IASO Biotechnology, a China-based biopharmaceutical company, announced on Thursday that it has received approval from the US Food and Drug Administration (FDA) for the Investigational New Drug (IND) application of its BCMA CAR-T (Equecabtagene Autoleucel) for use in US clinical trials for generalised myasthenia gravis (gMG), an autoantibody-mediated neuromuscular junction disorder.

The Chinese IND for this indication of Equecabtagene Autoleucel was approved by Chinese regulator, NMPA, in January this year.

Two patients with refractory MG were enrolled in an investigator-initiated open-label study to assess the safety and efficacy of Eque-cel intended for the treatment of relapsed/refractory antibody-mediated idiopathic inflammatory disorders of the nervous system (NCT04561557). Both patients were treated with a single infusion of Eque-cel at the doses of 1.0×106 CAR-T/Kg, respectively. No new safety risk was identified, and the safety profile was superior compared to that in multiple myeloma indication. The result also suggested that the long-term efficacy of CAR-T cell therapy may be related to the reconstitution of B cells with a predominantly naïve phenotype and the continued clearance of plasma cells.