Poseida Therapeutics has achieved a significant milestone in its mission to revolutionize cancer and rare disease treatment. The clinical-stage cell and gene therapy company has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for P-BCMA-ALLO1, marking a pivotal moment in the pursuit of innovative therapies for patients battling multiple myeloma.

P-BCMA-ALLO1 is a BCMA-targeted allogeneic, T stem cell memory (TSCM)-rich chimeric antigen receptor (CAR)-T therapy candidate, currently being investigated in collaboration with Roche for the treatment of relapsed/refractory multiple myeloma (RRMM).

"This Orphan Drug Designation underscores the high unmet medical need for a rapid and accessible off-the-shelf allogeneic CAR-T therapy for patients with multiple myeloma," emphasized Kristin Yarema, Ph.D., President and Chief Executive Officer of Poseida Therapeutics. "We believe that TSCM-rich allogeneic CAR-T therapies hold immense promise in delivering optimal clinical results, on-demand availability, and high-volume production, thereby facilitating broader access to CAR-T therapies. We eagerly anticipate sharing further clinical updates from the Phase 1 study of P-BCMA-ALLO1 later this year."

The Phase 1 clinical trial evaluating P-BCMA-ALLO1 has already demonstrated promising results. Positive early safety and preliminary efficacy data were presented at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2023. 

Notably, the therapy was well-tolerated with a favorable emerging safety profile, and it was administered to 100 percent of patients without the need for bridging chemotherapy or other anti-myeloma bridging therapies. Additionally, preliminary data showcased the trafficking of allogeneic TSCM-rich CAR-T cells to bone marrow, their differentiation into cell-killing effector T cells, and their persistence for at least six weeks after treatment, supporting the hypothesis of sustained cell presence at tumor-relevant sites.

Further insights into the efficacy of P-BCMA-ALLO1 will be unveiled at the American Association for Cancer Research (AACR) Annual Meeting in San Diego on April 8, 2024, during a poster presentation focusing on a subset of recently enrolled patients refractory to initial BCMA targeting therapy.

Looking ahead, Poseida Therapeutics plans to provide an additional clinical update on the P-BCMA-ALLO1 program at a scientific meeting in the second half of 2024, pending coordination with Roche.

The FDA's Orphan Drug Designation program is instrumental in fostering the development of treatments for rare diseases like multiple myeloma. Drugs or biologics granted Orphan Drug Designation receive various incentives, including tax credits for qualified clinical trials, prescription drug user-fee exemptions, and potential seven-year marketing exclusivity upon FDA approval.