Advancing Cell Therapeutic Products for Clinical Use
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Advancing cell therapeutic products for clinical use 1 Proprietary data of Pluristem Therapeutics Inc. 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. www.pluristem.com 2 Proprietary data of Pluristem Therapeutics Inc. 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. • Active with regulators in USA, EU, South Korea, Australia and Israel. 3 Proprietary data of Pluristem Therapeutics Inc. 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 • ~ 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 4 Proprietary data of Pluristem Therapeutics Inc. Collaborations Partner Indication Deal structure IC, CLI Joint Venture following marketing authorization by the South Korea only South Korean authorities Government Indication Deal structure Partnership Acute Radiation Syndrome U.S. National Institutes of Health (NIH) to Support Development of PLX-R18 Pluristem will contribute cells and scientific knowledge, FMU Acute Radiation Syndrome will conduct the studies and provide the required resources. Pluristem keeps IP and manufacturing rights in all collaborations 5 Proprietary data of Pluristem Therapeutics Inc. 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 US National Institutes of Health (NIH) to Further explore the role of the placenta in health and disease. 6 Proprietary data of Pluristem Therapeutics Inc. The PLX Technology 7 Proprietary data of Pluristem Therapeutics Inc. 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 conditions Culture conditions Angiogenesis Hematological PLX-CNS PLX-IMMUNE Neuronal Immunological 8 Proprietary data of Pluristem Therapeutics Inc. 3D manufacturing 150,000 doses annually 9 Proprietary data of Pluristem Therapeutics Inc. 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 10 Proprietary data of Pluristem Therapeutics Inc. 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 11 Proprietary data of Pluristem Therapeutics Inc. 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 12 Proprietary data of Pluristem Therapeutics Inc. Product 1: PLX-PAD • Reduces inflammation • Stimulates growth of collateral blood vessels • Stimulates repair of damaged muscle 13 Proprietary data of Pluristem Therapeutics Inc. 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 * U.S., France, Germany, U.K., Italy, Spain and Japan 14 Proprietary data of PluristemPluristem TherapeuticsTherapeuticsInc. Inc. 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 15 Proprietary data of PluristemPluristem TherapeuticsTherapeuticsInc. Inc. Clinical Data Support Shorten Time to CLI Market • Two Phase I/II Critical Limb Ischemia (N=27) Pre-Treatment Pre-Treatment 8 Weeks After6- 8Treatmentmonth follow-up 8 Weeks After Treatment 16 Proprietary data of Pluristem Therapeutics Inc. 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=0.0067 MVIC = Maximum Voluntary Isometric Construction 17 Proprietary data of Pluristem Therapeutics Inc. PLX-PAD - Strong Clinical Data • Muscle Injury following Total Hip Replacement (N=20) Change in Volume from Day 0 18 Proprietary data of Pluristem Therapeutics Inc. PLX-PAD - Strong Clinical Data • Muscle Injury following Total Hip Replacement (N=20) Injured (operated) Contralateral (non–operated) 19 Proprietary data of Pluristem Therapeutics Inc. Product 2: PLX-R18 • Stimulates regeneration of damaged bone marrow to produce blood cells (white, red and platelets) PLX-R18 Pipeline: 20 Proprietary data of Pluristem Therapeutics Inc. Collaboration for ARS with US Government U.S. National Institutes of Health to Support Development of Pluristem's PLX-R18 21 Proprietary data of Pluristem Therapeutics Inc. 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. U.S. National Institutes of Health to Support Development of Pluristem's PLX-R18 22 Proprietary data of Pluristem Therapeutics Inc. 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 (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 Proprietary data of PluristemPluristem TherapeuticsTherapeuticsInc. Inc. Investment Highlights Unmet Medical • Critical Limb Ischemia – limited treatment options Need • 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 • CLI – $12 billion global market Opportunity • Orthopedic indications – broad markets • Hematologic indications – broad markets • Incidence of many indications increasing in aging populations PLX cells • Off-the-shelf therapy, no tissue matching needed Differentiation • Positive data in 3 completed clinical trials • Well-described mechanism of action • Placenta-derived Pluristem • Expect to initiate 3 pivotal trials in 2016 Differentiation • Broad pipeline • FDA and EU – approved manufacturing facility • 3D cell product production technology • Strong balance sheet & IP 24 Proprietary data of Pluristem Therapeutics Inc. 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 Racheli Ofir, Ph.D. Lior Raviv VP Research & Intellectual Property Director of Development Sagi Moran Esther Lukasiewicz Hagai, M.D., Ph.D. VP Operations VP Clinical & Medical Affairs Erez Egozi Orly Amiran VP Finance VP Quality Assurance Karine Kleinhaus, M.D., MPH Divisional VP, North America 25 Proprietary data of Pluristem Therapeutics Inc. 26 Proprietary data of Pluristem Therapeutics Inc..