IASO Bio Receives Japan PMDA Approval for Phase III CAR-T Trial in Earlier-Line Multiple Myeloma
核心洞察
IASO Biotechnology (搜索) has received Clinical Trial Notification clearance from Japan's PMDA to initiate a Phase III study of equecabtagene autoleucel for relapsed/refractory multiple myeloma patients with 1-2 prior therapies.
The international, randomized trial will compare the BCMA (搜索)-targeted CAR-T therapy against standard treatments in lenalidomide-refractory patients, building on the study that began in China in June 2024.
IASO Bio plans to leverage a cost-effective regulatory strategy combining smaller domestic trials with global data packages to accelerate marketing approval in Japan.
IASO Biotechnology (搜索) has secured Clinical Trial Notification (CTN) clearance from Japan's Pharmaceuticals and Medical Devices Agency (PMDA) to advance its BCMA (搜索)-targeted CAR-T therapy into earlier treatment lines for multiple myeloma patients. The approval enables the company to initiate a Phase III study of equecabtagene autoleucel in patients with relapsed or refractory multiple myeloma who have received one to two prior therapies and are refractory to lenalidomide.
Expanding Treatment Access Through International Trial
The approved trial (CT103AC004) represents an international, multi-center, randomized, open-label, registrational Phase III study designed to evaluate the efficacy and safety of equecabtagene autoleucel compared to standard therapy. The study initially began in China in June 2024 and is progressing as planned, with Japanese enrollment now set to commence.
The therapy targets B-cell maturation antigen (BCMA (搜索)) using a fully human chimeric antigen receptor T-cell approach. Equecabtagene autoleucel uses lentivirus vectors to transfect autologous T cells and was developed through molecular structure screening and functional evaluations to achieve deep responses with manageable safety profiles.
Strategic Regulatory Approach in Japan
IASO Biotechnology (搜索) intends to pursue an efficient review and registration pathway that leverages smaller-sample domestic trials alongside global data packages. This approach is designed to reduce costs associated with standalone research and development efforts in Japan while expediting the potential marketing approval process for equecabtagene autoleucel.
"Following the CTN clearance for Eque-cel in late-line r/r MM in Japan in October 2025, this subsequent clearance for second- or third-line indications further demonstrates the high recognition by Japan's PMDA of the product's clinical value and the strength of China's clinical data," said Jinhua Zhang, founder, chairperson and CEO of IASO Biotechnology (搜索).
Addressing Unmet Medical Need in Multiple Myeloma
Multiple myeloma represents the second most common hematologic malignancy worldwide. According to Globocan data, the global incidence rate of multiple myeloma in 2022 was 1.8 per 100,000 population, with a 5-year prevalence rate of 6.8 per 100,000. In Japan specifically, the incidence rate was 1.5 per 100,000 population in 2022, with a 5-year prevalence rate of 14.9 per 100,000.
Despite progress in current anti-myeloma treatments, multiple myeloma remains largely incurable with multiple relapses and tendency to develop refractoriness to several drug classes, presenting a major therapeutic challenge. This creates an unmet need for new treatment options beyond current anti-myeloma therapies capable of achieving deep and durable responses.
Global Development Strategy
The approval supports IASO Bio's broader strategy to advance global development through multi-regional clinical trial pathways. Zhang emphasized the company's commitment to accelerating clinical trial progress and benefiting more patients in Japan and worldwide with the China-developed CAR-T therapy.
Equecabtagene autoleucel has already received approval in China for treating relapsed or refractory multiple myeloma in patients who have progressed after at least three prior lines of therapy. The therapy is also being developed for earlier treatment lines and autoimmune diseases, including myasthenia gravis, multiple sclerosis, and systemic lupus erythematosus.