Poseida Therapeutics, Inc. (Nasdaq: PSTX), a trailblazer in the field of clinical-stage cell and gene therapy, has achieved a significant milestone with the U.S. Food and Drug Administration (FDA) granting Orphan Drug Designation to its cutting-edge treatment for multiple myeloma, P-BCMA-ALLO1. This novel therapy, a B-cell maturation antigen (BCMA)-targeted allogeneic T stem cell memory (TSCM)-rich chimeric antigen receptor (CAR)-T candidate, is under investigation in partnership with Roche for addressing the challenges of relapsed/refractory multiple myeloma (RRMM), signaling a major advancement in the quest for more effective treatments for this formidable cancer.

“The Orphan Drug Designation for P-BCMA-ALLO1 underscores the high unmet medical need for a rapid and accessible off-the-shelf allogeneic CAR-T therapy for patients with multiple myeloma,” expressed Kristin Yarema, Ph.D., President and Chief Executive Officer of Poseida Therapeutics. This designation is a testament to the potential of TSCM-rich allogeneic CAR-T therapies to revolutionize patient care through their combination of clinical effectiveness, on-demand availability, and scalable production, which could significantly widen access to CAR-T therapies.

Currently in a Phase 1 clinical trial, P-BCMA-ALLO1 has shown promising early safety and preliminary efficacy outcomes, as revealed at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2023. The therapy demonstrated a well-tolerated profile and was successfully administered to 100% of patients without the need for bridging chemotherapy, showcasing its potential as an effective off-the-shelf option. Furthermore, preliminary findings indicated that allogeneic TSCM-rich CAR-T cells were capable of migrating to bone marrow, evolving into effector T cells capable of targeting cancer cells, and sustaining their presence for at least six weeks post-treatment.

Poseida Therapeutics plans to reveal more data from recently enrolled patients at the American Association for Cancer Research (AACR) Annual Meeting in San Diego on April 8, 2024. With additional clinical updates expected in the latter half of 2024, in collaboration with Roche, the company is poised to provide further insights into the efficacy and safety of P-BCMA-ALLO1.

The FDA’s Orphan Drug Designation is designed to encourage the development of treatments for rare diseases, affecting fewer than 200,000 people in the United States. This designation offers various incentives to sponsors, such as tax credits for clinical trials, exemptions from certain FDA fees, and the possibility of seven years of marketing exclusivity upon approval.

P-BCMA-ALLO1 represents a pivotal step forward in the fight against multiple myeloma, embodying hope for patients with limited treatment options. This collaboration between Poseida Therapeutics and Roche underscores the potential of innovative cell and gene therapies to meet critical healthcare needs and transform patient outcomes in the realm of cancer treatment.

The Orphan Drug Designation awarded to P-BCMA-ALLO1 by the FDA marks a significant milestone in the development of new treatments for multiple myeloma. This designation not only highlights the innovative approach of utilizing TSCM-rich allogeneic CAR-T cells but also emphasizes the urgent need for more effective and readily available treatments for this challenging disease. As the clinical trials progress, the medical community eagerly anticipates further updates, hopeful that this therapy will offer a new beacon of hope for patients battling relapsed or refractory multiple myeloma.

  AACR Annual Meeting, ASH Annual Meeting, CAR-T therapy, clinical-stage cell and gene therapy, FDA Orphan Drug Designation, multiple myeloma treatment, P-BCMA-ALLO1, Poseida Therapeutics, relapsed/refractory multiple myeloma, T stem cell memory