On November 4, 2021 Magenta Therapeutics, Inc. (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines designed to bring the curative power of stem cell transplants to more patients, reported financial results for the third quarter ended September 30, 2021, and recent program highlights (Press release, Magenta Therapeutics, NOV 4, 2021, View Source [SID1234594376]).
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"We are pleased with our recent execution across the portfolio as we look to continue to allocate our capital efficiently to our value-creating opportunities," said Jason Gardner, D.Phil., President and Chief Executive Officer, Magenta Therapeutics. "In mid-September, we cleared the IND process for the MGTA-117 targeted conditioning program and are now conducting the clinical trial start-up activities with the continued expectation of opening the trial this year. The MGTA-145 stem cell mobilization program also continues to advance with a fully enrolled investigator-initiated trial in multiple myeloma patients, a Phase 2 clinical trial in allogeneic transplant and the expected start of a Phase 2 clinical trial evaluating mobilization and collection of stem cells from patients with sickle cell disease."
Business Highlights:
In October 2021, Magenta welcomed Jeffrey Humphrey, M.D. to its Executive Team as Chief Medical Officer.
Program Highlights:
MGTA-145 Stem Cell Mobilization and Collection
Recent and Planned Activity:
Autologous Stem Cell Transplant: Multiple Myeloma
Investigator-Initiated Phase 2 Clinical Trial Design, Topline Data and Next Steps.
Trial Design: Surbhi Sidana, M.D., Assistant Professor of Medicine in the Division of Blood and Marrow Transplantation and Cellular Therapy at Stanford University School of Medicine led this investigator-initiated, Phase 2 open-label clinical trial. The trial evaluated the ability of MGTA-145, in combination with plerixafor, to mobilize stem cells for autologous stem cell transplantation in patients with multiple myeloma.
Top-Line Clinical Data in Poster Presentation at 2021 ASH (Free ASH Whitepaper) Annual Meeting: Top line clinical data from the fully enrolled investigator-initiated clinical trial will be included in a poster presentation at the ASH (Free ASH Whitepaper) Annual Meeting, held December 11-14, 2021. As disclosed separately, the clinical data showed that eighty-eight percent (88%) of patients (22/25) treated with MGTA-145 plus plerixafor met the primary endpoint of sufficient stem cell mobilization and collection for transplant. Also, as of the time of the data submission for the ASH (Free ASH Whitepaper) meeting, all patients (18/18) transplanted with stem cells mobilized by MGTA-145 plus plerixafor successfully engrafted. MGTA-145 plus plerixafor was well tolerated.
Next Steps in Multiple Myeloma: The results from this investigator-initiated trial represent a positive step forward in the development of MGTA-145, in combination with plerixafor, as a potential first line stem cell mobilization regimen. Based on the encouraging collection and engraftment data, the company intends to explore further development of MGTA-145 in a Phase 2b clinical setting. This approach would enable a comprehensive evaluation of the multiple myeloma patient population and allow for adjustments of dosing and administration which the company, in both cases, has identified as opportunities for optimization as a result of this investigator-initiated study and the company’s other MGTA-145 development efforts.
Allogeneic Stem Cell Transplant: Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL) and Myelodysplastic Syndromes (MDS)
Phase 2 Clinical Trial and Next Steps.
Trial Design. This Phase 2 clinical trial is designed to evaluate MGTA-145 in combination with plerixafor, in the mobilization and collection of stem cells from allogeneic donors for transplant in patients with AML, ALL and MDS.
Next Steps. Based on what Magenta has learned to date from the totality of the MGTA-145 program-related clinical trial data and other relevant information, Magenta believes it has identified an opportunity to optimize certain elements of the dosing and administration of the MGTA-145 mobilization regimen. Accordingly, Magenta intends to amend the Phase 2 allogeneic clinical trial to include a higher dose of MGTA-145 that matches the dose level used in the Phase 2 multiple myeloma clinical trial.
Stem Cell Mobilization of Patients with Sickle Cell Disease in Collaboration with bluebird bio. Magenta expects to open the Phase 2 clinical trial in December 2021. The trial is designed to evaluate the utility of MGTA-145 in combination with plerixafor, for the mobilization and collection of stem cells in patients with sickle cell disease where mobilization and collection is difficult and there is a clear unmet medical need.
MGTA-117 Targeted Conditioning
Recent and Planned Activity:
Phase 1/2 Clinical Trial Start-Up Activities Ongoing. The IND for the company’s MGTA-117 antibody-drug conjugate (ADC) targeted conditioning program is active with the U.S. Food and Drug Administration (FDA). The company expects to open the multi-center Phase 1/2 clinical trial in December 2021. The Phase 1/2 trial is designed to utilize dose escalating cohorts to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of MGTA-117 as a single dose with possible anti-tumor therapeutic benefit in patients with relapsed/refractory AML and MDS. As previously disclosed, Magenta expects to work with the FDA on an ongoing basis to transition the clinical trial to the primary target population of hematopoietic stem cell (HSC) transplant-eligible patients with AML and MDS after adequate data related to the safety, pharmacokinetics and pharmacodynamics of MGTA-117 have been collected in this initial patient population. As the program progresses, Magenta also plans to explore MGTA-117 as a targeted conditioning agent prior to the delivery of gene-corrected cells associated with stem cell gene therapy.
Oral Presentation at the 2021 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. As disclosed by Magenta in a separate press release, preclinical data showing that a single dose of a tool CD117 antibody drug conjugate (CD117-ADC) supports efficient engraftment of gene-modified CD34+ stem cells in a rhesus gene therapy model. The CD117-ADC utilized in this study had minimal toxicities unlike busulfan conditioning. The data will be the subject of an oral presentation at the ASH (Free ASH Whitepaper) Annual Meeting on December 13, 2021.
Financial Results:
Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2021, were $192.6 million, compared to $148.8 million as of December 31, 2020. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund operations and capital expenditures into the third quarter of 2023.
Research and Development Expenses: Research and development expenses were $10.8 million in the third quarter of 2021, compared to $11.8 million in the third quarter of 2020. The decrease was driven primarily by the completion of the GMP manufacturing activities to support the IND application for MGTA-117 and future clinical trials offset by an increase in personnel related costs.
General and Administrative Expenses: General and administrative expenses were $7.5 million for the third quarter of 2021, compared to $6.6 million for the third quarter of 2020. The increase was primarily due to an increase in personnel related costs.
Net Loss: Net loss was $17.4 million for the third quarter of 2021, compared to net loss of $17.7 million for the third quarter of 2020.