China-based IASO Biotherapeutics has announced approval from the US FDA for an Investigational New Drug (IND) application for its BCMA-targeted chimeric antigen receptor (CAR) T cell therapy CT103A (equecabtagene autoleucel). The therapy will be assessed in US-based clinical trials for relapsed/refractory multiple myeloma (r/r MM).
Drug Profile and Development
Equecabtagene autoleucel (CT103A) is a BCMA autologous CAR-T cell injection, a lentiviral vector containing a CAR structure with a fully human scFv, CD8a hinge and transmembrane, 4-1BB co-stimulatory, and CD3ζ activation domains. Developed on a proprietary in-house optimization platform and manufacturing process improvement, the construct of the BCMA CAR-T is potent, and equecabtagene autoleucel shows prolonged persistency in patients. Results from a clinical Phase I/II (NCT05066646) study in China showed excellent safety and efficacy for the therapy.
Regulatory Milestones and Future Prospects
Innovent and IASO filed equecabtagene autoleucel for approval in China for the r/r MM indication. China’s National Medical Products Administration (NMPA) gave the molecule a breakthrough therapy designation in February 2021, while the US FDA bestowed Orphan Drug Designation (ODD) in February 2022. In addition to multiple myeloma, the NMPA has received an IND application for equecabtagene autoleucel for the new expanded indication of neuromyelitis optica spectrum disorder (NMOSD). IASO Bio and Innovent Biologics, Inc. (1801.HK) are jointly developing CT103A for the treatment of r/r MM in mainland China.-Fineline Info & Tech