China’s National Medical Products (NMPA) has approved an investigational new drug application from IASO Bio for the new extended indication of neuromyelitis optica spectrum disorder (NMOSD).
Neuromyelitis optica spectrum disorders previously known as Devic disease or neuromyelitis optica are inflammatory disorders of the central nervous system characterized by severe, immune-mediated demyelination and axonal damage predominantly targeting optic nerves and the spinal cord.
The announcement that the Center for Drug Evaluation had given the go-ahead was made today (August 19) for fully human B-cell maturation antigen (BCMA) chimeric antigen receptor autologous T (CAR-T) cell injection, equecabtagene autoleucel.
This is the world’s first IND approval for CAR-T in NMOSD treatment.
The new IND application is based on clinical data from an investigator-initiated clinical study of equecabtagene autoleucel. Subjects in the study were NMOSD patients with poor symptom control who had at least one year of treatment with at least one immunosuppressant.
The study’s primary objective was to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetic (PK) and pharmacodynamic (PD) profiles in NMOSD patients treated with equecabtagene autoleucel.
Wen Maxwell Wang chief executive officer and chief medical officer of IASO Bio, said: “As one of the first companies to conduct research on CAR-T to treat autoimmune diseases worldwide, our BCMA CAR T-cell therapy represents a significant milestone for Investigator Initiated Trial (IIT) data of relapsed and refractory NMOSD, an autoimmune disease with serious complications, blindness, and paralysis.”
The existing treatment of NMOSD can only decrease the number of relapses within a certain period and has little effect on the functional recovery of sensory, nervous, and motor systems. BCMA CAR T-cell therapy can reduce the disability score and improve the functions of sensory, nervous, and motor systems, providing a milestone proof-of-concept for CAR-T therapy to treat autoimmune diseases caused by auto-antibodies produced by plasma cells.
The IND’s approval demonstrates further momentum for IASO Bio to promote the expansion of CAR-T therapy and launch products beyond the treatment of malignant tumors to the treatment of autoimmune diseases. IASO Bio will initiate and complete the clinical study per submitted protocol to prepare for new drug application (NDA) and to bring hope to NMOSD patients.