Vor Bio (NASDAQ:NOR) announced new data from its ongoing Phase 1/2 study of VBP101 in relapsed/refractory AML patients, demonstrating reliable engraftment, shielding from Mylotarg toxicity, a broadened therapeutic window, and early signs of patient benefit.
The data included 18 patients treated with trem-cel, with ten of those patients receiving Mylotarg as of the data cut-off date, July 19, 2024. The company plans to approach the FDA to discuss a pivotal design for trem-cel in combination with Mylotarg before the end of the year.
In a statement, Eyal Attar, M.D., CMO of Vor Bio, said, “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.”
Guenther Koehne, M.D., Ph.D., VBP101 study investigator and Deputy Director and Chief of Blood & Marrow Transplant and Hematologic Oncology at Miami Cancer Institute of Baptist Health South Florida, said, “All the hope I had in the safety of this approach has been supported by the data from this trial thus far. 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.”
