FDA Removed Clinical Hold from Cryopreserved NK Cell Therapy Candidate

The U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for natural killer (NK) cell therapy candidate GDA-201 and removed the clinical hold for a cryopreserved formulation of the therapy, according to a company press release.

GDA-201 is an off-the-shelf therapy candidate for the treatment of follicular lymphoma and diffuse large B-cell lymphoma.

“FDA clearance of our IND for the cryopreserved formulation of GDA-201 represents a significant milestone for the company and reflects our team’s expertise in the development of NAM-enabled cell therapies,” said Julian Adams, PhD, CEO of Gamida Cell, manufacturer of GDA-201. “Previously announced data from an investigator-sponsored study evaluating the fresh formulation of GDA-201 demonstrated durable complete responses in heavily pretreated patients with relapsed or refractory lymphoma. We are pleased to advance our plans to begin the company sponsored phase I/II study and progress our novel cryopreserved formulation of GDA-201 with objective to address the unmet need that exists for patients with follicular and diffuse large B-cell lymphomas.”

In the investigator-sponsored phase I/II study in patients with relapsed or refractory lymphoma, treatment with the fresh formulation of GDA-201 with rituximab demonstrated significant clinical activity.

Of the 19 patients with non-Hodgkin lymphoma (NHL), 13 had a complete response and one had a partial response. The overall response rate was 74%, with a complete response rate of 68%.

The company presented two-year follow-up data at the 2021 ASH Annual Meeting, including data on outcomes and cytokine biomarkers associated with survival. The data demonstrated a median duration of response of 16 months and an overall survival at two years of 78%.

GDA-201 was well-tolerated, and no dose-limiting toxicities were observed in 19 patients with NHL and 16 with multiple myeloma. The most common grade 3/4 adverse events were thrombocytopenia, hypertension, neutropenia, febrile neutropenia, and anemia. There was no incidents of cytokine release syndrome, neurotoxic events, graft-versus-host disease, or marrow aplasia.

Source: Gamida Cell Press Release, April 2022