On November 15, 2021 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, reported financial results for the quarter ended September 30, 2021 (Press release, Gamida Cell, NOV 15, 2021, View Source [SID1234595636]). Net loss for the third quarter of 2021 was $19.6 million, compared to a net loss of $14.8 million for the same period in 2020. As of September 30, 2021, Gamida Cell had total cash and cash equivalents of $120.8 million.
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During the past quarter, Gamida Cell:
Continued to execute on plans to submit a Biologic License Application (BLA) for omidubicel, a potentially life-saving treatment for patients with blood cancers in need of stem cell transplant. As previously disclosed, in a recent pre-BLA meeting, the FDA requested a revised analysis of the manufacturing data generated at Gamida Cell’s wholly owned commercial manufacturing facility to demonstrate the comparability to the omidubicel that was produced at the clinical manufacturing sites for the Phase 3 study. The FDA did not request additional clinical data to initiate the BLA submission once analytical comparability is demonstrated.
Progressed activities with objective to address the FDA’s Clinical Hold on the Investigational New Drug (IND) application for GDA-201, which was imposed based on questions about donor eligibility procedures and sterility assay qualification prior to the initiation of the study in patients with follicular and diffuse large B-cell lymphomas.
Expanded the company’s NAM-enabled natural killer (NK) cell pipeline targeting solid-tumor and hematological cancers, including genetically modified variants of proprietary NK therapies using both CRISPR/Cas9 and CAR methodologies.
"We are committed to advance our programs and bring our important potential therapies to patients as quickly as possible. We are working diligently to respond to the FDA’s information requests for omidubicel and GDA-201, and now expect to submit the BLA for omidubicel to the FDA in the first half of 2022 and we hope to promptly address outstanding issues regarding our IND application relating to GDA-201." said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "Additionally, at our recent NK-focused R&D Day, we provided details on our genetically modified NK cell immunotherapy programs leveraging CAR- and CRISPR-mediated strategies against hematologic malignancies and solid tumors. The company remains focused on our goal of bringing patients with cancer potentially curative cell therapies."
Recent Developments and Planned Presentations at ASH (Free ASH Whitepaper)
Omidubicel: Advanced Cell Therapy
BLA Submission: During a recent pre-BLA meeting, the FDA requested that Gamida Cell provide revised analysis of the manufacturing data generated at Gamida Cell’s wholly owned commercial manufacturing facility. Upon completing those requirements, the company anticipates submitting the BLA in the first half of 2022.
New data to be presented at ASH (Free ASH Whitepaper): Gamida Cell will have three omidubicel presentations – two presentations of additional data from the phase III randomized trial of omidubicel, and a poster presentation summarizing long term omidubicel data from multiple studies – at the 63rd American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (December 11-14, 2021).
Oral presentation of "Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel is Associated with Robust Immune Reconstitution and Lower Rates of Severe Infection Compared to Standard Umbilical Cord Blood Transplantation" on Saturday, December 11, 2021, at 4:30 p.m. ET. Data collected from a subset of 37 patients in the omidubicel Phase III trial shows that, in addition to more rapid short-term hematopoietic recovery, omidubicel-treated patients had more rapid recovery of a wide variety of immune cells including CD4+ T cells, B cells, monocytes, natural killer cells, and dendritic cells. The robust recovery of the broad range of the immune system correlated with and supports clinical data showing fewer severe bacterial, fungal, and viral infections in patients treated with omidubicel.
Poster presentation of "Hospitalization and Healthcare Resource Use of Omidubicel vs. Cord Blood Transplantation for Hematological Malignancies in a Global Randomized Phase III Clinical Trial" on Monday, December 13, 2021, 6:00-8:00 p.m. ET. Resource utilization data during the first 100 days after transplant were analyzed for 108 patients in the phase III trial and shows that omidubicel-treated patients has 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.
Poster presentation, "Allogeneic Stem Cell Transplantation with Omidubicel: Long-Term Follow-up from a Single Center" on Saturday, December 11, 2021, 5:30-7:30 p.m. ET. Analysis of outcomes of 22 patients with hematologic malignancies treated with omidubicel at Duke University over a 10-year period shows long-term sustained bone marrow function and immune recovery, with a 10-year overall survival of 48%. These data provide further support for the long-term clinical benefit of omidubicel with long-lasting hematopoietic recovery.
GDA-201: NAM-Enabled NK Cell Therapy
IND for Phase 1/2 Study: Gamida Cell is working to address the clinical hold on the IND for a Phase 1/2 study of GDA-201. As a result of the clinical hold, the initiation of our planned Phase 1/2 study of GDA-201 will be delayed beyond the end of 2021, as the company previously projected.
New data presented at SITC (Free SITC Whitepaper): Gamida Cell recently presented promising new preclinical data in two posters characterizing the NAM-enabled mechanisms of action that contribute to the metabolic modulation properties and enhanced tumor cytotoxicity activity of GDA-201 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 36th Annual Meeting (SITC 2021) held from November 10-14, 2021.
New data to be presented at ASH (Free ASH Whitepaper): A poster titled "GDA-201, A Novel Metabolically Enhanced Allogeneic Natural Killer (NK) Cell Product Yields High Remission Rates in Patients with Relapsed/Refractory Non-Hodgkin Lymphoma (NHL): 2-year survival and correlation with cytokine IL7" will be presented at the upcoming ASH (Free ASH Whitepaper) Annual Meeting and Exposition on Monday, December 13, 2021, 6:00-8:00 p.m. ET. This analysis provides longer follow-up in the investigator-led study of GDA-201 in patients with non-Hodgkin lymphoma and demonstrated an overall survival rate of 78% at two years, median duration of response of 16 months, and a safety profile that was similar to what had been previously reported.
NAM-Enabled NK Cell Pipeline Expansion
Advanced NAM-enabled genetically modified NK pipeline: During Gamida Cell’s NK-focused virtual R&D Day, the company presented new data and additional details on its genetically modified NK cell immunotherapy programs, which utilize CAR, membrane bound- and CRISPR-mediated strategies to increase targeting, potency and persistence against hematologic malignancies and solid tumors:
GDA-301: Knockout of CISH (cytokine inducible SH2 containing protein) in NK cells using CRISPR/Cas9 in combination with a membrane-bound IL-15/IL-15Ra;
GDA-501: anti HER2 CAR-engineered NK cells to target solid tumors expressing HER2, based on a single-chain variable fragment of the widely used humanized monoclonal antibody trastuzumab; and
GDA-601: CRISPR Knockout of CD38 on NK cells combined with anti CD38 CAR. CD38 is an established immunotherapeutic target in multiple myeloma, but its expression on NK cells and its further induction during ex vivo NK cell expansion represents a barrier to the development of an anti CD38 CAR-NK cell therapy. Gamida Cell recently announced a research collaboration with the Dana-Farber Cancer Institute to study the in vitro cytotoxicity of GDA-601 in fresh samples from multiple myeloma patients.
New data presented at PEGS Europe: Data from early-stage studies of GDA-501 demonstrated enhanced potency and cytotoxicity against a HER2-expressing tumor cell line. Data presented on GDA-301 showed cytotoxic activity against a chronic myelogenous leukemia cell line (K562) and a multiple myeloma cell line (RPMI). These data were presented at the 13th Annual Protein and Antibody Engineering Summit (PEGS) in Barcelona, Spain November 2-4, 2021.
Third Quarter 2021 Financial Results
Research and development expenses in the third quarter of 2021 were $12.4 million, compared to $10.5 million for the same period in 2020. The increase was mainly due to omidubicel commercial manufacturing readiness activities, and the advancement of the GDA-201 program, including broadening scientific capabilities and talent.
Commercial expenses in the third quarter of 2021 were $6.0 million, compared to $1.9 million for the third quarter of 2020. The increase was mainly attributed to progress with omidubicel commercial readiness activities. Going forward, the company anticipates reducing its near-term commercial readiness expenses in line with the revised omidubicel BLA submission timing.
General and administrative expenses were $4.8 million for the third quarter of 2021, compared to $2.7 million for the same period in 2020. The increase was mainly due to professional services and the hiring of key management positions, to support business growth.
Finance income, net, was $3.5 million for the third quarter of 2021, compared to $0.3 million for the third quarter of 2020. The increase was primarily due to non-cash income, resulting from revaluation of warrants offset by convertible note interest expenses.
Net loss for the third quarter of 2021 was $19.6 million, compared to a net loss of $14.8 million for the same period in 2020.
2021 Financial Guidance
Gamida Cell is re-assessing its planned spending and prior financial guidance as a result of the revised timing of the expected omidubicel BLA submission.
Expected Milestones in 2022
Omidubicel
BLA submission to the FDA in the first half of 2022
GDA-201
Initiation of a company-sponsored Phase 1/2 clinical study in NHL in 2022
NK cell pipeline expansion
Establish preclinical proof of concept studies of the NAM-enabled, genetically modified NK therapeutic targets in 2022
Select pipeline candidate(s) for IND enabling studies by end of 2022
Conference Call Information
Gamida Cell will host a conference call today, November 15, 2021, at 8:00 a.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com. To participate in the live call, please dial 866-930-5560 (domestic) or 409-216-0605 (international) and refer to conference ID number 4347485. A recording of the webcast will be available approximately two hours after the event, for approximately 30 days.
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 blood cancers. Omidubicel is the first bone marrow transplant graft to receive Breakthrough Therapy Designation from the U.S. FDA and has also received Orphan Drug Designation in the U.S. and EU. For more information about omidubicel, please visit View Source
Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
About GDA-201
Gamida Cell applied the capabilities of its nicotinamide (NAM)-enabled cell expansion technology 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 about GDA-201, please visit View Source
GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.