On December 9, 2024 Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, reported updated clinical data from its ongoing Phase 1/2 VBP101 study of patients with relapsed/refractory AML receiving trem-cel followed by MylotargTM (Press release, Vor BioPharma, DEC 9, 2024, View Source [SID1234648920]). The data, which was presented in a poster at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting on Sunday, December 8th, demonstrated durable engraftment, shielding from Mylotarg on-target toxicity, a broadened Mylotarg therapeutic window, and early evidence of improved relapse free survival compared to published high-risk AML comparators.
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"With additional maturity, we are even more encouraged by this data and the potential of offering AML and MDS patients the opportunity to receive post-transplant maintenance therapy while still maintaining healthy blood count levels," said Dr. Eyal Attar, Vor Bio’s Chief Medical Officer.
The data released today included 25 patients treated with trem-cel of which 15 had received Mylotarg (six at the 2 mg/m2 dose) as of the data cut-off date of November 1, 2024. The data demonstrated:
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Preliminary evidence of improved relapse-free survival (median RFS not reached with median follow-up duration of 7.4 months) compared to published groups of AML patients at high risk of relapse post hematopoietic stem cell transplant (HCT)1.
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Shielding of the blood system, with maintained neutrophil and platelet counts across multiple Mylotarg doses of 0.5, 1, and 2 mg/m2.
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Broadened therapeutic index for Mylotarg when administered after trem-cel.
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Reliable engraftment, with 100% of patients achieving primary neutrophil engraftment (median 9.5 days), robust platelet recovery (median 16 days), and full myeloid donor chimerism at Day 28.
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Trem-cel continues to be manufactured with high CD33 editing efficiency (median 90%, range 71-94%).
Company received supportive feedback from the FDA in a Type C meeting
The Company had the opportunity to interact with the FDA regarding data from the trem-cel + Mylotarg study alongside a proposed registrational clinical trial synopsis. The FDA agreed that trem-cel engrafts neutrophils and platelets and has a similar safety profile to unedited CD34+ grafts. In addition, there was agreement with the trem-cel + Mylotarg registrational clinical trial design with respect to study population, control arm, primary endpoint, stratification factors, and statistical design. The Company agreed to provide further updates to the FDA alongside submission of the full clinical trial protocol.