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Poseida Therapeutics Receives US FDA Orphan Drug Designation For P-BCMA-101 For The Treatment Of Multiple Myeloma

By: Press Release Reporter; Published: May 13, 2019 @ 8:00 am | Comments Disabled

San Diego, CA (Press Release) – Poseida Therapeutics Inc., a clin­i­cal-stage bio­pharma­ceu­tical com­pany leveraging pro­pri­e­tary non-viral gene engi­neer­ing tech­nolo­gies to create life-saving thera­peutics, today announced the United States Food and Drug Admin­istra­tion (FDA) has granted orphan drug desig­na­tion to P-BCMA-101 for the treat­ment of re­lapsed and/or refractory multiple myeloma. P-BCMA-101 is an au­tol­o­gous CAR-T ther­apy devel­oped using Poseida’s piggyBac® plat­form tech­nology. P-BCMA-101 is com­prised of a high per­cent­age of long-lived, self-renewing stem cell memory T cells targeting cancer cells expressing B-cell maturation an­ti­gen (BCMA).

“FDA orphan desig­na­tion is an im­por­tant regu­la­tory mile­stone in the con­tinued devel­op­ment and com­mer­cial­iza­tion of P-BCMA-101,” said Eric Ostertag, M.D., Ph.D., chief exec­u­tive officer of Poseida. “P-BCMA-101 has dem­onstrated outstanding potency, with strikingly low rates of toxicity in our phase 1 clin­i­cal trial. In fact, the FDA has approved fully outpatient dosing in our Phase 2 trial starting in the second quarter of 2019.”

Poseida’s non-viral piggyBac DNA Modification System results in CAR-T prod­uct can­di­dates with a high per­cent­age of stem cell memory T cells (Tscm), the only T cell that is self-renewing and long-lived, leading to CAR-T prod­ucts with im­proved efficacy, lower toxicity and poten­tially greater durability than earlier generation CAR-T ther­a­pies.

Orphan drug desig­na­tion is granted by the FDA Office of Orphan Products Development to drugs and biologics which are in­tended for the treat­ment, diag­nosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Under the Orphan Drug Act, the FDA may provide grant funding to­ward clin­i­cal trial costs, tax ad­van­tages, FDA user-fee benefits and seven years of mar­ket exclusivity in the United States fol­low­ing mar­ket­ing approval by the FDA.

Poseida has received grant funding from the California Institute for Regenerative Medicine to sup­port the clin­i­cal devel­op­ment of P-BCMA-101.

About Poseida Therapeutics, Inc.

Poseida Therapeutics is a clin­i­cal-stage bio­technology com­pany translating best-in-class gene engi­neer­ing tech­nolo­gies into lifesaving treat­ments 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, as well as gene ther­a­pies for orphan genetic diseases. Poseida has assembled a suite of industry-leading gene editing tech­nolo­gies, in­­clud­ing the piggyBac® DNA Modification System, Cas-CLOVER™ and TAL-CLOVER™ site-specific nucleases and Footprint-Free® Gene Editing. For more in­for­ma­tion, visit www.poseida.com.

Source: Poseida Therapeutics.


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