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1

White paper

Jefferies 2012: Global Healthcare Conference Review

By Caroline Richards & Maura MusciaccoNovember 2012



2

DatamGlobaLondo This analy

Prod

Lundbec

The

breast ca

(insulin d

trastuzum

rheumato

hypercho

bowel sy

84]) for t

ceftoloza

complica

infusion

monitoal Healton oveysis review co

duct-specific

ck, ImmunoG

status of late

ancer, Novo

degludec + in

mab emtans

oid arthritis, A

olesterolemia

yndrome and

he treatment

ane/tazobact

ated intra-abd

or attenthcare

er Noveovers the key

and overall

Gen, Medivat

e-stage agen

Nordisk’s Tr

nsulin aspart

ine (TDM1) f

Aegerion’s lo

a, NPS Phar

d Natpara (re

t of adult hyp

tam for comp

dominal infec

nded th Confe

ember y highlights o

company up

ion, Novo No

nts such as R

resiba (insuli

t) for type 2 d

for breast ca

opidomide fo

rmaceuticals’

ecombinant h

poparathyroid

plicated urina

ctions, and U

he Jeffeerence 14–15,of the confer

pdates for: Ae

ordisk, NPS

Roche’s Perj

n degludec)

diabetes, Imm

ncer, Rigel’s

or homozygo

’ Gattex (ted

human parath

dism, Cubist

ary tract infec

United Thera

eries 2held in, 2012.

rence includin

egerion, Cub

jeta (pertuzu

and Ryzode

munoGen’s

s fostamatinib

us familial

uglutide) for

hyroid hormo

t’s

ctions and

peutics’ impl

2012 n . ng:

bist,

mab) for

eg

b for

short

one [1-

lantable


3

The quanswe

directly Aeg

prescreat

inte

Key prgloba

Lomitapide

homozygou

characteriz

treatments

nature of its

developme

date of Dec

adults with

that the dru

(LDL-C) an

Aegerion a

EU a MAA

company e

uestion aner sessionproceedi

gerion’s entation ted some eresting

resental healt

e is Aegerion

us familial hy

zed by extrem

. Aegerion d

s promising

ental and reg

cember 29, 2

HoFH and p

ug led to a 50

nd had a favo

also briefly dis

has been file

expects appro

nd n ing

ations thcare

’s lead produ

ypercholeste

mely high cho

edicated its 4

new candida

ulatory timel

The US

curr

A

c

A

r

to

make

drug on the

2012. Aegeri

presented 78

0.2% reducti

orable risk-be

scussed lopi

ed with the E

oval to come

Aegappdatdiso

from Jconfer

uct, and has

rolemia (HoF

olesterol and

40-minute pr

ate for the dis

ines.

S Food and D

rently review

Application (M

company hig

Endocrinolog

Advisory Co

ecommende

two vote in f

es it likely tha

e Prescription

on has comp

8-week data f

on in low-de

enefit profile

idomide’s sta

European Me

e about in mi

gerion: hoproval chee for drugorder loom

Jefferierence

just complet

FH), a rare d

d with no cur

resentation to

sease and su

Drug Adminis

ing a Market

MAA) for lopid

ghlighted that

gic and Meta

mmittee (EM

ed approval o

favor of back

at the FDA w

n Drug User

pleted a pivo

from this stu

ensity lipopro

.

atus in ex-US

edicines Age

d-2013. Mea

opes for neeer intensig to treat rms

es 2012

ted Phase III

disorder

rrent effective

o a round-up

ummarized e

stration (FDA

ting Authoriz

domide. The

t the FDA's

abolic Drugs

MDAC) recen

of lopidomide

king the drug

ill now appro

Fee Act (PD

otal Phase III

dy, which sh

tein choleste

S countries.

ency (EMA), a

anwhile, Aeg

ew-year ify as PDUrare chole

2

I trials in

e

p of the

expected

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ation

e

ntly

e in a 13

, which

ove the

DUFA)

trial in

howed

erol

In the

and the

erion is

UFA esterol


4

gearing up

year. This w

The questio

spun up so

Pharmaceu

approachin

lopidomide

factor betw

oral drug w

drug and so

to start Phas

will be a sma

on and answ

ome interestin

uticals, whos

ng approval.

e, at the end o

ween its HoFH

with a half-life

o carries the

se I trials in J

all study, in fe

wer session d

ng discussio

se late-stage

Mipomersen

of January 2

H product an

e of 20–30 ho

e risk of inject

Japanese pa

ewer than 10

directly proce

n on Aegerio

HoFH cand

n’s PDUFA d

2013. Aegerio

nd ISIS’s is th

ours, wherea

tion-site reac

atients towar

0 patients.

eeding Aeger

on’s main co

idate, mipom

ate is a little

on said that a

hat lopidomid

as Mipomers

ctions.

rds the end o

rion’s presen

mpetitor, ISI

mersen, is als

later than th

a differentiat

de is a once-

en is an inje

of this

ntation

S

so

hat of

ting

-daily

ctable


5

Cubist star

time it hope

late-stage t

income. It i

three cand

treatment f

receptor an

and its intra

(CXA-201)

urinary and

While it is t

in 5 years'

revenues w

while non-

to $77.7m,

The compa

product Cu

drug was a

treatment o

infections,

Staphyloco

usually occ

product in a

Cubist attri

rted its prese

es to have ge

trials, and to

s already pa

idates in Pha

for Clostridiu

ntagonist, CB

avenously ad

for the treat

d reproductiv

too early to s

time, it certa

were up 18%

GAAP adjus

compared to

any turned ne

ubicin (daptom

approved in t

of complicate

including me

occus aureus

cur in hospita

a new class

buted its stro

Cubunvdeli

entation by ou

enerated $2b

have achiev

art of the way

ase III develo

m difficile-as

B-5945, for th

dministered c

ment of com

ve tract infect

surmise on w

ainly posted s

% in Q3 2012

sted operatin

o $71.2m in

ext to discus

mycin for inje

he US in 200

ed skin and s

ethicillin-resis

s (MRSA). Th

alized patient

of antibiotics

ong Q3 2012

bist: ambiveiled as Civer reven

utlining its vis

bn in global r

ved $700m in

y there with it

opment: an o

ssociated dia

he treatment

combination

mplicated intra

tions.

whether Cubis

solid Q3 201

over the sam

g income inc

Q3 2011.

ss its approve

ection). This

03 for the

skin structure

stant

hese serious

ts and Cubic

s called cyclic

2 revenue gro

itious 5-yeCubicin conue growt

g

sion for the f

revenue, hav

n non-GAAP

ts pipeline as

oral lipopepti

arrhea (CDAD

t of opioid-ind

of ceftolozan

a-abdominal

st will achiev

2 financial re

me period in

creased 9%

ed

e

s infections

cin was the fi

c lipopeptide

owth

ear plan ontinues tth

In 5 yea[Cubist]

to hgenerate

in glo

reve

future: in 5 ye

ve four candi

adjusted op

spirations. It

de, CB-315,

D); a mu-opio

duced consti

ne and tazob

infections an

ve its financia

esults. Its tot

2011, at $23

rst approved

e antibiotic ag

to

ars' time ] hopes

have ed $2bn obal nue

ears'

idates in

perating

has

for the

oid

ipation;

bactam

nd

al goals

al net

38.2m,

d

gents.


6

Cubist fina

ceftolozane

infections a

product has

against a n

particular b

explained,

currently av

infections.

qualified in

Antibiotic In

from the FD

designation

Hatch-Wax

lized its pres

e/tazobactam

and complica

s the potenti

number of ba

bacterium is e

and has dev

vailable antib

In October 2

fectious dise

ncentives No

DA is expect

ns would qua

xman exclusi

sentation by d

m, which is in

ated intra-abd

al to be more

acterial strain

extremely pr

veloped num

biotics, makin

2012, Cubist

ease product

ow (GAIN) ac

ted within 60

alify CXA-20

ivity, priority

discussing it

n Phase III tr

dominal infec

e successful

ns, including

revalent in th

erous resista

ng it one of p

submitted ap

t (QIDP) des

ct for both CX

days of sub

1 and CB-31

review, and

s combinatio

ials for comp

ctions. Cubis

than Cubici

Pseudomona

e hospital se

ance mechan

physicians’ m

pplications to

ignation und

XA-201 and

mission. If g

5 for a 5-yea

eligibility for

on drug

plicated urina

st stated that

n, since it is

as aeruginos

etting, Cubist

nisms agains

most feared

o the FDA fo

er the Gener

CB-315. A re

ranted, the

ar extension

fast-track sta

ary tract

t this

active

sa. This

t

st

r

rating

esponse

of

atus.


7

PerjetT-DM

expecteparticprom

Roche’s CF

illustrating

solid growt

company in

has a subs

are in Phas

three cand

approved in

of Europe;

Europe; an

Europe. Pe

enable Roc

franchise. P

potential to

metastatic

antibody-dr

activity whi

(see Immu

sales grew

experience

Europe wa

impact of g

Bonviva (ib

ta and M1 are ed to be cularly

mising

FO, Alan Hip

a considerab

th in the eme

nvests nearly

stantial pipeli

se I, 24 are in

idates are un

n the US and

Erivedge (vi

nd T-DM1 (tra

erjeta and T-

che to build o

Perjeta in co

o create a ne

breast cance

rug conjugat

le targeting i

noGen below

L

Centra

w by 12% in b

ed only single

s the only re

generic erosio

bandronic ac

ppe, presente

ble pipeline a

erging marke

y half of this

ne with 71 n

n Phase II, a

ndergoing re

d Switzerland

smodegib), w

astuzumab e

DM1 are exp

on its human

mbination w

w standard o

er. T-DM1, w

tes, or “arme

intracellular d

w).

Year-to-da

generated

company’

Asia grew

Latin Americ

al Asia, Midd

both regions.

e-digit growth

egion to unde

on of produc

id).

F. Hoffmaassets to

ed an overvie

and strong fo

ts. With a tot

into its late-s

ew molecula

and eight are

gistration: Pe

d and is curr

which is app

emtansine), w

pected to be

n epidermal g

ith Herceptin

of care for wo

which is an e

d antibodies

delivery of a

ate sales illus

d in the emer

’s pharmaceu

by 14% (driv

ca and CEMA

le East, Afric

In stark con

h (6% and 1%

ergo a contra

cts such as C

an La Rocbuild on

ew of the Ro

ocus on onco

tal R&D budg

stage assets

ar entities (NM

e in Phase III

erjeta (pertuz

rently being r

roved in the

which is filed

particularly p

growth factor

n (trastuzuma

omen with H

merging clas

s” – retains H

potent cell-k

strate the stro

rging market

uticals divisio

ven mostly b

AI (Central an

ca, and India

trast, sales i

%, respective

action in sale

CellCept (myc

che: late-sits Hercep

oche Group,

ology, as wel

get of CHF8

. Currently R

MEs), of whi

. The remain

zumab), whi

reviewed in t

US and filed

in the US an

promising as

r receptor 2 (

ab; Roche) h

HER2-posiitve

ss of therapie

Herceptin’s bi

killing agent,

ongest growt

ts. Looking a

on only, sale

by China), wh

nd Eastern E

an subcontine

n the US and

ely), while W

es (-2%) due

cophenolate

tage pipeptin franc

ll as

bn, the

Roche

ch 36

ning

ch is

he rest

d in

nd in

s they

(HER2)

has the

e

es –

iological

DM1

th being

at the

es in

hile in

Europe,

ent)

d Japan

Western

to the

) and

line hise


8

Lundbeck i

products in

portfolio an

substantial

major oppo

Revenue fr

Sabril (viga

episodes a

in Japan, O

lymphocytic

revenues. I

comparing

in sales in t

launched in

Lundbeck’s

than 50% o

by three lat

launch duri

(aripiprazo

(nalmefene

antidepress

US, Europe

2013. The

registration

2013, while

addition the

bipolar diso

of alcohol c

feedback fr

expected in

s shifting aw

n the Europea

nd a balance

unrealized p

ortunity for Lu

rom new pro

abatrin; epile

associated wi

Onfi (clobaza

c leukemia) d

Its key brand

Q3 2011 sa

this market d

n Japan in 20

s new produc

of revenues b

te-stage pipe

ing 2013: vo

le; schizophr

e; alcohol de

sant vortioxe

e, and Canad

antipsychotic

n with the FD

e in Europe s

e drug is und

order. Selinc

consumption

rom the Com

n Q4 2012, w

way from its o

an markets,

d geographic

potential outs

undbeck, and

ducts – Xena

psy], Sycres

ith bipolar I d

am; seizures]

doubled in Q

d Lexapro ha

les to Q3 20

due to gener

011, where it

cts are expec

by 2015. Thi

eline assets t

rtioxetine (de

renia) once m

pendency). I

etine was sub

da, and Lund

c treatment A

DA in Septem

submission o

dergoing Pha

cro is expecte

n; an MAA wa

mmittee for M

with approval

H. LuLexap

old business

and instead

c footprint, g

side of Europ

d it will conti

azine (tetrab

st (asenapine

disorder], Lex

], and Treand

Q3 2012 and

as lost patent

12, this bran

ic erosion. N

t is still unde

cted to contr

s will be driv

that are expe

epression), A

monthly, and

In 2012 the

bmitted for re

dbeck is exp

Abilify once m

mber 2012, w

of an MAA is

ase III trials f

ed to be the f

as submitted

Medicinal Pro

l in H1 2013.

undbeck: spro expiry

model focus

building a m

iven that the

pe. As such,

nue its expa

benazine; Hu

e; moderate t

xapro (escita

da (bendamu

now represe

t protection i

nd experience

Nevertheless,

ergoing solid

ribute more

ven in part

ected to

Abilify

d Selincro

egistration in

ecting appro

monthly was

with approval

expected by

for acute sch

first treatmen

d in Decembe

ducts for Hu

.

shifting foy

Lundproexp

contrtha

reven

sed on a han

more diversifie

e company ha

China repre

nsion in this

ntington’s dis

to severe ma

alopram; dep

ustine; chron

ent 17% of co

n the US, an

ed an 89% d

, Lexapro wa

uptake.

the

oval by the en

submitted fo

expected du

y the end of 2

izophrenia a

nt to target re

er 2011, and

man Use (C

ocus after

dbeck’s newoducts are pected to ribute moran 50% of

nues by 201

ndful of

ed

as

esents a

market.

sease],

anic

pression]

nic

ompany

nd by

decline

as

nd of

or

uring H1

2012. In

and

eduction

HMP) is

w

re

15


9

Its leadT-DM1

regismetas

positiveand Im

expeapprov

ImmunoGe

proprietary

licenses its

antibodies;

Healthcare

trastuzuma

attached to

DM1 is und

ImmunoGe

in H2 2013

T-DM1’s co

In addition

companies

these are:

cancer, Ph

targets fola

ding candiis undergstration fostatic HER breast ca

mmunoGeecting FDAval in H1 2

en is a leadin

y Targeted An

s technology

in fact it has

e, and Sanofi

ab emtansine

o the HER2-b

dergoing reg

en is expectin

3.

ommercial po

to several pi

s, ImmunoGe

IMGN901, w

ase II, and m

ate receptor a

idate going or R2-ancer, en is A

2013

ng player in th

ntibody Paylo

to other com

s secured ke

. ImmunoGe

e (T-DM1), w

binding antib

istration for m

ng FDA appr

In add

an

ex

antibodi

otential throu

ipeline candi

en has three

which targets

multiple myel

alfa, which is

he antibody-

oad (TAP) te

mpanies for u

ey deals with

en’s leading p

which acts as

ody, Hercep

metastatic H

roval in H1 2

dition, the co

n adjuvant th

positive bre

expected

mechanis

also show

gastric ca

are expec

successful t

pansion stra

ies, ImmunoG

ugh its strate

idates that a

wholly owne

CD56-expre

oma, Phase

s over-expres

Imne

-drug conjuga

echnology. Im

use with their

Roche, Amg

pipeline cand

s a cancer-kil

ptin (trastuzum

ER2-positive

013, followed

mpound is b

herapy for ea

east cancer,

for 2018. Th

sm of action

wn potential

ancer, and re

cted in 2015.

track record

ategies for its

Gen should b

gic partner o

re being co-d

ed compound

essing cance

I); IMGN853

ssed on the

mmunoGeears the m

ate field than

mmunoGen o

r engineered

gen, Bayer

didate is

ling agent w

mab; Roche)

e breast canc

d by EMA ap

being investig

arly-stage HE

with results

hanks to its

, the therapy

in HER2-pos

esults from th

Given Roch

of indication

s own monoc

be able to m

of choice.

developed w

ds. Specifica

ers (small cel

3 (Phase I) w

most prevale

en: first prmarket

nks to its

out-

when it is

). T-

cer, and

pproval

gated as

ER2-

y has

sitive

his trial

e’s

clonal

aximize

with other

lly,

ll lung

which

ent

roducts


10

types of no

carcinomas

cell maligna

leukemia. I

on-small cell

s; and IMG52

ancies such

nitial data fro

lung cancers

29 (Phase I)

as non-Hodg

om these thr

s, ovarian ca

, a CD37-tar

gkin’s lymph

ree compoun

ancers, as we

rgeting TAP c

oma and chr

nds are expe

ell as other

conjugate to

ronic lympho

cted in 2013

treat B-

ocytic

3.


11

Medivation

treatment o

docetaxel i

following th

Dimebon (l

(although t

approved w

and since b

impressive

for Xtandi,

part to its s

drug by onc

physicians

Medivation

prechemot

most impor

represente

opportunity

expected in

positive, he

therapy, wh

would resu

for the drug

earlier-stag

that the ong

patients wit

declines of

naïve and c

induce tum

disease rat

’s lead produ

of metastatic

n August 20

he previous f

atrepirdine),

he company

with a hefty w

becoming av

$14m in sal

Astellas. At t

substantial pr

cologists 4 w

reporting po

emphasized

herapy trial,

rtant study fo

d a "gateway

y." This trial,

n the second

elp Xtandi to

hich is Mediv

lt in greater

g, thereby inc

ge clinical da

going Phase

th progressiv

f prostate-spe

chemotherap

mor regressio

te of 49% in

uct Xtandi (e

c castrate-res

12, allowing

failure with its

which led to

y did not allud

wholesale ac

vailable on Se

es, half of w

the conferen

romotional ef

weeks after it

ostchemother

d that the on

PREVAIL, w

or the compa

y to a larger

results for w

d half of 2014

move up to

vation’s ultim

numbers of p

creasing its s

ata with Xtand

e III trial will b

ve CRPC, Xt

ecific antigen

pyexperience

ns with a pa

soft tissues f

enzalutamide

sistant prosta

the compan

s late-stage A

o it being dro

de to this at t

cquisition cos

eptember 13

hich has gon

nce, Medivati

fforts, which

t was launch

rapy details f

going Phase

was now the

any, as it

commercial

which are

4, will, if

earlier lines

mate goal. Th

patients bein

sales potenti

di in the prec

be successfu

tandi was fou

n at all dosag

ed patients. T

rtial respons

for the overa

Medivateyes turprechem

emt

pre

e) was approv

ate cancer (C

y to breathe

Alzheimer’s

opped from M

the conferen

st of $7,450 f

3, the drug ha

ne to the com

ion attributed

led to an 88

ed as well as

for Septemb

e III

of

is

ng eligible

ial. The com

chemotherap

ul. In a Phase

und to induce

ges and in bo

The drug wa

se rate of 22%

all patient pop

tion: with rn to potemotherapy

Medivatimphasizedthe ongo

Phase Iechemoth

trial

ved for the

CRPC) follow

a sigh of rel

disease can

Medivation’s p

ce). Xtandi w

for a 30-day

as brought in

mpany’s co-li

d such high s

8% awarenes

s over 50% o

er.

pany discuss

py setting ind

e I/II trial in 1

e substantial

oth chemoth

as also found

% and stable

pulation.

Xtandi launtial in y setting

ion d that

oing III herapy

wing

ief

didate

pipeline

was

supply,

n an

censor

sales in

ss of the

of

sed how

dicate

40

l

erapy-

d to

e

unched,


12

Medivation

leading dru

Zytiga (abir

uptake in p

Medivation

Xtandi. In a

improveme

better than

prechemot

with metas

randomized

twice daily

primary end

statistically

arm of the

prednisone

versus a pr

However, t

since it was

significant

Medivation

including a

who have n

combinatio

discussed

Medivation

2012.

also pointed

ug for metast

raterone; Joh

prechemothe

believes this

addition, if Xt

ent in overall

Zytiga, whic

herapy trial.

tatic CRPC w

d to receive Z

or placebo p

dpoints. Inte

y significant im

study compa

e resulted in

respecified p

the endpoint

s an interim

OS benefit a

went on to d

Phase II tria

not yet been

on trial with X

its plans to e

began enro

d to the patte

tatic CRPC p

hnson & Joh

rapy patients

s indicates th

tandi can de

survival (OS

ch just misse

This trial, kn

who had not

Zytiga 1,000

plus predniso

rim analysis

mprovement

ared to the co

an estimated

p-value of 0.0

that was mis

analysis, and

at the end of

discuss the a

al in hormone

castrated, a

Xtandi in com

expand into a

lling patients

erns of use o

patients who

nson), which

s in the US s

hat a similar

monstrate a

S) in PREVA

ed significanc

nown as COU

received pri

0mg orally on

one 5mg twic

showed that

t in progress

ontrol arm, tr

d 33% impro

0008 to achie

ssed had a v

d so Zytiga c

the trial.

additional tria

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