FDA Granted Hopstem’s hNPC01 for Chronic Ischemic Stroke Approval to Enter Phase 2/3 Adaptive Pivotal Clinical Trial with Bridging Study
HOUSTON, Jan. 21, 2026 (GLOBE NEWSWIRE) -- Hopstem Biotechnology Inc. (hereinafter referred to as "Hopstem"), a company dedicated to the development of induced pluripotent stem cell (iPSC)-derived allogeneic neural cell therapy products, announced today that it has successfully convened an End-of-Phase 1 (EOP1) meeting with the U.S. Food and Drug Administration (FDA) recently. The two parties reached a high level of consensus on the accelerated clinical development pathway of the core product, hNPC01 Injection, for the treatment of chronic motor dysfunction due to ischemic stroke.
Based on the positive data from the Phase 1 clinical trials in China, FDA has approved Hopstem to initiate a dose-bridging, randomized-controlled, double-blinded, Phase 2 clinical trial in the United States. Leveraging the rolling meeting mechanism conferred by the interim analysis and Fast Track Designation[1], Hopstem can further determine the key design elements of the Phase 3 clinical trial, such as sample size, in collaboration with the FDA, and then directly expand the Phase 2 trial into the final pivotal study, thus implementing a combined Phase 2/3 adaptive confirmatory clinical development program.
The approval of this accelerated pathway not only demonstrates the FDA’s full recognition of the quality, safety, and efficacy of the Phase 1 clinical data of hNPC01 in China, but also will significantly shorten the product’s time-to-market, enabling chronic ischemic stroke patients worldwide to benefit from this innovative cell therapy.
Stroke is a major neurological disorder with high incidence, disability, and mortality rates globally, imposing a heavy burden on patients’ families and society at large. Clinical data show that approximately 50%–70% of stroke patients suffer from varying degrees of hemiplegia sequelae, leading to severe consequences such as motor dysfunction and loss of daily-living abilities. At present, there remains a huge unmet medical need in the treatment of post-stroke chronic dysfunctions.
Developed by Hopstem, hNPC01 Injection is currently the most advanced iPSC allogeneic forebrain neural progenitor cell product in clinic development, with IND approved by NMPA in June 2023[2] and FDA in March 2024[3]. Through stereotactic intracerebral transplantation, hNPC01 can further differentiate into functional neurons and glial cells, replace damaged neural cells, and promote neural circuit reconstruction, thereby providing an innovative approach and direction for the treatment of chronic dysfunctions after stroke and other neural injury diseases[4].
The 12-month results of the Phase 1 clinical trials of hNPC01 treating chronic ischemic stroke in China, previously released by Hopstem, have demonstrated the product’s manageable safety profile and excellent efficacy[5]. A total of 23 chronic ischemic stroke patients with stroke onset ranging from 6 months to 5 years were enrolled in the study. To date, the participants under continuous follow-ups have completed 18 months of safety follow-up, with some patients followed up for up to 2 years. No dose-limiting toxicity, abnormal overgrowth, or tumorigenesis has been observed. In addition, the 18-month follow-up data show that 90% of the followed-up patients continued to achieve further sustained improvements in motor function compared with the 12-month visit; for patients with 2-year follow-up, the efficacy has entered a stable plateau phase without any sign of efficacy decline.
Dr. Shuning Zhang, Vice President of Medical Affairs at Hopstem, stated: "The smooth progress and outcomes of this meeting with FDA have exceeded our expectations. This accelerated development pathway granted by the FDA not only allows hNPC01 to bypass redundant Phase 1 clinical development in the United States, but also saves the waiting time and redundant enrollment between the two phases through the seamless adaptive design of Phase 2/3 trials, which can significantly improve the efficiency of clinical development. The FDA’s endorsement of core protocol elements, including patient population, dose selection, clinical endpoint settings, statistical plan, without raising any safety concerns, has cleared critical obstacles for the efficient advancement of subsequent international multicenter studies.
The adoption of adaptive design enables the dynamic accumulation of clinical data and protocol optimization, maximizing R&D efficiency while ensuring the scientific validity of the study. To our knowledge, this is the first case in the pluripotent stem cell field where U.S. Phase 1 clinical trials are waived based on Chinese Phase 1 data, and also the first China-developed pluripotent stem cell product approved overseas to bridge into pivotal clinical trial. This not only marks a significant breakthrough in the clinical development of hNPC01, but also provides highly valuable practical reference for the international clinical pathway of China’s innovative cell therapy products. Our clinical team will strictly adhere to international standards, efficiently advance the conduct of the study, focus on addressing the unmet needs of patients, and verify the product’s value with solid clinical data."
As the world’s first iPSC-derived forebrain neural progenitor cell product with IND approvals in both China and the United States, hNPC01 has evolved from being the first neural cell product in China to obtain IND approval, accumulated the largest number of clinical cases with the longest observation, to now gaining FDA’s recognition for accelerated development based on the rigorous, safe, and effective Phase 1 data from China, thus maintaining its leading position in China’s iPSC neural cell therapy track.
Hopstem’s successful experience not only validates the clinical value and international competitiveness of in-China-developed iPSC-derived cell therapy products, but also conveys the scientific rigor and reliability of China’s innovative cell therapy clinical research to global regulatory authorities and markets. It serves as a paradigm of collaboration between regulatory agencies and biotechnology companies to address major unmet medical needs for global neural injury patients.
Contact:
Tel: +1-732-822-1963
Email: BD@hopstem.com
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