IASO Biotherapeutics’ Equecabtagene Autoleucel, the World’s First CAR-T for the Treatment of NMOSD, Receives Acceptance of IND Application from NMPA

SAN JOSE, California, NANJING, China and SHANGHAI, May 28, 2022 /PRNewswire/ — IASO Biotherapeutics (IASO Bio), a clinical-stage biopharmaceutical company engaged in the discovery, development and manufacturing of innovative cell therapies and antibody products, announced today that China The National Medical Products Administration (NMPA) has accepted its Investigational New Drug Application (IND) for the new extended indication of Neuromyelitis Optic Spectrum Disorder (NMOSD) for an injection of autologous BCMA chimeric antigen receptor T cells Fully Human (CAR-T) (Equecabtagene AutoleucelCT103A). This NMPA acceptance, the world’s first IND application for CAR-T in NMOSD.

(PRNewsfoto/IASO Biotherapeutics)

IASO Bio’s IND application is based on the Equecabtagene Autoleucel Investigator Initiated Trial (IIT) recruiting patients with refractory NMOSD who were positive for Aquaporin 4 (AQP4) antibodies. The subjects included were patients whose symptoms were poorly controlled despite at least one year of treatment with at least one immunosuppressant. The primary endpoint of this study was to observe safety and tolerability, as well as preliminary efficacy, pharmacokinetic (PK) and pharmacodynamic (PD) indicators in subjects with relapsed/refractory NMOSD treated with Equecabtagene Autoleucel. From March 20, 2022 deadline, 12 subjects received reinfusion of Equecabtagene Autoleucel cells, three of them in the 0.5 × 106 CAR-T cell dose group/kg, three in the 1.0 × 106 Dose group of CAR-T cells/kg at the dose escalation stage and six subjects at the expansion stage.

Preliminary data presented the safety and efficacy of Equecabtagene Autoleucel in the 0.5 × 106 CAR-T cells/kg and 1.0×106 Dose groups of CAR-T cells/kg in the treatment of NMOSD patients with conditions poorly controlled by conventional therapies. All underwent 1-2 CRS and no protocol-mandated ICANS events were found during the 12-subject dose exploration period. Equecabtagene Autoleucel generally reduced the Expanded Disability Status Scale (EDSS) scores of NMOSD subjects, with improved visual acuity in 50% of subjects, improved walking ability in 67% of subjects, and improved bladder function in 75% of subjects.

“The use of CAR-T cell products has been widely accepted in the treatment of hematological malignancies, but has rarely been reported in the treatment of autoimmune diseases. As one of the first companies to use the CAR-T cells to treat autoimmune diseases, IASO Bio files the first IND application in China, marking the beginning of the expansion of their indication from oncology to autoimmune diseases, which is of great importance for the field of cell therapy. BCMA-directed CAR-T cell products targeting plasma cells are available as a new option to treat autoimmune diseases caused by autoantibodies produced by plasma cells. Neuromyelitis Optic Spectrum Disorder (NMOSD) is characterized by high rates of recurrence and disability, etc., and often causes blindness, paralysis, defecation, and other serious physical impairments at a later stage” , said dr. Wen Wangthe managing director and the medical director of IASO Bio. “In contrast, the new therapeutic strategy proposed by IASO Bio with Equecabtagene Autoleucel may provide an opportunity to reverse and ameliorate organ damage and improve the quality of life of patients with NMOSD. It may provide a new option treatment for patients with poorly controlled NMOSD We anticipate that this product candidate will soon be available for NMOSD patients in China and all over the world.”

About Neuromyelitis Optic (NMO) and Neuromyelitis Optic spectrum disorder (NMOSD)

Neuromyelitis optica (NMO) is an acute or subacute inflammatory demyelinating disorder of the central nervous system, an idiopathic antibody-mediated inflammatory disease of the nervous system. Neuromyelitis optica spectrum disorder (NMOSD) is marked by the presence of NMO-IgG antibodies in serum, covering both NMO and NMO-related diseases. The first onset of NMOSD is observed at all ages, mainly in young and middle-aged people, with a median age of 39 years. The prevalence of NMOSD is high in populated areas of Asia, and middle-aged women are the most prevalent group. According to Frost & Sullivan, the number of NMOSD cases in China was 49,200, and the total number of NMOSD cases worldwide was approximately 171,000. AQP4-Ab is one of the main pathogenic antibodies of NMOSD, and a large number of basic and clinical studies have confirmed that this antibody can cause pathological damage to the central nervous system in animals and humans. Its diagnostic specificity is up to more than 90%, with an AQP4-Ab positivity rate 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 evolution, approximately 60% relapse within one year and 90% relapse within three years, with sequelae found in most patients such as severe visual impairment and/or physical dysfunction, and bladder and bowel dysfunction.

About IASO Bio

IASO Bio is an innovative biopharmaceutical company specializing in the development and industrialization of cellular therapeutics and antibody drugs. The company is expanding into solid tumors and autoimmune diseases with cell-based and antibody-based hematology oncology drug development as a cornerstone of innovation. It has full platform capability for the entire process from early discovery, registration, clinical development to commercial production, and owns many technology platforms, including a fully human antibody discovery form, a high-throughput CAR-T drug preference platform, a general CAR technology platform. , a production technology platform and a translational clinical research platform. There are more than 10 varieties in different stages of development, and Equecabtagene Autoleucel (CT103A, fully human BCMA chimeric antigen receptor autologous T cell injection) with rapid growth is in advanced clinical development. In February 2021it has been included in the “Breakthrough Drugs Designated for Therapy” by the Center of Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) and has been recognized as an “Orphan Drug” by the Food and Drug Administration (FDA ) the United States. in February 2022. The innovative product CT120 (injection of fully human CD19/CD22 dual-targeted CAR-T cells) developed by the company independently has entered the clinical research phase with indications for relapsed CD19/CD22 positive non-Hodgkin’s lymphoma/ B-cell (B-NHL) and relapsed/refractory acute lymphoblastic leukemia (B-ALL), respectively, and has received Orphan Drug Designation (ODD) from the FDA.

Relying on its management team with strong execution, rich product pipeline, unique innovative R&D and business model, IASO Bio is determined to become an influential innovative pharmaceutical company in the industry, trying to introduce innovative drugs that truly solve clinical problems and have a competitive market to clinical practice and even to the market, opening up a new treatment path for subjects and bringing them new hope. For more information, please visit www.iasobio.com or www.linkedin.com/company/iasobiotherapeutics.

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