FDA Grants Regenerative Medicine Advanced Therapy and Fast Track Designations to C-CAR039 for Relapsed/Refractory DLBCL

A bi-specific anti-CD19/CD20 CAR T-cell therapy, C-CAR039, received a regenerative medicine advanced therapy designation and fast track designation from the FDA for treating patients with relapsed/refractory diffuse large B cell lymphoma.

The FDA granted a regenerative medicine advanced therapy designation and fast track designation to the bi-specific anti-CD19/CD20 CAR T-cell therapy, C-CAR039, for the treatment of patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), according to a press release from the therapy’s developer, Cellular Biomedicine Group Inc. (CBMG).

Favorable efficacy and safety data were observed in an investigator-initiated trial across multiple sites in China utilizing C-CAR039 to treat patients with relapsed/refractory B-cell non-Hodgkin’s lymphoma. Data from the study highlight a best reported overall response rate (ORR) of 92.6%, and complete response rate (CRR) of 85.2% following treatment with C-CAR039. The median time to response for this patient population was 1.0 month, and 74.1% of patients remained in complete remission at a median follow-up of 7 months. The estimated 6-month progression-free survival (PFS) rate was 83.2% (95% CI, 69.1%-100.0%).

“This is great news for Cellular Biomedicine Group (CBMG) that the FDA has granted C-CAR039 both regenerative medicine advanced therapy and fast track designations based on its potential to increase objective and complete response rates in R/R DLBCL,” Tony (Bizuo) Liu, chairman and chief executive officer at CBMG, said in the press release. “The clinical data based on our clinical trials in China continue to support the hypothesis that C-CAR039 is the best-in-class CAR T asset for patients in this indication. We are working towards initiating 1b/2 trials for C-CAR039 in the U.S. soon. And we will work closely with the FDA to seek the best path forward to deliver the drug to patients in the U.S. and EU.”

A total of 34 patients received C-CAR039 cell therapies as of April 20, 2021, 28 of whom were evaluable for safety and 27 were evaluable for efficacy. The median age for patients receiving treatment was 55.5 years and patients had undergone a median of 3 prior lines of therapy. The majority of patients had Ann Arbor stage III/IV disease (75%). A total of 17.9% of patients received bridging therapy.

In terms of safety, cytokine release syndrome (CRS) was observed in 96% of patients, with most cases of CRS being grade 1 or 2 (92%); one patient had grade 3 CRS. Two patients experienced grade 1 immune effector cell-associated neurotoxicity syndrome, with no reports of grade 2 or higher neurologic toxicities in the study.

Previously, orphan drug designation was granted to C-CAR039 by the FDA Office of Orphan Products Development for the treatment of patients with follicular lymphoma.

CBMG, the biopharmaceutical company that develops cellular immunotherapies, plans to study C-CAR039 in longer follow-up trials moving forward.

Reference

CBMG receives FDA regenerative medicine advanced therapy and fast track designations for bi-specific anti-CD19/CD20 CAR-T cell therapy for relapsed/refractory B-cell non-Hodgkin lymphoma. News release. CBMG Holdings. January 12, 2022. Accessed January 12, 2022. https://tinyurl.com/3kbwj6kx