Gamida Cell — Technical Presentation (December 2022)
19 slides from Gamida Cell's technical presentation, December 2022
Slide 1: Clinical Outcomes Following Allogeneic HCT With Omidubicel or Other Donor Sources
CLINICAL OUTCOMES FOLLOWING ALLOGENEIC HEMATOPOIETIC CELL TRANSPLANTATION (HCT) WITH OMIDUBICEL OR OTHER DONOR SOURCES IN PATIENTS WITH HEMATOLOGIC MALIGNANCIES: COMPARISON OF CLINICAL TRIAL RESULTS TO CENTER FOR INTERNATIONAL BLOOD AND MARROW TRANSPLANT RESEARCH (CIBMTR) DATABASE CONTROLS SMITHA SI
Slide 2: Background
BACKGROUND ¡ In a global randomized Phase 3 trial, omidubicel, an investigational advanced cell therapy donor source for allogeneic HCT in patients with hematologic malignancies, has demonstrated significantly accelerated time to engraftment, reduced risk of infections and shortened hospital stays v
Slide 3: Objective
OBJECTIVE ¡ Comparative efficacy of omidubicel vs. other donor sources (MUD, MMUD, Haplo) used in clinical practice is unknown − CIBMTR registry data can provide valuable evidence in the absence of head-to-head clinical trial data ¡ The objective of this study was to compare outcomes of patients in
Slide 4: Methods: Patient Inclusion and Exclusion Criteria
METHODS: PATIENT INCLUSION AND EXCLUSION CRITERIA ¡ CIBMTR cohort selection criteria were harmonized with the omidubicel trial − Key inclusion/exclusion criteria from the trial were applied to the CIBMTR cohort • First allo-HCT in the US between January 1, 2017, and December 31, 2019 • Age at transp
Slide 5: Methods: Statistical Analyses
METHODS: STATISTICAL ANALYSES ¡ The following baseline characteristics were included in all adjusted analyses: − Age − Sex − Weight − Race − Primary diagnosis − Karnofsky/Lansky performance score − Hematopoietic cell transplant-comorbidity index (HCT-CI) − Disease risk index (DRI)* − Year of transpl
Slide 6: Results: Baseline Characteristics
RESULTS : BASELINE CHARACTERISTICS Omidubicel MUD MMUD Haplo UCB (N=52) (N=450) (N=65) (N=328) (N=56) P value Age at transplant (years), mean ± SD 38.9 ± 16.0 45.8 ± 13.8 42.9 ± 15.2 40.0 ± 15.3 35.6 ± 15.1 < 0.001 Male, n (%) 27 (51.9) 268 (59.6) 32 (49.2) 186 (56.7) 35 (62.5) Caucasian, n (%) 31 (
Slide 7: Results: Baseline Characteristics (Cont'd)
RESULTS : BASELINE CHARACTERISTICS (CONT’D) Omidubicel MUD MMUD Haplo UCB (N=52) (N=450) (N=65) (N=328) (N=56) P value Karnofsky/Lansky PS <90, n (%) 12 (23.1) 170 (37.8) 24 (36.9) 131 (39.9) 14 (25.0) HCT-CI ≥3, n (%) 27 (51.9) 201 (44.7) 29 (44.6) 137 (41.8) 28 (50.0) Body weight (kg), mean ± SD 7
Slide 8: Neutrophil Engraftment
RESULTS: NEUTROPHIL ENGRAFTMENT ¡ Omidubicel was associated with faster rates of neutrophil engraftment compared with other donor sources after adjusting for baseline variables 1.0 Omidubicel MUD MMUD Haplo 0.8 UCB Probability 0.6 HR relative to omidubicel P value (95% CI) 0.4 MUD 0.65 (0.47-0.89) 0
Slide 9: Platelet Engraftment
RESULTS: PLATELET ENGRAFTMENT ¡ Omidubicel was associated with a slower rate of platelet engraftment compared with other donor sources except UCB after adjusting for baseline variables 1.0 Omidubicel MUD MMUD Haplo 0.8 UCB Probability 0.6 HR relative to omidubicel P value 0.4 (95% CI) MUD 2.72 (2.07
Slide 10: Acute GvHD
RESULTS: ACUTE GVHD ¡ Compared with other donor sources, omidubicel had significantly higher rates of acute GvHD Grade II-IV, however no significant differences were observed for severe acute GvHD (grade III-IV) Grade II-IV Grade III-IV Omidubicel Omidubicel P = 0.14 0.7 MUD P < 0.001 0.7 MUD MMUD M
Slide 11: Chronic GvHD
RESULTS: CHRONIC GVHD ¡ No statistically significant difference for chronic GvHD between omidubicel and other donor sources after adjusting for baseline variables HR relative to omidubicel P value (95% CI) MUD 0.82 (0.52-1.30) 0.40 0.7 Omidubicel MUD MMUD 1.21 (0.69-2.09) 0.51 MMUD Haplo 0.67 (0.42-
Slide 12: Non-Relapse Mortality and Disease Relapse
RESULTS: NON-RELAPSE MORTALITY AND DISEASE RELAPSE ¡ The differences in relapse and non-relapse mortality between omidubicel and other donor sources were not statistically significant after adjusting for baseline variables Non-relapse mortality Disease relapse Omidubicel Omidubicel 0.7 MUD HR relati
Slide 13: Disease-Free Survival (DFS)
RESULTS: DISEASE-FREE SURVIVAL (DFS) ¡ No statistically significant difference between omidubicel and other donor sources for DFS after adjusting for baseline variables HR relative to 100% omidubicel P value (95% CI) 1.23 (0.72-2.09) 0.45 Proportion Free of Event MUD MMUD 1.33 (0.71-2.48) 0.38 Haplo
Slide 14: Overall Survival
RESULTS: OVERALL SURVIVAL ¡ No statistically significant differences for overall survival between omidubicel and other donor sources after adjusting for baseline variables HR relative to omidubicel P value (95% CI) 100% MUD 1.44 (0.75-2.76) 0.27 MMUD 1.46 (0.68-3.14) 0.33 Proportion Free of Event Ha
Slide 15: Limitations of Current Research
LIMITATIONS OF CURRENT RESEARCH ¡ Comparisons between clinical trial and registry data are limited by: − Heterogeneity in baseline characteristics − Inconsistent definitions of outcomes − Differences in standard of care between patients in registry vs clinical trial setting • Intensity and schedule
Slide 16: Conclusions
CONCLUSIONS Compared with results from existing donor sources (MUD, MMUD, Haplo) reported to CIBMTR, omidubicel was associated with ¡ More rapid rate of neutrophil engraftment ¡ Slower rate of platelet engraftment ¡ Comparable rates of grade III-IV acute GvHD and chronic GvHD ¡ Comparable overall su
Slide 17: Acknowledgements
ACKNOWLEDGEMENTS ¡ We thank: − The investigators and patients who participated in the clinical trial − The centers who contributed data to the CIBMTR database − The patients who provided consent for their data to be used by the CIBMTR in research − Everyone at CIBMTR who contributed to this research
Slide 18: Backup
BACKUP 18
Slide 19: Subgroup Analysis of Neutrophil Engraftment Among Those Receiving Growth Factors
RESULTS: SUBGROUP ANALYSIS OF NEUTROPHIL ENGRAFTMENT AMONG THOSE RECEIVING GROWTH FACTORS (N=708) ¡ There were similar results for 1.0 neutrophil engraftment in subgroup Omidubicel MUD analyses restricting to patients who MMUD Haplo received G-CSF/GM-CSF (n=108 in 0.8 UCB omidubicel trial, n=600 acr