Gamida Cell — EBMT 2021
26 slides from Gamida Cell's conference presentation, 2021
Slide 1: Improved Clinical Outcomes with Omidubicel versus Standard Myeloablative Cord Blood Transplantation
Improved Clinical Outcomes with Omidubicel versus Standard Myeloablative Umbilical Cord Blood Transplantation: Results of a Phase III Randomized, Multicenter Study Guillermo F. Sanz, Patrick J. Stiff, Corey S. Cutler, Claudio G. Brunstein, Andrew R. Rezvani, Rabi Hanna, William Y.K. Hwang, Richard T
Slide 2: Disclosures
Disclosures • Consulting or advisory role: AbbVie, Amgen, Boehringer- Ingelheim, Celgene/BMS, Helsinn Healthcare, Janssen, Novartis, Roche, Takeda. • Speakers’ Bureau: Takeda. • Honoraria: Celgene/BMS • Research funding: Celgene/BMS, Gamida Cell, Novartis. • Travel, accommodations, expenses: Celgene
Slide 3: Umbilical cord blood stem cell grafts
Umbilical cord blood stem cell grafts Advantages • Readily available stem cells source • Tolerance across HLA barriers • Less chronic GvHD vs. Matched Unrelated donor • Potent anti-tumor activity Disadvantages Potential solution • Low stem cell dose Ex-vivo expansion of cord • Delayed hematopoietic
Slide 4: Omidubicel
Omidubicel Cellular product consisting of two cryopreserved fractions derived from a single entire cord blood unit (CBU) and thawed at the transplant center immediately before infusion • Cells obtained after CD133+ selection CD133- CD133+ ex vivo expanded for 21 days in the Fraction Fraction presenc
Slide 5: Omidubicel: phase I/II trial (N=36)
Omidubicel: phase I/II trial (N=36) Patients: 36 patients with high-risk hematologic malignancies (78% with intermediate/high DRI) undergoing myeloablative conditioning UCB grafts: • CD34+ cells infused (median): 6.3 x 106/kg Results • Very fast hematopoietic engraftment • Median time to neutrophil
Slide 6: Phase III trial of omidubicel
Phase 3 trial of omidubicel Primary Endpoint RANDOMIZE 1:1 • Age 12-65 Time to neutrophil Omidubicel engraftment • High-risk hematologic malignancies: AML, ALL, MDS, CML, lymphoma • Eligible for allogeneic stem cell Secondary transplantation Control Endpoints Time to platelet • No matched donor Stan
Slide 7: CBU selection criteria
CBU selection criteria Omidubicel Randomization Standard Cord 4-6/6 HLA Match TNC count ≥ 1.8x109 cells CBU #1 TNC dose ≥ 1.5x107 cells/kg CBU #1 CD34+ count ≥ 8x106 cells Red cell and plasma reduced At least 1 allele match at DRB1 OR Production (per EBMT 2006 CBU selection guidelines)* Omidubicel C
Slide 8: Patient disposition
Patient disposition Randomized (33 centers [US, Europe, (n=125) South America & Asia]) Randomized to Randomized to ITT Omidubicel Standard Cord (N=125) (n=62) (n=63) Transplanted with Transplanted with AT Omidubicel Standard Cord (N=108) (n=52) (n=56) 8 ITT: Intent to treat; AT: As treated populatio
Slide 9: Demographics
Demographics Omidubicel Control (N=62) (N=63) Gender Female 30 (48%) 23 (37%) Male 32 (52%) 40 (63%) Age (y) Median (range) 40 (13-62) 43 (13-65) 12-17 8 (13%) 6 (10%) 18-39 23 (37%) 23 (36%) 40-59 27 (44%) 31 (49%) 60-65 4 (7%) 3 (5%) Weight Median (range) 78.6 (43-134) 77.4 (46-133) Race White 35
Slide 10: Patient and transplant characteristics
Patient and transplant characteristics Omidubicel Control (N=62) (N=63) AML 27 (44%) 33 (52%) ALL 20 (32%) 21 (33%) MDS 6 (10%) 3 (5%) Disease CML 4 (7%) 2 (3%) Lymphoma 3 (5%) 2 (3%) Rare Leukemia 2 (3%) 2 (3%) Low 15 (24%) 15 (24%) Disease Risk Index Moderate 27 (44%) 25 (40%) High/Very High 20 (3
Slide 11: Graft characteristics: HLA match & intended number of CBUs to be transplanted
Graft characteristics: HLA match & intended number of CBUs to be transplanted Omidubicel Control (N=62) (N=63) 4/6 46 (74%) 46 (73%) HLA match (CBU #1) 5/6 15 (24%) 16 (25%) 6/6 1 (2%) 1 (2%) 4/6 31 (74%) HLA match (CBU #2) 5/6 10 (24%) 6/6 1 (2%) Intended CBU Single 20 (32%) 21 (33%) transplant Dou
Slide 12: Graft characteristics: cell dose
Graft characteristics: cell dose Median CD34+ cell expansion: 130-fold (range 32-233) 12
Slide 13: Primary endpoint: Time to neutrophil engraftment (ITT population)
Primary endpoint Time to neutrophil engraftment (ITT population) Median Time to Intent-to-treat Neutrophil 95% CI P Value Engraftment (Days)* Omidubicel (N = 62) 12.0 (10.0 – 15.0) <0.001** Control (N = 63) 22.0 (19.0 – 25.0) *Patients not transplanted or who did not engraft by Day 42 post transplan
Slide 14: Neutrophil engraftment (treated population, N = 108)
Neutrophil engraftment (treated population, N = 108) Median Time to Neutrophil P value Engraftment (Days) Omidubicel: 10.0 (95% CI: 8, 13) p<0.001 Control: 20.5 (95%CI: 18, 24) 14
Slide 15: Secondary endpoint: Time to platelet engraftment (ITT population)
Secondary endpoint: Time to platelet engraftment (ITT population) Cumulative Difference in Intent-to-treat Day 42 Cumulative Incidence 95% CI P Value Incidence Omidubicel (N = 62) 0.55 0.20 (0.03 – 0.35) 0.028 Control (N = 63) 0.35 15
Slide 16: Platelet engraftment (treated population, N = 108)
Platelet engraftment (treated population, N = 108) Median Time to Platelet Engraftment P value (Days) Omidubicel: 37 (95% CI: 33, 42) P=0.023 Control: 50 (95%CI: 42, 58) 16
Slide 17: Secondary endpoint: Grade 2-3 bacterial or invasive fungal infection by 100 days
Secondary endpoint: Grade 2-3 bacterial or invasive fungal infection by 100 days (ITT Population) 17
Slide 18: Viral infections (ITT population)
Viral infections (ITT population) 18
Slide 19: Secondary endpoint: Days alive and out of the hospital in the first 100 days
Secondary endpoint: Days alive and out of the hospital in the first 100 days post-transplant (ITT) Omidubicel: Median 60.5 days Control: Median 48.0 days P = 0.005 19
Slide 20: Acute GvHD
Acute GvHD Grade II-IV Acute GVHD Day 100 Grade III-IV Acute GVHD Day 100 20
Slide 21: Chronic GvHD
Chronic GvHD All Chronic GVHD at One Year 21
Slide 22: Non-relapse mortality and relapse (ITT)
Non-relapse mortality and relapse (ITT) Non-Relapse Mortality Relapse 22
Slide 23: Disease-free and overall survival (ITT)
Disease-free and overall survival (ITT) Disease-Free Survival Overall Survival HR 0.59 (95% CI = 0.29 – 1.12) 23
Slide 24: Conclusions
Conclusions • This global phase III randomized study demonstrated that transplantation with omidubicel compared to standard cord blood transplantation results in − Faster hematopoietic recovery − Fewer infections − Fewer days in hospital • Omidubicel should be considered as the new standard of care
Slide 25: Acknowledgements
Acknowledgements Co-Investigators Gamida Cell Mitchell Horwitz Edward Peres Dennis Cooper Tony Peled Patrick Stiff Einat Galamidi-Cohen Corey Cutler Leonid Volodin Irit Segalovich Claudio Brunstein Joanne Kurtzberg David Valcarcel Rabi Hanna Isabel Badell Emmes Corp. Richard Maziarz William Hwang An
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