Gamida Cell — Investor Presentation (September 2019)
39 slides from Gamida Cell's investor presentation, September 2019
Slide 1: Company Overview
Company Overview September 2019 1
Slide 2: Disclaimer
Disclaimer This Presentation includes certain projections and forward-looking statements as of the date of this Presentation provided by Gamida Cell Ltd (the “company”). The information in this Presentation is current only as of its date and may have changed since that date. These projections and fo
Slide 3: Omidubicel, GDA-201, NAM Technology
“Much remains to be done because strategies for engineering immunity are Omidubicel* not yet optimized, and adverse events, limitations, and mechanisms of resistance to cellular immunotherapy are evident. What will shape the immunotherapy field in the next 10 years? GDA-201 NAM Technology Future bre
Slide 4: Gamida Cell Has Worldwide Rights to Its Clinical Pipeline
Gamida Cell Has Worldwide Rights to Its Clinical Pipeline PRODUCT PRECLINICAL PHASE 1/2 PHASE 3 MILESTONES Top-line data High-Risk Hematologic Malignancies 1H20 Omidubicel Severe Aplastic Anemia* Initiate Cohort 2 1H19 Additional data GDA-201 Hematologic Malignancies 2H19 *The Aplastic Anemia Inve
Slide 5: Cell Expansion Platform Based on Nicotinamide (NAM) Mechanism of Action
Cell Expansion Platform Based on Nicotinamide (NAM) Mechanism of Action NAM can expand any cell type, including stem cells and natural killer (NK) cells Importance of NAM Histone O NAD-dependent • Plays a key role in metabolic Acetylation enzymes reprogramming of cells NH2 N NAM • Is a master regula
Slide 6: Omidubicel
Omidubicel Hematologic Malignancies and Serious Blood Disorders 6 6
Slide 7: Omidubicel Is Designed to Enhance the Life-Saving Benefits of Allogeneic Bone Marrow Transplant
Omidubicel Is Designed to Enhance the Life-Saving Benefits of Allogeneic Bone Marrow Transplant “The study… is the first to our knowledge to show that an expanded CB unit can be infused as a stand- alone graft and is capable of providing robust and durable hematopoiesis.” ─ Mehta et al., JCO editori
Slide 8: Bone Marrow Transplant May Be Curative in Hematologic Malignancies
Bone Marrow Transplant May Be Curative in Hematologic Malignancies ~13,000 ~8,000 Patients with Allogeneic stem cell transplants (Annually in U.S.) ~40% of patients don’t hematologic receive a malignancies eligible transplant for transplant annually Internal Market Research Studies and Data Analysis
Slide 9: Bone Marrow Transplantation Must Be Improved
Bone Marrow Transplantation Must Be Improved Bone Marrow Transplant Process Today’s Challenges in Transplantation Finding an optimal match Lengthy time to engraftment, which can lead to infections and hospitalizations Conditioning Transplant Engraftment Complications, such as graft vs. host disease
Slide 10: There Are Currently No FDA-Approved Graft Sources for Bone Marrow Transplant
There Are Currently No FDA-Approved Graft Sources for Bone Marrow Transplant Limitations of Certain HSCT Graft Sources Haploidentical Matched Unrelated Umbilical Donor Donor (MUD) Cord Blood Not available for all patients Small number of stem cells Age of donor Relapse rates1 Time required to find a
Slide 11: Omidubicel Is a Pharmaceutical-Grade Advanced Cell Therapy Product
Omidubicel Is a Pharmaceutical-Grade Advanced Cell Therapy Product Manufacturing and Treatment Process For Omidubicel Cord Blood Unit (CBU) Omidubicel Selected Omidubicel Infusion CBU selected by NAM-Expanded Uncultured physician from public cord blood bank Cells Fraction Stem cells cultured using I
Slide 12: Phase 1/2 Study of Omidubicel in Hematologic Malignancies
Phase 1/2 Study of Omidubicel in Hematologic Malignancies Age 12-65 Hematologic Malignancies Myeloablative Transplantation Follow-up AML Conditioning with omidubicel MDS N=36 ALL CML Day Lymphoma Eligible for Transplant -7 0 +100 No matched donor 13 sites: U.S., EU, Asia • Primary endpoint: Neutroph
Slide 13: Rapid Neutrophil Engraftment with Omidubicel
Rapid Neutrophil Engraftment with Omidubicel ~50% reduction in time to engraftment is associated with clinical benefit Cumulative Incidence of Neutrophil 94% of patients engrafted Engraftment 85% of patients engrafted Median days to engraftment Omidubicel: 11 days (95%CI: 9-13d) CIBMTR Cohort: 21 da
Slide 14: Rapid Engraftment Is Associated with Fewer Infections and Shorter Hospitalizations
Rapid Engraftment Is Associated with Fewer Infections and Shorter Hospitalizations Infection Mean Infections per 100 patient-days Omidubicel Standard Cord Total Infections Bacterial Infections 14 Anand et al. BBMT 23:1151-7, 2017.
Slide 15: Omidubicel Recipients Experienced Rapid and Robust Immune Reconstitution
Phase 1/2 Omidubicel Study: Omidubicel Recipients Experienced Rapid and Robust Immune Reconstitution (IR) Following Transplantation Day 100 350 Omidubicel 323 300 Unmanipulated Dual Cord 250 Cells/µl (median) 200 207 203 150 159 100 100 50 53 15 27 0 CD4+ CD8+ B-cells NK-cells 15 de Koning et al., A
Slide 16: Omidubicel Phase 1/2 Study: Key Takeaways
Omidubicel Phase 1/2 Study: Key Takeaways Significant reduction in: Acceptable safety profile • Time to neutrophil • Low incidence engraftment of acute and chronic GvHD • Rate and severity of infections • Days of hospitalization FDA Breakthrough Therapy Designation 16 Journal of Clinical Oncology 20
Slide 17: Phase 3 Registration Study of Omidubicel for Allogeneic Transplantation
Phase 3 Registration Study of Omidubicel for Allogeneic Transplantation in 120 Patients with Hematologic Malignancies Omidubicel (60 patients) • 120 patients with hematologic malignancies • Eligible for allogeneic bone marrow transplantation R • No suitable donor Standard Cord Blood (60 patients) Pr
Slide 18: Omidubicel Has Potential to Help Many Patients in Need
OPT 3 Omidubicel Has Potential to Help Many Patients in Need Not Matched/ Not Referred 40% ~13,000 Matched Unrelated Donor 25% Haploidentical Patients with Donor 10% hematologic malignancies eligible for transplant annually 5% Umbilical Cord Blood 20% Family-Related Matched Donor Not matched / not r
Slide 19: Omidubicel Has Potential to Help Many Patients in Need
OPT 3 Omidubicel Has Potential to Help Many Patients in Need Not Matched/ Not Referred 40% 80% Matched Unrelated Donor 25% Addressable Haploidentical Donor 10% by omidubicel 5% Umbilical Cord Blood 20% Family-Related Matched Donor Not matched / not referred based on: Besse K, et al. Estimating Deman
Slide 20: Preparing to Bring Omidubicel to Patients
Preparing to Bring Omidubicel to Patients Approximately 70 transplant centers account for ~80% of bone marrow transplants in U.S. 1https://bloodcell.transplant.hrsa.gov/research/transplant_data/us_tx_data/data_by_center/center.aspx Allogeneic and select blood cancers (ALL, AML, CML, HL, NHL, MDS, MM
Slide 21: Preparing for a Successful Omidubicel Launch
Preparing for a Successful Omidubicel Launch 1 Engaging top transplant centers Preparing to submit 2 Building patient and hospital support services Biologics License Application to FDA in 2020* 3 Working with payers to ensure reimbursement 4 Ensuring commercial manufacturing readiness 21 *If data ar
Slide 22: GDA-201
GDA-201 Harnessing Innate Immunity Using Natural Killer Cells to Treat Cancer 22
Slide 23: GDA-201 Is Designed to Enhance the Power of Natural Killer Cells
GDA-201 Is Designed to Enhance the Power of Natural Killer Cells Natural killer cells (yellow) attack a cancer cell. Eye of Science/Science Source Leslie M. ScienceMag.org; doi:10.1126/science.aav4154 23
Slide 24: GDA-201 Immunotherapy
GDA-201 Immunotherapy NAM Expansion of Natural NK Cells • NK cells infusion is a promising immune therapy for cancer: – No HLA-matching required NAM – Synergy with antibodies • Expansion is necessary to obtain GDA-201 + Tumor-Specific Antibodies clinically meaningful doses 24
Slide 25: Phase 1 Study of GDA-201 in Non-Hodgkin Lymphoma and Multiple Myeloma
Phase 1 Study of GDA-201 in Patients with Non-Hodgkin Lymphoma and Multiple Myeloma Patients with Non-Hodgkin Fludarabine/cyclophosphamide lymphoma (NHL) or multiple Lymphodepleting preparative GDA-201 myeloma (MM) regimen (N = 24) Rituximab (NHL) or Elotuzumab (MM) • Primary endpoint: Maximum toler
Slide 26: GDA-201 Phase 1 Study Data: Select Patient Demographics
GDA-201 Phase 1 Study Data: Select Patient Demographics Number of Patients Patient and Disease Characteristics (N = 14) Age [median (range)] 62 (47-75 years) Diagnosis Non-Hodgkin lymphoma* 6 Multiple myeloma 8 Disease status Relapsed 10 Refractory 4 Prior Therapies Number of lines of prior therapy
Slide 27: GDA-201 Generally Well Tolerated: Phase 1 Safety Summary
GDA-201 Generally Well Tolerated: Phase 1 Safety Summary • No dose limiting toxicities, GVHD, or neurotoxicity • Most common related grade 3 or 4 adverse events were neutropenia (n=4) and hypertension (n=4) • One patient with MM had grade 3 cytokine release syndrome (fever, hypoxemia, hypotension),
Slide 28: GDA-201 Phase 1 Data: Multiple Complete Responses in Non-Hodgkin Lymphoma
GDA-201 Phase 1 Study Data: Multiple Complete Responses Observed in Patients with Non-Hodgkin Lymphoma Patients with Non-Hodgkin Lymphoma Legend Patient Number (Diagnosis) 002 (FL) Cohort/Dose Level 1 2 3 004 (FL) Complete response 005 (FL) Partial response 003 (tDLBCL) Progressive disease 009 (tDLB
Slide 29: Phase I Study of GDA-201: Non-Hodgkin Lymphoma
Phase I Study of GDA-201: Non-Hodgkin Lymphoma Patient 002: Radiographic Complete Response • 67-year-old man with Stage IVA follicular lymphoma diagnosed in 2012, previously treated with multiple rituximab-containing regimens (R-CVP, R-bendamustine, R-ICE, R-EPOCH) • Presented with bulky adenopathy
Slide 30: Phase I Study of GDA-201: Non-Hodgkin Lymphoma
Phase I Study of GDA-201: Non-Hodgkin Lymphoma Patient 004: Radiographic Complete Response • 60-year-old man with Stage IV follicular lymphoma diagnosed in 2015, previously treated with R-bendamustine, R- CHOP, and R-ICE • Received GDA-201 dose level 2: Complete response • Went on to allogeneic bone
Slide 31: Phase I Study of GDA-201: Multiple Myeloma
Phase I Study of GDA-201: Multiple Myeloma Patient 010: Radiographic Complete Response One complete response observed in patients with multiple myeloma • Pt 010: 70-year-old woman with multiple myeloma diagnosed in 2012, previously treated with lenalidomide/bortezomib/dexamethasone; Pre-treatment le
Slide 32: GDA-201: Encouraging Clinical Activity Supports Continued Development
GDA-201: Encouraging Clinical Activity Supports Continued Development Key Takeaways1 Next Steps Future Directions • Well tolerated • Complete Phase 1 • Combine with a broad study range of antibodies • Clinically active • Cryopreservation • Evaluate in solid • Maximum target dose tumors achieved • In
Slide 33: Corporate Information
Corporate Information gamida (ell
Slide 34: The Gamida Cell Executive Team Has Substantial Experience
The Gamida Cell Executive Team Has Substantial Experience Julian Adams, Ph.D. ─ Chief Executive Officer Josh Hamermesh ─ Chief Business Officer Thomas Klima ─ Chief Commercial Officer Shai Lankry ─ Chief Financial Officer Tracey Lodie, Ph.D. ─ Chief Scientific Officer Tzvi Palash ─ Chief Operating O
Slide 35: Financial Snapshot
Financial Snapshot • Total cash position of $41.7 million as of June 30, 2019 ─ Subsequently closed $40.3 million follow-on equity offering in July* • Anticipated cash used for operating activities in 2019: $35-$40 million • Cash supports capital needs through data readout for the omidubicel Phase 3
Slide 36: Expected 2019-2020 Milestones
Expected 2019-2020 Milestones Omidubicel - Hematologic malignancies 2H19: Complete enrollment in Phase 3 study 1H20: Report topline Phase 3 data 2H20: Complete BLA submission Omidubicel - Severe aplastic anemia 1H19: Present preliminary data from Cohort 1 1H19: Initiate Cohort 2 evaluating
Slide 37: Appendix
Appendix gamida Cell
Slide 38: Omidubicel: Phase 1/2 Study in Severe Aplastic Anemia
Omidubicel: Phase 1/2 Study in Severe Aplastic Anemia 600-900 Americans are diagnosed with aplastic anemia each year1 Cohort 1 successfully completed Neutrophil recovery data from Cohort 1* – Consisted of 3 patients who received omidubicel + haploidentical stem cell graft following Median time 6 v
Slide 39: Company Overview
Company Overview September 2019 39