Jefferies Healthcare Conference 2016

Transcription

1 Jefferies Healthcare Conference 2016

2 Forward looking Statement This presentation concerning Pluristem Therapeutics may include forward-looking statements which represent Pluristem Therapeutics' expectations or beliefs regarding future events. I caution that such statements involve risks and uncertainties that may cause actual results to differ materially from those in the forward-looking statements. Consequently, all such forward-looking statements are qualified by the cautionary language and risk factors set forth in Pluristem Therapeutics' periodic and other reports filed with the SEC. There can be no assurance that the actual results, events or developments referenced in such forwardlooking statements will occur or be realized. Pluristem Therapeutics assumes no obligation to update these forward-looking statements to reflect actual results, changes in assumptions or changes in factors affecting such forward-looking statements.

3 Pluristem corporate overview Placenta-derived cell products target serious diseases with poor treatment options & require no tissue matching Patented 3D cell culturing technology enables efficient, controlled production of cell products in commercial quantities Manufacturing process & facility approved by U.S., Europe & Japan Initiating clinical trials targeting marketing authorization

4 Financial glance Public company: Market Cap: ~ $130 million PSTI PSTI/ PLTR Cash and marketable securities: $38 million (March 31, 2016) No debt IP Ownership: over 60 granted patents and ~150 pending applications Fully owned and operated manufacturing facility

5 The PLX technology

6 3D Manufacturing In-house cell production Promotes consistency and limits risk Allows us to control our cell supply Reduces costs 150,000 doses annual capacity

7 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

8 Product 1: PLX-PAD Reduces inflammation Stimulates growth of collateral blood vessels Stimulates repair of damaged muscle

9 CLI & Market size 2 million people in the U.S. and 3 million people in Western Europe 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

10 Clinical data support shortened time to CLI market Two Phase I/II Critical Limb Ischemia (N=27) 6-8 month follow-up Pre-Treatment 8 Weeks After Treatment

11 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 targeting conditional marketing authorization via Japan s rapid regulatory pathway for regenerative medicine

12 PLX-PAD Strong Clinical Data Muscle Injury following Total Hip Replacement (N=20) Change at week 26 in Mean (±SE) Gluteus Medius MVIC from Day 0 (mitt) Improvement of 500% P= MVIC = Maximum Voluntary Isometric Construction

13 PLX-PAD Strong clinical data Muscle Injury following Total Hip Replacement (N=20) Improvement of 300% P= Change in Volume from Day 0

14 Product 2: PLX-R18 Stimulates regeneration of damaged bone marrow to produce blood cells (white, red and platelets)

15 Collaboration for ARS with U.S. Government U.S. National Institutes of Health to Support Development of Pluristem's PLX-R18 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

16 Human placenta a platform for cell products Each PLX Product Secretes a Different Range of Proteins to Address Different Indications PLX-PAD Angiogenesis Culture conditions Culture conditions PLX-R18 Hematological PLX-CNS Neuronal Culture conditions Culture conditions PLX-IMMUNE Immunological

17 How is Pluristem different? Broad platform with products tailored to indications 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 Strong Clinical Data

18 How is Pluristem different? Distinctive Mechanism of Action Superior Manufacturing Targeting Near Term Product Approval for Critical Limb Ischemia

19 Collaborations Partner Indication Deal structure IC, CLI South Korea only Joint Venture following marketing authorization by the South Korean authorities Government Partnership Indication Deal structure Acute Radiation Syndrome U.S. National Institutes of Health (NIH) to Support Development of PLX-R18 Acute Radiation Syndrome Pluristem will contribute cells and scientific knowledge, FMU will conduct the studies and provide the required resources. Pluristem keeps IP and manufacturing rights in all collaborations

20 Company milestones 2016 Initiation of pre-marketing trials Critical Limb Ischemia (CLI)- Europe Critical Limb Ischemia (CLI)- Japan Completion of U.S. NIH Acute Radiation Syndrome (ARS) dose selection trial Completion of enrollment for Intermittent Claudication (IC) trial ongoing in U.S, Germany, Israel and South Korea Initiation of Phase 1 trial in incomplete engraftment of hematopoietic cell transplant Add clinical development program for orthopedic indication into Adaptive Pathways project

21 Management team Zami Aberman Chairman & CEO Yaky Yanay President & COO Efrat Livne-Hadass VP Human Resources Racheli Ofir, Ph.D. VP Research & Intellectual Property Sagi Moran VP Operations Hillit Mannor Shachar, M.D., M.B.A. VP Business Development Lior Raviv Director of Development Esther Lukasiewicz Hagai, M.D., Ph.D. VP Clinical & Medical Affairs Erez Egozi VP Finance Orly Amiran VP Quality Assurance Karine Kleinhaus, M.D., MPH Divisional VP, North America

22 Investment Highlights Unmet Medical Needs Significant Market Opportunity Critical Limb Ischemia limited treatment options Muscle Injury need to improve muscle function and rehab times after trauma, sports injuries Damaged or poorly functioning Bone Marrow ARS, HCT CLI $12 billion global market Orthopedic indications broad markets Hematologic indications broad markets Incidence of many indications increasing in aging populations PLX cells No tissue matching needed Positive data in 3 completed clinical trials Well-described mechanism of action Placenta-derived Pluristem Expect to initiate pre-marketing trials in 2016 Broad pipeline FDA and EU-approved manufacturing facility 3D cell product production technology Strong balance sheet & IP

23 Development Partners 23 Franziskus Hospital Berlin Berlin-Brandenburg Center for Regenerative Therapies Fraunhofer Institute for Cell Therapy and Immunology IZI in Leipzig NYU Langone Medical Center Hadassah Medical Center Stanford University School of Medicine Rambam Health Care Campus Duke University School of Medicine Sheba Medical Center Center for Therapeutic Angiogenesis Technion Israel Institute of Technology The Hebrew University Jerusalem Tel Aviv University

24 Thank you!