Company Presentation September 2016

Transcription

1 Company Presentation September 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 Cell therapy (Bio-therapy) company using off-the-shelf, placental-expanded cells to achieve local and systemic therapeutic effects

4 Pluristem corporate overview Cell therapy company entering late-stage trials for 2 products in 3 indications No tissue matching or immunosuppression is required to administer our placenta-derived cell products First in class 3D cell culturing technology allowing for efficient, controlled production of different cell products in commercial quantities. Regulatory approval for clinical trials in US, EU, Japan, South Korea, Australia and Israel.

5 Financial glance Public company, Traded in: Market Cap: ~ $140 million PSTI PSTI/ PLTR Cash and marketable securities: $33 million (June 30, 2016) No debt Net burn: ~ $22 million No significant royalty liability (other than Israeli government) 170 employees (18 PhD, 5 MD) IP Ownership: over 75 granted patents and ~135 pending applications

6 Company Pipeline Pivotal pre-marketing trials Indication Product Location Pre-Clinical Phase 1 Phase 2 Phase 3 Market Critical Limb Ischemia (CLI)* PLX-PAD U.S. Europe Japan Conditional Approval Pathway Femoral Neck Fracture** Acute Radiation Syndrome (ARS) PLX- PAD PLX- R18 U.S./ Europe U.S. Pivotal study via FDA Animal Rule * One Multinational trial- U.S- phase 3, Europe- via adaptive pathway allowing early marketing approval ** Pending FDA/EMA approval

7 CLI & Market size 3-4 million people in major pharmaceutical markets* suffer from CLI (2010) Estimated cost for treating CLI is $12 billion per year Obstruction of arteries in the leg High mortality High amputation rates Poor treatment options Source: Lifecells LLC * (U.S., Europe)

8 Status of CLI Pivotal Trials Pivotal Phase III trial design approved by the FDA and planning to submit BLA following single positive pivotal study Pivotal phase III trial accepted to the Adaptive Pathways project in Europe- only six programs approved worldwide! Pivotal trial in Japan approved by the PMDA under an accelerated approval pathway U.S / Europe trial- N=250 Japan trial- N=75

9 CLI Development Plan Achievements Accepted to accelerated regulatory pathways by the European and Japanese regulatory authorities, significantly shortening time to market Targeting regulatory approval in both U.S. and Europe using data from a single pivotal Phase III trial in 250 patients Awarded $8 Million Grant from Europe s Horizon 2020 Program Program supported by the Israeli Chief Scientist

10 Study Design for U.S. Pivotal Phase 3 Trial (N=250) Primary endpoint is time to amputation or death Other methods of efficacy include: AFS, Quality of life, TcPO 2, Pain Score Dosing regimen: two doses of 300 million cells, two months apart No HLA matching or immunosuppression required

11 Early Clinical Studies Support Design of Pivotal Phase 3 Trial Amputation Free Survival at 6 months: US (total n=12) - 100% Germany (total n=15) - 93% Comparison to published data on no-option CLI: Pre-Treatment TASC II: 20% death and 40% major amputations in 6m TAMARIS (n=259 control pts.): 76% AFS in 6m (196/259) Meta-analyses (Benoit 2011, Weems 2015): 67%-77% AFS at 6m The majority of events usually occur in the first 6m 8 Weeks After Treatment

12 Early Clinical Studies Support Design of Pivotal Phase 3 Trial Amputation Free Survival at 12 months: US (total n=12) - 100% Germany (total n=15) - 73% Comparison to published data on no-option CLI: TASC II - (all CLI): 45% AFS, 30% MA & 25% death at 1y Reinecke (all CLI): 68% AFS at 1y R4+R5

13 Additional Supportive Data Quality of life (VascuQoL) - USA Quality of life (VascuQoL) - Germany Improvement of the quality of life within the first 3m Higher improvement at 2m repeated dosing Improvement in quality of life maintained for 12m

14 Additional Supportive Data Increase of mean TcPO 2 - USA Increase of mean TcPO 2 - Germany Improvement of TcPO 2 - after 1 month Earlier and higher improvement at 2m repeated dosing Improvement in TcPO 2 maintained for 24 months in German study

15 Additional Supportive Data Reduction of Pain Score (VAS) - USA Reduction of Pain Score (VAS) - Germany Overall pain decrease at month 3 Most notable decrease with repeated dose

16 Company Pipeline Pivotal pre-marketing trials Indication Product Location Pre-Clinical Phase 1 Phase 2 Phase 3 Market Critical Limb Ischemia (CLI) PLX-PAD U.S. Europe* Japan Conditional Approval Pathway Femoral Neck Fracture** Acute Radiation Syndrome (ARS) PLX- PAD PLX- R18 U.S./ Europe U.S. Pivotal study via FDA Animal Rule * One Multinational trial- U.S- phase 3, Europe- via adaptive pathway allowing early marketing approval ** Pending FDA/EMA approval

17 Orthopedic 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

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

19 Orthopedic Strong clinical data Muscle Injury following Total Hip Replacement (N=20) Improvement of 4000% P=0.012 Injured (operated) Contralateral (non operated)

20 Company Pipeline Pivotal pre-marketing trials Indication Product Location Pre-Clinical Phase 1 Phase 2 Phase 3 Market Critical Limb Ischemia (CLI) PLX-PAD U.S. Europe* Japan Conditional Approval Pathway Femoral Neck Fracture** Acute Radiation Syndrome (ARS) PLX- PAD PLX- R18 U.S./ Europe U.S. Pivotal study via FDA Animal Rule * One Multinational trial- U.S- phase 3, Europe- via adaptive pathway allowing early marketing approval ** Pending FDA/EMA approval

21 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

22 Company Pipeline Additional Clinical Trials Indication Intermittent Claudication (IC) Support for Hematopoietic Cell Transplantation Hematopoietic Cell Transplantation failure Pulmonary Arterial Hypertension (PAH) Product PLX-PAD PLX-R18 PLX- R18 PLX-PAD Pre-Clinical Phase 1 Phase 2 Phase 3 Market Cleared for U.S. FDA phase 1 study

23 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 NIH Placenta Project Launched by the U.S. National Institutes of Health (NIH) to Further explore the role of the placenta in health and disease.

24 Human placenta- a platform for cell products Each PLX Product Secretes a Different Range of Proteins to Address Different Varieties of Indications PLX-PAD Angiogenesis Culture conditions Culture conditions PLX-R18 Hematological PLX-CNS Neuronal Culture conditions Culture conditions PLX-IMMUNE Immunological Reduces inflammation Stimulates growth of collateral blood vessels Stimulates repair of damaged muscle Stimulates regeneration of damaged bone marrow to produce blood cells (white, red and platelets)

25 3D Manufacturing, in-house cell production 150,000 doses annually

26 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

27 Key Differentiators Late-stage bio-therapy company preparing for 3 pivotal trials PLX cell products each target different indications with customized cytokine secretion profiles Low immunogenicity confirmed and NO HLA-MATCHING required PLX products do not induce in vivo priming of Th1 response Repeated IM injections of PLX cells from the same placenta do not induce activation of patients memory T-cells Designed to be used off-the-shelf convenience in many medical settings

28 Key Differentiators In-house manufacturing with certified batch-to-batch consistency Proprietary 3D cell production process CMC & Manufacturing Facility for PLX-PAD approved by FDA and EU Regulators for 3D culturing for Phase II, III clinical trials and marketing

29 Collaborations Partner Indication Deal structure IC, CLI South Korea only Joint Venture following marketing authorization by the South Korean authorities Acute Radiation Syndrome U.S. National Institutes of Health (NIH) to Support Development of PLX-R18 Pluristem keeps IP and manufacturing rights in all collaborations Acute Radiation Syndrome Acute Radiation Syndrome CLI, Immunology, Cardiovascular, Orthopedic Pluristem will contribute cells and scientific knowledge, FMU will conduct the studies and provide the required resources. Conducting trials to test PLX-R18 cells in the treatment of ARS and understanding of MOA Research to test the unique immunology of the placenta and cells MOA

30 Company milestones- 12 months Initiate pivotal pre-marketing clinical trials in: Critical Limb Ischemia (CLI)- U.S and Europe- Phase 3 study (N=250) Japan- pivotal trial (N=75) via Conditional Approval pathway for regenerative medicine Acute Radiation Syndrome (ARS) Large animal study via FDA Animal Rule, funded by the NIH Hip Fracture Repair- Entering advanced clinical trials in U.S and Europe subject to regulatory approvals Complete enrollment In Intermittent Claudication (IC) trial ongoing in U.S, Germany, Israel and South Korea Patients recruitment for Phase 1 trial in incomplete engraftment of hematopoietic cell transplant cleared by the FDA

31 Investment Highlights Unmet Medical Need Significant Market Opportunity PLX cells Products Critical Limb Ischemia limited treatment options Muscle Injury need to improve muscle function following trauma 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 Off-the-shelf therapy, no tissue matching or immunosuppression Well-described mechanism of action Placenta-derived Convenient for use in most medical settings Pluristem Late-stage bio-therapy company preparing for 3 pivotal trials Primary endpoint met in all completed clinical trials FDA, EU and Japan approved manufacturing facility Highly efficient 3D cell production technology Broad platform with tailored products Strong balance sheet & IP

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

33 Thank you!