company presentation may 2016 forward looking statement

Company Presentation - May 2016 Forward looking Statement This - PowerPoint PPT Presentation

Company Presentation - May 2016 Forward looking Statement This presentation concerning Pluristem Therapeutics may include forward-looking statements which represent Pluristem Therapeutics' expectations or beliefs regarding future events. I

  1. Company Presentation - May 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 forward- looking 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 • 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. • Entering 3 pivotal pre-marketing clinical trials. • Active with regulators in USA, EU, Japan, South Korea, Australia and Israel.

  4. Financial glance • Public company, Traded in: PSTI PSTI/ PLTR • Market Cap: ~ $130 million • Cash and marketable securities: $38 million (March 31, 2016) • No debt • Net burn: ~ $22 million • No significant royalty liability (other than Israeli government) • 165 employees (15 PhD, 5 MD) • IP Ownership: over 60 granted patents and ~150 pending applications

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

  6. Placenta derived cells • Rich & Diverse • Highly potent • pro-angiogenic • immunoregulatory • Young donors • Unlimited source • Easy to collect The NIH Placenta Project Launched by the US National • Ethically accepted Institutes of Health (NIH) to Further explore the role of the • Over 20,000 Doses of 300 million cells per placenta placenta in health and disease.

  7. The PLX technology

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

  9. 3D Manufacturing, in-house cell production 150,000 doses annually

  10. 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

  11. How is PLX different from the competition?  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 •

  12. 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 •

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

  14. CLI & Market size  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 Source: Lifecells LLC * U.S., France, Germany, U.K., Italy, Spain and Japan

  15. 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

  16. Clinical data support shorten time to CLI market Two Phase I/II Critical Limb Ischemia (N=27) Pre-Treatment 8 Weeks After Treatment

  17. PLX-PAD – Strong clinical data Muscle Injury following Total Hip Replacement (N=20) Change at week 26 in Mean ( ± SE) Improvement of Gluteus Medius 500% MVIC from Day 0 P=0.0067 (mITT) MVIC = Maximum Voluntary Isometric Construction

  18. PLX-PAD – Strong clinical data Muscle Injury following Total Hip Replacement (N=20) Change in Volume from Day 0 Improvement of 300% P=0.004

  19. PLX-PAD – Strong clinical data Muscle Injury following Total Hip Replacement (N=20) Contralateral Injured (operated) (non–operated)

  20. Product 1: PLX-R18 Stimulates regeneration of damaged bone marrow to produce • blood cells (white, red and platelets)

  21. Collaboration for ARS with US 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

  22. Company milestones 2016 Initiate pivotal pre-marketing clinical trials: Critical Limb Ischemia (CLI)- Europe • • Critical Limb Ischemia (CLI)- Japan • Acute Radiation Syndrome (ARS) – U.S. trial (via Animal 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 •

  23. Investment Unmet Medical Critical Limb Ischemia – limited treatment options • • Muscle Injury – need to improve muscle function in the elderly Need Highlights Damaged or poorly functioning Bone Marrow – ARS, HCT • • Preeclampsia – no treatment exists, orphan drug status Significant • CLI – $12 billion global market Orthopedic indications – broad markets • Market Hematologic indications – broad markets • Opportunity • Incidence of many indications increasing in aging populations PLX cells • Off-the-shelf therapy, no tissue matching needed Positive data in 3 completed clinical trials • Differentiation • Well-described mechanism of action Placenta-derived • Expect to initiate 3 pivotal trials in 2016 Pluristem • Broad pipeline • Differentiation • FDA and EU – approved manufacturing facility 3D cell product production technology • • Strong balance sheet & IP

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

  25. Thank you!


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