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

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This NMPA acceptance, the world's first IND application for CAR-T in NMOSD.

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PR Newswire

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星期六, 五月 28, 2022

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CALIFORNIA, CHINA

摘要

Key Points:

This NMPA acceptance, the world's first IND application for CAR-T in NMOSD.
IASO Bio's IND application is based on the Equecabtagene AutoleucelInvestigator Initiated Trial (IIT) enrolling patients with refractory NMOSD who were positive for Aquaporin 4 antibodies (AQP4).
Subjects enrolled were patients with poor symptom control despite at least one year of treatment with at least one immunosuppressant.
"Theuse of CAR-T cell products has been widely accepted in the treatment of hematologic malignancy but has been rarely reported in the treatment of autoimmune diseases.

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

SAN JOSE, Calif., NANJING, China and SHANGHAI, May 28, 2022 /PRNewswire/ -- IASO Biotherapeutics (IASO Bio), a clinical-stage biopharmaceutical company engaged in discovering, developing, and manufacturing innovative cell therapies and antibody products, announced today that China's National Medical Products Administration (NMPA) has accepted its investigational new drug (IND) application 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 NMPA acceptance, the world's first IND application for CAR-T in NMOSD.

IASO Bio's IND application is based on the Equecabtagene Autoleucel Investigator Initiated Trial (IIT) enrolling patients with refractory NMOSD who were positive for Aquaporin 4 antibodies (AQP4). Subjects enrolled were patients with poor symptom control despite at least one year of treatment with at least one immunosuppressant. The primary endpoint of this study was to observe the safety and tolerability, as well as preliminary efficacy, pharmacokinetic (PK), and pharmacodynamic (PD) related indicators in subjects with relapsed/refractory NMOSD treated with Equecabtagene Autoleucel. As of March 20, 2022 cut-off date, 12 subjects received Equecabtagene Autoleucel cell reinfusion, including three in the 0.5×10⁶ CAR-T cells/kg dose group, three in the 1.0×10⁶ CAR-T cells/kg dose group in the dose escalation stage and six subjects in the expansion stage.

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

"The use of CAR-T cell products has been widely accepted in the treatment of hematologic malignancy but has been rarely reported in the treatment of autoimmune diseases. As one of the first company to use CAR-T cell to treat autoimmune diseases, IASO Bio submits the first IND application in China, marking the initiation of the expansion of their indication from oncology to autoimmune diseases, which is of great important for the cell therapy field. BCMA-directed CAR-T cell products targeting plasmocyte are available as a new option to treat the autoimmune diseases caused by plasmocyte-produced autoantibody. Neuromyelitis optica spectrum disorder (NMOSD) is characterized by high recurrence and disability rates, etc., and often cause blindness, paralysis, defecation and other serious physical disorders in a later stage" said Dr. Wen Wang, the Chief Executive Officer and Chief Medical Officer of IASO Bio. "In contrast, the new therapeutic strategy offered by IASO Bio with Equecabtagene Autoleucel can provide an opportunity to reverse and ameliorate organ damage and improve the quality of life in NMOSD patients. It may provide a new treatment option for patients with poorly controlled NMOSD by existing drugs. We expect that this product candidate will be available soon to NMOSD patients in China and around the world."

About Neuromyelitis Optica (NMO) and Neuromyelitis Optica Spectrum Disorder (NMOSD)

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. Neuromyelitis Optica Spectrum Disorder (NMOSD) is marked by the presence of 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 49,200，and the total number of NMOSD cases in the worldwide was about 171,000. AQP4-Ab is a foremost pathogenic antibody of NMOSD, and a large number of basic and 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, about 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 specialized in the development and industrialization 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 has a complete 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 platform, a high-throughput CAR-T drug preference platform, a general CAR technology platform, a production technology platform, and a clinical translational research platform. There are more than 10 varieties at different stages of development, and Equecabtagene Autoleucel (CT103A，fully human BCMA chimeric antigen receptor autologous T cell injection) with a rapid growth is in the late stage of clinical development. In February 2021, it was included in the "breakthrough therapy-designated drugs" by the Center of Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) and was recognized as an "orphan drug" by the U.S. Food and Drug Administration (FDA) in February 2022. The innovative product CT120 (fully human CD19/CD22 dual-target CAR-T cell injection) developed by the company independently has entered the clinical research stage with indications for CD19/CD22 positive relapsed/refractory B-cell non-Hodgkin's lymphoma (B-NHL) and relapsed/refractory acute B-lymphoblastic leukemia (B-ALL), respectively, and has received FDA orphan drug designation (ODD).

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