Shanghai, China (Press Release) – CARsgen Thera­peutics Co. Ltd., a clin­i­cal-stage bio­pharma­ceu­tical com­pany to­day an­nounced that the United States Food and Drug Admin­istra­tion (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) desig­na­tion to its inves­ti­ga­tional CT053 CAR-T cell ther­apy. CT053 is a fully human anti-BCMA (B Cell Maturation Antigen) au­tol­o­gous chi­meric an­ti­gen re­cep­tor (CAR) T Cell ther­apy for the treat­ment of re­lapsed and/or re­frac­tory mul­ti­ple myeloma (rrMM).

RMAT desig­na­tion was based on clin­i­cal data from an on­go­ing CT053 phase 1 study in heavily pre-treated mul­ti­ple myeloma patients in China. Updated data from CT053 will be pre­sented at the 61th annual meeting of the American Society of He­ma­tol­ogy in Orlando on De­cem­ber 9.

"RMAT eligibility is an im­por­tant regu­la­tory mile­stone for CARsgen in the con­tinued devel­op­ment and com­mer­cial­iza­tion of CT053 anti-BCMA CAR T cell ther­apy," said Zonghai Li, M.D., Ph.D., the chief exec­u­tive officer of CARsgen. "The RMAT desig­na­tion in­di­cates that CT053 has dem­onstrated poten­tial to address unmet med­i­cal needs for patients with rrMM. The desig­na­tion is a remarkable achieve­ment to­wards expediting the prod­uct devel­op­ment and review of our planned bio­log­ics license appli­ca­tion (BLA) and will be invaluable to bringing this cutting-edge ad­vance to patients as quickly as possible. RMAT as well as the PRIority MEdicines (PRIME) eligibility re­ceived from the Euro­pean Medicines Agency (EMA) empower us to col­lab­o­rate closely with the U.S. FDA and EMA to rapidly ad­vance the CT053 devel­op­ment pro­gram to­ward global regu­la­tory ap­prov­als." The CT053 anti-BCMA CAR-T pro­gram has also re­ceived Inves­ti­ga­tional New Drug (IND) clear­ance and Orphan Drug desig­na­tion from the U.S. FDA and authori­za­tion of its Clinical Trial Appli­ca­tion (CTA) from Health Canada.

Established under the 21st Century Cures Act, RMAT desig­na­tion is a ded­i­cated pro­gram de­signed to expedite the drug devel­op­ment and review processes for promising regenerative med­i­cines and ad­vanced ther­a­pies, in­clud­ing CAR T cell ther­a­pies. The desig­na­tion in­cludes all the benefits of the FDA's Fast Track and Break­through Therapy desig­na­tions, providing the benefits of intensive FDA guidance on efficient drug devel­op­ment, in­clud­ing the ability for early inter­actions with FDA senior man­agement to discuss surrogate or intermediate end­points, poten­tial ways to sup­port ac­cel­er­ated ap­prov­al and satisfy post-approval re­quire­ments, poten­tial priority review of the BLA and other oppor­tu­ni­ties to expedite devel­op­ment and review. Between De­cem­ber 13, 2016 and Sep­tem­ber 30, 2019, the FDA re­ceived and assessed a total of 115 re­quests for eligibility. Of these, only 44 have been granted RMAT desig­na­tion.

About CARsgen Thera­peutics, Inc.

CARsgen Thera­peutics is a clin­i­cal-stage immune-oncology com­pany com­mit­ted to the devel­op­ment and com­mer­cial­iza­tion of CAR-T thera­peutics for unmet med­i­cal need. The com­pany has col­lab­o­rated with top hos­pi­tals in China to launch several other First-in-Human stud­ies such as anti-GPC3 CAR-T cell ther­apy for hepato­cellular carcinoma and squamous lung cancer, anti-EGFR/EGFRvIII CAR-T cell ther­apy for glio­blas­toma multiforme and anti-Claudin18.2 CAR-T cell ther­apy for gastric and pancreatic cancer.

For more in­for­ma­tion, please visit: www.carsgen.com

Source: CARsgen.