Magenta Therapeutics and Beam Therapeutics Announce Collaboration to Evaluate Targeted Antibody-Drug Conjugate (ADC) MGTA-117 as Conditioning Regimen for Base Editing Therapies
– MGTA-117 conditioning for precise depletion of cells in the bone marrow could potentially increase patients’ benefit from Beam’s base editing therapies for sickle cell disease and beta thalassemia –
Conditioning is a critical component necessary to prepare a patient’s body to receive the edited cells, which carry the corrected gene and must engraft in the patient’s bone marrow in order to be effective. Today’s conditioning regimens rely on nonspecific chemotherapy or radiation, which are associated with significant toxicities. MGTA-117 precisely targets only hematopoietic stem and progenitor cells, sparing immune cells, and has shown high selectivity, potent efficacy, wide safety margins and broad tolerability in non-human primate models. MGTA-117 may be capable of clearing space in bone marrow to support long-term engraftment and rapid recovery in patients.
Beam has demonstrated the ability to edit individual DNA bases in hematopoietic stem cells at high efficiency and with little impact on the viability of edited cells relative to unedited cells using its novel base editing technology. Combining MGTA-117 with Beam’s HPFH and Makassar base editors could meaningfully advance the treatment of patients with sickle cell disease or beta-thalassemia.
“We believe patients will benefit from a more precise process to remove hematopoietic stem cells and prepare them to receive genetic medicines. Magenta has developed targeted ADCs as the preferred modality for our conditioning programs, and we have designed MGTA-117 specifically to optimize it for use with a genetically-modified cell product delivered in a transplant setting,” said
“Base editing has the potential to offer lifelong treatment for patients with many diseases, including sickle cell disease and beta-thalassemia. Our novel base editors create precise single base changes in genes without cutting the DNA, enabling durable correction of hematopoietic stem cells with minimal effects on cell viability or genomic integrity,” said
Beam will be responsible for clinical trial costs related to development of Beam’s base editors when combined with MGTA-117, while Magenta will continue to be responsible for all other development costs of MGTA-117. Magenta will also continue to develop MGTA-117 in other diseases, including blood cancers and genetic diseases. Each company will retain all commercial rights to their respective technologies.
MGTA-117, Magenta’s most advanced conditioning program, is a CD117-targeted antibody engineered for the transplant setting and conjugated to amanitin, a toxin in-licensed from Heidelberg Pharma. It is designed to precisely deplete only hematopoietic stem and progenitor cells and has shown high selectivity, potent efficacy, wide safety margins and broad tolerability in non-human primate models, suggesting that it may be capable of clearing space in bone marrow to support long-term engraftment and rapid recovery in patients. Magenta plans to complete IND-enabling studies this year and initiate clinical studies in 2021. Magenta will continue to develop MGTA-117 in other diseases, including blood cancers and genetic diseases.
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This press release contains forward-looking statements. Investors are cautioned not to place undue reliance on these forward-looking statements, including statements about the timing, progress and success of the collaboration contemplated under the agreement between Beam and Magenta, the successful evaluation of MGTA-117 in conjunction with Beam’s base-editing therapies under the agreement, the expected timing of filing INDs applications and the therapeutic applications of Beam’s technology. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include the risks and uncertainties, among other things, regarding: the success in development and potential commercialization of our product candidates; Beam’s ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; whether preclinical testing of our product candidates and preliminary or interim data from preclinical and clinical trials will be predictive of the results or success of ongoing or later clinical trials; that enrollment of clinical trials may take longer than expected; that Beam’s product candidates will experience manufacturing or supply interruptions or failures; that Beam will be unable to successfully initiate or complete the preclinical and clinical development and eventual commercialization of product candidates; that the development and commercialization of Beam’s product candidates will take longer or cost more than planned; the impact of COVID-19 on Beam’s business and the other risks and uncertainties identified under the heading “Risk Factors” and in Beam’s Annual Reports on Form 10-K for the year ended