Press Release Details
Magenta Therapeutics Announces IND Clearance for MGTA-117 Targeted Conditioning Clinical Trial
– Phase 1/2 clinical trial expected to open in Q4 2021 in patients with relapsed/refractory acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) –
“We are very pleased that our collaboration with the FDA has resulted in the clearance of the MGTA-117 IND. We have addressed the FDA’s request for a bioassay to be incorporated into the clinical trial protocol,” said
The multi-center, open label Phase 1/2 clinical trial with single-dose escalating cohorts will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of MGTA-117 as a single agent in relapsed/refractory AML and MDS patients. Magenta will continue to engage with the FDA to transition the trial to the intended primary target population of hematopoietic stem cell transplant-eligible AML and MDS patients. In addition, Magenta has planned gene therapy clinical trial collaborations with
Magenta’s MGTA-117 program is the company’s lead targeted conditioning product candidate, an antibody-drug conjugate (ADC) designed to selectively deplete hematopoietic stem cells (HSCs) from patients prior to transplant or HSC-based gene therapy to reduce the need for high-dose or high-intensity chemotherapeutic agents or, in the case of gene therapy applications, to potentially eliminate the need for chemotherapeutic agents altogether. MGTA-117 targets the CD117 receptor, which is highly expressed on the cell surface of HSCs and leukemia cells, making it a promising target for conditioning across broad sets of diseases, including certain blood cancers, hemoglobinopathies (sickle cell disease and beta thalassemia) and inherited metabolic disorders.
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