex is in phase III development of

ipilimumab (MDX-010), a fully human anti-

body targeting CTLA-4, a molecule on T cells

that suppresses the immune response. An

open label, single arm trial of ipilimumab

monotherapy is underway in patients with

previously treated Stage 3 or Stage 4 metasta-

tic melanoma unresponsive to at least one

other regimen of another melanoma therapy.

A phase III pivotal trial is also ongoing in

combination with the melanoma peptide

vaccine MDX-1379.

Allovectin-7, a plasma/lipid complex con-

taining the human DNA gene sequences that

encode HLA-B7 antigen and Beta2

microglobulin, is in phase III testing by Vical.

Together, these two antigens form a Class I

Major Histocompatibility Complex (MHC-

I) antigen. Injecting Allovectin-7 directly into

melanoma lesions may boost the patient’s

immune response to both local and metasta-

tic tumors.

Synta is in phase IIb trials with STA-4783,

an inducer of heat shock protein 70 (Hsp70)

on the surface of tumor cells that attracts nat-

ural killer (NK) immune cells and activates

NK-mediated tumor cell death. This novel,

injectable, small molecule induces an oxida-

tive stress response in cells, which is charac-

terized by increased production of gene fam-

ilies that protect against different cellular

stresses such as intense heat, oxygen radicals

or heavy metal exposure.

In a double-blind, randomized phase IIb

trial at 21 U.S. sites in the U.S., 81 patients

Eye On: Melanoma

see Melanoma on page 22

Page 22: August 2007 A Thomson CenterWatch Publication

Grant Opportunities

Thomson22 August 2007

In the Pipeline: Melanoma

Drug Company Contact Additional Information

Phase I

Exherin (ADH-1) Adherex (919) 484-8484 An angiolytic, it binds to N-cadherin, a cell-surface www.adherex.com adhesion molecule that encourages blood vessel cells

to bind to each other. Selectively causes tumor blood vessels to rupture and slows or stops the flow ofblood necessary for cancer cells to grow. Is being investigated in combination with limb infusion melphalan

plasmid DNA Inovio (858) 597-6006 encodes a tumor antigen for delivery using Inovio’s vaccine www.innovio.com MedPulser electroporation technology

Provecta Provectus (865) 769-4011 a concentrated drug formulation of rose Bengal,www.pvct.com a radiochemosensitizer

were randomized to treatment with STA-

4783 in combination with paclitaxel or pacli-

taxel alone. Combination therapy was associ-

ated with a 50% reduced risk of disease pro-

gression and a three times higher objective

response rate. STA-4783 was well tolerated,

with adverse events for combination therapy

typical for those expected with paclitaxel

alone (fatigue, hair loss, constipation, nausea,

numbness, arthralgia, insomnia, diarrhea and

anemia).

Another therapeutic that alters the func-

tion of a heat shock protein is tanespimycin,

in phase II testing by Kosan. This ansamycin

antibiotic binds to and inhibits Hsp90, pre-

venting the proper folding and stability of

many proteins involved in cancer and leading

to cancer cell death. In clinical studies, Hsp90

inhibitors appear to re-sensitize cancer cells

to drugs to which they have developed resist-

ance, and they may also enhance the initial

activity of existing cancer therapies.

OncoVEX, which is intended for use in

solid tumors, is in phase II development by

BioVex. This modified oncolytic version of the

herpes simplex virus type-1 (HSV-1) incor-

porates Biovex’s OncoVEX platform with the

granulocyte-macrophage colony stimulating

factor (GM-CSF) gene. After reproducing

within cells, the HSV-1 virus lyses or ruptures

the cell membrane, killing the cell. Deleting

the gene encoding the ICP34.5 protein from

the HSV-1 DNA allows HSV to replicate in

tumor cells but not in normal cells. Adding

the GM-CSF gene enhances stimulation of

anti-tumor response by immune system cells.

In early trials, OncoVEXGM-CSF was well

tolerated with few serious side effects.

SciClone and Sigma-Tau are developing

Zadaxin (thymalfasin), a 28-amino acid syn-

thetic peptide resembling a natural peptide

isolated from the thymus gland. In a phase II

trial of combination therapy with Zadaxin

plus chemotherapy in 488 patients with stage

IV malignant melanoma, thymalfasin plus

DTIC without interferon alpha was associat-

ed with triple the overall response rate and

three-month improvement in overall survival

compared with DTIC plus interferon alpha

treatment.

ZymoGenetics and Novo Nordisk are in

phase IIa testing of IL-21, an injectable

recombinant human Interleukin 21 cytokine

with regulatory effects on cytotoxic T cells

(CTL) and Natural Killer (NK) cells. In ani-

mal models of cancer, IL-21 increases NK cell

activity and disease-specific CTLs and

reduces the number of tumors.

Amplimexon, an injectable formulation

of the cyanoaziridine compound imexon, is

in phase I/II development by AmpliMed. By

targeting an enzyme involved in DNA syn-

thesis, this drug inhibits cancer cell division;

other mechanisms of action include disrup-

tion of mitochondria and increased uptake of

other chemotherapies into cancer cell DNA.

The dismal prognosis and adverse events

associated with conventional and currently

available treatments for melanoma mandates

an innovative approach. Time will tell

whether the target biological agents in the

pipeline will ultimately be more effective and

less toxic than standard treatments.

—Laurie Barclay, M.D.

Melanomacontinued from page 21

Page 23: August 2007 A Thomson CenterWatch Publication

Grant Opportunities

23August 2007CenterWatch

Drug Company Contact Additional Information

Phase I/II

Amplimexon AmpliMed (520) 529-1000 an injectable formulation of imexon, which inhibits www.amplimed.com cancer cell division and targets an enzyme involved in

DNA synthesis; being evaluated in combination with dacarbazine

BNP 1350 BioNumerik (210) 614-1701 a silicon-containing agent from a novel class ofwww.bionumerik.com lipophilic camptothecins

Phase IIa

IL-21 ZymoGenetics/ (206) 442-6600 an injectable recombinant human IL-21; has Novo Nordisk www.zymogenetics.com regulatory effects on cytotoxic T cells (CTL) and

Natural Killer (NK) cells

Phase II

OncoVEX BioVex (617) 444-8445 a modified oncolytic version of the herpes simplex www.biovex.com virus type-1 (HSV-1) that incorporates Biovex’s

OncoVEX platform with the granulocyte-macrophage colony stimulating factor (GM-CSF) gene

Uvidem IDM Pharma (949) 470-4751 a vaccine composed of autologous dendritophages www.idm-biotech.com loaded in vitro with specific tumor antigens derived

from the patients’ cancer cell extracts

tanespimycin Kosan (510) 732-8400 an ansamycin antibiotic which binds to Hsp90 www.kosan.com (Heat Shock Protein 90) and alters its function

Hi8 melanoma Oxford Biomedica (858) 677-6500 a vaccine based on Oxford’s the Hi8 PrimeBoostvaccine www.oxfordbiomedica.com technology platform. It utilizes a DNA plasmid

(DNA.Mel3) and a MVA viral vector (MVA.Mel3)containing seven human HLA-A2 or HLA-A1-restricted CTL epitopes; designed to enhance the body’s own ability to recognize and destroy cancerous cells

Zadaxin SciClone/Sigma-Tau (650) 358-3456 a 28-amino acid peptide originally isolated from the www.sciclone.com thymus gland and now produced synthetically

Phase IIb

STA-4783 Synta (781) 274-8200 an inducer of heat shock protein 70 (Hsp70) on the www.syntapharma.com surface of tumor cells; attracts natural killer (NK)

immune cells and activates NK-mediated tumor cell death

Phase III

Oncophage Antigenics (212) 994-8200 a personalized vaccine based on tumor antigens www.antigenics.com extracted from the patient’s tumor mixed with

endoplasmin (heat shock protein)

M-Vax Avax (215) 241-9760 an autologous cell hapten-modified vaccinewww.avax-tech.com

Page 24: August 2007 A Thomson CenterWatch Publication

Grant Opportunities

Thomson24 August 2007

Multiple TherapeuticAreasStill Seeking Investigators

Kendle International1200 Carew Tower, 441 Vine StreetCincinnati, OH 45202

Contact: Karen KempfEmail: [email protected]

Drug name: Not applicableIndication: AnySpecialty: Some key specialities being sought

are: Rheumatologists, Gastro-enterologists, Oncologists,Hematologists, Pediatricians,Allergists, Acute Neurologists,Internal Medicine, OB/GYN,Women’s Health

Phase: I-IVNotes: Join the Kendle investigator team

as we study breakthrough thera-peutic agents. Our teams ofInvestigator professionals play acritical role in the advancement ofmedical science across the world.Please send an email to [email protected] to be contactedregarding joining our Investigatorteam.

Welch Allyn, Inc.4341 State Street Rd., Box 220Skaneateles, NY 13153-0220

Contact: Kathi DurdonEmail: [email protected]

Device name: Not availableIndication: Cardiac, pulmonary, neonatology,

pediatrics, emergency medicine,internal medicine, family medi-cine, acute care, critical care

Specialty: Device experienced researchersPhase: Class I & II DeviceNotes: Seeking device-experienced/inter-

ested clinical researchers forupcoming studies. Class I & II inneonatal, pediatric and adultpopulations; AMC, hospital andprivate practice settings.

TrialWatch is designed to help

sponsors and CROs identify a

pool of investigators for their

upcoming trials. Each sponsor that is listed

here has confirmed that it will be actively

selecting sites during the next few weeks,

and would like to receive inquiries from

investigative sites. Sponsors and CROs

that would like to use this service should

contact Tamar Skowronski at (617) 856-5974

or email [email protected].

Visit our web site at www.centerwatch.com/

professional/trialwatch.html to use Trial-

Watch online.

For investigators, this listing provides pre-

qualified leads for clinical grants. Please note:

Unless a phone or fax number is given, do

not call the sponsor or CRO. Sponsors have

provided this information to CenterWatch

with the understanding that investigative sites

will mail cover letters, CVs and other infor-

mation about their facilities, staff and

patients. Please inform the sponsor or CRO

that you learned of the project through

CenterWatch.

Investigators—Have you called a sponsor in

this issue of TrialWatch and learned that the

sponsor is no longer seeking sites? If so, please

call (617) 856-5974 to let us know so that we

may contact the sponsor and update our list-

ings accordingly.

TrialWatch

Note: If you would like further information on any drug listed above, or to review our comprehensive database of drugs in development, please visit www.centerwatch.com.

Drug Company Contact Additional Information

Phase III (continued)

Genasense Genta (908) 286-9800 targets Bcl-2, a protein over-expressed in many www.genta.com forms of cancer.

Ipilimumab Medarex (609) 430-2880 a fully human antibody that targets CTLA-4,(MDX-010) www.medarex.com a molecule on T cells that suppresses the immune

response

Allovectin-7 Vical (858) 646-1100 a DNA/lipid complex containing the human www.vical.com gene that encodes HLA-B7 antigen

Page 25: August 2007 A Thomson CenterWatch Publication

Grant Opportunities

25August 2007CenterWatch

Cardiology/Vascular DiseaseStill Seeking Investigators

Duke Clinical Research Institute2400 Pratt St.Durham, NC 27705

Contact: Kifu FaruqEmail: [email protected]

Drug name: Oral direct Factor Xa inhibitorIndication: Non-valvular atrial fibrillation in

patients at high risk for eventsSpecialty: Cardiology, Neurology, Internal

Medicine, Hematology(Anticoagulation Clinic)

Phase: IIINotes: Primary objective is to demon-

strate that the efficacy of a oncedaily direct factor Xa inhibitor, isnon-inferior to dose-adjusted war-farin for the prevention of throm-boemoblic events in pts with non-valvular atrial fibrillation meas-ured by the composite of strokeand non-CNS systemic embolism.

Nicholas Piramal India Ltd.

Contact: Anjali SonawaneEmail: [email protected];

[email protected]

Drug name: Niyat (Atrovastatin+Niacin)Indication: HypercholesterolemiaSpecialty: CardiologyPhase: IVComments: Seeking Indian investigators

ProMedDx, LLC10 Commerce WayNorton, MA 02766

Contact: Marion M. Santa InesEmail: [email protected]

Drug name: Not applicableIndication: Congestive Heart FailureSpecialty: CardiologyPhase: Not applicableNotes: Only one study visit will be

required for this study. The onlyrisks to participants are those asso-ciated with a routine blood draw.

Tasly Pharmaceuticals, Inc.One Research Court, Suite 450-54Rockville, MD 20850

Contact: Jason GuoEmail: [email protected]

Drug name: T89Indication: Prevention of occurrence of stable

anginaSpecialty: PI specializing in cardiovascular

disease (angina), in a hospital, uni-versity, or medical research centers

Phase: II/IIbNotes: 2-herb drug for chronic stable

angina. Open, 500mg, baseline-controlled. 40 patients. Patientstake T89, stop long term nitrate,reduce current anti-angina to 50%on day 0, and to 0% on day 28 ifjustified, free to use on-demandnitrate as needed. Exercise tread-mill testing on days -14, 0, 28 & 56.

DermatologyStill Seeking Investigators

Light BioScience933 First Colonial Rd. Suite 204Virginia Beach, VA, 23454

Contact: Susan Kaplan RN, PhDEmail: [email protected]

Device name: GentleWavesIndication: Acne treatmentSpecialty: DermatologyPhase: II/ IIINotes: This is a 6-8 week trial of a new

treatment for facial acne inpatients ages 14-60 using LEDtechnology.

Emergency MedicineStill Seeking Investigators

Intranasal Therapeutics, Inc.1513 Bull Lea BlvdLexington, KY 40511

Contact: Kim NewEmail: [email protected]

Drug name: Not availableIndication: Acute PainSpecialty: ER Physician with research experi-

ence treating acute pain.Phase: II

EndocrinologyNew Leads

Anaclim6325 Digital Way, Suite 401Indianapolis, IN 46278

Contact: Randy BrownEmail: [email protected]

Drug name: Not availableIndication: Type II DiabetesSpecialty: EndocrinologyPhase: IVNotes: Insulin study for patients who are

currently receiving oral meds forthe treatment of Type II Diabetes.

Still Seeking Investigators

AlthoTech Pharmaceutical Inc.310 Glen Manor Dr. W.Toronto, Ontario, Canada M4E 2X7

Contact: Peter TomlinsonEmail: [email protected]

Drug name: ATPI-C01Indication: Treatment of ischemic and neuro-

pathic foot ulcersSpecialty: Clinical diabetologist; clinical der-

matologistPhase: INotes: ATPI-C01 is a patented intrale-

sional formualation of EGF fortreating diabetic foot ulcers.Treatment effective in end-stageDFU sized from 1-80 cm2, wherethe amputation is the only avail-able choice.

Genaera Corporation5110 Campus DrivePlymouth Meeting, PA 19462

Contact: Jim EllisEmail: [email protected]

Drug name: Not availableIndication: Diabetes Type IISpecialty: Phase I unit Obese Diabetes Type

IIPhase: INotes: We are looking for a Phase I unit

that can recruit 28 Obese Type IIDiabetes patients (7 subjects in 4dose groups), for a single IV dosestudy, patients will be housed for72 hours.

ProMedDx, LLC10 Commerce WayNorton, MA 02766

Contact: Marion M. Santa InesEmail: [email protected]

Drug name: Not applicableIndication: DiabetesSpecialty: EndocrinologyPhase: Not applicableNotes: Only one study visit will be

required for this study. The onlyrisks to participants are those asso-ciated with a routine blood draw.

Page 26: August 2007 A Thomson CenterWatch Publication

Grant Opportunities

Thomson26 August 2007

GastroenterologyStill Seeking Investigators

Advanced Biologics580 Union Square DriveNew Hope, PA 18939

Contact: Wen LinEmail: [email protected]

Drug name: Not availableIndication: C. Difficile-associated diarrheaSpecialty: Infectious disease,

GastroenterologyPhase: IIINotes: Looking for sites in US and

Canada.

Alba Therapeutics800 West Baltimore St., Suite 800Baltimore, MD 21201

Contact: Sarah CappelloEmail: [email protected]

Drug name: AT-1001Indication: Celiac DiseaseSpecialty: Gastroenterology; Immunology-

with access to adult celiac diseasepatients. Experienced in conduct-ing clinical trials.

Phase: IIbNotes: Alba is recruiting investigators for

the following clinical trial: Subjects18-65, diagnosed with celiac dis-ease by small bowel biopsy, glutenfree for up to 6 mons and anti-tTGneg. Will evaluate the efficacy ofAT-1001 vs. drug placebo/gluten,in preventing exacerbation of celi-ac symptoms and intestinal per-meability when exposed to gluten.

Duke Clinical Research InstitutePO Box 17969Durham, NC 27715

Contact: Ron RoddyEmail: [email protected]

Drug name: Not availableIndication: Moderate to severe Crohn’s

DiseaseSpecialty: IBD specialist, GastroenterologyPhase: IIINotes: Looking for subjects refractory to

steroids, immunosupressants, andbiologicals. We need at least fivesubjects recruited over a 10 monthperiod.

ProMedDx, LLC10 Commerce WayNorton, MA 02766

Contact: Marion M. Santa InesEmail: [email protected]

Drug name: Not applicableIndication: Non-viral Liver DiseaseSpecialty: GastroenterologyPhase: Not applicableNotes: Only one study visit will be

required for this study. The onlyrisks to participants are those asso-ciated with a routine blood draw.

Wyeth Research500 Arcola Road, B4002Collegeville, PA 19426

Contact: Maria DattiloEmail: [email protected]

Drug name: PantoprazoleIndication: GERDSpecialty: Gastroenterology; MetabolismPhase: IIINotes: The study is seeking patients

between the ages of 1 through 11months with a diagnosis of pre-sumptive GERD requiring phar-macologic treatment.

Wyeth Research500 Arcola Road, B-4021Collegeville, PA 19426

Contact: Stacey LoReEmail: [email protected]

Drug name: pantoprazole/Protonix OralSuspension

Indication: GERDSpecialty: Neonatologist/Neonatal/Pediatric

GastroenterologistPhase: III with PK and pH assessmentsNotes: Need sites with access to a

Neonatal ICU. Protocol will studypHmetry and pharmacokinetics inpatients who are either term orpost term infants with presumedGERD who are within the neona-tal period of less than or equal to28 days. Procedures include twopHmetry assessments (1 atBaseline and 1 at Steady State)along with Single and MultipleDose PK collection. Study isenrolling but needs additionalinvestigators experienced in con-ducting pHmetry in infants, inorder to bolster recruitment.

HematologyStill Seeking Investigators

Cylene Pharmaceuticals5820 Nancy Ridge Drive, Suite 200San Diego, CA 92121

Contact: Claire PadgettEmail: [email protected]

Drug name: CX-3543Indication: Relapsed or refractory CLLSpecialty: Hematology; oncologyPhase: II

PRA International630 Dresher RoadHorsham, PA 19044

Contact: Toni BaileyEmail: [email protected]

Drug name: Not availableIndication: Graft vs. host diseaseSpecialty: Hematology; oncology; gastroen-

terologyPhase: IIINotes: Recruiting sites with patients who

have had a Bone MarrowTransplant (BMT) or Blood StemCell Transplant (PBSCT) and havea new onset of acute GvHD. Thisis a Phase III study. Each site isexpected to recruit 2-3 patients.The target populations arebetween ages 18-75 years.

Immunology/Infectious DiseaseNew Leads

BDpath Group310 Queen Street South, Unit 208Kitchener, ONCanada N2G 1K2

Contact: Yong ZhangEmail: [email protected]

Drug name: Not availableIndication: AIDSSpecialty: Immunology / infectious diseasePhase: II

Still Seeking Investigators

Advanced Biologics580 Union Square DriveNew Hope, PA 18939

Contact: Wen LinEmail: [email protected]

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27August 2007CenterWatch

Drug name: Not availableIndication: C. Difficile-associated diarrheaSpecialty: Infectious disease,

GastroenterologyPhase: IIINotes: Looking for sites in US and

Canada.

Catalyst Pharmaceuticals1111 S. Arroyo Parkway, Suite 200Pasadena, CA 91105

Contact: Antoinette TorresEmail: [email protected]

Drug name: Not availableIndication: Herpes ZosterSpecialty: Immunology/infectious diseasePhase: IIb

Covance Inc210 Carnegie Center BlvdPrinceton, NJ

Contact: Elizabeth PircEmail: [email protected]

Drug name: Not availableIndication: Influenza txSpecialty: Infectious Desease in a hospital

settingPhase: II

ICON CRO100 Commerce DriveNewark, DE 19713

Contact: Jillian CrillyEmail: [email protected]

Drug name: Not availableIndication: Hepatitis BSpecialty: Immunology, infectious diseasePhase: III

NephrologyStill Seeking Investigators

Advanced Magnetics, Inc.Clinical Trials Dept.61 Mooney StreetCambridge, MA 02138

Contact: Carrie DelaneyEmail: [email protected]

Drug name: ferumoxytolIndication: anemiaSpecialty: NephrologyPhase: IIINotes: Multicenter study

Angiotech Pharmaceuticals13921 Park Center Road, Suite 100Herndon, VA 20171

Contact: Andrew HarrisonEmail: [email protected]

Drug name: Vascular WrapTM paclitaxel andLifespan ePTFE Vascular Graft

Indication: Vascular Access for End StageRenal Disease

Specialty: Nephrology, Vascular Surgery,Interventional Radiology

Phase: PivotalNotes: A randomized, single blind trial to

assess the effectiveness of main-taining patency and safety of thepaclitaxel-eluting Vascular Wrapafter surgical implantation withthe Lifespan Graft in the upperextremity for hemodialysis vascu-lar access.

MedSource16902 El Camino RealHouston, TX 77058

Contact: Hope McPhersonEmail: [email protected]

Drug name: Not availableIndication: Renal AdenocarcinomaSpecialty: Nephrology, OncologyPhase: IIINotes: I am looking for investigators who

might be interested in participat-ing in a Phase III Vaccine study forpatients with Renal CellAdenocarcinoma. This is a greatdrug with a wonderful safety pro-file. Please let me know if any ofyour sites might be interested inmore information.

PRA International630 Dresher Rd.Horsham, PA 19044

Contact: Monica VolpeEmail: [email protected]

Drug name: Not availableIndication: Chronic kidney diseaseSpecialty: Nephrology – must be able to con-

duct phase I trials in addition tophases II & III

Phase: I-III

NeurologyNew Leads

Ortho-McNeil Janssen Scientific Affairs(OMJSA)62 Crescent Creek WaySelkirk, NY 12158

Contact: Rhonda LichtenwalnerEmail: [email protected]

Drug name: Not availableIndication: Essential tremorSpecialty: NeurologyPhase: IINotes: Seeking investigators that can use

central IRBs

Schering-Plough2000 Galloping Hill RoadMailstop K-15-3-3113Kenilworth, NJ 07033

Contact: Bohang Chen, MDEmail: [email protected]

Drug name: SCHIndication: Movement DisordersSpecialty: Tardive Dyskinesia, Drug Induced

Movement Disorder,Schizophrenia

Phase: IINotes: We are looking for investigators

who have conducted or are con-ducting studies for TardiveDiskinesia, Drug InducedMovement Disorder orSchizophrenia. The PIs can beinternational, but we prefer NorthAmerica.

Still Seeking Investigators

D.L. Anderson International3100 McKinnon Str, Ste #100Dallas, TX 75201

Contact: Nadia FarragEmail: [email protected]

Drug name: Not availableIndication: Partial epilepsySpecialty: NeurologyPhase: III

Ovation Pharmaceuticals, Inc.Four Parkway North, Suite 200Deerfield, IL 60015

Contact: Carmen MiceliEmail: [email protected]

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Grant Opportunities

Thomson28 August 2007

Drug name: Anti-epilepsy drugIndication: Pediatric and adult patients with

active epilepsySpecialty: Neurologists with in-patient phase

I pK capabilitiesPhase: I

PrecisionMed132 N. Acacia AveSolana Beach, CA 92075

Contact: Carole MarksEmail: [email protected]: No DrugIndication: Probable Alzheimer’s Disease or

Mild Cognitive ImpairmentSpecialty: Neurology; PsychiatryPhase: Biological Sample CollectionNotes: This is a long term follow-up of

subjects with Mild Alzheimer’sDisease or Mild CognitiveImpairment. Subjects are seenevery 6 months and have a blooddraw, spinal tap, and rating scales.Any medications are allowed.

Teva Neuroscience999 de Maisonneuve Blvd. West, Suite 550Montreal, QuebecH3A 3L4

Contact: Lynda MillerEmail: [email protected]

Drug name: CopaxoneIndication: Relapsing Remitting Multiple

SclerosisSpecialty: NeurologyPhase: IV

OB/GynNew Leads

Covance Inc.Site Activation Services150 4th Avenue North, Suite 600Nashville, TN 37219

Contact: Erin GreunkeEmail: [email protected]

Drug name: PhenoxodiolIndication: Ovarian CancerSpecialty: Gynecologic OncologyPhase: IIINotes: The purpose of this project is to

see if phenoxodiol in combinationwith weekly carboplatin, comparedto a placebo with weekly carbo-platin, is effective against late stageovarian cancer. Ages Eligible forStudy: Females 18 years and above

Still Seeking Investigators

Ciphergen Biosystems, Inc.6611 Dumbarton CircleFremont, CA 94555

Contact: Gillian CrutcherEmail: [email protected]

Drug name: Not applicableIndication: Ovarian CancerSpecialty: Ob/Gyn; Gyn/OncPhase: Not applicableNotes: This study aims to differentiate

cancer from non-cancer inpatients with pelvic mass. The testis specifically directed towardsovarian cancer. We require onepre-op blood draw and the study iscompleted after the pathologyreport.

Cytokine PharmaSciences, Inc.150 S. Warner Road, Suite 420King of Prussia, PA 19406

Contact: Barbara PowersEmail: bpowers@

cytokinepharmasciences.com

Drug name: Misoprostol vaginal insertIndication: Cervical ripening and reducing

time to deliverySpecialty: Obstetrician or family practice

doctor with hospital privileges andin-patient clinial research capabili-ties

Phase: IIINotes: Have 35 sites, looking for 5 addi-

tional sites; study will recruit 900women requiring induction oflabor. Must induce at least 5women per week to qualify. Studystarts in April, 2006.

OncologyNew Leads

Covance Inc.Site Activation Services150 4th Avenue North, Suite 600Nashville, TN 37219

Contact: Erin GreunkeEmail: [email protected]

Drug name: PhenoxodiolIndication: Ovarian CancerSpecialty: Gynecologic OncologyPhase: III

Notes: The purpose of this project is tosee if phenoxodiol in combinationwith weekly carboplatin, comparedto a placebo with weekly carbo-platin, is effective against late stageovarian cancer. Ages Eligible forStudy: Females 18 years and above

Pharmatech, Inc.789 Sherman St., Suite 600Denver, CO 80203

Contact: Erin BeaverEmail: [email protected]

Drug name: CoFactorIndication: Metastatic colorectal cancerSpecialty: OncologyPhase: IIINotes: Official Title: A Phase III Multi-

Center Randomized Clinical Trialto Evaluate the Safety and Efficacyof CoFactor and 5-Fluorouracil (5-FU) Plus Bevacizumab VersusLeucovorin and 5-FU PlusBevacizumab as Initial Treatmentfor Metastatic ColorectalCarcinoma

Still Seeking Investigators

Accentia Biopharmaceuticals Inc.450 Park Avenue, South 12th FloorNew York, NY 10016

Contact: William Calhoun, MSEmail: [email protected]

Drug name: BioVaxidIndication: Follicular, Non-Hodgkin’s

LymphomaSpecialty: Immunology; Hematology;

OncologyPhase: IIINotes: The objective of this Phase 3 study

is to confirm the safety and effica-cy of BIOVAXID, an autologoustumor derived idiotype vaccine, asmeasured by prolongation of dis-ease-free survival of patients withfollicular (NHL) during their firstcomplete remission induced byPACE. Seeking several investigativesites in US, Canada and Mexico.Sites must be willing to use onlyPACE for remission induction. NoCHOP-R or Rituxin permitted.Patient-specific vaccine manufac-tored from biopsy materials.

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29August 2007CenterWatch

A.P. Pharma Inc.123 Saginaw DriveRedwood City, CA 94063

Contact: Erin O’BoyleEmail: [email protected]

Drug name: APF530: TEG-POE polymerBased Formulation Containing2% Granisetron

Indication: Prevention of Chemo-InducedNausea and Vomiting (CINV)

Specialty: OncologyPhase: IIINotes: The comparator in this trial is

Aloxi. Patients will be allowed toparticipate in up to 4 treatmentcycles and do not have to bechemotherapy naive.

Cephalon, Inc.CCRIChildren’s Hospital of Philadelphia3535 Market St., Suite 1200Philadelphia, PA 19104

Contact: Eileen DorseyPhone: (215) 590-1295Email: [email protected]

Drug name: Not availableIndication: Pediatric breakthrough painSpecialty: Pediatric oncologyPhase: II

Ciphergen Biosystems, Inc.6611 Dumbarton CircleFremont, CA 94555

Contact: Gillian CrutcherEmail: [email protected]

Drug name: Not applicableIndication: Ovarian CancerSpecialty: Ob/Gyn; Gyn/OncPhase: Not applicableNotes: This study aims to differentiate

cancer from non-cancer inpatients with pelvic mass. The testis specifically directed towardsovarian cancer. We require onepre-op blood draw and the study iscompleted after the pathologyreport.

Covance Inc.210 Carnegie CenterPrinceton, NJ 08540

Contact: Brenda RolfeEmail: [email protected]

Drug name: Phenoxodiol with carboplatinIndication: Relapse / recurrent carboplatin

resistent ovarian cancerSpecialty: Gynecology, OncologyPhase: IIINotes: Efficacy Study Comparing

Phenoxodiol in Combination withCarboplatin versus Carboplatinwith Placebo in Patients withPlatinum-Resistant or Platinum-Refractory Late-Stage EpithelialOvarian, Fallopian or PrimaryPeritoneal Cancer Following atLeast Second Line PlatinumTherapy

Cylene Pharmaceuticals5820 Nancy Ridge Drive, Suite 200San Diego, CA 92121

Contact: Claire PadgettEmail: [email protected]

Drug name: CX-3543Indication: Relapsed or refractory CLLSpecialty: Hematology; oncologyPhase: II

Delcath Systems1100 Summer StreetStamford, CT 06905

Contact: Dr. Samuel HerschkowitzPhone: (718) 624-6277Email: [email protected]

Drug name: MelphalanIndication: Cancer (melanoma)Specialty: Oncology, Interventional radiologyPhase: IIINotes: Delivery system for the isolated

hepatic arterial infusion ofchemotherapy to patients withmetastatic liver tumors.

GenVec, Inc.65 West Watkins Mill RoadGaithersburg, MD 20878

Contact: Renee Riggs-GarrettPhone: (240) 632-5582Email: [email protected]

Drug name: TNFeradeIndication: Pancreatic CancerSpecialty: OncologyPhase: II/IIINotes: Please contact us for additional

information to participate in thisstudy.

GTx , Inc.3 North DunlapMemphis, TN 38163

Contact: Susan FowlerEmail: [email protected]

Drug name: GTx-024Indication: Non-Small Cell Lung CancerSpecialty: OncologyPhase: IIbNotes: This study is for patients with

stage 2, 3, and 4 NSCLC who haveexperienced weight loss / musclewasting (cachexia).

ICON Clinical Research Canada Inc.7405 Transcanada Hwy, Suite 300St. Laurent, QC, H4T 1Z2 Canada

Contact: Stefan MilenkovEmail: [email protected]

Drug name: Not availableIndication: Non-small cell lung cancer

(NSCLC)Specialty: OncologyPhase: II

Imaging Diagnostic Systems6531 NW 18th CourtPlantation, FL 33313

Contact: Deborah O’BrienEmail: [email protected]

Device name: Laser Breast imaging deviceIndication: Detection of breast cancerSpecialty: Radiology mammographyPhase: Clinical collection phaseComments: Looking for clinical sites to collect

patient data in the dense breastclassification scheduled for biopsy.Need high volume sites.

Introgen Therapeutics, Inc.2250 HolcombeHouston, TX 77030

Contact: Julie Sicam, MT (ASCP) MSHSEmail: [email protected]

Drug name: Advexin (INGN 201)Indication: Squamous Cell Carcinoma of the

Head and NeckSpecialty: Oncologist/ENT SurgeonPhase: III

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Grant Opportunities

Thomson30 August 2007

Notes: A Phase III, Multi-Center, Open-Label, Randomized Study toCompare the Effectiveness andSafety of IntratumoralAdministration of INGN 201 inCombination with ChemotherapyVersus Chemotherapy Alone inPatients with RecurrentUnresectable Squamous CellCarcinoma of the Head and Neck(SCCHN).

Javelin Pharmaceuticals125 CambridgePark DriveCambridge, MA 02140

Contact: Jennifer BurgEmail: [email protected]

Drug name: Intranasal KetamineIndication: Breakthrough pain in cancer

patientsSpecialty: Anesthesia; Pain Management;

OncologyPhase: III

MedSource16902 El Camino RealHouston, TX 77058

Contact: Hope McPhersonEmail: [email protected]

Drug name: Not availableIndication: Renal AdenocarcinomaSpecialty: Nephrology, OncologyPhase: IIINotes: I am looking for investigators who

might be interested in participat-ing in a Phase III Vaccine study forpatients with Renal CellAdenocarcinoma. This is a greatdrug with a wonderful safety pro-file. Please let me know if any ofyour sites might be interested inmore information.

Pharmacyclics999 East Arques AvenueSunnyvale, CA 94085

Contact: Priscilla HornEmail: [email protected]

Drug name: Xcytrin (Motexafin Gadolinium)Injection

Indication: Non-Small Cell Lung CancerSpecialty: Medical OncologyPhase: II

Notes: Randomized Phase II study ofMotexafin Gadolinium as secondline treatment of unresected local-ly advanced (Stage IIIB) or metas-tastic non-small cell lung cancer.Do not respond if you are a CROor SMO.

PPD3900 Paramount ParkwayMorrisville, NC 27560

Contact: Peidi GuEmail: [email protected]

Drug name: Not availableIndication: CTM; head & neck cancer therapySpecialty: OncologyPhase: I and II

Schering-Plough2015 Galloping Hill RoadKenilworth, NJ 07033

Contact: Siu-Long YaoEmail: [email protected]

Drug name: Not availableIndication: CancerSpecialty: Oncologist or physician experi-

enced with oncology trials.Phase: INotes: We are interested in sites that can

utilize central IRBs and have expe-rience with Phase I trials.

Seattle Genetics21823 30th Drive SEBothell, WA 98021

Contact: Dr. Eric SieversEmail: [email protected]

Drug name: SGN-30Indication: Cutaneous ALCL (Anaplastic

Large Cell Lymphoma)Specialty: Hematologist OncologistPhase: II/IIINotes: This is a rare form of lymphoma.

We would be interested in contact-ing research sites even if they onlysee 1-3 cases per year.

Vical Incorporated10390 Pacific Center CourtSan Diego, CA 92121

Contact: Linda Strause, PhDEmail: [email protected]

Drug name: Allovectin-7Indication: Metastatic MelanomaSpecialty: Medical OncologistPhase: III

Notes: This is a head-to-head superioritytrial of Allovectin-7 vs. first linechemotherapy in metastaticmelanoma patients where surgeryis not a curative option. This trialwill enroll 375 patients, random-ized 2: 1 to Allovectin-7. Pleasecontact Vical at [email protected] or call (877) 343-6389.

OphthalmologyStill Seeking Investigators

Rx Development Resources3110 Cherry Palm Drive, Suite 350Tampa, FL 33619

Contact: Penny CobbEmail: [email protected]

Drug name: Not availableIndication: Adenovirus/conjunctivitisSpecialty: Ophthalmology; Infectious diseasePhase: IINotes: Please contact me as soon as possi-

ble if you feel your site couldpotentially enroll 4-5 subjects in a3 month period.

Rx Development Resources3104 Cherry Palm Drive, Suite 260Tampa, FL 33619

Contact: Penny CobbEmail: [email protected]

Drug name: Not availableIndication: Dry AMDSpecialty: OphthalmologyPhase: IINotes: We are currently seeking potential

retinal research sites that have aMP-1 MicroPerimeter and a HRAII. At least one piece is required tobe considered as a potential site.

OtolaryngologyStill Seeking Investigators

Alcon Research, Ltd6201 South Freeway, TC-40Fort Worth, TX 76134

Contact: Sally F. BeezleyEmail: [email protected]

Drug name: Not availableIndication: Acute Otitis Media with

Tympanostomy Tube (AOMT)Specialty: Ear Nose and Throat (ENT) or

PediatricianPhase: III

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Grant Opportunities

31August 2007CenterWatch

Notes: Must have access to the followingequipment: Vacuum suctioning,tympanometry, and audiology. Noplacebo arm in study; all activetreatment.

Pain ManagementStill Seeking Investigators

Javelin Pharmaceuticals125 CambridgePark DriveCambridge, MA 02140

Contact: Jennifer BurgEmail: [email protected]

Drug name: Intranasal KetamineIndication: Breakthrough pain in cancer

patientsSpecialty: Anesthesia; Pain Management;

OncologyPhase: III

Pediatrics/NeonatologyNew Leads

Biosolve638 Via CristinaNewbury Park, CA 91320

Contact: Andrea FriedmanEmail: [email protected]

Product: Infant formulaIndication: Infant nutritionSpecialty: Investigator with access to a popu-

lation of healthy infants followedfrom 3 months to a year. Infantsmay be breastfed or formula fed;either exclusively, or mixed.

Phase: II/IIINotes: Investigator must have access to

Hologic DEXA and/or Peapd.Anthropometrics will be moni-tored for this growth study. Inaddition, two blood draws, ataproximately 3 and 6 months ofage will be required.

GlaxoSmithKline - Vaccines/Biologicals2301 Rennaissance BoulevardKing of Prussia, PA 19406

Contact: Kerri MalloryEmail: [email protected]

Drug name: Not applicableIndication: VaccineSpecialty: PediatricsPhase: II-IV

Notes: GlaxoSmithKline Biologicals-Vaccines has a continuous risingneed for investigators and researchsites to assist with clinical researchtrials. We are interested in develop-ing mutually successful relation-ships with qualified sites.

Still Seeking Investigators

Boehringer Ingelheim Pharmaceuticals,Inc.900 Ridgebury Road, PO Box 368Ridgefield, CT 06877-0368

Contact: Patricia NormanEmail: [email protected]

heim.com

Drug name: Micardis (telmisartan)Indication: Pediatric HypertensionSpecialty: Pediatricians / Pediatric

NephrologistsPhase: IINotes: 4 week active treatment trial.

Children 6-17. Investigators withPK experience preferred.

Cephalon, Inc.CCRIChildren’s Hospital of Philadelphia3535 Market St., Suite 1200Philadelphia, PA 19104

Contact: Eileen DorseyPhone: (215) 590-1295Email: [email protected]

Drug name: Not availableIndication: Pediatric breakthrough painSpecialty: Pediatric oncologyPhase: II

Wyeth Research500 Arcola Road, B4002Collegeville, PA 19426

Contact: Maria DattiloEmail: [email protected]

Drug name: PantoprazoleIndication: GERDSpecialty: Gastroenterology; MetabolismPhase: IIINotes: The study is seeking patients

between the ages of 1 through 11months with a diagnosis of pre-sumptive GERD requiring phar-macologic treatment.

Wyeth Research500 Arcola Road, B-4021Collegeville, PA 19426

Contact: Stacey LoReEmail: [email protected]

Drug name: pantoprazole/Protonix OralSuspension

Indication: GERDSpecialty: Neonatologist/Neonatal/Pediatric

GastroenterologistPhase: III with PK and pH assessmentsNotes: Need sites with access to a

Neonatal ICU. Protocol will studypHmetry and pharmacokinetics inpatients who are either term orpost term infants with presumedGERD who are within the neona-tal period of less than or equal to28 days . Procedures include twopHmetry assessments (1 atBaseline and 1 at Steady State)along with Single and MultipleDose PK collection. Study isenrolling but needs additionalinvestigators experienced in con-ducting pHmetry in infants, inorder to bolster recruitment.

Phase I/Healthy VolunteersStill Seeking Investigators

Bristol-Myers SquibbPO Box 4000Princeton, NJ 08543-4000

Contact: Diane RandallEmail: [email protected]

Drug name: Not availableIndication: SchizophreniaSpecialty: PsychiatryPhase: I

Genaera Corporation5110 Campus DrivePlymouth meeting, PA 19462

Contact: Jim EllisEmail: [email protected]

Drug name: Not availableIndication: Diabetes Type IISpecialty: Phase I unit Obese Diabetes Type

IIPhase: I

Page 32: August 2007 A Thomson CenterWatch Publication

Notes: We are looking for a Phase I unitthat can recruit 28 Obese Type IIDiabetes patients (7 subjects in 4dose groups), for a single IV dosestudy, patients will be housed for72 hours.

Johnson & Johnson PRD

Contact: Yvonne Tan Phone: (800) 362-6345, x 74060 Email: [email protected]

Drug name: Not available Indication: Healthy volunteers Specialty: Experience with healthy volunteersPhase: I Notes: Very interested in seeking cen-

ters/CROs on the West Coast thatrun phase I healthy-volunteerstudies, preferably in SouthernCalifornia.

Johnson & Johnson PRD

Contact: Juli E. LeDuc, M.A., CCRC,CCRA

Email: [email protected]

Drug name: variousIndication: Schizophrenia; sarcoma; HIV;

healthy-patient studiesSpecialty: Phase I unitsPhase: INotes: Seeking MDs with some Phase I

research experience who are ableto utilize central IRB for patientstudies as well as healthy-volunteerstudies. Sites should be located inthe United States only, Northeastregion—as far West as Indiana,and as far South as Virginia andEastern Canada.

Schering-Plough2015 Galloping Hill RoadKenilworth, NJ 07033

Contact: Siu-Long YaoEmail: [email protected]

Drug name: Not availableIndication: CancerSpecialty: Oncologist or physician experi-

enced with oncology trialsPhase: INotes: We are interested in sites that can

utilize central IRBs and have expe-rience with Phase I trials.

Psychiatry/PsychologyStill Seeking Investigators

Appian Services, LLC10107 White Cascade DriveCharlotte, NC 28269

Contact: Grier Harris, PharmDEmail: [email protected]

Drug name: Not availableIndication: Cocaine abuse and addictionSpecialty: PsychologyPhase: IINotes: Study: new agent for cocaine

addiction. Population: adults withestablished history of cocaine use.Enrollment goal is 60, to completea minimum of 40. Study duration:14-16 weeks. Total of 34-40 visits.Target enrollment period is 6months.

Bristol-Myers SquibbPO Box 4000Princeton, NJ 08543-4000

Contact: Diane RandallEmail: [email protected]

Drug name: Not availableIndication: SchizophreniaSpecialty: PsychiatryPhase: I

PulmonologyStill Seeking Investigators

Forest Research InstituteHarborside Financial Center, Plaza VJersey City, NJ 07311

Contact: Jessica HughesEmail: [email protected]

Drug name: Not availableIndication: Chronic Obstructive Pulmonary

DiseaseSpecialty: PulmonologyPhase: IIINotes: Experienced with exercise toler-

ance studies. Site needs to haveaccess to plethysmograph and elec-tronically braked cycle ergometer.

Novartis1 Health PLaza, 435-1175East Hanover, NJ

Contact: Sandeep AthalyeEmail: [email protected]

Drug name: Not availableIndication: COPD, Cystic FibrosisSpecialty: MD with Respiratory

SpecializationPhase: I-IIComments: CROs with strength in COPD or

Cystic Fibrosis fields preferred.

Pacific Clinical Center17337 Ventura Blvd., Suite #226Encino, CA 91316

Contact: Janet JohnsonEmail: [email protected]

Drug name: Not availableIndication: AsthmaSpecialty: PulmonologyPhase: IIINotes: Investigator must be Board certi-

fied and located in the Los Angelesarea.

RheumatologyStill Seeking Investigators

CombinatoRx, Inc.245 First St., 16th Fl.Cambridge, MA 02142

Contact: Gretchen L. ChildsEmail: [email protected]

Drug name: CRx-102Indication: OsteoarthritisSpecialty: Osteoarthritis; Internal MedicinePhase: IINotes: Study Objectives: To investigate

the efficacy and dose response ofCRx-102, a novel oral synergisticfixed dose combination product,in Osteoarthritis of the Knee.

CombinatoRx, Inc.245 First St., 16th Fl.Cambridge, MA 02142

Contact: James KeaneEmail: [email protected]

Drug name: CRx-102Indication: Rheumatoid ArthritisSpecialty: Rheumatology; Internal MedicinePhase: IIbNotes: Study Objectives: To investigate

the efficacy of CRx-102, a noveloral synergistic fixed dose combi-nation product, in RheumatoidArthritis patients on stableDMARD therapy.

Grant Opportunities

Thomson32 August 2007

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Grant Opportunities

33August 2007CenterWatch

Pacific Clinical Center17337 Ventura Blvd., Suite #226Encino, CA 91316

Contact: Janet JohnsonEmail: [email protected]

Drug name: Not availableIndication: ArthritisSpecialty: RheumatologyPhase: IIINotes: Investigator must be Board certi-

fied and located in the Los Angelesarea.

Strategic Science & Technologies58 Charles StreetCambridge, MA 02141

Contact: Eric FosselEmail: [email protected]

Drug name: Transdermal ibuprofenIndication: Arthritis and painSpecialty: Any who deal with arthitis, joint or

muscle painPhase: PilotNotes: Our transdermal ibuprofen prod-

uct is potentially revolutionary asit provides effective localized reliefof pain at a much lower total bodydose than oral ibuprofen and itavoids the complication of stom-ach irritation and ulceration.

Wyeth Research500 Arcola Rd.Collegeville, PA 19426

Contact: Patti ShottEmail: [email protected]

Drug name: Not availableIndication: Rheumatoid ArthritisSpecialty: RheumatologyPhase: II and III

SurgeryNew Leads

Auxilium Pharmaceuticals40 Valley Stream ParkwayMalvern, PA 19355

Contact: Brian WalkerEmail: [email protected]

Drug name: AA4500Indication: Dupuytren’s ContractureSpecialty: Orthopedic Surgeon; Hand

SurgeonPhase: III

Notes: Auxilium Pharmaceuticals is cur-rently recruiting doctors to partici-pate in a Phase III clinical trialinvolving patients withDupuytren’s Contracture. Thistrial is studying an enzyme injec-tion as a non-surgical treatmentfor patients who have Dupuytren’sContracture for Dupuytren’sDisease.

Still Seeking Investigators

Angiotech Pharmaceuticals13921 Park Center Road, Suite 100Herndon, VA 20171

Contact: Andrew HarrisonEmail: [email protected]

Drug name: Vascular WrapTM paclitaxel andLifespan ePTFE Vascular Graft

Indication: Vascular Access for End StageRenal Disease

Specialty: Nephrology, Vascular Surgery,Interventional Radiology

Phase: PivotalNotes: A randomized, single blind trial to

assess the effectiveness of main-taining patency and safety of thepaclitaxel-eluting Vascular Wrapafter surgical implantation withthe Lifespan Graft in the upperextremity for hemodialysis vascu-lar access.

Anika Therapeutics, Inc.Clinical Department236 West Cummings ParkWoburn, MA 01801

Contact: Clinical DepartmentEmail: [email protected]

Device name: UnavailableIndication: OsteoarthritisSpecialty: Orthopedic surgeryPhase: IIINotes: Anika Therapeutics, Inc. is cur-

rently seeking orthopedic surgeonsfor a Phase III trial of OA.Preference will be given to investi-gator with prior research experi-ence. Please email current CV andcontact information if interested.

SkyePharma Inc.10450 Science Center DriveSan Diego, CA 92121

Contact: Marius Ardeleanu, MDEmail: [email protected]

Device name: DepoBupivacaineIndication: Postoperative analgesiaSpecialty: Anorectal surgeryPhase: IINotes: Looking for suitable clinical site to

conduct a dose-ranging study inpatients undergoing 2- or 3-col-umn excisional hemorrhoidecto-my using Milligan-Morgan orFerguson-type techniques undergeneral anesthesia, includingnewer approaches with specializedinstruments, such as LigaSure andharmonic scalpel. Need site withlarge volume of cases to ensuretimely enrollment (target enroll-ment for each site is 10 subjects in3 months).

UrologyStill Seeking Investigators

PPD, Inc.7551 Metro Center Dr, Ste 300Austin, TX 78744

Contact: Dave MatthewsEmail: [email protected]

Drug name: Not availableIndication: Benign Prostatic Hyperplasia

(BPH)Specialty: UrologyPhase: II

Sanofi-Aventis9 Great Valley ParkwayMalvern, PA 19355

Contact: Martha RitzEmail: [email protected]

Drug name: Not availableIndication: Overactive bladder/urge inconti-

nenceSpecialty: Urology; OB/GYN; Family CarePhase: IIb

Page 34: August 2007 A Thomson CenterWatch Publication

Grant Opportunities

Thomson34 August 2007

Opportunities Initiating

Indication Sponsor Drug/Device Date Planned Additional Information

Phase I

cancer Kinex KX2-391 September 2007 Plans to enroll subjects with solid tumors and lymphoma in the US

Phase II

Gram-positive Arpida iclaprim (iv) H2, 2007 Plans to enroll 130 subjects with pneumonia hospital-acquired, ventilator-associated

or healthcare-associated pneumonia

organ rejection LifeCycle Pharma LCP-Tacro 2007 Plans to enroll 60 kidney transplant recipients in the US and Canada

acute ischemic stroke Nuvelo alfimeprase H2, 2007 Plans to enroll 100 subjects within 3-9 hours of stroke onset.

non-small cell Peregrine bavituximab 2007 Plans to enroll up to 49 subjects in Indialung cancer

Phase III

chronic renal failure Affymax Hematide Q4, 2007 Plans to 2,200 subjects with chronic renalfailure, including those on dialysis and not on dialysis.

type 1 diabetes Generex Oral-lyn Q3/Q4, 2007 Plans to enroll 750 subjects internationally

liver cancer Progen PI-88 2007 Plans to enroll 800 subjects with hepatocellular carcinoma internationally

obesity Vivus Qnexa H2, 2007 Plans to enroll 4,500 obese subjects (BMI greater than 30) and those with associated co-morbidities

This section provides investigative sites with a listing of phase

I through phase IV programs that are being initiated by

sponsors. Sites can use this information to track up and

coming studies that may offer grant opportunities. Please note:

these companies have not announced that they are actively seeking

candidates sites.

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T H O M S O N C E N T E R WAT C H

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The 2007 Edition is Here!State of the Clinical Trials Industry 2007State of the Clinical Trials Industry 2007 is a comprehensive resource that examines all aspects of the global clinical trials industry and provides original CenterWatch data. Included are over 500 charts and graphs, a review of the top clinical research stories of 2006 as reported by the editorial staff of The CenterWatch Monthly; as well as drug pipeline information and updates on specific disease areas. Much of the data is based on CenterWatch’s clinical research benchmarking surveys that have been conducted across global segments and represents an analysis of current sponsor and CRO practices.

The 2007 edition includes:100 new charts Hundreds of updated charts from the 2006 editionNew articles on each chapter topicUpdated pipeline activity for major therapeutic areas

To preview a table of contents, a chart index and sample charts, visit our bookstore onwww.centerwatch.com.

Order your copy today for only $599 (+s/h)! To order, call us at (800) 765-9647 and ask for discount code 3554.

For more information:

Visit our bookstore onwww.centerwatch.com

Phone:(866) 219-3440, ext. 4

Email:[email protected]

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At Inclinix, we take your perspective – the clinicalperspective – and integrate it into a recruitment processthat delivers results. Well before the trial begins, Inclinixuses our technology solutions to make informed investigator selections. We equip each site with the tools necessary to convert interested study candidates into consented patients. Because enrollment time lines are critical, we providedirect-to-patient advertising and contact center solutions to refer even more quali�ed candidates into your trial.

With productive investigators and quali�ed patients,we keep them interested and motivated to ensure trial completion using customized retention strategies.

Don’t leave your Clinical Trial Recruitment to chance.Call Inclinix today.

Enrollment Feasibility : : Investigator Recruitment : : Site Support : : Patient Recruitment : : Retention : : Monitoring with ECRAs

3534 South College Road, Suite I • Wilmington, North Carolina 28412ph: 910.332.2001 • www.inclinix.com • email: [email protected]

The Standard for Clinical Trial Recruitment.

The Enrollment CRO.