Our R&D

High-risk Hematologic Malignancies

About Omidubicel

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). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU.

Development status

Omidubicel has been studied in an international, multi-center, randomized Phase 3 clinical study evaluating its safety and efficacy compared to standard umbilical cord blood transplant.

• The study achieved its primary endpoint (p<0.001). In the intent-to-treat analysis, median time to neutrophil engraftment was 12 days for patients receiving omidubicel (95% CI: 10-15 days) compared to 22 days for the comparator group (95% CI: 19-25 days).
• Omidubicel was generally well tolerated. Among patients who were transplanted per protocol, rates of acute and chronic graft-versus host disease were similar and cumulative incidence of infections was significantly smaller in omidubicel compared to controls for both viral infections and bacterial or invasive fungal infections.
• The study also met all three of its secondary endpoints, improving platelet engraftment, and reductions in infections and hospitalizations, which are key measures for success for bone marrow transplant.

The Phase 3 data also show that during the first 100 days of treatment, omidubicel-treated patients had significantly shorter durations of hospitalization, intensive care unit time, consultant visits, procedures, and transfusions than the control arm. These data provide further evidence of the clinical benefit associated with the more rapid hematopoietic recovery in patients treated with omidubicel and the corresponding reduction in healthcare resource utilization.

Full safety and efficacy data from this Phase 3 study have been published in Blood.

In February 2022, we initiated the Biologics License Application (BLA) rolling submission process for omidubicel with the U.S. Food and Drug Administration. We are on track to complete the BLA submission in the second quarter of 2022. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia, a rare and life-threatening blood disorder.

Non-Hodgkin Lymphoma, Multiple Myeloma

About GDA-201

GDA-201 is an innate natural killer (NK) cell immunotherapy candidate for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. When combined with targeted antibodies, GDA-201 has shown enhanced antibody-dependent cellular toxicity, or ADCC. In April 2022, the U.S. Food and Drug Administration (FDA) cleared our investigational new drug (IND) application and removed the clinical hold for a cryopreserved formulation of GDA-201.

Clinical Trial Information

The study of the cryopreserved formulation of GDA-201 is currently open to enrollment at Henry Ford Cancer Institute (Detroit, MI) and the Masonic Cancer Center at the University of Minnesota, and additional sites will be added in the coming months. The Phase 1 portion of the study is designed to evaluate the safety of GDA-201 in patients with follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL)/high grade B-cell lymphoma (HGBCL), marginal zone lymphoma or mantle cell lymphoma. The Phase 2 expansion phase is designed to evaluate the safety and efficacy of GDA-201 in two patient cohorts, FL and DLBCL/HGBCL. The study will include patients who have relapsed or refractory lymphoma after at least two prior treatments. For more information about the study, please visit www.clinicaltrials.gov (NCT03019666).

GDA-201 has been evaluated in a Phase 1 investigator-led study in patients with refractory non-Hodgkin lymphoma (NHL) and multiple myeloma, demonstrating that GDA-201 generally was well tolerated in 35 patients. Of the 19 patients with NHL, 13 complete responses and one partial response were observed (CRR = 68%, ORR = 74%). No dose-limiting toxicities were observed.

Two-year follow up data in the same Phase 1 study demonstrated an overall survival rate of 78% at two years, with a median duration response of 16 months (Bachanova, et al. ASH 2021. Abstract #3854).