Vor Bio announces new data, plans to approach FDA on trial design by year-end

Vor Bio announced new clinical data from its ongoing Phase 1/2 VBP101 study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg . The data demonstrated reliable engraftment, shielding from Mylotarg on-target toxicity, a broadened Mylotarg therapeutic window, and early evidence of patient benefit. “We are encouraged by this data and the potential benefit that trem-cel in combination with Mylotarg may offer to patients in a disease that has extremely poor outcomes even after transplant,” said Dr. Eyal Attar, Vor Bio’s Chief Medical Officer. “With this data, we plan to explore a registrational trial while we continue to pursue other synergistic opportunities for Vor Bio’s platform such as VCAR33ALLO and VADC45.” The data released today included 18 patients treated with trem-cel of which ten had received Mylotarg as of the data cut-off date of July 19. “All the hope I had in the safety of this approach has been supported by the data from this trial thus far,” said Guenther Koehne, MD, PhD, an investigator on the VBP101 study and Deputy Director and Chief of Blood & Marrow Transplant and Hematologic Oncology at Miami Cancer Institute of Baptist Health South Florida. “I look forward to treating my next patients at high risk of relapse on this trial as their outcomes are otherwise limited with standard transplants.” Vor Bio plans to approach the U.S. Food & Drug Administration to discuss a pivotal trial design for trem-cel + Mylotarg by around year end.