Pluri Inc. · NASDAQ:PLUR
Pluri — Pluristem Therapeutics — Investor Presentation (May 2015)
44 slides from Pluri's investor presentation, May 27, 2015
Slide 1: Advancing cell therapeutic products for clinical use
1 Advancing cell therapeutic products for clinical use
Slide 2: Forward-looking statements
Proprietary data of Pluristem Therapeutics Inc. 2 This presentation contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and federal securities laws. For example, we are using forward-looking statements when we
Slide 3: Corporate Overview
Proprietary data of Pluristem Therapeutics Inc. 3 • Cell therapy company (NasdaqCM: PSTI, TASE: PLTR) • Using off-the-shelf placental expanded cells to achieve both local and systemic therapeutic effects • First in class 3D cell culturing technology allowing for efficient, controlled production of d
Slide 4: Strong Clinical Data
Proprietary data of Pluristem Therapeutics Inc. 4 Strong Clinical Data Muscle Injury following Total Hip Replacement N=20
Slide 5: Correlation Between Improvement in the Muscle Force of Injured and Contralateral Leg
Proprietary data of Pluristem Therapeutics Inc. 5 Correlation Between Improvement in the Muscle Force of Injured and Contralateral Leg Contralateral (non-operated) Injured (operated)
Slide 6: PLX Technology
Proprietary data of Pluristem Therapeutics Inc. 6 PLX Technology
Slide 7: Four Distinct Products Derived from Placenta
Proprietary data of Pluristem Therapeutics Inc. 7 Culture conditions Culture conditions PLX-R18 Hematological PLX-PAD PLX-CNS Neuronal PLX-IMMUNE Immunological Human Placenta- A platform for distinct cell products Four Distinct Products Derived from Placenta
Slide 8: PLX-PAD CMC approved by 5 Regulatory agencies
Proprietary data of Pluristem Therapeutics Inc. 8 Manufacturing facility and PLX-PAD CMC (3D culturing) for Phase II/III and marketing* approved by FDA, PEI (Germany), EMA, Korean & Israeli regulatory agencies Batch-to-Batch comparability confirmed, multiple donors * Subject to supplier approval PLX
Slide 9: Proprietary data of Pluristem Therapeutics Inc. 9
Proprietary data of Pluristem Therapeutics Inc. 9
Slide 10: Selected for EU Adaptive Pathway - potential for marketing approval in 2018
Proprietary data of Pluristem Therapeutics Inc. 10 Selected for EU Adaptive Pathway - potential for marketing approval in 2018
Slide 11: Progression of Atherosclerotic Lesions
Proprietary data of Pluristem Therapeutics Inc. 11 Progression of Atherosclerotic Lesions From: Peripheral Arterial Disease. Source: Decision Resources Created for Hillit Mannor Shachar, Pluristem Therapeutics. IP Address: (Unknown) © 2011 Decision Resources, Inc.
Slide 12: Therapeutic Angiogenesis by Growth Factors
Proprietary data of Pluristem Therapeutics Inc. 12 Therapeutic Angiogenesis by Growth Factors From: Peripheral Arterial Disease. Source: Decision Resources Created for Hillit Mannor Shachar, Pluristem Therapeutics. IP Address: (Unknown) © 2011 Decision Resources, Inc.
Slide 13: PLX-PAD secretion profile
Proprietary data of Pluristem Therapeutics Inc. 13 Proangiogenic VEGF, HGF, TGF b, Angiogenin, IL-8, Angiopoietin, IL-6, MMP-1, TIMP-1, PAI-1, IGFBPs, Immunomodulatory HGF, IDO, IL-28, IL-22, IL-10RA, Antifibrotic HGF, Decorin Protrophic - LIF, Angiopoietin, b-NGF PLX-PAD secretion profile
Slide 14: PLX-PAD increased blood flow in the tibia
Proprietary data of Pluristem Therapeutics Inc. 14 Placebo PLX-PAD treated PLX-PAD increased blood flow in the tibia Live Cellvizio imaging following IV administration of FITC labeled Dextran
Slide 15: PLX-PAD in Peripheral Artery Disease
Proprietary data of Pluristem Therapeutics Inc. 15 • July 2009 - First CLI patient injected in Berlin (N=15) • October 2010 - last CLI Patient injected in US (N=12) • April 2012 - EMA declaration - Adaptive Pathways program • May 2012 - End of 2 years patients safety follow-up in Europe • April 2012
Slide 16: Base scenario peak sales and NPV
Proprietary data of Pluristem Therapeutics Inc. 16 Without the EMA approval, Peak sales in 2033. Reduce the NPV by $200M (~25%) COMPANY CONFIDENTIAL In the base scenario, PLX-PAD has the potential to achieve peak sales of ~$350M in the US And ~ $290M in Europe NPV - Base Scenario Peak sales in 2030
Slide 17: Upside scenario peak sales and NPV
Proprietary data of Pluristem Therapeutics Inc. 17 Without the EMA approval, Peak sales in 2033. Reduce the NPV by $700M (~ 25%) COMPANY CONFIDENTIAL In the upside scenario, PLX-PAD has the potential to achieve peak sales of ~$1.4B in the US and ~$1B in Europe NPV Upside Scenario Peak sales in 2030
Slide 18: PLX-PAD CLI program status in Europe
Proprietary data of Pluristem Therapeutics Inc. 18 PLX-PAD CLI program status in Europe • 3-4 years earlier in a multi-billion market • Manufacturing facility approved and inspected by European Qualified Person for Phase III and marketing • Granted European patent for the use of PLX in ischemic dise
Slide 19: Proprietary data of Pluristem Therapeutics Inc. 19
Proprietary data of Pluristem Therapeutics Inc. 19
Slide 20: Management Team
Proprietary data of Pluristem Therapeutics Inc. 20 Zami Aberman Chairman & CEO Efrat Livne-Hadass VP Human Resources Racheli Ofir, Ph.D. VP Research & Intellectual Property Sagi Moran VP Operations Erez Egozi VP Finance Karine Kleinhaus, M.D., MPH Divisional VP, North America Yaky Yanay President &
Slide 21: New Regulatory Approaches for Accelerated Approval of PLX-PAD in Critical Limb Ischemia
Proprietary data of Pluristem Therapeutics Inc. 21 New Regulatory Approaches for Accelerated Approval of PLX-PAD in Critical Limb Ischemia Dr Esther Lukasiewicz-Hagai, MD, PhD VP Medical and Clinical Affairs
Slide 22: Introduction
Proprietary data of Pluristem Therapeutics Inc. 22 • Pluristem is highly committed to provide early access of PLX-PAD to Critical limb ischemia (CLI) patients all over the world • Pluristem is taking advantage of each new regulatory opportunity to achieve this goal: Ø In Europe : New Adaptive Pathwa
Slide 23: Completed, ongoing and planned studies in PAOD
Proprietary data of Pluristem Therapeutics Inc. 23 Completed, ongoing and planned studies in PAOD • 2 completed Phase I studies in Critical Limb Ischemia (n= 27) • 1 ongoing Phase 2 study in Intermittent claudication (n=150) • 2 planned studies in CLI: ü 1 Phase II/III in Europe via Adaptive Pathway
Slide 24: Completed Phase I Studies in CLI
Proprietary data of Pluristem Therapeutics Inc. 24 PLX-PAD dose U.S. study n=12 ( 8 Ruth 4/4 Ruth 5) German study n=15 (9 Ruth 4/6 Ruth 5) 200x 10 6 - Single course 50 injections (n=3) 300x 10 6 Single course 30 injections (n=5) Single course 50 injections (n=6) 600x 10 6 2 courses of 300 10 6 at 2
Slide 25: Strong Clinical Data from 2 Phase I/II CLI Trials
Proprietary data of Pluristem Therapeutics Inc. 25 Event Rate USA data (AFS- 100%) Germany data (AFS- 73%) Average (85%) Published historical data Strong Clinical Data from 2 Phase I/II Critical Limb Ischemia Trials(N=27) 12 month - 85% Amputation-Free Survival (AFS)
Slide 26: Main studies results
Proprietary data of Pluristem Therapeutics Inc. 26 • Good Safety profile • Trends in efficacy with improvement from baseline in: – Transcutaneous Oxygen Pressure (limb perfusion) – Quality of Life – Pain score Main studies results Pre-Treatment 8 Weeks After Treatment
Slide 27: EMA Adaptive Pathways
Proprietary data of Pluristem Therapeutics Inc. 27 EMA Adaptive Pathways
Slide 28: EMA Adaptive Pathways - Background
Proprietary data of Pluristem Therapeutics Inc. 28 EMA Adaptive Pathways - Background Pilot project launched by EMA on March 19 th , 2014 : • European initiative intended to grant earlier access to drugs meant to treat debilitating and/or life-threatening diseases with unmet medical need • Only for
Slide 29: EMA Adaptive Pathways - General principles
Proprietary data of Pluristem Therapeutics Inc. 29 EMA Adaptive Pathways - General principles: • Early approval of a drug for a restricted patient population followed by progressive adaptations of the marketing authorization to expand access to the drug to broader patient populations based on data g
Slide 30: Adaptive vs. traditional Pathway to Market
Proprietary data of Pluristem Therapeutics Inc. 30 Adaptive vs. traditional Pathway to Market Phase II RCT CLI (niche population) Conditional MA in niche population 2016 2017 2018 2019 2020 2021 Confirmation for the CLI subgroup (Phase III/post- marketing registries) + Additional studies in broader
Slide 31: Adaptive Pathways for PLX-PAD in CLI
Proprietary data of Pluristem Therapeutics Inc. 31 Adaptive Pathways for PLX-PAD in CLI • Severely debilitating and life-threatening disease : overall 30% of CLI patients have amputations and 25% die by 1 year after diagnosis (higher rates in CLI patients that cannot undergo revascularization) • Unm
Slide 32: Clinical development of PLX-PAD
Proprietary data of Pluristem Therapeutics Inc. 32 Clinical development of PLX-PAD Subgroup of CLI patients at high risk (Phase II trial /Surrogate endpoint) Initial approval in selected subgroup of CLI patients Expansion to broader CLI population (RCT / RWD) Expansion to additional indication/s Bas
Slide 33: Expedited approval for regenerative therapy in Japan
Proprietary data of Pluristem Therapeutics Inc. 33 Expedited approval for regenerative therapy in Japan
Slide 34: Background
Proprietary data of Pluristem Therapeutics Inc. 34 Background New Japanese regulations from Nov 2014 to accelerate development of drugs in the field of regenerative therapy: • Only for Regenerative therapy • Seriously-debilitating / Life-threatening indication • Unmet medical need
Slide 35: Accelerated vs. traditional approval process
Proprietary data of Pluristem Therapeutics Inc. 35 Accelerated vs. traditional approval process • Conditional time-limited approval can be obtained based on proof of safety and “ limited proof of efficacy ” (Phase I/II, surrogate endpoint) • Up to 7 years to confirm efficacy post conditional approva
Slide 36: Clinical development of PLX-PAD for CLI
Proprietary data of Pluristem Therapeutics Inc. 36 Clinical development of PLX-PAD for CLI Phase I/II trial (limited proof of efficacy) Time-limited conditional approval Final/Additional Approval Registry of patients treated with PLX-PAD for pharmacovigilance
Slide 37: Regulatory timeline 2015-2021 for EU and Japan
Proprietary data of Pluristem Therapeutics Inc. 37 2015 2016 2017 2018 2019 2020 2021 EMA initiation PIP Waiver ATMP Class. SA on CMC SA with EMA/HTA Phase II/III (EU) CTA Subm. Phase II Phase III/Long term follow up Registry of patients treated with PLX-PAD MA (EMA) CMA Presub. meeting MAA subm. MA
Slide 38: Conclusion
Proprietary data of Pluristem Therapeutics Inc. 38 Conclusion In the framework of the 2 new regulatory approaches that exist in Europe and in Japan , Pluristem plan to initiate 2 clinical studies in CLI in 2016 with the aim of obtaining initial approval already in 2018
Slide 39: So, what's next?
Proprietary data of Pluristem Therapeutics Inc. 39 • Time to Market (TTM)- we will be focus on adaptive pathways Ø Europe Ø Japan Ø Our goal is to file early/conditional approval by the end of 2017/early 2018 • Launching PLX-R18 studies for hematology and ARS • Building platform for new products- se
Slide 40: Proprietary data of Pluristem Therapeutics Inc. 40
Proprietary data of Pluristem Therapeutics Inc. 40
Slide 41: Collaborations
Proprietary data of Pluristem Therapeutics Inc. 41 Partner Indication Deal structure Upfront payment of $7M, additional $48M in milestones, cells supply (cost +) and royalties in gross margin IC, CLI South Korea only JV following marketing authorization of the Korean authorities Acute Radiation Synd
Slide 42: Company milestones
Proprietary data of Pluristem Therapeutics Inc. 42 IC CLI (Adaptive EU) CLI (Japan) Orthopedic indication Hematology ARS PAH Preeclampsia End of enrollment Proprietary data of Pluristem Therapeutics Inc. indication End of enrollment Data Readout Licensing deal Study initiation Adaptive Pathway appro
Slide 43: Company milestones
Proprietary data of Pluristem Therapeutics Inc. 43 Proprietary data of Pluristem Therapeutics Inc. 43 indication Technologies New products (Tox ready) Thawing device Plurispheare MP4 NewPro1 PLX-R18 MP5 NewPro2 NewPro3 Company milestones
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