On November 10, 2020 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, reported financial results for the quarter ended September 30, 2020 (Press release, Gamida Cell, NOV 10, 2020, View Source [SID1234570552]). The company also highlighted progress with omidubicel, an advanced cell therapy in Phase 3 clinical development as a potentially life-saving treatment option for patients in need of bone marrow transplant, and GDA-201, a natural killer (NK) cell immunotherapy in Phase 1 development in patients with non-Hodgkin lymphoma (NHL).

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"Gamida Cell continued to make strong progress during the quarter. Most importantly, we recently announced that our Phase 3 study of omidubicel met all three secondary endpoints, further underscoring the potential clinical benefit of this novel graft source for bone marrow transplant," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We look forward to reporting the data in further detail and to initiating the biologics license application (BLA) for omidubicel to the FDA on a rolling basis, both in the fourth quarter of this year."

"Our second program, GDA-201, a natural killer cell therapy, is also advancing. NK cell immunotherapies offer tremendous potential for transforming the care of hematologic malignancies. Last week, we announced that updated data from the Phase 1 study in patients with non-Hodgkin lymphoma will be presented next month in an oral session at the American Society for Hematology (ASH) (Free ASH Whitepaper) annual meeting. The data continue to be compelling, with multiple complete responses and a favorable tolerability profile reported in patients with advanced disease," Dr. Adams continued.

Omidubicel, an investigational advanced cell therapy for allogeneic bone marrow transplant

During the quarter, Gamida Cell continued to advance its Phase 3 product candidate, omidubicel, which is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) and which has the potential to be the first FDA-approved engineered bone marrow transplant graft.

Last month, Gamida Cell announced that all three pre-specified secondary endpoints in the company’s Phase 3 study of omidubicel demonstrated a statistically significant improvement among patients who received omidubicel compared to the comparator group (who received standard umbilical cord blood). These secondary endpoints were platelet engraftment, infections and hospitalization.

In May, Gamida Cell reported that omidubicel achieved its primary endpoint, demonstrating a statistically significant reduction in time to neutrophil engraftment, a key milestone in recovery from a bone marrow transplant. The international, multi-center, randomized Phase 3 study was designed to evaluate the safety and efficacy of omidubicel in patients with hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. Gamida Cell expects to report the full data set later in the fourth quarter of 2020.

Additional omidubicel highlights:

New Phase 1 data from study of omidubicel in patients with severe aplastic anemia to be presented at ASH (Free ASH Whitepaper): Gamida Cell is evaluating omidubicel in an investigator-sponsored, Phase 1/2 study in patients with severe aplastic anemia and will present new data from the ongoing study at the 62nd ASH (Free ASH Whitepaper) Annual Meeting. Data from the abstract published in Blood showed that omidubicel was generally well tolerated and could result in rapid engraftment and achieve sustained hematopoiesis in heavily transfused patients with severe aplastic anemia, a rare and life-threatening blood disorder. The poster presentation, "Rapid Engraftment, Immune Recovery, and Resolution of Transfusion Dependence in Treatment-Refractory Severe Aplastic Anemia Following Transplantation with Ex Vivo Expanded Umbilical Cord Blood (Omidubicel)" (Abstract #1531) will take place on Saturday, December 5, 2020, 7:00 a.m. – 3:30 p.m. PT (10:00 a.m. – 6:30 p.m. ET).
Expanded collaboration with Be The Match BioTherapies: In October, Gamida Cell and Be The Match BioTherapiesannounced an expansion of their existing strategic collaboration for omidubicel. The original partnership agreement between the organizations focused on the omidubicel development program and leveraged a wide range of Be The Match BioTherapies’ capabilities and services. In building upon the collaboration, Gamida Cell will work with Be The Match BioTherapies for the ordering and supply of cord blood units, which serve as the starting material for omidubicel. The expanded agreement is designed to provide a smooth process for the supply of cord blood units throughout the supply chain to deliver omidubicel to patients.
Presented initial observational data from collaboration with CIBMTR: In September,Gamida Cell reported initial data from an observational study that includes data contemporaneous to the Phase 3 study of omidubicel. The study was the result of a research agreement between Gamida Cell and the CIBMTR (Center for International Blood and Marrow Transplant Research) designed to collect and analyze health outcomes data in patients with hematologic malignancies who receive a hematopoietic stem cell transplant or cellular therapy infusion, including bone marrow transplant graft from various donor sources. The study evaluated clinical outcomes for 660 patients in the CIBMTR registry who underwent a bone marrow transplant with a matched unrelated, mismatched unrelated or haploidentical graft source contemporaneous to the Phase 3 study of omidubicel. The study data demonstratd that key clinical outcomes, including time to neutrophil engraftment and overall survival, were improved for patients with donors under the age of 30.
Continued focus on activities required to successfully bring omidubicel to patients: Gamida Cell is continuing to advance key activities required to bring omidubicel to patients in a commercial setting, including building out manufacturing infrastructure, assembling an experienced commercial team with expertise in cell therapy and transplant, establishing hospital services and patient assistance programs.
GDA-201, an innate NK cell immunotherapy

Updated Phase 1 data to be presented at the 62nd ASH (Free ASH Whitepaper) Annual Meeting: Last week, Gamida Cell announced that updated data from the ongoing Phase 1 study of GDA-201 in patients with non-Hodgkin lymphoma and multiple myeloma will be presented at the ASH (Free ASH Whitepaper) Annual Meeting. Data from the abstract published in Blood demonstrated that GDA-201 in combination with a monoclonal antibody was generally well tolerated and clinically active. Among 15 patients with NHL, there were 10 complete responses and 1 partial response. Additional details will be presented in an oral session, "Results of a Phase 1 Trial of GDA-201, Nicotinamide-Expanded Allogeneic Natural Killer (NK) Cells in Patients with Refractory Non-Hodgkin Lymphoma (NHL) and Multiple Myeloma" (Abstract #63) on Saturday, December 5, at 7:30 a.m. PT (10:30 a.m. ET).
Continued advancing Phase 1 study of GDA-201: Gamida Cell continues to advance activities to enable the submission of an investigational new drug (IND) application for GDA-201 in the first half of 2021. The company continues to be on track to initiate a multi-center, Phase 1/2 clinical study in patients with NHL next year. Gamida Cell is pioneering a novel approach that harnesses the power of its cell expansion technology, which uniquely improves antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells.
Corporate Highlights

Responded to COVID-19 pandemic: Gamida Cell is committed to the health and safety of its employees while continuing to advance its business, including regularon-site testing.
Expanded team with key appointments in information technologies and market access: Today, Gamida Cell announced the appointment of Stephen Jamieson to the newly created role of senior vice president, information technologies. Mr. Jamieson brings 25 years of information technologies experience, primarily in the life sciences industry. Prior to joining Gamida Cell, Mr. Jamieson served as chief information officer and head of commercial operations at Verastem Oncology. He also held prior information technologies roles at several life sciences companies, including Infinity Pharmaceuticals, Inc., ARIAD Pharmaceuticals, Inc. and OSI Pharmaceuticals, Inc. (acquired by Astellas Pharma, Inc.). Mr. Jamieson holds a B.S. in Computer Science/Mathematics from Widener University.

Today, the company also announced the appointment of Rocio Manghani to the role of senior vice president, market access. Ms. Manghani has nearly 20 years of experience in market access and patient support roles. In this role, she will lead the strategic planning and implementation of Gamida Cell’s market access initiatives. Ms. Manghani joins the company from Tricida, Inc., where she served as senior vice president, market access. Prior to Tricida, she worked in various commercial positions with increasing responsibility, including roles at Kite Pharma, Inc. (acquired by Gilead Sciences, Inc.), Celgene (acquired by Bristol-Myers Squibb), Abraxis Oncology and Roche Laboratories. Ms. Manghani holds a M.P.H. from Emory University and a B.S. in Sociology, Healthcare and Social Issues from University of California, San Diego.
Third Quarter 2020 Financial Results

Research and development expenses in the third quarter of 2020 were $10.5 million, compared to $7.5 million for the same period in 2019. The increase was mainly due to BLA readiness preparation, increased clinical activities relating to the advancement of GDA-201, and the initiation of the omidubicel expanded access study.
Commercial expenses in the third quarter of 2020 were $1.9 million compared to $1.7 million for the same period in 2019. The increase was mainly attributed to commercial readiness activities for omidubicel.
General and administrative expenses were $2.7 million for the third quarter of 2020 and compared to $2.8 million for the same period in 2019. The decrease was mainly due to reduced travel expenses.
Finance income, net, was $0.3 million for the third quarter of 2020, compared to finance income, net, of $1.7 million for the third quarter of 2019. The decrease was primarily due to noncash income resulting from revaluation of warrants owned by certain shareholders.
Net loss for the third quarter of 2020 was $14.8 million, compared to a net loss of $10.1 million for the same period in 2019.
As of September 30, 2020, Gamida Cell had total cash and cash equivalents of $73.3 million, compared to $55.4 million as of December 31, 2019.
2020 Financial Guidance

Gamida Cell expects cash used for ongoing operating activities in 2020 to range from $60 million to $65 million.

Gamida Cell expects that its current cash and cash equivalents will support the company’s ongoing operating activities into the second half of 2021. This cash runway guidance is based on the company’s current operational plans and excludes any additional funding and any business development activities that may be undertaken.

Expected 2020-2021 Milestones

Gamida Cell plans to achieve the following milestones during 2020-2021:

Omidubicel

Present data from the Phase 3 study at a medical meeting in the fourth quarter of 2020
Initiate the submission of the BLA to the FDA, on a rolling basis, in the fourth quarter of 2020
Report additional data from the Phase 1/2 study in patients with severe aplastic anemia in the fourth quarter of 2020
Commercial launch of omidubicel in the second half of 2021, contingent upon FDA approval
GDA-201

Present additional data from the Phase 1 study at the ASH (Free ASH Whitepaper) Annual Meeting
Submit company-sponsored IND application to the FDA in the first half of 2021
Initiate a Phase 1/2 clinical study in patients with NHL in 2021
Conference Call Information

Gamida Cell will host a conference call today, November 10, 2020, at 8:30 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 +1-409-216-0605 (international) and refer to conference ID number 9998754. A recording of the webcast will be available approximately two hours after the event, for approximately 30 days.

About Omidubicel

Omidubicel, the company’s lead clinical program, 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 FDA and has also received Orphan Drug Designation in the U.S. and EU. In both Phase 1/2 and Phase 3 clinical studies (NCT01816230 and NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

About GDA-201

Gamida Cell applied the capabilities of its NAM-based 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 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. GDA-201 is in Phase 1 development through an investigator-sponsored study in patients with refractory non-Hodgkin lymphoma and multiple myeloma (NCT03019666).

Omidubicel and GDA-201 are investigational therapies, and their safety and efficacy have not been evaluated by the U.S. Food and Drug Administration or any other health authority.