Gamida Cell — Investor Presentation (August 2017)
43 slides from Gamida Cell's investor presentation, August 2017
Slide 1: Company Overview
August 2017 Company Overview Curative Treatments for Cancer and Orphan Genetic Diseases
Slide 2: Curative Treatments for Orphan Indications
Curative Treatments for Orphan Indications • NiCord® - a bone marrow transplantation treatment for patients with high risk leukemia and lymphoma ─ Breakthrough Therapy designation by FDA ─ Phase 3 registration study is enrolling patients ─ Positioned to become the graft of choice for patients with n
Slide 3: Clinical Stage Therapies Addressing Orphan Indications
Clinical Stage Therapies Addressing Orphan Indications Platform Technology with Multiple Pipeline Products Product Research Pre-Clinical Phase 1/2 Phase 3 Status Phase 3 is NiCord® High Risk Hematological Malignancies enrolling Sickle Cell Disease, Thalassemia Data presented at ASH 2016 CordIn™ Apla
Slide 4: NiCord®
NiCord® Breakthrough Therapy Designation (First in BMT) Orphan Drug Designation Phase 3 is enrolling patients
Slide 5: BMT is a Potential Cure for High Risk Blood Cancers, But Limited by Availability
BMT is a Potential Cure for High Risk Blood Cancers, But Limited by Availability and Quality of Donor Cells • Re-shaped treatment and prognosis of cancer patients • A curative therapy also for rare genetic diseases such as sickle cell disease and thalassemia • Requires full tissue matching between t
Slide 6: Half of Indicated Patients Never Get to Transplant
Half of Indicated Patients Never Get to Transplant 70% of Patients Have a Low or Intermediate Probability of Finding a Match need donor derived ~70,000 (allogeneic) patients bone marrow matched each year transplantation unrelated donor 30% 40,000 in U.S., Europe & Japan 40% patients never get altern
Slide 7: Cord Blood Stem Cells Offer a Potential Solution
Cord Blood Stem Cells Offer a Potential Solution No Need for Full Tissue Matching, Can Practically be Available to all Patients in Need Advantages Limitations • Cord Blood (CB) is readily and Limiting Cell Dose Higher Morbidity and Non-Relapse Mortality rapidly available from public CB banks worldwi
Slide 8: NAM Technology Expands Cord Blood Stem Cell Population While Preserving Functionality
NAM Technology Expands Cord Blood Stem Cell Population While Preserving Functionality • Epigenetic regulation by the small molecule, nicotinamide (NAM), a Successful homing to the bone marrow multifunctional derivative of Rolling Integrin Activation vitamin B3 Arrest Transendothelial • Addresses the
Slide 9: NAM Technology Improves Graft Functionality
NAM Technology Improves Graft Functionality Platform to Pipeline Products Preserved Gene Expression Active molecule is NAM Non- • Precursor of NAD, inhibits enzymes that use cultured NAD, including Sir2 (SIRT1-7), class-III NAD-dependent histone-deacetylase Cultured • Modulates cell energy and metab
Slide 10: NiCord® Manufacturing Process is Validated and Cost Effective
NiCord® Manufacturing Process is Validated and Cost Effective Product is Shipped to the Hospital Ready for Infusion Cord CliniMACS Blood selection of Unit CD133+ cells NiCord is transfused at Cultured Non-cultured + fraction - fraction the transplant center CD133+ cells are cultured for 3 CD133- cel
Slide 11: NiCord® Smoothly Integrates into Current Transplant Practice and Procedures
NiCord® Smoothly Integrates into Current Transplant Practice and Procedures Patient is Eligible cord blood Cord blood unit Manufacturing NiCord® designated for unit selected shipped from of NiCord® cryopreserved transplantation cord blood bank to (3 weeks) formulation is with NiCord® manufacturing s
Slide 12: NiCord® Pilot Study Demonstrated Prompt and Durable Engraftment
NiCord® Pilot Study Demonstrated Prompt and Durable Engraftment • 11 adult, high-risk hematological malignancy patients, with myeloablative conditioning, at Duke and Loyola universities (U.S.) • All patients transplanted with NiCord® together with unmanipulated CB unit • Prompt, robust and durable l
Slide 13: NiCord® Phase 1/2 Study Provides Basis for Phase 3
NiCord® Phase 1/2 Study Provides Basis for Phase 3 • 24 Adult, high risk hematological malignancy patients with myeloablative conditioning, treated in 6 sites in the US and Europe • NiCord® transplanted as a standalone product, cryo preserved formulation • Meaningful improvement in time to engraftme
Slide 14: NiCord® Neutrophil Engraftment is Significantly More Rapid Than Cord Blood
NiCord® Neutrophil Engraftment is Significantly More Rapid Than Cord Blood Time to Neutrophil Engraftment is The Primary Endpoint in the Phase 3 Study 1.0 NiCord® median = 11 days CIBMTR median = 21 days Incidence of Neutrophil Engraftment 0.8 p<0.0001 (Wilcoxon rank sum test) 0.6 0.4 NiCord® at Day
Slide 15: NiCord® Non-Relapse Mortality is Lower than Matched Controls
NiCord® Non-Relapse Mortality is Lower than Matched Controls 1.0 Incidence of Non-Relapse Mortality NiCord® at 6 Month 6: 14.5% (95% Cl: 3.3%, 33.5%) p=0.02 (difference in the 0.8 cumulative incidence estimates) ClBMTR at Month 6: 31.2% (95% Cl: 23.3%, 39.4%) 0.6 NiCord® at Year 1: 14.5% (95% Cl: 3.
Slide 16: NiCord® Positive Impact on Morbidity and Mortality Predicts Potential for Widespread Adoption
NiCord® Positive Impact on Morbidity and Mortality Predicts Potential for Widespread Adoption Improved Pharmacoeconomics; Validating Breakthrough Therapy designation • Transplantation of NiCord® is associated with a significant improvement in: ─ Recovery of neutrophils and platelets (Horwitz et al.
Slide 17: NiCord® is Positioned to Shift BMT Paradigm
NiCord® is Positioned to Shift BMT Paradigm A Potential Graft of Choice for Patients with no Matched Related Donor Clinical Factors of Interest Peripheral Blood – MUD1 NiCord® 41 patients treated2 Availability of graft to transplant 30% of patients (3-4 months) Most patients (1 month) Time to neutro
Slide 18: NiCord® Phase 3 Registration Study is Enrolling
NiCord® Phase 3 Registration Study is Enrolling Breakthrough Therapy Designation Granted by FDA Randomized controlled Phase 3 Registration Study 120 patients Adult patients with high risk hematological malignancies including AML, ALL, MDS and CML 60 patients 60 patients + NiCord® Control unmanipulat
Slide 19: NiCord® Business Opportunity
NiCord® Business Opportunity
Slide 20: NiCord® Provides Significant Benefits to Patients, Hospitals and Payers
NiCord® Provides Significant Benefits to Patients, Hospitals and Payers Patients • Better clinical outcomes • Rapid hematopoietic recovery • Reduced morbidity • Reduced transplant related mortality Hospital • Shorter hospital stay by ~20 days • Improved efficiency in beds utilization (census) • Decr
Slide 21: NiCord® Can Become the Graft of Choice for Patients With No Suitable Donor
NiCord® Can Become the Graft of Choice for Patients With No Suitable Donor Target Population in the US, Europe and Japan Alone: 35,800 Patients in Peak Year Based on Penetration Assumptions, Peak Sales ~9,000 Patients Annually 44,800 Indicated Patients (US, Europe and Japan only) (a) NiCord Target M
Slide 22: NiCord® Revenues Potential (USA, Europe and Japan)
NiCord® Revenues Potential (USA, Europe and Japan) Over $ 1 billion in potential annual sales (peak year 2025) % Number of Graft Source(a) U.S.A. Europe Japan Total(b) Penetration patients (peak) treated (peak) Matched Related Donor 2,700 5,070 1,240 9,010 0% 0 Matched Unrelated Donor 6,110 11,075 2
Slide 23: Low COGS Ensures Margins of Profitability
Low COGS Ensures Margins of Profitability In House Manufacturing Capability; Logistical Infrastructure is Efficient • In-house GMP manufacturing capacity in Israel, approved by the Israeli Ministry of Health • A central manufacturing site to accommodate commercial needs will be established prior to
Slide 24: NiCord® Market Exclusivity Supported by Three Strategic Barriers
NiCord® Market Exclusivity Supported by Three Strategic Barriers REGULATORY EXCLUSIVITY NiCord® has an orphan drug INTELLECTUAL PROPERTY designation in the US and EU 55 Granted patents worldwide, of which 25 granted and 2 pending applications related to NiCord® . Until 2028-2029 GRANTED CLAIMS Compo
Slide 25: CordIn™
CordIn™ A Potential Cure for Sickle Cell Disease
Slide 26: CordIn™ - a Cure of Rare Genetic Diseases Where Bone Marrow Transplantation is the Only Established Cure
CordIn™ - a Cure of Rare Genetic Diseases Where Bone Marrow Transplantation is the Only Established Cure • BMT is the only cure of Sickle Cell Disease (SCD) and thalassemia which has been clinically established − Gene therapy does not provide a long term cure to many of the patients • SCD and thalas
Slide 27: Clinical Proof of Concept in Curing Sickle Cell Disease
Clinical Proof of Concept in Curing Sickle Cell Disease Data Presented at ASH 2016 • First evidence of successful engraftment in patients with Sickle Cell Disease (SCD) ─ Eleven patients treated ─ Very rapid engraftment of donor cells (median 7 days) ─ 2 patients died (one of secondary graft failure
Slide 28: CordIn™ in Bone Marrow Failure Syndromes: Aplastic Anemia and MDS
CordIn™ in Bone Marrow Failure Syndromes: Aplastic Anemia (AA) and Myelodysplastic Syndrome (MDS) • Investigator initiated study in collaboration with Prof. Richard Childs at the U.S. National Heart, Lung and Blood Institute (NHLBI) • Approximately 30 patients age 4-55 • First part of the study: up
Slide 29: Natural Killer (NK) Cell Product
Natural Killer (NK) Cell Product Immune Therapy of Cancer
Slide 30: NAM-NK Cells for Immunotherapy of Blood and Solid Cancers
NAM-NK Cells for Immunotherapy of Blood and Solid Cancers NK • NK cells play an important role in the immune system fight of cancer NK • Unlike T cells, NK cells do not need prior sensitization and do not increase the risk of NK GvHD • NK cells in cancer patients are exhausted • Functionality is com
Slide 31: Robust and Cost Effective Manufacturing of NAM-NK Cells
Robust and Cost Effective Manufacturing of NAM-NK Cells Potential Off-the-shelf Cell Product Based on Common Haplotypes Leukapheresis CD3 depletion Seeding using CliniMACS NK cells ~ 5-30% B cells Granulocytes, Monocytes Residual T and NKT Efficient ex-vivo expansion process - Only one cell selectio
Slide 32: NAM Technology Improves Efficacy of NK Cells
NAM Technology Improves Efficacy of NK Cells Enhanced Direct and Adaptive Immune Killing Excellent Homing and Retention of NK Cells in Enhanced Direct and Adaptive Immune Animal Models Tumor Killing Degranulation Activation Bone Marrow Spleen Lung of death Cytokine 0.30 0.30 0.6 of cytotoxic granule
Slide 33: NAM Reduces Expression of PD-1 in NK Cells
NAM Reduces Expression of PD-1 in NK Cells May be Associated With Reduced Tumor Escape PD-1 (Programmed Death receptor-1) CD200R 12 40 35 10 30 8 25 6 20 15 4 10 2 5 0 0 0 2.5 5 7.5 NAM (mM) 0 2.5 5 7.5 NAM (mM) PD-1 Ligation of CD200 / CD200R or PD-L1 / PD1 suppresses NK cell Tumor Cells function a
Slide 34: Improved In Vivo Proliferation of NK Cells Cultured with NAM
Improved In Vivo Proliferation of NK Cells Cultured with NAM NK Cells Proliferation in the BM is an Important Measure of Their Activity BrdU assay for detection of Percent of in vivo proliferating cells proliferating cells by FACS 20.0 0mM NAM 5mM NAM Proliferating cells 15.0 %BrdU+ of NK 10.0 5.0 0
Slide 35: NAM-NK Cells Phase 1/2 Study to Begin
NAM-NK Cells Phase 1 Study to Begin H1/2017 • Patients with B-cell lymphoma and multiple myeloma • Donor derived NK cells are expected to be more effective, in particular for patients resistant to treatment • in combination with Rituximab (for B-cell lymphoma) or with Elotuzumab (for multiple myelom
Slide 36: Financials and Business Overview
Financials and Business Overview
Slide 37: Financials and Shareholders
Financials and Shareholders • Cash, June 30, 2017: $10 M • $40 M financing closed in July 2017 • Approved 2017 support by Israeli government: $3.5M • Use of proceeds: − Complete Phase 3 study of NiCord® and prepare for product commercialization − Continue pipeline development − Hire additional senio
Slide 38: Value Creation During 2016-2018
Value Creation During 2016-2018 R&D Indication 2016 2017 2018 Value Phase 1/2 data Phase 1/2 Clinical First in class cure for Hematological Phase 3 complete NiCord® readout Day 180 final hematological malignancies; malignancies enrollment; Begin Phase 3 analysis standalone graft No gene manipulation
Slide 39: Management Team in Israel and in the US
Management Team in Israel and in the US • Dr. Yael Margolin ─ President & CEO since 2005. Previously VP of Denali Ventures LLC, and manager of new R&D initiatives at Teva Pharmaceuticals. Ph.D. in Biology from the Weizmann Institute of Science and was a post-doctoral associate at the Yale University
Slide 40: Scientific and Clinical Advisors are Internationally Preeminent
Scientific and Clinical Advisors are Internationally Preeminent • Joanne Kurtzberg, MD • Guillermo Sanz, MD Director, Carolinas Cord Blood Bank Professor and Head of Clinical Hematology Department Chief Scientific Officer, Robertson Clinical and Hospital Universitario La Fe, Valencia, Spain Translat
Slide 41: Board of Directors
Board of Directors • Dr. Julian Adams - Chairman of the Board More than 30 years of experience in drug discovery and development with a strong focus on cancer research. Senior vice president of drug discovery and development at Millennium Pharmaceuticals, heading the successful Velcade® program. Pre
Slide 42: Curative Treatments for Orphan Indications
Curative Treatments for Orphan Indications • NiCord® - a bone marrow transplantation treatment for patients with high risk leukemia and lymphoma ─ Breakthrough Therapy designation by FDA ─ Phase 3 registration study is enrolling patients ─ Positioned to become the graft of choice for patients with n
Slide 43: Yael Margolin, Ph.D President & CEO
Yael Margolin, Ph.D President & CEO Yael@gamida-cell.com www.gamida-cell.com