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 allogeneic CAR-T cell therapy, for the treatment of patients with relapsed/refractory multiple myeloma. This designation aims to expedite the development and review of regenerative medicine therapies for serious conditions. The RMAT application was evaluated based on encouraging early data from the ongoing Phase 1 study of P-BCMA-ALLO1.
Clinical Data and Unmet Needs
The RMAT designation was granted based on early clinical data from Poseida's Phase 1 study, which demonstrated P-BCMA-ALLO1's potential to offer promising efficacy, a manageable safety profile, and rapid 'off-the-shelf' patient access. Multiple myeloma remains a challenging disease with significant unmet needs, especially in patients who have relapsed or become refractory to standard treatments. P-BCMA-ALLO1 represents a novel approach, utilizing stem cell memory T cells (T SCM) to target B-cell maturation antigen (BCMA) on myeloma cells.
Management Commentary
"The RMAT designation for P-BCMA-ALLO1, our lead program, 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," said Kristin Yarema, Ph.D., president and chief executive officer of Poseida Therapeutics. "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."
Upcoming Data Presentation and Collaboration
Poseida Therapeutics will present new clinical data from the P-BCMA-ALLO1 Phase 1 study at the 21st International Myeloma Society Annual Meeting in Rio de Janeiro from September 25-28, 2024. The company also plans to provide additional clinical updates in the second half of 2024, in coordination with Roche, which has a strategic collaboration with Poseida covering multiple investigational 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 Regenerative Medicine Advanced Therapy (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.
