BCMA CAR T Therapy Recognized for R/R Multiple Myeloma

The FDA has granted regenerative medicine advanced therapy (RMAT) and fast track designations to IASO Bio’s chimeric antigen receptor (CAR) T-cell therapy CT103A (equecabtagene autoleucel) for treating relapsed/refractory multiple myeloma (RRMM).¹

CT103A is a BCMA-targeted, autologous, lentiviral vector, CAR T-cell therapy. China’s National Medical Products Administration (NMPA) has accepted the therapy’s new drug application and granted it breakthrough therapy designation. The FDA accepted the therapy’s investigational new drug application (IND) in December 2022 and granted it orphan drug designation in February 2022. The NMPA has also accepted the IND of CT103A for a new expanded indication of neuromyelitis optica spectrum disorder (NMOSD).

"The IND approval of CT103A in the United States is an important milestone of IASO Bio and a new starting point outside China,” Wen (Maxwell) Wang, chief executive officer, IASO Bio, said in a statement regarding the IND clearance in RRMM.² “IASO Bio will accelerate its overseas clinical trials and the development and implementation of cellular immunotherapy drugs to benefit more patients globally."

CT103A recently demonstrated a manageable safety profile in patients with NMOSD recently published data from an ongoing open-label, single-arm, phase 1 clinical trial (NCT04561557) in January 2023.³ All 12 treated patients experienced adverse events of grade 3 or higher, the most common of which were hematological toxic effects, including leukopenia (100%), neutropenia (100%), anemia (50%), and thrombocytopenia (25%), and most of which resolved within 4 weeks. Serious adverse effects included cytomegalovirus infection (25%), coagulation disorder (8%), and pneumonia (8%).

READ MORE: Ide-cel Beats SOC in Relapsed/Refractory Multiple Myeloma PFS

The study participants were mostly women (83.3%) and had a median age of 49.5 years (range, 30–67). Seven (58%) patients developed an infection during the course of the study; these were all under grade 4. Eleven patients had not relapsed at a median follow-up of 5.5 months, and all participants generally reported improvements in disability and quality of life outcomes. Investigators also observed a downward trend in AQP4 serum antibodies in 11 patients and an association between CAR T-cell expansion and responses in 17% of patientsthat continued through 6 months post-infusion.

"Our data show that CAR-BCMA T-cell therapy might induce clinical remission of NMOSD associated with the rapid removal of AQP4 autoantibodies, and longer-term observation will be warranted," lead author Chuan Qin, MD, associate professor, department of neurology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China, and colleagues wrote.³

REFERENCES

1. IASO Bio announces CT103A granted regenerative medicine advanced therapy (RMAT) and fast track (FT) Designations by the FDA. News release. IASO Bio. February 12, 2023. https://www.prnewswire.com/news-releases/iaso-bio-announces-ct103a-granted-regenerative-medicine-advanced-therapy-rmat-and-fast-track-ft-designations-by-the-fda-301744775.html
2. IASO Bio announces US FDA approval of clinical trial application for BCMA CAR-T CT103A for relapsed/refractory multiple myeloma. News release. IASO Biotherapeutics. December 22, 2022.http://www.iasobio.com/info.php?id=209
3. Qin C, Tian DS, Zhou LQ, et al. Anti-BCMA CAR T-cell therapy CT103A in relapsed or refractory AQP4-IgG seropositive neuromyelitis optica spectrum disorders: phase 1 trial interim results. Signal Transduct Target Ther. 2023;8(1):5. Published 2023 Jan 4. doi:10.1038/s41392-022-01278-3