Gamida Cell — Technical Presentation (2022)
16 slides from Gamida Cell's technical presentation, Jul 14, 2022
Slide 1: Allo-HSCT transplant with omidubicel versus standard UCBT: final Phase III results
ALLOGENEIC HEMATOPOIETIC STEM CELL (ALLO-HSCT) TRANSPLANT WITH OMIDUBICEL DEMONSTRATES SUSTAINED CLINICAL IMPROVEMENT VERSUS STANDARD MYELOABLATIVE UMBILICAL CORD BLOOD TRANSPLANTATION (UCBT): FINAL RESULTS OF A PHASE III RANDOMIZED, MULTICENTER STUDY MITCHELL E. HORWITZ, MD1, PATRICK STIFF, MD2, CO
Slide 2: Disclosures
DISCLOSURES ¡ Gamida Cell – Research funding ¡ Equillium – Data and Safety Monitoring Board member ¡ Allovir – Advisory Board 2
Slide 3: Nicotinamide (NAM): preserves stem cells by mimicking the bone marrow niche
NICOTINAMIDE (NAM): PRESERVES STEM CELLS BY MIMICKING THE BONE MARROW NICHE Histone NAD+ acetylation enzymes NICOTINAMIDE Gene expression st Ce ¡ Master regulator of NAD-related signaling pathways s re llu as lm ss la on d ri a ol is m ,R r O NAD+ ¡ Enhances cellular functionality and phenotype ch a
Slide 4: Omidubicel manufacturing
OMIDUBICEL MANUFACTURING Omidubicel CBU selected by NAM-Expanded Non-Cultured Omidubicel Infusion treating physician from Cells* Fraction* public cord blood bank Stem cells cultured Immune cells, using proprietary including T cells NAM technology *Fractions are cryopreserved for transport and thawed
Slide 5: Phase 3 global, randomized study of omidubicel compared to standard UCB transplantation
PHASE 3 GLOBAL, RANDOMIZED STUDY TO EVALUATE THE EFFICACY OF OMIDUBICEL COMPARED TO STANDARD UCB TRANSPLANTATION N=125 Omidubicel (n=62) ¡ Age 12-65 ¡ High-risk hematologic malignancies R ¡ Eligible for allogeneic hematopoietic transplantation UCB (n=63) ¡ No readily available matched donor (standar
Slide 6: Transplant with omidubicel: significantly faster time to engraftment
TRANSPLANT WITH OMIDUBICEL: SIGNIFICANTLY FASTER TIME TO ENGRAFTMENT* Neutrophil Engraftment † by Day 42 Platelet Recovery † by Day 100 1.0 Randomized treatment 1.0 Randomized treatment Omidubicel Omidubicel 0.8 UCB 0.8 UCB Cumulative Incidence Cumulative Incidence 0.6 0.6 0.4 0.4 0.2 0.2 0.0 0.0 0
Slide 7: Reduced risk of bacterial/fungal infections and hospitalization in first 100 days
TRANSPLANT WITH OMIDUBICEL: REDUCED RISK OF BACTERIAL/FUNGAL INFECTIONS AND HOSPITALIZATION IN FIRST 100 DAYS* Grade 2/3 Bacterial or Invasive Fungal Infections Days Alive and Out of the Hospital 1.0 Randomized treatment 100 Difference 12.5 days (P=0.005) Omidubicel Omidubicel 90 UCB UCB Cumulative
Slide 8: Robust early recovery for T cell, B cell, and NK cell subsets
ROBUST EARLY RECOVERY OBSERVED FOR T CELL, B CELL, AND NK CELL SUBSETS (DAY 0 TO DAY 28) CD4+ cells B cells NK cells Day 7 Day 14 Day 21 Day 28 Day 7 Day 14 Day 21 Day 28 Day 7 Day 14 Day 21 Day 28 200 400 10 50 10 700 P=0.022 180 350 9 45 9 P=0.013 600 160 P=0.003 8 40 8 P=0.026 300 140 7 35 7 500
Slide 9: 1-year update analysis
1-YEAR UPDATE ANALYSIS For this 1-year update analysis*: 1. All patients have completed 15 months of follow-up from randomization − Median follow-up (ITT population): 13.9 mo (omidubicel arm) and 14.1 mo (UCB arm) 2. The following endpoints were analyzed at 1 year post transplant: − Infectious compl
Slide 10: Infectious complications at year post transplant
INFECTIOUS COMPLICATIONS AT 1 YEAR POST TRANSPLANT* First Grades 2-3 Bacterial or Invasive Fungal Infection First Grade 3 Viral Infection 1.0 Randomized treatment 1.0 Randomized treatment Omidubicel Omidubicel UCB UCB Cumulative Incidence Cumulative Incidence 0.8 P=0.007 70% 0.8 0.6 0.6 0.4 45% 0.4
Slide 11: Chronic GvHD
CHRONIC GVHD* Chronic GvHD (All Grades) Moderate/Severe Chronic GvHD 1.0 Randomized treatment 1.0 Randomized treatment Omidubicel Omidubicel UCB UCB Cumulative Incidence Cumulative Incidence 0.8 0.8 0.6 0.6 0.4 34% 0.4 P=0.550 P=0.984 23% 0.2 29% 0.2 23% 0.0 0.0 0 90 180 270 365 0 90 180 270 365 Day
Slide 12: Non-relapse mortality
NON-RELAPSE MORTALITY* 1.0 Randomized treatment Omidubicel 0.8 UCB Cumulative Incidence 0.6 Difference 14% (95% CI: 0 to 28; P=0.07) 0.4 29% 0.2 15% 0.0 0 60 120 180 240 300 360 420 480 Days Post Randomization *Intent-to-treat population 1 year following transplantation (N=125). CI, confidence inter
Slide 13: Overall survival
OVERALL SURVIVAL* 1.0 Probability of Overall Survival 0.8 73% 0.6 60% 0.4 Difference 13% (95% CI: –5% to 28%; P=0.13)† 0.2 Randomized treatment Omidubicel UCB 0.0 0 60 120 180 240 300 360 420 480 Days Post Randomization No. at risk Omidubicel 62 62 57 54 49 49 46 45 10 UCB 63 61 54 43 43 41 39 38 16
Slide 14: Disease-free survival and relapse
DISEASE-FREE SURVIVAL AND RELAPSE* Disease-Free Survival Relapse† 1.0 1.0 Randomized treatment Probability of Disease-Free Survival Omidubicel Cumulative Incidence of Relapse UCB 0.8 P=0.448 0.8 63% 0.6 0.6 56% 0.4 0.4 P=0.320 23% 0.2 Randomized treatment 0.2 Omidubicel UCB 16% 0.0 0.0 0 60 120 180
Slide 15: Adverse events
ADVERSE EVENTS For this 1-year update: ¡ No unexpected severe adverse events ¡ Low secondary graft failure − Stable hematopoiesis has been observed (Lin C, et al. Poster #322) − One patient in omidubicel arm at ~6 months following transplantation, concurrent with a diagnosis of ALL relapse − No case
Slide 16: Conclusions
CONCLUSIONS HCT with omidubicel is associated with rapid hematopoietic recovery, reduced rates of infections, and no increase in acute or chronic GvHD rates compared with standard UCB No unexpected adverse events attributable to ex vivo expansion were observed Infectious complications remain signifi