12 August 2022 - - The first patient in a company-sponsored Phase 1/2 study evaluating a cryopreserved, readily available formulation of GDA-201 for the treatment of follicular and diffuse large B cell lymphomas (NCT05296525), Israel-based biotechnology company Gamida Cell Ltd. (NASDAQ: GMDA) said.

The Phase 1 portion of the study is a dose escalation phase, designed to evaluate the safety of GDA-201, and will include patients with follicular lymphoma, diffuse large B-cell lymphoma (DLBCL)/high grade B-cell lymphoma, marginal zone lymphoma or mantle cell lymphoma.

The Phase 2 expansion phase is designed to evaluate the safety and efficacy of GDA-201 in 63 patients comprised of two cohorts of patients with either FL or DLBCL.

The study will include patients who have relapsed or refractory lymphoma after at least two prior treatments, which may include CAR-T or stem cell transplant.

GDA-201 leverages Gamida Cell's proprietary NAM (nicotinamide) technology platform to expand the number and functionality of NK cells to direct tumor cell killing properties and antibody-dependent cellular cytotoxicity.

In an investigator-sponsored Phase 1/2 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, 13 complete responses and one partial response were observed, with an overall response rate of 74% and a complete response rate of 68%. Two-year data on outcomes and cytokine biomarkers associated with survival data demonstrated a median duration of response of 16 months (range 5-36 months) and an overall survival at two years of 78% (95% CI, 51%–91%).

In this study, GDA-201 was well-tolerated and no dose-limiting toxicities were observed in 19 patients with NHL and 16 patients with multiple myeloma.

The most common Grade 3/4 adverse events were thrombocytopenia, hypertension, neutropenia, febrile neutropenia, and anemia.

There were no incidents of cytokine release syndrome, neurotoxic events, graft versus host disease or marrow aplasia, the company said.

Its NAM-enabled technology, supported by positive Phase 3 data for omidubicel, is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies.

Leveraging the unique properties of NAM, we can expand and metabolically modulate multiple cell types -- including stem cells and NK cells -- with appropriate growth factors to maintain the cells' active phenotype and enhance potency.

Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

Gamida Cell applied the capabilities of its NAM-enabled cell expansion technology to develop GDA-201, an innate NK cell immunotherapy candidate for the treatment of hematologic and solid tumors in combination with standard-of-care antibody therapies.

GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical trial results.

GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, GDA-201 improves ADCC and tumor targeting of NK cells.

There are approximately 40,000 patients with relapsed/refractory lymphoma in the US and EU, which is the patient population that will be studied in the currently ongoing GDA-201 Phase 1/2 clinical trial.

GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.

Gamida Cell is developing an immunotherapy pipeline of potentially curative cell therapy candidates for patients with solid tumor and blood cancers and other serious blood diseases.