The US Food and Drug Administration (FDA) has awarded regenerative medicine advanced therapy (RMAT) designation to P-BCMA-ALLO1, a treatment being developed by Poseida Therapeutics for relapsed or refractory multiple myeloma.
Poseida Therapeutics is engaged in the development of allogeneic cell therapies and genetic medicines. P-BCMA-ALLO1 is an investigational allogeneic CAR-T cell therapy that is currently in Phase 1/1b clinical trials.
Licensed to Roche, P-BCMA-ALLO1 is designed to target B-cell maturation antigen (BCMA) for treating multiple myeloma that does not respond to other treatments or has returned after treatment. The therapy candidate includes a VH-based binder that specifically targets BCMA.
Data shared at the American Society of Hematology (ASH) in December 2023 suggested that this investigational therapy, which is rich in T stem cell memory, could meet the needs of patients with multiple myeloma who lack other treatment options.
The RMAT designation offers benefits similar to those of the FDA’s fast track and breakthrough therapy designations, including opportunities for early and frequent communication with the FDA.
The decision to grant RMAT status to P-BCMA-ALLO1 was based on preliminary results from an ongoing Phase 1 study, which showed potential in terms of safety, efficacy, and the ability to be readily available for patients.
Poseida Therapeutics president and CEO Kristin Yarema said: “The RMAT designation for P-BCMA-ALLO1, our lead programme, is based on impressive early clinical data from our ongoing Phase 1 study and further validates its potential to address the unmet needs of patients with relapsed/refractory multiple myeloma. Importantly, our data has shown clinical responses in very sick, refractory patients, including those that have received prior BCMA-targeted therapies.
“With both RMAT and Orphan Drug designations for P-BCMA-ALLO1, we look forward to working closely with the FDA as we continue to advance this next-generation, off-the-shelf allogeneic CAR-T therapy, including the recently initiated Phase 1b portion of the trial.”
Poseida Therapeutics plans to present additional findings from the P-BCMA-ALLO1 Phase 1 trial at the upcoming 21st International Myeloma Society Annual Meeting in Rio de Janeiro, Spain.
More clinical updates are anticipated in the latter half of 2024, subject to coordination with Roche. Roche and Poseida Therapeutics have a partnership focused on various investigational allogeneic CAR-T therapies for blood cancers, including P-BCMA-ALLO1.
