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.

The European Medicines Agency (EMA) has granted PRIority MEdicines (PRIME) eligibility to its investigational CAR-T cell therapy fully human anti-BCMA (B Cell Maturation Antigen) autologous chimeric antigen receptor (CAR) T Cells (ct053) for the treatment of relapsed or refractory multiple myeloma.

PRIME eligibility was based on clinical data from an ongoing CT053 BCMA CAR-T phase 1 study in China. The results from the trial were presented at an oral presentation on September 14, 2019 in Boston at the 17^th International Myeloma Workshop. As of June 30, 2019, 21 out of 24 myeloma patients (87.5%) who received a median of 4.5 prior lines of myeloma therapy showed objective response. 19 out of 24 patients (79.2%) achieved complete response. There was no grade 3 or higher cytokine release syndrome. The duration of response data will be reported at a future date.

“PRIME eligibility is an important regulatory milestone in the continued development and commercialization of CT053 anti-BCMA CAR T cells,” said Zonghai Li, M.D., Ph.D., chief executive officer of CARsgen. “CT053 has demonstrated the potential to become the best-in-class BCMA CAR-T therapy. PRIME designation is invaluable to the advancement of this cutting-edge therapeutic to potential market approval and being available to patients as quickly as possible.” The CT053 anti-BCMA CAR-T program has received Investigational New Drug (IND) clearance and Orphan Drug designation from the U.S. Food and Drug Administration and authorization of its Clinical Trial Application (CTA) from Health Canada.

EMA’s voluntary PRIME scheme supports the development of promising medicines that show a high potential to benefit patients and target a significant unmet medical need. The status is granted to medicines that may offer a major therapeutic advantage over existing treatments. The program provides developers with enhanced interaction and early dialogue with the EMA to expedite drug development. Between the launch of PRIME in March 2016 through July 25, 2019, the EMA has received and assessed a total of 246 requests for eligibility. Of these, only 24 out of a total of 76 requests in oncology and hematology have been accepted into the scheme.