Hode Biologics Receives FDA IND Approval for hNPC01, the World’s First iPSC-Derived Cell Therapy for Motor Dysfunction Following Hemorrhagic Stroke

On June 18, Hopstem Biotechnology Inc. (Hopstem Biotechnology) announced that the U.S. Food and Drug Administration (FDA) has officially approved its Investigational New Drug (IND) application for hNPC01, an allogeneic universal forebrain neural precursor cell injection derived from induced pluripotent stem cells (iPSCs) independently developed by the company, for a new indication: motor dysfunction following hemorrhagic stroke (cerebral hemorrhage). This marks the first IND globally for a pluripotent stem cell-derived product targeting the sequelae phase of cerebral hemorrhage, and represents an expansion of indications based on positive Phase I clinical data of hNPC01 in treating hemiplegia sequelae after ischemic stroke (cerebral infarction).

Hemorrhagic stroke accounts for approximately 30% of all stroke types; however, due to the ineligibility for thrombolysis and thrombectomy, as well as additional neural injury caused by hemorrhage, about 70% of patients are left with long-term sequelae one year after onset, a prognosis that has not shown significant improvement over the past 15 years.^【1】. According to a systematic review in The Lancet Regional Health^【1】, the global annual incidence of hemorrhagic stroke is approximately 24.6 cases per 100,000 population, with around 3.41 million new cases each year. There are currently about 20.6 million prevalent cases, imposing a substantial burden of approximately 68.57 million disability-adjusted life years (DALYs) worldwide. Patients in the chronic phase of intracerebral hemorrhage commonly suffer from persistent unilateral motor dysfunction. Currently, there are no effective neurorestorative therapies available in clinical practice, representing a significant unmet medical need.

The Phase I clinical study in China of hNPC01 for the treatment of chronic motor dysfunction resulting from ischemic stroke has achieved a maximum follow-up duration of 2.5 years. No product-related adverse events other than immune rejection were observed, and no functional abnormalities compared to baseline were detected. At the 12-month endpoint, the target subgroup showed an average improvement of 16 points on the Fugl-Meyer Motor Scale (FMMS), with nearly 80% of patients achieving a clinically significant improvement of more than 10 points on the FMMS. By 18 months, over 92% of patients in the target subgroup attained clinically significant improvement, and 90% of the followed-up patients demonstrated further sustained improvement in motor function compared to the 12-month assessment. Two-year follow-up data indicated that therapeutic efficacy had entered a stable plateau phase, with no evidence of waning effects. Based on the preliminary safety and efficacy data from 20 cases of ischemic stroke, and recognizing that patients with hemorrhagic and ischemic stroke share similar pathologies and equally urgent needs during the chronic motor dysfunction phase (occurring six months or even more than one year after onset), Hopstem Biotechnology submitted an Investigational New Drug (IND) application to the FDA based on non-clinical and clinical data from ischemic stroke studies, which received direct approval.

Following the successful IND approval for hemorrhagic stroke indications, hNPC01 has become the only therapy globally addressing the two major subtypes of stroke—ischemic stroke^【5】and hemorrhagic stroke—both are forebrain neural cell products that have received U.S. FDA approval for clinical trials. Meanwhile, this indication expansion strategy helps to more efficiently accelerate the clinical development of the hemorrhagic stroke indication, building on the already explored treatment regimens, patient populations, and dosing for ischemic stroke in clinical settings, thereby striving to promptly meet the urgent needs of patients with neurological injuries for affordable innovative cell therapies.

References:

1. Incidence, case fatality, and functional outcome of intracerebral haemorrhage, according to age, sex, and country income level: a systematic review and meta-analysis 

2. [Breakthrough Progress in iPSC Cell Therapy] Hopstem Biotechnology’s hNPC01 Injection Achieves 12-Month Follow-Up Endpoint in Phase I Clinical Study for Hemiplegia Following Ischemic Stroke, Demonstrating Significant Clinical Improvement

3. Xiao H, et al. Forebrain neural progenitors effectively integrate into host brain circuits and improve neural function after ischemic stroke. Nature Communications. 2025; 16:5132. 

4. Important Mechanisms of hNPC01 Neural Progenitor Cell Transplantation in a Rat Stroke Model Published in Nature Communications 

5. World’s First: FDA Grants Implicit Approval for Clinical Trials of Hopstem Biotechnology’s iPSC-Derived hNPC01 Injection for the Treatment of Hemiplegia Sequelae Following Ischemic Stroke

6. Hopstem Biotechnology’s hNPC01 Injection Receives FDA Fast Track Designation, Accelerating Breakthroughs in the Treatment of Neurological Brain Injury Disorders

7. Hopstem Biotechnology’s hNPC01 Pipeline for the Treatment of Sequelae of Ischemic Stroke Receives FDA Approval to Enter Adaptive Phase 2/3 Confirmatory Clinical Trials 

8. Hopstem Biotechnology’s iPSC-derived hNPC01 Injection Becomes the World’s First Forebrain Neural Progenitor Cell Product to Receive FDA Regenerative Medicine Advanced Therapy (RMAT) Designation

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