SHANGHAI, NANJING, China and SAN JOSE, Calif., Aug. 19, 2022 -- IASO Biotherapeutics (IASO Bio), a clinical-stage biopharmaceutical company engaged in discovering, developing, and manufacturing innovative cell therapies and antibody products, announced today that the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) approved its investigational new drug (IND) application (Acceptance No.: CXSL2200233，CXSL2200234) for the new extended indication of Neuromyelitis Optica Spectrum Disorder (NMOSD) for a fully human BCMA chimeric antigen receptor autologous T (CAR-T) cell Injection (Equecabtagene Autoleucel, CT103A). 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. As of March 20, 2022, 12 subjects received Equecabtagene Autoleucel cell infusion, including three in the 0.5×106 CAR-T cells/kg dose group and nine in the 1.0×106 CAR-T cells/kg dose group. The study data initially showed that the Equecabtagene Autoleucel injection was safe in patients with relapsed/refractory NMOSD in the 0.5×106 CAR-T cells/kg and 1.0×106 CAR-T cells/kg dose groups. All patients experienced Grade 1-2 CRS (Cytokine Release Syndrome) and no Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) events. In terms of efficacy, all subjects observed improved Expanded Disability Status Scale (EDSS) scores after infusion. Fifty percent experienced improved visual acuity, 67% improved their walking ability, and 75% experienced improved bladder function. After a median follow-up of 5.5 months, 11 / 12 (92%) subjects did not observe any disease recurrence.
Wen (Maxwell) Wang, M.D., Ph.D., 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.
About Equecabtagene Autoleucel (CT103A)
Equecabtagene Autoleucel (CT103A) is a BCMA chimeric antigen receptor autologous T cell injection, a lentiviral vector containing a CAR structure with a fully human scFv, CD8a hinger and transmembrane, 4-1BB co-stimulatory and CD3ζ activation domains. Based on strict selection and screening, utilizing a proprietary in-house optimization platform, and integrated in-house manufacturing process improvement, the construct of the BCMA CAR-T is potent and Equecabtagene Autoleucel shows prolonged persistency in patients. The NMPA accepted the New Drug Application for Equecabtagene Autoleucel for the treatment of elapsed/refractory multiple myeloma (R/R MM). Equecabtagene Autoleucel also received Breakthrough Therapy Designation by the NMPA in February 2021 and Orphan Drug Designation (ODD)by the U.S. FDA in February 2022.
In addition to multiple myeloma, the NMPA has received IND application of Equecabtagene Autoleucel for the new expanded indication of Neuromyelitis Optica Spectrum Disorder (NMOSD).
About Neuromyelitis Optica (NMO)
Neuromyelitis Optica (NMO) is an acute or subacute inflammatory demyelination disorder of the central nervous system, an antibody-mediated idiopathic inflammatory disease of the nervous system. NMO Spectrum Disorders (NMOSD) are marked by NMO-IgG antibodies in the serum, covering NMO and NMO-related diseases. The first onset of NMOSD is seen at all ages, mostly in young and middle-aged people, with a median age of 39 years. The prevalence of NMOSD is high in Asian populated areas, and middle-aged women are the most prevalent group. According to Frost & Sullivan, the number of NMOSD cases in China was around 49,300 and 173,000 worldwide in 2021. AQP4-Ab is a foremost pathogenic antibody of NMOSD, and many clinical studies have confirmed that this antibody may cause pathological damage to the central nervous system in animals and humans. Its diagnostic specificity is up to more than 90%, with the positive rate of AQP4-Ab in NMOSD patients ranging from 40% to 90%. NMOSD is a highly recurrent disease with a high disability rate. More than 90% of patients have a multitemporal course, 60% relapse within one year and 90% relapse within three years, with sequelae found in most patients such as severe visual impairment, physical dysfunction, and urination and defecation disorders.
About IASO Bio
IASO Bio is an innovative biopharmaceutical company specializing in the development and manufacture of cellular therapeutics and antibody drugs. The company is expanding into solid tumors and autoimmune diseases with the development of hematologic oncology cell-based drugs and antibody drugs as the cornerstone of innovation. It offers a complete platform from early discovery, registration, and clinical development to commercial production. IASO Bio owns many technology platforms, including a fully human antibody discovery platform, a high-throughput CAR-T drug preference platform, a general CAR technology platform, a production technology platform, and a clinical translational research platform. It has more than 10 products at different stages of development, including Equecabtagene Autoleucel (CT103A)，fully human BCMA chimeric antigen receptor autologous T cell injection, which received NDA acceptance of the China NMPA for the treatment of elapsed/refractory multiple myeloma (R/R MM). Equecabtagene Autoleucel also received Breakthrough Therapy Designation by the NMPA in February 2021 and Orphan Drug Designation (ODD) by the U.S. FDA in February 2022. In addition to multiple myeloma, the NMPA has received IND application of Equecabtagene Autoleucel for the new expanded indication of Neuromyelitis Optica Spectrum Disorder (NMOSD). Additionally, its product CT120 (fully human CD19/CD22 dual-target CAR-T cell injection) has entered the clinical research stage for the treatment of CD19/CD22-positive relapsed/refractory B-cell non-Hodgkin lymphoma (B-NHL) and relapsed/refractory acute B-lymphoblastic leukemia (B-ALL) and granted FDA Orphan Drug Designation (ODD).
Leveraging its strong management team, rich product pipeline, cutting-edge R&D, and business model, and with the introduction of innovative drugs that truly solve clinical pain points and open new treatment paths, IASO Bio is becoming one of the industry's most influential and innovative pharmaceutical companies.