IASO Bio Secures FDA Approval for BCMA CAR-T Therapy in Multiple Autoimmune Diseases
核心洞察
IASO Bio (搜索) received FDA IND approval for Equecabtagene Autoleucel (Eque-cel (搜索)) to treat multiple sclerosis (搜索), marking the second autoimmune indication approved in 2024.
The fully human anti-BCMA (搜索) CAR-T therapy has now secured five total IND approvals across China and the US for various autoimmune diseases including lupus and myasthenia gravis (搜索).
Multiple sclerosis (搜索) affects approximately 3.07 million patients worldwide with 400,000 in the US, representing a significant unmet medical need in neuroinflammatory diseases.
IASO Biotechnology (搜索) has received U.S. Food and Drug Administration (搜索) approval for its investigational new drug application to test Equecabtagene Autoleucel (Eque-cel (搜索)) in multiple sclerosis (搜索) patients, expanding the fully human anti-BCMA (搜索) CAR-T therapy's reach into neuroinflammatory diseases. This marks the second FDA IND approval for autoimmune diseases in 2024, following approval for refractory generalized myasthenia gravis (搜索).
The Shanghai-based biopharmaceutical company has now secured five total IND approvals for Eque-cel (搜索) across autoimmune indications in China and the United States. The FDA has approved three autoimmune indications including myasthenia gravis (搜索), multiple sclerosis (搜索), and systemic lupus erythematosus (搜索) with lupus nephritis (搜索), while China's National Medical Products Administration (搜索) has approved two indications for neuromyelitis optica spectrum disorder (搜索) and myasthenia gravis.
Targeting Multiple Sclerosis Through B Cell Depletion
Multiple sclerosis (搜索) represents a significant therapeutic challenge, affecting approximately 3.07 million patients worldwide, including 400,000 in the United States according to Frost & Sullivan data. The neuroinflammatory disease of the central nervous system causes demyelination and neuronal injury, making it one of the most common causes of non-traumatic disability among young adults aged 18-40 years.
The disease demonstrates a strong female predominance with a 3:1 female to male ratio. Approximately 85-90% of MS patients develop a relapsing-remitting form characterized by periods of symptom exacerbation followed by remission. Around 50% of patients eventually progress to secondary progressive MS, which involves progressive, irreversible accumulation of neurologic disability.
Dr. Yongke Zhang, Chief Scientific Officer of IASO Bio (搜索), stated: "In an investigator initiated trial conducted in China, Eque-cel (搜索) has shown promising efficacy in 6 autoimmune diseases. The IND approval of Eque-cel in the treatment of MS from the FDA is another strong evidence of IASO Bio's ongoing dedication and technological advancements in the treatment of autoimmune diseases."
Expanding Into Lupus Indications
The company has also received FDA approval to test Eque-cel (搜索) in systemic lupus erythematosus (搜索) and lupus nephritis (搜索), conditions affecting approximately 8.049 million patients worldwide, including 289,000 in the United States. Lupus nephritis represents the most common severe complication of SLE, with studies showing that 60% of SLE patients develop LN during their disease course, making it a major cause of high mortality.
Current standard-of-care treatments for SLE demonstrate limited efficacy, with 14-33% of lupus nephritis (搜索) patients showing no response to treatment, highlighting the significant unmet medical need in this patient population.
Mechanism of Action and Clinical Development
Equecabtagene Autoleucel employs second-generation CAR technology to precisely identify and eliminate cells expressing BCMA (搜索) (B-cell maturation antigen). The therapy works by clearing activated B cells and plasma cells from patients' bodies, reducing pathogenic cells and autoantibodies to suppress autoimmune inflammation.
The individually tailored, BCMA (搜索)-targeted genetically modified autologous T cell immunotherapy has already received marketing approval from China's NMPA in June 2023 for treating patients with relapsed and/or refractory multiple myeloma (搜索) who received at least three prior therapy lines containing a proteasome inhibitor and an immunomodulatory agent.
Global Development Strategy
IASO Bio (搜索)'s comprehensive drug development capabilities span from early discovery through clinical development, regulatory approval, and commercial production. The company maintains a diversified pipeline of over 10 novel products, with Equecabtagene Autoleucel representing a key asset in both oncology and autoimmune disease applications.
Zhang emphasized the company's commitment to global expansion: "We will continue to adhere to the research and development philosophy that prioritizes clinical value to address unmet clinical needs and will place great importance on implementing a global strategy. Through close collaboration and in-depth exchanges with international clinical research institutions, we aim to accelerate the development and commercialization of more innovative drugs, bringing greater benefits to patients worldwide."