FDA Clearance to Gamida Cell for GDA-201 Treatment of Follicular & Diffuse Large B Cell Lymphomas

Gamida Cell Ltd. (Nasdaq: GMDA), the leader in nicotinamide (NAM) -enabled cell therapies for patients with hematologic and solid cancers, has received the green light from the U.S. Food and Drug Administration (FDA) for its investigational new drug (IND) application and removed the clinical hold for a cryopreserved formulation of GDA-201. Pre-clinical data was also presented on Omidubicel, GDA-301, and GDA-601 at professional meetings on cell therapy taking place in the USA.

GDA-201 is an off-the-shelf cell therapy candidate for the treatment of patients with follicular and diffuse large B cell lymphomas. GDA-201 is an innate natural killer (NK) cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. Gamida Cell expects to initiate a company-sponsored Phase 1/2 clinical study in patients with follicular and diffuse large B-cell lymphomas in this calendar year.

“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, Ph.D., chief executive officer of Gamida Cell. “Previously announced data from an investigator-sponsored (IS) 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 1/2 study and progress our novel cryopreserved formulation of GDA-201 with the objective to address the unmet need that exists for patients with follicular and diffuse large B cell lymphomas.”

GDA-201 leverages Gamida Cell’s proprietary NAM technology platform to expand the number and functionality of NK cells to direct tumor cell killing properties and antibody-dependent cellular cytotoxicity (ADCC). 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 (NHL), 13 complete responses and one partial response were observed, with an overall response rate of 74% and a complete response rate of 68%.

At the December 2021 Annual Meeting of the American Society of Hematology, two-year follow-up data were reported on outcomes and cytokine biomarkers associated with survival. The data demonstrated a median duration of response of 16 months (range 5-36 months) and overall survival at two years of 78% (95% CI, 51%–91%). In the IS 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 (CRS), neurotoxic events, GvHD, or marrow aplasia.

In another promising development, Gamida Cell also presented the results of a new study demonstrating the potential impact of access to Omidubicel on health disparities in allogeneic hematopoietic stem cell transplant in a poster presentation at the 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings (TCT), being held in Salt Lake City, UT, April 23-26, 2022. This demonstrated that Omidubicel has the ability to provide therapy as a donor source to minority groups struggling to find a donor match for a transplant,

“Today, minority groups comprise only about 30% of all allogeneic hematopoietic stem cell transplant transplants, indicating that lack of access to a matched donor is a significant barrier to treatment in the current landscape,” said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. “This study is encouraging in that it projects that broad access to Omidubicel has the potential to open up allo-HSCT as an effective treatment for more patients and address the barriers that have contributed to this alarming health disparity. These data are particularly encouraging as we continue to advance our rolling BLA submission to the FDA and move closer to bringing the ther-apy to more patients in need.Gamida Cell initiated a rolling Biologics License Application (BLA) submission for omidubicel in the first quarter of 2022 and is on-track to complete submission of all modules of the BLA in the second quarter of 2022.

On May 5, at the International Society for Cell & Gene Therapy (ISCT) 2022,  held in San Francisco, CA, May 4-7, 2022 Gamida Cell presented preclinical data on their GDA-301 and GDA-601, two product candidates in the Company’s NAM-enabled genetically modified natural killer (NK) pipeline.

“The preclinical data generated from our expanding pipeline of NAM-enabled cell therapies is al-ready showing signs of meaningful potential as a future approach to fighting cancer,” said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. “With evidence of enhanced cytotoxicity demonstrated across hematologic cancers and solid tumors with these diverse, genetically modi-fied NK cell immunotherapy programs, we look forward to continuing our progress toward open-ing new frontiers in cancer immunotherapy.”

About GDA-201

Gamida Cell’s nicotinamide (NAM)-enabled cell expansion technology was applied to develop GDA-201, an innate NK cell immunotherapy 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 antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells.

For more information on the Phase 1/2 clinical trial of GDA-201, please visit www.clinicaltrials.gov.

 

In addition to GDA-201, Gamida Cell is working on two other therapy pipelines,  GDA-301, and the GDA-601, that are based on the NAM-NK cell therapy developed by Gamida Cell.

About GDA-301

GDA-301 is an investigational genetically modified NAM-NK cell therapy candidate aimed at targeting hematologic malignancies and solid tumors. It was demonstrated that after six hours of co-culture with chronic myelogenous leukemia (K562) or multiple myeloma (RPMI) cell line, GDA-301, a combined genetic manipulation of CISH gene editing and the engineered expression of mb IL-15, showed increased cytotoxicity compared with control NAM-NK cells. Additional in vitro assays showed elevation of degranulation marker CD107a, and intracellular proinflammatory cytokines interferon-γ and tumor necrosis factor-α, suggesting increased potency of GDA-301 compared with control. The potency and cytotoxicity data suggest that GDA-301 represents a novel potential immunotherapeutic targeting hematologic malignancies as well as solid tumors.

About GDA-601

GDA-601 is an investigational genetically engineered NAM-NK cell therapy candidate designed to target multiple myeloma (MM) cells. It has been shown that in vitro killing assays performed six hours after co-culture of GDA-601 with a MM (RPMI) cell line showed increased cytotoxicity compared with control NAM-NK cells. Fratricide attributable to CD38 antigen was effectively eliminated with GDA-601. There was a significant enhancement of potency against CD38-positive MM cells demonstrated by elevation of the degranulation marker CD107a and intracellular proinflammatory cytokines interferon-γ and tumor necrosis factor-α in vitro. These results suggest that GDA-601 displays superior antitumoral responses against MM cells and represent a promising adoptive cell therapeutic strategy against MM.

About NAM Technology

Gamida Cell’s NAM-enabling technology, supported by positive Phase 3 data, is designed to enhance the number and functionality of targeted cells, enabling a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (Nicotinamide), it is possible to expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, Gamida Cell’s NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

About Omidubicel

Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant for individuals with hematologic malignancies. It has been granted breakthrough status and orphan drug designation by the FDA as well as orphan drug designation by the EU.

Additionally, Omidubicel is being evaluated in a phase 1/2 clinical study in individuals with severe aplastic anemia (NCT03173937). For more information on clinical trials of Omidubicel, please visit the Gamida Cell website.

Positive 1-Year, Post-Transplant Data on Omidubicel

Advanced cell therapy is under development as an allogeneic hematopoietic stem cell transplant for patients with hematologic malignancies.

Gamida Cell announced 1-year post-transplant data presented on Omidubicel at the 2022 Transplantation & Cellular Therapy Meetings of the American Society for Transplantation and Cellular Therapy and Center for International Blood & Marrow Transplant Research Tandem Meetings, held in Salt Lake City, Utah, from April 23 to April 26, 2022.

In the oral presentation titled “Allogeneic Hematopoietic Stem Cell (allo-HSCT) Transplant with Omidubicel Demonstrates Sustained Clinical Improvement Versus Standard Myeloablative Umbilical Cord Blood Transplantation (UCBT): Final Results of a Phase III Randomized, Multicenter Study,” Mitchell Horwitz, MD, a professor of Medicine at Duke Cancer Institute, shared data that showed sustained clinical benefits in the first-year post-transplant with the therapy, as demonstrated by a significant reduction in infectious complications.

The findings also showed a reduction in non-relapse mortality and no significant increase in relapse rates with Omidubicel at 23% compared with UCBT at 18%.

Investigators concluded that hematopoietic stem cell transplantation (HSCT) with Omidubicel resulted in rapid hematopoietic recovery, reduced rates of infection, and no increase in graft versus host disease rates compared with standard UCBT.

There was a continued trend toward improvement of overall survival with Omidubicel at 73% compared with UCBT at 60%.

The overall and sustained clinical benefit of Omidubicel makes the drug an important addition to options for allogeneic HSCT, investigators said.

“In allo-HSCT, early engraftment and lower infections are the key predictors of long-term success for patients,” Julian Adams, Ph.D, CEO of Gamida Cell, said in a statement.

“We are encouraged by the continuous positive and sustained results from patients involved in the phase 3 trial of Omidubicel, now 1-year out from treatment. These results provide promising rationale that Omidubicel could become a compelling treatment option for patients in need of an allo-HSCT transplant,” Adams said.

The company initiated a rolling biologics license application (BLA) submission for Omidubicel in the first quarter of 2022 and is on track to complete the submission of all modules of the BLA in the second quarter. In total, the company presented 2 oral and 6 poster presentations at TCT 2022 including an oral presentation that was selected as a TCT Best Abstract. All poster presentations are publicly available at www.ASTCT.org.

About Gamida Cell

Gamida Cell is pioneering a diverse immunotherapy pipeline of potentially curative cell therapies for patients with solid tumors and blood cancers and other serious blood diseases. We apply a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapies with the potential to redefine standards of care. These include Omidubicel, an investigational product with potential as a life-saving alternative for patients in need of bone marrow transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumors and hematological malignancies. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook, or Instagram at @GamidaCellTx.

Contacts https://www.gamida-cell.com/

For investors:

Courtney Turiano – Stern Investors Relations, Inc.
Courtney.Turiano AT sternir.com Tel: 1-212-362-1200

For media:
Rhiannon Jeselonis – Ten Bridge Communications
Rhiannon AT tenbridgecommunications.com Tel: 1-978-417-1946

Source: Businesswire April 2020

About JLM- BioCity

News on the many life science and healthcare companies in the Jerusalem area can be seen at https://jlm-biocity.org/jlm-biocity-innovations/

JLM-BioCity is a non-profit organization focused on developing and excelling Jerusalem’s bio-community.

We invite you, researcher, professional, investor, and executive to become part of our vision, in building Jerusalem’s Biomed eco-system.

If you want your company featured in our blogs, or news releases, please contact: info at jlm-biocity.org