China-based IASO Biotherapeutics announced that the National Medical Products Administration (NMPA) has approved its Investigational New Drug (IND) application for equecabtagene autoleucel (CT103A), a BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, for the treatment of neuromyelitis optica spectrum disorder (NMOSD). This marks the first global trial of a BCMA CAR-T for this indication.
Trial Design
The trial focuses on patients with relapsed/refractory NMOSD who are positive for Aquaporin 4 antibodies (AQP4) and have poor symptom control despite immunosuppressant treatment. The primary endpoint is to evaluate the safety, tolerability, and preliminary efficacy of equecabtagene autoleucel.
Preliminary Results
As of March 20, 2022, 12 patients received treatment, showing improved Expanded Disability Status Scale (EDSS) scores, visual acuity, walking ability, and bladder function. No disease recurrence was observed in 92% of subjects after a median follow-up of 5.5 months.
Collaboration and Licensing
CT103A, co-developed with Innovent Biologics (HKG: 1801), received breakthrough therapy designation (BTD) for recurrent/refractory multiple myeloma (r/r MM) in February 2021. In January 2022, Sana Biotechnology Inc. (NASDAQ: SANA) licensed the BCMA CAR construct for in vivo and ex vivo gene therapy applications, with potential milestone payments of up to USD 204 million.-Fineline Info & Tech
