San Diego, CA (Press Release) – Poseida Therapeutics Inc., a clin­i­cal-stage bio­pharma­ceu­tical com­pany focused on leveraging pro­pri­e­tary next-generation, non-viral gene engi­neer­ing tech­nolo­gies to create life-saving thera­peutics, today announced the U.S. Food and Drug Admin­istra­tion (FDA) has granted a Regenerative Medicine Advanced Therapy (RMAT) desig­na­tion to P-BCMA-101, Poseida’s lead CAR-T thera­peutic can­di­date cur­rently in a Phase 1 clin­i­cal trial for the treat­ment of patients with re­lapsed / refractory multiple myeloma. RMAT desig­na­tion in­cludes all of the benefits of the Fast Track and Break­through Therapy desig­na­tion pro­grams, in­­clud­ing early inter­actions with the FDA.

“P-BCMA-101 is the first anti-BCMA CAR-T ther­apy to receive RMAT desig­na­tion from the FDA and underscores the urgent need for new treat­ment options for multiple myeloma,” said Eric Ostertag, M.D., Ph.D., chief exec­u­tive officer of Poseida Therapeutics. “Initial Phase 1 data presented at the CAR-TCR Summit earlier this year in­cluded en­cour­ag­ing response rates and safety data, in­­clud­ing meaningful responses in a heavily pre­treated pop­u­la­tion, with some patients reaching VGPR and stringent CR. We ex­pec­t to have an addi­tional data update by the end of the year and look for­ward to work­ing closely with the FDA to expedite devel­op­ment of P-BCMA-101.”

The RMAT desig­na­tion is a pro­gram under the 21st Century Cures Act that is in­tended to expedite the devel­op­ment and review of regenerative med­i­cines for the treat­ment of serious or life-threatening diseases and con­di­tions. A regenerative med­i­cine ther­apy is eli­gible for the desig­na­tion if it is in­tended to treat, modify, reverse or cure a serious or life-threatening disease or con­di­tion, and pre­lim­i­nary clin­i­cal evi­dence indicates that the prod­uct has the poten­tial to address unmet medical needs for such a disease or con­di­tion.

RMAT desig­na­tion in­cludes all Break­through Therapy desig­na­tion features, in­­clud­ing early inter­actions to discuss any poten­tial surrogate or intermediate end­points. RMATs may be eli­gible for accelerated approval based on pre­vi­ously agreed-upon surrogate or intermediate end­points that are reason­ably likely to predict long-term clin­i­cal benefit, or reliance upon data obtained from a meaningful number of sites, in­­clud­ing through expansion to addi­tional sites, as appro­pri­ate.

About P-BCMA-101

P-BCMA-101 is an au­tol­o­gous CAR-T thera­peutic can­di­date being devel­oped to treat patients with re­lapsed / refractory multiple myeloma. P-BCMA-101 targets cells that express B cell maturation an­ti­gen, or BCMA, which is ex­pressed on essentially all multiple myeloma cells. P-BCMA-101 is engi­neered with Poseida’s non-viral piggyBac™ DNA Modification System, resulting in a high per­cent­age of T stem cell memory cells. Preliminary results from the com­pany’s ongoing Phase 1 clin­i­cal trial suggest that P-BCMA-101 may have im­proved response rates with a favorable safety profile com­pared to published results from clin­i­cal trials of other CAR-T ther­a­pies at similar doses. Low to no levels of cytokine release syn­drome or neurotoxicity have been seen. The Phase 1 study is funded in part by the California Institute for Regenerative Medicine.

About Poseida Therapeutics, Inc.

Poseida Therapeutics is a clin­i­cal-stage bio­technology com­pany leveraging pro­pri­e­tary next-generation non-viral, gene engi­neer­ing tech­nolo­gies to create life-saving thera­peutics for patients with high unmet medical need. The com­pany is devel­op­ing a wholly-owned pipe­line of au­tol­o­gous and allo­geneic CAR-T prod­uct can­di­dates, initially focused on the treat­ment of hema­to­logical malig­nan­cies and solid tumors. Poseida’s prod­uct can­di­dates are designed to address the limitations of other CAR-T ther­a­pies, in­­clud­ing duration of response, the ability to treat solid tumors and safety con­cerns. P-BCMA-101 is Poseida’s lead CAR-T ther­apy cur­rently in Phase 1 clin­i­cal devel­op­ment for the treat­ment of re­lapsed / refractory multiple myeloma.

Source: Poseida Therapeutics.