On November 3, 2022 Magenta Therapeutics (Nasdaq: MGTA), a clinical-stage biotechnology company developing novel medicines designed to bring the curative power of stem cell transplant to more patients, reported financial results for the third quarter ending September 30, 2022, and recent program highlights (Press release, Magenta Therapeutics, NOV 3, 2022, View Source [SID1234622920]).
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"We are building momentum in the MGTA-117 clinical trial with new clinical results and are making progress across our pipeline, including our second targeted conditioning program CD45-ADC," said Jason Gardner, President and Chief Executive Officer of Magenta Therapeutics, Inc. "In the MGTA-117 Phase 1/2 clinical trial, we continue to enroll and generate clinical data in higher-dose cohorts. Our preclinical work has been particularly helpful in predicting our clinical experience in these early dose cohorts regarding MGTA-117 activity at different exposure levels and the overall tolerability profile. We look forward to presenting available clinical data at ASH (Free ASH Whitepaper) and using our data to support interactions with regulators as we plan to advance MGTA-117 to stem cell transplant-eligible patients and into gene therapy."
Program Highlights:
MGTA-117 Phase 1/2 Clinical Trial Progression and Data Disclosure Expectations
MGTA-117 is Magenta’s most advanced targeted conditioning product candidate designed to deplete target cells prior to a patient undergoing stem cell transplant or receiving an ex vivo gene therapy product. The program is currently enrolling patients with relapsed/refractory acute myeloid leukemia (AML), and myelodysplastic syndromes (MDS), in a Phase 1/2 dose escalation clinical trial. MGTA-117 is an anti-CD117 antibody conjugated to an amanitin payload. CD117, also known as c-Kit receptor, is highly expressed on hematopoietic stem cells, progenitor cells, and leukemic cells.
Enrollment Progress. Magenta has completed enrollment in Cohort 1 and Cohort 2. In addition, Magenta has enrolled a sufficient number of patients to complete Cohort 3, provided that the patients complete their respective dose-limiting toxicity (DLT) observation periods. No serious adverse events have been deemed to be related to MGTA-117, and no dose-limiting toxicities have been observed to date. Our clinical experience has confirmed that patients with relapsed/refractory AML are at high risk for multiple disease complications, including susceptibility to infection, all of which can rapidly progress at any time leading to severe morbidity or mortality. All enrolled patients have contributed data to the clinical trial independent of the completion of the DLT period.
Proof-of-Mechanism. Available clinical data, including new data since the ASH (Free ASH Whitepaper) abstract submission in August 2022, support proof-of-mechanism for MGTA-117 due to evidence of its ability to bind CD117-expressing cells, deplete CD117-expressing cells, clear the body rapidly and be well-tolerated.
Regulatory Plans. Magenta has initiated requests for formal engagement with multiple regulatory authorities for the purpose of transitioning the clinical program into transplant-eligible AML and MDS patients. The pending regulatory interactions are expected to focus on MGTA-117’s clinical data relating to target binding, drug clearance and stability and tolerability across multiple dose levels. All available clinical data will be used to support these regulatory interactions, as well as the predictive preclinical modeling in non-human primates that has closely matched our clinical experience to date.
Gene Therapy. Magenta expects data from the Phase 1/2 trial to also inform clinical development planning and enable regulatory engagements for MGTA-117 as a potential monotherapy prior to patients undergoing autologous ex vivo gene therapy. Magenta has existing clinical collaborations with gene therapy companies and anticipates entering into additional collaborations as data progresses.
ASH Presentations. As disclosed separately, Magenta will present clinical and preclinical data at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2022. The MGTA-117 clinical presentation will include data on pharmacokinetics, pharmacodynamic activity and the tolerability of MGTA-117. In light of the need to collect and finalize complete Cohort 3 data, Magenta currently anticipates presenting Cohort 1 and Cohort 2 clinical data at ASH (Free ASH Whitepaper). Any data not included in the ASH (Free ASH Whitepaper) presentation is anticipated to be available at a scientific conference in Q1 2023.
CD45-Antibody Drug Conjugate (ADC): Second Targeted Conditioning Program
Magenta’s CD45-ADC is designed to selectively target and deplete both stem cells and immune cells and is intended to replace the use of chemotherapy and radiation-based conditioning prior to stem cell transplant in patients with blood cancers and autoimmune diseases.
Magenta has completed a dose-ranging toxicology preclinical study successfully with no unexpected findings. The data inform dosing for a Good Laboratory Practices toxicology study intended to support a planned Investigational New Drug (IND) application.
Manufacturing and other IND-enabling activities are ongoing, and Magenta is preparing for regulatory interactions and clinical development activities.
Magenta expects to provide a further update on the CD45-ADC program in December 2022.
MGTA-145 Stem Cell Mobilization and Collection
Magenta is developing MGTA-145, in combination with plerixafor, to improve the process by which stem cells are released out of the bone marrow and into the bloodstream, known as stem cell mobilization. The mobilized cells are then collected and available for transplant. This is the first step for patients and is required for the majority of transplants and stem cell gene therapies.
Magenta, in partnership with bluebird bio, has initiated a Phase 2 clinical trial in sickle cell disease 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. Mobilization and collection are difficult in sickle cell disease patients where granulocyte colony-stimulating factor (GCSF) cannot be used, and there is a clear unmet medical need.
Magenta anticipates generating initial data from this clinical trial in December 2022 followed by a more comprehensive data set in H1 2023.
Financial Results:
Cash Position: Cash, cash equivalents and marketable securities as of September 30, 2022, were $128.3 million, compared to $176.9 million as of December 31, 2021. The September cash balance includes gross proceeds of $3.0 million from our "at-the-market" facility. Magenta anticipates that its cash, cash equivalents and marketable securities will be sufficient to fund its current operational plan into Q2 2024.
Research and Development Expenses: Research and development expenses were $11.2 million in the third quarter of 2022, compared to $10.8 million in the third quarter of 2021. The increase was driven primarily by higher preclinical and manufacturing costs to support our IND-enabling studies for CD45-ADC, offset by a decrease in clinical trial costs related to our mobilization program.
General and Administrative Expenses: General and administrative expenses were $6.1 million for the third quarter of 2022, compared with $7.5 million in the third quarter of 2021. The decrease was primarily due to a decrease in stock-based compensation.
Net Loss: Net loss was $16.1 million for the third quarter of 2022, compared to net loss of $17.4 million for the third quarter of 2021.