Company Overview. Investor Presentation. August 2018

Transcription

1 Company Overview Investor Presentation August

2 Notice to Investors The sole purpose of this Presentation (this Presentation ) is to assist recipients in deciding whether to proceed with a further investigation of Gamida Cell Ltd. (the Company ) and considering an investment in a private placement of securities by the Company (a Transaction ). This Presentation is not to be used, in whole or in part, for any other purpose. This Presentation does not constitute an offer or invitation for the sale or purchase of securities. Any reproduction or distribution of this Presentation, in whole or in part, or the disclosure of any of its contents is prohibited. The Company and its affiliates and representatives, expressly disclaim any and all liability based, in whole or in part, on such information, errors therein or omissions therefrom. None of the Company or their respective affiliates or representatives, makes any representation or warranty, expressed or implied, as to the accuracy or completeness of the information contained herein or any other written or oral communication transmitted or made available to any recipient. The information contained in the Presentation is provided as of the date of the Presentation and the Company has no obligation to update such information. Only those representations and warranties that are made in a definitive agreement relating to the Transaction shall have any legal effect. In addition, this Presentation includes certain projections and forward-looking statements as of the date of this Presentation provided by the Company. The information in this Presentation is current only as of its date and may have changed since that date. These projections and forward-looking statements include, but are not limited to, those regarding the Company s future financial position and results of operations, the Company s commercialization, marketing and manufacturing capabilities and strategy, the Company s intellectual property position, regulatory matters, market size and opportunity and the Company s estimates regarding expenses, future revenues, capital requirements and needs for additional financing. These projections and forward-looking statements are based on the beliefs of the Company s management as well as assumptions made and information currently available to the Company. Such statements reflect the current views of the Company with respect to future events and are subject to business, regulatory, economic and competitive risks, uncertainties, contingencies and assumptions about the Company and its subsidiaries and investments, including, among other things, the development of its business, trends in the industry, the legal and regulatory framework for the industry and future expenditures. In light of these risks, uncertainties, contingencies and assumptions, the events or circumstances referred to in the forward-looking statements may not occur. None of the future projections, expectations, estimates or prospects in this presentation should be taken as forecasts or promises nor should they be taken as implying any indication, assurance or guarantee that the assumptions on which such future projections, expectations, estimates or prospects have been prepared are correct or exhaustive or, in the case of the assumptions, fully stated in the presentation. The actual results may vary from the anticipated results and the variations may be material. 2

3 We Envision a World Where No Patient Has to Forgo a Safe and Effective Transplant Waiting for a Donor Match NiCord : Most advanced product candidate for bone marrow transplant Phase 3 registration study under way in patients with hematologic malignancies Received FDA Breakthrough Therapy Designation and Orphan Designation from FDA and EMA Preparing U.S. launch to deliver NiCord to patients Proven, proprietary nicotinamide (NAM)-based technology Recently expanded executive team and established U.S. headquarters Wholly-owned clinical pipeline sets the stage for multiple milestones through

4 NiCord Hematologic Malignancies 4 4

5 Bone Marrow Transplant May Be Curative in Hematologic Malignancies Lymph nodes Bone marrow Treatment Complete Remission Relapse or refractory Eligible for bone marrow transplant Blood and lymph node cancers (e.g., AML, ALL, CLL, CML, HL, NHL)* 5 *AML: acute myelogeneous leukemia; ALL: acute lymphoblastic leukemia; CLL: chronic lymphocytic leukemia; CML: Chronic myelogenous leukemia; HL: Hodgkin lymphoma; NHL: non-hodgkin lymphoma.

6 Allogeneic Bone Marrow Transplantation Conditioning Stem Cell Infusion Engraftment Day 0-7 Patient discharged 100 The Problem (*) Complications can occur during infusion, engraftment, and post-engraftment (e.g., infections, GvHD) Many patients never get to transplant or have a significant wait to get a match 6

7 HSCT Candidate Patient Segmentation 50,000 patients each year are eligible for a transplant Never get transplant 40% Matched unrelated donor 25% Family-related matched donor 20% Haploidentical donor 10% Umbilical cord blood 5% 7 *HSCT patient candidate percentages for the US and rounded to the nearest 5% Sources: 1) Besse, et al. Ests Demand and Unmet Need for Allo-HCT in the US. J. of Onc Practice V 11 I 2 Mar ) Gale, et al. HCT in Latin America. BMT Jul; 51(7): doi: /bmt ) Horowitz. Trends in Graft Sources for Allo-HCT, CIBMTR, Med Col of WI Sep, ) JDCHCT. Activities and Outcomes of HCT in Japan. Summary Slides. 5) Passweg, et al. HCT in Europe BMT. (2016) 51, ) Nemetz Group. HCT Physician Quant Survey. Data on file. Spring 2018.

8 HSCT Candidate Patient Segmentation 50,000 patients each year are eligible for a transplant Never get transplant 40% Matched unrelated donor 25% Family-related matched donor 20% Haploidentical donor 10% Umbilical cord blood 5% 8 *HSCT patient candidate percentages for the US and rounded to the nearest 5% Sources: 1) Besse, et al. Ests Demand and Unmet Need for Allo-HCT in the US. J. of Onc Practice V 11 I 2 Mar ) Gale, et al. HCT in Latin America. BMT Jul; 51(7): doi: /bmt ) Horowitz. Trends in Graft Sources for Allo-HCT, CIBMTR, Med Col of WI Sep, ) JDCHCT. Activities and Outcomes of HCT in Japan. Summary Slides. 5) Passweg, et al. HCT in Europe BMT. (2016) 51, ) Nemetz Group. HCT Physician Quant Survey. Data on file. Spring 2018.

9 Nicotinamide (NAM) Technology: Mechanism of Action NAM causes metabolic reprogramming, leading to cell proliferation while preserving stem cells O NH 2 N NAM Preserved Gene Expression Increased Engraftment Efficacy Noncultured Cultured w/o NAM Cultured +NAM 9

10 NiCord Manufacturing Process Is Validated and Cost Effective Product Is Cryopreserved, Shipped to the Hospital Ready for Infusion Cord Blood Unit CliniMACS selection of CD133 + cells + Cultured fraction - Noncultured fraction NiCord is transfused at the transplant center CD133 + cells are cultured for 3 weeks using proprietary NAM technology CD133- cells, including T- cells, is cryopreserved until the day of transplantation Cryopreserved formulation, ready for infusion 10

11 Phase 1/2 Study of NiCord in Patients with Hematologic Malignancies Age Hematologic Malignancies AML MDS ALL Lymphoma Eligible for Transplant No matched donor 13 sites: US, EU, Asia N=36 Myeloablative Conditioning Day Transplantation with NiCord Primary endpoint: Neutrophil engraftment Follow-up Secondary endpoints: Platelet engraftment, acute GvHD, chronic GvHD, infections, hospitalization, non-replase mortality, overall survival, disease-free survival 11 Clinicaltrials.gov identifier NCT

12 Cumulative Incidence of Neutrophil Engraftment Rapid Neutrophil Engraftment with NiCord ~50% reduction in time to engraftment is an FDA/EMA approved surrogate for clinical benefit 94% of patients engrafted 85% of patients engrafted Median days to engraftment) NiCord : 11 days (95%CI: 9-13d) CIBMTR: 21 days (95%CI: 20-23d) p<0.001 NiCord CIBMTR Cohort Days Post Transplant 12 Horwitz et al., ASH Note: Center for International Blood and Marrow Transplant Research (CIBMTR) registry data of 146 patients who received myeloablative conditioning and unmanipulated cord blood transplantation.

13 Cumulative Incidence of Platelet Engraftment Incidence of Platelet Engraftment Rapid Platelet Engraftment with NiCord 81% of patients engrafted 63% of patients engrafted Days to engraftment (median) NiCord: 34 days (95%CI: 32-42d) CIBMTR: 46 days (95%CI: 42-50d) p<0.001 NiCord CIBMTR Cohort Days Post Transplant 13 Horwitz et al., ASH Note: CIBMTR registry data of 146 patients who received myeloablative conditioning and unmanipulated cord blood transplantation.

14 Mean Infections per 100 patient-days Days Alive and out of Hospital by Day 100 Rapid Engraftment Is Associated with Improved Clinical Outcomes Infection Hospitalization NiCord Standard Cord Total Infections Bacterial Infections 14 Anand et al. BBMT 23:1151-7, 2017.

15 Phase 1/2 NiCord Study: Secondary Endpoint Results Chimerism agvhd: Grade III-IV at 100 days Full donor (>95%): 97% Mixed chimerism: 3% 11% cgvhd: Moderate-Severe at 1 year 10% Number of days of primary hospitalization 19d Number of days alive and out of hospital by Day 100 (median) 73d *Median follow-up 14 months (range 5-37m) 15 Horwitz et al., ASH 2017.

16 Cells/µl (median) Phase 1/2 NiCord Study: NiCord Recipients Experienced Rapid and Robust Immune Reconstitution (IR) Following Transplantation At 100 days post transplantation with NiCord, CD4+ reconstitution is at least as fast as transplant with unmanipulated CB and BM in young adolescents NiCord Day 100 Unmanipulated Dual Cord* 323 NK, B-cells and monocyte reconstitution faster than transplant with unmanipulated CB and BM In depth immune monitoring may lead to better predictors for outcome Optimal comparison of IR in randomized Phase 3 study of NiCord is underway CD4+ CD8+ B-cells NK-cells 16 Horwitz et al., ASH 2017; Nierkens et al., EBMT 2018; Barker et al: Brit J Haem 2015.

17 Probability of Overall Survival NiCord Phase 1/2 Outcome: Overall Survival ~ Half the patients who did not have a matched donor received NiCord were alive after two years No significant difference in OS from CIBMTR cohort Nicord population small, heterogeneous, high risk Analysis not stratified for disease risk Phase 3 randomized trial stratifies for disease risk and other factors to allow for comparisons NiCord Year 2 Estimate: 51.2% (95% CI: 32.9%, 66.8%) CIBMTR Year 2 Estimate: 48.3% (95% CI: 39.7%, 56.4%) p=0.72 FDA accepted neutrophil engraftment as primary endpoint for Phase 3 Months Post Transplant 17 Horwitz et al., ASH 2017.

18 NiCord Phase 1/2 Case Study: Patient #1 63 y/o retired male and avid golfer with acute myeloid leukemia, first complete remission No matched donors in National Marrow Donor Program (NMDP) registry Neutrophil recovery on Day 14 and discharged from hospital early on Day 16 Grade II GvHD, resolved with short course prednisone Disease free for 7 years and returns to Duke for annual visits 18

19 Phase 3 Registration Study of NiCord for Allogeneic Transplantation in ~120 Patients with Hematologic Malignancies 120 patients with hematologic malignancies Eligible for allogeneic bone marrow transplantation No suitable donor R NiCord (60 patients) Standard Cord Blood (60 patients) Primary endpoint: Time to neutrophil engraftment Secondary endpoints: Platelet engraftment, acute GvHD, chronic GvHD, infections, hospitalization, non-relapse mortality, overall survival, disease-free survival 14 sites initiated in the U.S., EU and Asia; Expect to open additional sites Expected enrollment completion: 2H19; Topline data anticipated 1H20 19 Clinicaltrials.gov identifier NCT

20 Understanding Real World Outcomes Collaboration with CIBMTR to collect real world outcomes in patients following bone marrow transplantation Registry data collection concurrent with the Phase 3 randomized NiCord trial and based on similar endpoints Health economics outcomes research (HEOR) expected to inform price, reimbursement and adoption 20

21 NiCord Designed to Provide Significant Benefits to Patients, Physicians, Providers and Payers Patients and Physicians Better clinical outcomes Rapid hematopoietic recovery Reduced morbidity Reduced transplant related mortality Providers (Hospitals) Shorter hospital stay by ~20 days Improved efficiency in beds utilization (census), allows more patients to get to transplant Decrease in resource utilization Additional carved out budget for the graft itself is available in many cases $ Payers Improved outcomes for their beneficiaries Value for money Cost offsets (no double cords, shorter hospital stays, fewer readmissions) 21

22 Intellectual Property and Exclusivity Orphan drug designation in the U.S. and in Europe resulting in patent exclusivity 12 years exclusivity in U.S. 10 years exclusivity in Europe NiCord patents coverage with NAM technology includes Method of expanding HSC Population of expanded HSC Method of transplanting expanded HSC Exclusivity protection due to trade secrets 22

23 Gamida Cell Has Worldwide Rights to Its Clinical Pipeline PRODUCT PRECLINICAL PHASE 1/2 PHASE 3 MILESTONES High-Risk Hematologic Malignancies Top-line data H NiCord Severe Aplastic Anemia* Preliminary data 2019 NAM-NK Hematologic Malignancies Preliminary data *The Aplastic Anemia Investigational New Drug (IND) application is currently filed with the FDA under the brand name, CordIn, which is the same investigational development candidate as NiCord.

24 NiCord Advanced Hematologic Disorders 24 #

25 NiCord Potential in Severe Aplastic Anemia Bone Marrow Transplant is the Only Known Cure for Severe Aplastic Anemia Cohort 1 Patients with severe aplastic anemia (N = 3-6) Reduced-intensity conditioning NiCord + haploidentical stem cell graft Cohort 2 Patients with severe aplastic anemia (N = 23) Reduced-intensity conditioning NiCord Americans are diagnosed with aplastic anemia each year 1 Bone marrow failure disease: marrow unable to produce enough blood cells Patients with severe to very severe aplastic anemia are at risk for life-threatening infections Cohort 1 ongoing Next steps: Evaluate outcomes after first 3 patients; expansion to additional study sites Registration strategy: single arm study following FDA discussion 25 1 Aplastic Anemia and MDS International Foundation: Note: The Aplastic Anemia Investigational New Drug (IND) application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as NiCord.

26 Additional Potential HSCT Indications for Nicord Potential to Treat Orphan Disease Indications in Which HSCT is Utilized Bone Marrow Diseases Approximate Annual Incidence (US) Severe aplastic anemia Fanconi anemia 50 Paroxysmal nocturnal hemoglobinuria (PNH) 350 Inherited Immune System Disorders Severe combined immunodeficiency (SCID) 100 Wiskott-Aldrich syndrome (WAS) 25 Hemoglobinopathies Beta thalassemia major 3,000 Sickle cell disease (SCD) 1 in 365 African American births Inherited Metabolic Disorders Krabbe disease (GLD) 40 Hurler syndrome (MPS-IH) 40 Adrenoleukodystrophy (ALD) 200 Metachromatic leukodystrophy (MLD) Note: Transplant data: Incidence data: and

27 Natural Killer (NK) Cell Development Candidate Harnessing Innate Immunity in Cancer 27 #

28 NK Cell Immunotherapy Program Historically, functionality of adoptively transferred NK cells has been limited Healthy donor apheresis Gamida Cell s NAM-NK development candidate demonstrated: In vivo migration, survival and proliferation Cytotoxicity and anti-tumor activity Scalability and clinical adaptability NAM-NK cell expansion Infusion 28

29 Killing (%) NAM Technology Has Been Adapted to NK Cells High affinity antibodies to specific targets on tumor and NK cells, bring the NK and cancer cells in proximity, trigger the killing cascade and increase the specificity and efficacy of NK cells NK NK + Rituximab Rituximab Antibody dependent cellular cytotoxicity (ADCC) Ab ADCC Tumor cells 10 0 Tumor cells NK cells 29 Brachya et al., ASH 2017.

30 Phase 1 Study of NAM-NK Cells in Patients with Multiple Myeloma or Non-Hodgkin Lymphoma Patients with multiple myeloma or Non-Hodgkin lymphoma (N = 24) Fludarabine/cyclophosphamide Lymphodepleting preparative regimen NAM-NK Cells Elotuzumab (Myeloma) or Rituximab (NHL) Primary endpoint: Maximum tolerated dose of NAM-NK cells Secondary endpoints: Overall response, toxicity 30 ClinicalTrials.gov Identifier NCT

31 Phase I Study of NAM-NK: Patient Outcomes Preliminary Safety Results No cytokine release syndrome or neurotoxicity observed in the first patients treated (n=2) Expected short-term neutropenia and thrombocytopenia observed No dose limiting toxicity; no grade 3 or 4 adverse events Patient y/o patient with follicular lymphoma diagnosed in Oct 2012; Stage IVA Adenopathy in upper and lower abdomen; bone marrow involved Ongoing treatments since 2012; tumor continued to progress 31 Bachanova et al., AACR Advances in Malignant Lymphoma 2018.

32 Phase I Study of NAM-NK: Patient Outcomes Patient 002: Radiographic Complete Response PRE-TREATMENT POST-NK CELL TREATMENT Key findings: Symptomatic resolution of bulky inguinal lymphadenopathy Complete response by CT/PET scan Biopsy of residual mass showed no evidence of lymphoma Evidence of expansion of donor NK cells in peripheral blood Study continues to actively enroll patients with multiple myeloma and NHL 32 Bachanova et al., AACR Advances in Malignant Lymphoma 2018.

33 The Gamida Cell Executive Team Julian Adams, Ph.D. Chairman & Chief Executive Officer Josh Hamermesh Chief Business Officer Shai Lankry Chief Financial Officer Tzvi Palash Chief Operating Officer Tony Peled Chief Scientific Officer Ronit Simantov, M.D. Chief Medical Officer 33

34 Financial Snapshot Total cash position is $36 million as of March 31, 2018 Raised more than $135 million since inception IFRS calendar year basis 58 employees Supported by dedicated syndicate of investors 34

35 Steady Momentum Over Past 12 Months Corporate Made key management hires: CEO, CMO, CFO, CBO, COO Secured lease to commercial manufacturing facility Nicord Received Orphan Drug Designation from FDA as a treatment for hematopoietic stem cell transplantation (HSCT) Presented Phase 1/2 data at ASH and Tandem Initiated Phase 3 study in high-risk hematologic malignancies Initiated Phase 1/2 study in severe aplastic anemia NAM-NK Initiated Phase 1 study 35

36 Anticipated Milestones Nicord : Phase 3 study in high-risk hematologic malignancies Complete enrollment in 2H19 Topline data in 1H20 Nicord: Phase 1/2 study in severe anaplastic anemia Topline data in 2019 NAM-NK Phase 1 study Complete patient enrollment in 2019 Topline data in

37 Company Overview Investor Presentation August