Gamida Cell — Technical Presentation (December 2021)
12 slides from Gamida Cell's technical presentation, December 2021
Slide 1: HSCT With Omidubicel Is Associated With Robust Immune Reconstitution and Lower Rates of Severe Infection
Hematopoietic Stem Cell Transplantation (HSCT) With Omidubicel Is Associated With Robust Immune Reconstitution and Lower Rates of Severe Infection Compared to Standard Umbilical Cord Blood Transplantation Paul Szabolcs, MD1, Stuart Levy, MPH2, Dima Yackoubov, MSc2, Aviad Pato, PhD2, Einat Galamidi-C
Slide 2: Acknowledgments
Acknowledgments • Patients and families who participated in this research • Co-investigators and centers who participated in the substudy – Isabel Badell, Hospital Sant Pau, Spain – Claudio Brunstein, University of Minnesota, MN, USA – Olga Frankfurt, Northwestern University Hospital, IL, USA – Nels
Slide 3: Limitations of Cord Blood Transplantation
Limitations of Cord Blood Transplantation • Delayed hematopoietic recovery Potential solution: ex vivo manipulation • Delayed immune recovery of cord blood stem cells (omidubicel) • Increased transplant related mortality Historical data show that there is a delay in However, a robust recovery of NK
Slide 4: Omidubicel Using Nicotinamide Technology Delivers Full Complement of CD34+ Progenitors Cells and Immune Cells
Omidubicel Using Nicotinamide Technology Delivers Full Complement of CD34+ Progenitors Cells and Immune Cells • Using nicotinamide-based proprietary technology to expand UCB CD34+ cells preserves the multipotency of progenitor cells for long-term repopulation, while increasing cell quantity for tran
Slide 5: Transplant With Omidubicel: Significantly Faster Time to Engraftment
Transplant With Omidubicel: Significantly Faster Time to Engraftment Completed global phase III trial (n=125); 108 patients with hematologic malignancies treated with omidubicel vs standard UCB transplantation following myeloablative conditioning and with no serotherapy Neutrophil engraftment by 42
Slide 6: Transplant With Omidubicel: Reduced Risk of Bacterial, Fungal, and Viral Infections
Transplant With Omidubicel: Reduced Risk of Bacterial, Fungal, and Viral Infections Incidence of first grade 2/3 bacterial or Incidence of first grade 3 invasive fungal infections (ITT; N=125) viral infection (ITT; N=125) 1.0 Randomized treatment Randomized treatment 1.0 First Grade 2/3 Bacterial or
Slide 7: Immune Reconstitution Substudy Following Omidubicel Transplantation
Immune Reconstitution Substudy Following Omidubicel Transplantation Research questions: 1. Is rapid neutrophil engraftment followed by rapid recovery of other cell lineages? 2. What is driving the reduced risk of infections? Methods • Optional substudy – 14 participating clinical centers • 37 patien
Slide 8: Substudy: Baseline Patient and Graft Characteristics
Substudy: Baseline Patient and Graft Characteristics Omidubicel (n=17) UCB (n=20) Median age (range), years 30 (13–62) 43 (19–55) Male, n (%) 10 (59) 12 (60) Primary diagnosis, n (%) Acute leukemia (ALL, AML) 14 (82) 18 (90) Other (CML, MDS, lymphoma) 3 (18) 2 (10) Disease risk, n (%) Medium – high/
Slide 9: Substudy: Patient Outcomes
Substudy: Patient Outcomes Omidubicel UCB (n=20) P-value (n=17) Median time to neutrophil engraftment (range), 10 (6–28) 18.5 (14–40) <0.001 days Grade 2/3 infections over 365 days, n (%) Bacterial Infections 7 (41) 14 (70) =0.037 Viral Infections 1 (6) 9 (45) =0.010 Patients with steroid use in fir
Slide 10: Robust Early Recovery Observed for T cell, B cell, NK Cell and Dendritic Cell Subsets (Day 0 to Day 28)
Robust Early Recovery Observed for T cell, B cell, NK Cell and Dendritic 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 10 700 350 P=0.013 600 P=0.026 P=0.003 8 40 8 150 300 P=0.022 500 P=0.001
Slide 11: Durability of Recovery Observed for up to 1 Year Post-transplant (Day 100 to Day 365)
Durability of Recovery Observed for up to 1 Year Post-transplant (Day 100 to Day 365) CD4+ cells B cells NK cells Day 100 Day 180 Day 365 Day 100 Day 180 Day 365 Day 100 Day 180 Day 365 3000 6000 1000 2500 5000 P=0.028 P=0.001 P=0.017 800 2000 4000 600 1500 3000 400 1000 2000 500 1000 200 0 0 0 mDC
Slide 12: Conclusions
Conclusions • HSCT with omidubicel results in rapid hematopoietic recovery, reduced rates of infections, and no increase in GvHD rates compared with standard UCB • In the immediate post-transplant period (up to Day 28), immune cell recovery was significantly faster with omidubicel compared with stan