Poseida Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to P-BCMA-ALLO1, an investigational stem cell memory T cell (TSCM)-based allogeneic CAR-T cell therapy. This therapy is currently in Phase 1/1b clinical development for patients with relapsed/refractory multiple myeloma. The RMAT designation aims to expedite the development and review of regenerative medicine therapies for serious conditions.
The FDA's decision was influenced by positive early clinical data from the ongoing Phase 1 study of P-BCMA-ALLO1. The data suggested that P-BCMA-ALLO1 has the potential to offer promising efficacy, a favorable safety profile, and rapid 'off-the-shelf' patient access. This designation includes all the benefits of Fast Track and Breakthrough Therapy designations, facilitating early and frequent interactions with the FDA.
Clinical Data and Unmet Needs
Kristin Yarema, Ph.D., president and chief executive officer of Poseida Therapeutics, stated that the RMAT designation validates the potential of P-BCMA-ALLO1 to address the unmet needs of patients with relapsed/refractory multiple myeloma. Clinical responses have been observed in heavily pre-treated, refractory patients, including those who have previously received BCMA-targeted therapies.
Upcoming Data Presentation
Poseida Therapeutics plans to present new clinical data from the P-BCMA-ALLO1 Phase 1 study at the 21st International Myeloma Society Annual Meeting in Rio de Janeiro, scheduled for September 25-28, 2024. Further clinical updates are anticipated in the second half of 2024, contingent upon coordination with Roche, which has a strategic collaboration with Poseida covering multiple allogeneic CAR-T therapies targeting blood cancers, including P-BCMA-ALLO1.
About P-BCMA-ALLO1
P-BCMA-ALLO1 is an allogeneic CAR-T product candidate licensed to Roche, targeting B-cell maturation antigen (BCMA) for the treatment of relapsed/refractory multiple myeloma. The FDA has granted P-BCMA-ALLO1 RMAT designation for adult patients with relapsed/refractory multiple myeloma after three or more prior lines of therapies including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD), and anti-CD38 antibody, in addition to Orphan Drug designation for multiple myeloma. Additional information about the Phase 1/1b study is available at www.clinicaltrials.gov using identifier: NCT04960579.
RMAT Designation Details
The RMAT designation, established under the 21st Century Cures Act, is designed to expedite the development and review of regenerative medicine therapies for serious or life-threatening diseases. Eligible therapies must demonstrate preliminary clinical evidence indicating the potential to address unmet medical needs. The designation includes features of the Breakthrough Therapy designation, such as opportunities for early discussions regarding surrogate or intermediate endpoints. RMAT-designated therapies may be eligible for accelerated approval based on agreed-upon surrogate endpoints that are reasonably likely to predict long-term clinical benefit.
