Gamida Cell Provides Pipeline Update, Including Detailed Results of Pivotal Phase 3 Clinical Study of Omidubicel, and Prepares to Start BLA Submission by End of 2020
—Omidubicel results in improved clinical outcomes as measured by reduction in time to neutrophil engraftment, time in hospital and infections following bone marrow transplant—
— Company announces omidubicel commercial readiness plan, including the creation of Gamida Cell Assist, to support a positive patient and transplant center experience in preparation for potential launch of omidubicel as early as Q4 2021, subject to ongoing FDA discussions—
—Provides update on Phase 1 study of investigational natural killer cell therapy GDA-201 and that Phase 2 study of omidubicel in patients with severe aplastic anemia has demonstrated sustained early engraftment, as reported at ASH Annual Meeting—
“Our goal with omidubicel is to revolutionize the field of bone marrow transplantation and bring a potentially curative cell therapy option to thousands of patients who are in need of a bone marrow transplant, but lack a suitable stem cell donor. These results bring us one step closer towards that goal,” said
These pivotal data form the basis of a Biologics License Application (BLA) that
The live event will be available here. More information about the Phase 3 study of omidubicel and the other updates included in this release can be found in the Pipeline Deep Dive presentation on the
Details of Phase 3 Endpoints
As previously reported,
- Platelet engraftment was significantly accelerated with omidubicel, with 55 percent of patients randomized to omidubicel achieving platelet engraftment at day 42, compared to 28 percent for the comparator (p = 0.028).
- Infection rates were significantly reduced for patients randomized to omidubicel. The cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel was 37 percent, compared to 57 percent for the comparator (p = 0.027).
- Total days in hospital were reduced in patients randomized to omidubicel. The median number of days alive and out of hospital for patients randomized to omidubicel was 60.5 days, compared to 48.0 days for the comparator (p = 0.005).
The international, multi-center, randomized Phase 3 study for omidubicel was designed to evaluate the safety and efficacy of omidubicel in patients with hematologic malignancies undergoing allogeneic bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant.
The company anticipates reporting the full data set in a peer-reviewed setting in the first half of 2021.
The company discussed the market potential for omidubicel and launch plans. These included quantifying the market opportunity and keys aspects for a successful launch.
As it prepares for the potential commercial launch of omidubicel, the company also announced plans for the Gamida Cell Assist program, which has been designed to focus on patient access and support of every individual and their caregiver at each step of the transplant process. Once the program is launched, the Gamida Cell Assist case management team would provide a consistent, single point of contact for patients and health care professionals. This team would work with the transplant center to track each individual patient’s omidubicel therapy and provide real-time updates on the status of the therapy. Gamida Cell Assist is also designed to provide additional services, including coverage and reimbursement support, and patient and caregiver support, which may include financial, travel, and lodging assistance.
“At Gamida Cell we are inspired to cure, with the goal of pioneering new standards of care for patients with blood cancers and serious blood diseases,” said
Update on Natural Killer Cell Therapy GDA-201
In an oral presentation at the recent
Phase 2 Study of Omidubicel in Patients with Severe Aplastic Anemia
In a poster presentation at ASH, it was shown that patients with severe aplastic anemia treated with omidubicel achieved sustained early engraftment. These data, which were presented on
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.12 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
GDA-201 is an investigational therapy, and its safety and efficacy has not been established by the
About the NAM Therapeutic Platform
Gamida Cell’s proprietary NAM-based cell expansion platform is designed to enhance the number and functionality of donor cells in culture, enabling the creation of potentially transformative therapies that move beyond what is possible with existing approaches. The NAM therapeutic platform leverages the unique properties of nicotinamide to enable the expansion of multiple cell types — including stem cells and natural killer (NK) cells — with appropriate growth factors to maintain the cells' original phenotype and potency. This can enable the administration of a therapeutic dose of cells with the potential to improve patient outcomes.
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 initiation and progress of and data reported from the clinical trials of Gamida Cell’s product candidates, anticipated regulatory filings, launch readiness and FDA approval, commercialization efforts and Gamida Cell’s expectations regarding its projected ongoing operating activities, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Gamida Cell’s clinical trials and ramifications for the cost thereof; 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.
3 Clinicaltrials.gov identifier NCT03019666
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