FDA Grants RMAT Designation to Poseida Therapeutics for P-BCMA-ALLO1 in Relapsed/Refractory Multiple Myeloma

The U.S. Food and Drug Administration (FDA) has granted Poseida Therapeutics' investigational therapy, P-BCMA-ALLO1, the Regenerative Medicine Advanced Therapy (RMAT) designation. P-BCMA-ALLO1 is an allogeneic CAR-T cell therapy currently in Phase 1/1b clinical development for treating patients with relapsed or refractory multiple myeloma, a severe form of blood cancer.

The RMAT designation is designed to expedite the development and review of regenerative medicinal products that show potential in treating serious or life-threatening conditions. Therapies under this designation benefit from increased interaction with the FDA, facilitating a smoother and potentially faster development process. P-BCMA-ALLO1’s RMAT application was evaluated based on promising early data from its ongoing Phase 1 study. This study illustrated P-BCMA-ALLO1's potential effectiveness and safety, as well as rapid availability for patients.

Kristin Yarema, Ph.D., President and CEO of Poseida Therapeutics, highlighted the importance of this designation, noting that the early clinical data from the Phase 1 study supports P-BCMA-ALLO1’s potential to meet the needs of patients with relapsed/refractory multiple myeloma. These patients often have limited treatment options, especially those who failed prior BCMA-targeted therapies. The RMAT designation comes in addition to the Orphan Drug designation already granted to P-BCMA-ALLO1, further supporting its development.

Poseida is set to present new clinical data from the Phase 1 study at the 21st International Myeloma Society Annual Meeting in Rio de Janeiro later this month. Additional clinical updates are anticipated later in the year, in collaboration with Roche, which has a strategic partnership with Poseida covering several investigational allogeneic CAR-T therapies for blood cancers, including P-BCMA-ALLO1.

The RMAT designation, part of the 21st Century Cures Act, aims to accelerate the development and review of regenerative therapies that treat, modify, reverse, or cure serious or life-threatening diseases. To qualify for this designation, a therapy must demonstrate preliminary clinical evidence indicating its potential to address unmet medical needs for the condition it targets. RMAT designation encompasses all the features of the Breakthrough Therapy designation, such as early discussions with the FDA about potential surrogate or intermediate endpoints. These therapies may also gain accelerated approval based on such endpoints if they are considered likely to predict long-term clinical benefits.

P-BCMA-ALLO1 is an allogeneic CAR-T product targeting B-cell maturation antigen (BCMA) for the treatment of multiple myeloma. The ongoing clinical development involves a Phase 1/1b study, with early data supporting its potential as an effective and safe treatment. This product candidate has already been granted both RMAT and Orphan Drug designations by the FDA, specifically for adults who have undergone at least three prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.

Poseida Therapeutics is a clinical-stage biopharmaceutical company focused on advancing allogeneic cell therapies and genetic medicines to potentially cure certain cancers and rare diseases. Their portfolio includes investigational therapies for both solid tumors and hematologic cancers, leveraging proprietary genetic editing platforms such as the piggyBac DNA Delivery System, Cas-CLOVER Gene Editing System, and various nanoparticle gene delivery technologies. The company has established strategic partnerships with Roche and Astellas to further explore the promise of cell therapies for cancer treatment.