advancing cell therapeutic products for clinical use · advancing cell therapeutic products for...

Proprietary data of Pluristem Therapeutics Inc.1

Advancing cell therapeutic products for clinical use


This presentation concerning Pluristem Therapeutics may include forward-lookingstatements which represent Pluristem Therapeutics' expectations or beliefsregarding future events. I caution that such statements involve risks anduncertainties that may cause actual results to differ materially from those in theforward-looking statements. Consequently, all such forward-looking statementsare qualified by the cautionary language and risk factors set forth in PluristemTherapeutics' periodic and other reports filed with the SEC.

There can be no assurance that the actual results, events or developmentsreferenced in such forward-looking statements will occur or be realized. PluristemTherapeutics assumes no obligation to update these forward-looking statements toreflect actual results, changes in assumptions or changes in factors affecting suchforward-looking statements.

www.pluristem.com

Forward Looking Statement


• Cell therapy (Biotechnology) company.

• Using off the shelf placental expanded cells to achieve local and

systemic therapeutic effect.

• First in class 3D cell culturing technology allowing for efficient,

controlled production of different cell products in commercial

quantities.

• Active with regulators in USA, EU, South Korea, Australia andIsrael.

Pluristem Corporate Overview


• Public company, Traded in:

• Market Cap: ~ $130 million

• Cash and marketable securities: $38 million (March 31, 2016)

• No debt

• Net burn: ~ $22 million

• ~ 2 years of liquidity based on last 6 months burn rate

• No royalty liability (other than Israeli government)

• 165 employees (15 PhD, 5 MD)

• IP Ownership: over 60 granted patents and ~150 pending applications

Financial Glance

PSTI PSTI/ PLTR


Partner Indication Deal structure

IC, CLISouth Korea only

Joint Venture followingmarketing authorization by theSouth Korean authorities

Pluristem keeps IP and manufacturing rights in all collaborations

Collaborations

Government Partnership

Indication Deal structure

Acute Radiation Syndrome U.S. National Institutes ofHealth (NIH) to SupportDevelopment of PLX-R18

Acute Radiation Syndrome

Pluristem will contribute cellsand scientific knowledge, FMUwill conduct the studies andprovide the required resources.


Placenta derived cells• Rich & Diverse

• Highly potent

- pro-angiogenic

- immunoregulatory

• Young donors

• Unlimited source

• Easy to collect

• Ethically accepted

• Over 20,000 Doses of 300 million cells per placenta

The Placenta Project-Launched by the USNational Institutes ofHealth (NIH) toFurther explore therole of the placenta inhealth and disease.


The PLX Technology


Culture conditionsPLX-R18Hematological

PLX-PADAngiogenesis

Culture conditions

PLX-CNSNeuronal

PLX-IMMUNEImmunological

Human Placenta - A platform for cell productsEach PLX Product Secretes a Different Range of Proteins to Address

Different Varieties of Indications


3D manufacturing 150,000 doses annually


CMC & Manufacturing Facility for PLX-PAD approved by FDA,

German, EU, South Korean, Japanese & Israeli Regulatory Agencies for 3D culturing for

Phase II, III trials and marketing


• Broad platform with tailored products per indication• PLX cells can be customized to secrete a specific cytokine

secretion profile to target different indications• Real off the shelf product requiring no additional manipulation

at bedside

• Unique Immunological Properties• No HLA- matching required• Low immunogenicity confirmed :

• PLX does not induce in vivo priming of Th1 responses • Repeated IM injections of PLX cells from the same placenta

does not induce patients memory T-cells activation.

• Distinctive MOA• Products demonstrate both local and systemic efficacy via

intramuscular injection

How is PLX Different from the Competition


• Superior Manufacturing• In-house manufacturing which guarantees batch to

batch consistency and high margins

• Near Term Product Approval• First product approval expected 2018 for CLI

How is PLX Different from the Competition


Product 1: PLX-PAD• Reduces inflammation

• Stimulates growth of collateral blood vessels

• Stimulates repair of damaged muscle

Proprietary data of Pluristem Therapeutics Inc.14 Proprietary data of Pluristem Therapeutics Inc.

• 1.7 million people in the major pharmaceutical markets* suffer from CLI (2010) & Expected to increase to 2 million in 2020

• Estimated cost for treating CLI in the U.S. is $10 billion per year

• Obstruction of arteries in the leg• High mortality• High amputation rates• Poor treatment options

* U.S., France, Germany, U.K., Italy, Spain and Japan

CLI & Market Size


Strategy to Shorten Time to CLI Market

Positive clinical data

Manufacturing facility & production process approved by U.S., Europe and Japan

Target regulatory pathways that allow for early entry into the market

Selected for Europe’s Adaptive Pathways project

Cleared to begin clinical trial via Japan’s rapid regulatory pathway for regenerative medicine targeting conditional marketing authorization


• Two Phase I/II Critical Limb Ischemia (N=27)

Pre-Treatment 8 Weeks After Treatment

Pre-Treatment

8 Weeks After

Treatment

Clinical Data Support Shorten Time to CLI Market

6-8 month follow-up


• Muscle Injury following Total Hip Replacement (N=20)

PLX-PAD - Strong Clinical Data

Improvement of 500%

P=0.0067

Change at week 26 in Mean (±SE) Gluteus Medius MVIC from Day 0 (mITT)

MVIC = Maximum Voluntary Isometric Construction




Change in Volume from Day 0




Injured (operated) Contralateral(non–operated)


Product 2: PLX-R18

• Stimulates regeneration of damaged bonemarrow to produce blood cells(white, red and platelets)

PLX-R18 Pipeline:


U.S. National Institutes of Health to Support Development of Pluristem's PLX-R18

Collaboration for ARS with US Government


U.S. National Institutes of Health to Support Development of Pluristem's PLX-R18

Collaboration for ARS with US Government

Collaboration with Fukushima Medical University and Science Center to develop PLX-R18 cells for the treatment

of other component of ARS ( GI, Lung and Skin), and for morbidities following radiotherapy in cancer patients.


Company Milestones 2016• Initiate three pre-marketing clinical trials

Critical Limb Ischemia (CLI)- Europe

Critical Limb Ischemia (CLI)- Japan

Acute Radiation Syndrome (ARS) – U.S. trial (viaAnimal Rule)

• Complete enrollment In Intermittent Claudication (IC) trial ongoing in U.S, Germany, Israel and South Korea

• Initiate Phase 1 trial in incomplete engraftment of hematopoietic cell transplant – U.S. FDA cleared IND

• Add clinical development program for orthopedic indication into Adaptive Pathways project


Investment HighlightsUnmet Medical Need

• Critical Limb Ischemia – limited treatment options• Muscle Injury – need to improve muscle function in the elderly• Damaged or poorly functioning Bone Marrow – ARS, HCT• Preeclampsia – no treatment exists, orphan drug status

Significant Market Opportunity

• CLI – $12 billion global market• Orthopedic indications – broad markets• Hematologic indications – broad markets• Incidence of many indications increasing in aging populations

PLX cells Differentiation

• Off-the-shelf therapy, no tissue matching needed• Positive data in 3 completed clinical trials• Well-described mechanism of action• Placenta-derived

Pluristem Differentiation

• Expect to initiate 3 pivotal trials in 2016• Broad pipeline• FDA and EU – approved manufacturing facility• 3D cell product production technology • Strong balance sheet & IP


Zami AbermanChairman & CEO

Efrat Livne-Hadass VP Human Resources

Racheli Ofir, Ph.D.VP Research & Intellectual Property

Sagi MoranVP Operations

Erez Egozi

VP Finance

Karine Kleinhaus, M.D., MPHDivisional VP, North America

Yaky YanayPresident & COO

Hillit Mannor Shachar, M.D., M.B.A.VP Business Development

Lior RavivDirector of Development

Esther Lukasiewicz Hagai, M.D., Ph.D. VP Clinical & Medical Affairs

Orly AmiranVP Quality Assurance

Management Team

advancing cell therapeutic products for clinical use · advancing cell therapeutic products for...

Documents