Gamida Cell Presents Efficacy and Safety Results of Phase 3 Study of Omidubicel in Patients with Hematologic Malignancies at the 2021 TCT Meetings of ASTCT and CIBMTR
— Omidubicel represents a potentially transformative cell therapy treatment option for patients in need of a bone marrow transplant —
—Newly presented data support clinical benefit demonstrated by primary and secondary endpoints—
—Company anticipates BLA submission in second half of 2021—
This clinical data set was from the international, multi-center, randomized Phase 3 study of omidubicel that was designed to evaluate the safety and efficacy of omidubicel in patients with high-risk hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. This is the first presentation of these data in a peer-reviewed conference. The full presentation is available on the
“The results of this global Phase 3 study of omidubicel in patients with hematologic malignancies show that omidubicel resulted in faster hematopoietic recovery, fewer bacterial and viral infections and fewer days in hospital, all of which are meaningful results and represent potentially important advancements in care when considering the patient experience following transplant,” said
Details of Phase 3 Efficacy and Safety Results Shared at the TCT Meetings
Patient demographics including racial and ethnic diversity and baseline characteristics were well-balanced across the two study groups. The study’s intent-to-treat analysis included 125 patients aged 13–65 years with a median age of 41. Diseases included acute lymphoblastic leukemia, acute myelogenous leukemia, chronic myelogenous leukemia, myelodysplastic syndrome or lymphoma. Patients were enrolled at more than 30 clinical centers in
All three secondary endpoints demonstrated a statistically significant improvement among patients who were randomized to omidubicel in relation to patients randomized to the comparator group (intent-to-treat). Platelet engraftment was significantly accelerated with omidubicel, with 55 percent of patients randomized to omidubicel achieving platelet engraftment at day 42, compared to 35 percent for the comparator (p = 0.028). The rate of infection was significantly reduced for patients randomized to omidubicel, with the cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel of 37 percent, compared to 57 percent for the comparator (p = 0.027). Hospitalization in the first 100 days after transplant was also reduced in patients randomized to omidubicel, with a median number of days alive and out of hospital for patients randomized to omidubicel of 60.5 days, compared to 48.0 days for the comparator (p = 0.005). The details of these data were first reported in
Previously unpublished data from the study relating to exploratory endpoints also support the clinical benefit demonstrated by the study’s primary and secondary endpoints. There was no statistically significant difference between the two patient groups related to grade 3/4 acute GvHD (14 percent for omidubicel, 21 percent for the comparator) or all grades chronic GvHD at one year (35 percent for omidubicel, 29 percent for the comparator). Non-relapse mortality was shown to be 11 percent for patients randomized to omidubicel and 24 percent for patients randomized to the comparator (p=0.09).
These clinical data results will form the basis of a Biologics License Application (BLA) that
“We believe that omidubicel has the potential to transform the field of hematopoietic bone marrow transplant by expanding access to this potentially curative cell therapy treatment for thousands of patients who are in need of a transplant but lack access to a matched related donor,” said
Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated.1,2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn®, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.
Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
Cautionary Note Regarding Forward Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of anticipated regulatory submissions, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the progress and expansion of Gamida Cell’s manufacturing capabilities and other commercialization efforts and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cell’s Annual Report on Form 20-F, filed with the
1 Horwitz M.E., Wease S., Blackwell B., Valcarcel D. et al. Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol. 2019 Feb 10;37(5):367-374.
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