Note: This article does not constitute any investment advice or recommendations; please refer to official/company announcements for authoritative information. This article is solely for the purpose of introducing drugs related to medical and health care, and does not recommend treatment plans (if involved), nor does it represent the platform's position.Authorization is required for any article reprint.
On June 18, Hopstem Biotechnology Inc. (Hopstem Biotechnology) announced that the U.S. Food and Drug Administration (FDA) has officially approved a new Investigational New Drug (IND) application for its self-developed iPSC-derived allogeneic universal forebrain neural progenitor cell injection, hNPC01, for the indication of motor dysfunction following hemorrhagic stroke (cerebral hemorrhage).
This is currently the world’s first Investigational New Drug (IND) application for a pluripotent stem cell-derived product targeting the sequelae phase of intracerebral hemorrhage., which is also an expansion to a new indication based on the positive Phase I clinical data of hNPC01 in the treatment of hemiplegic sequelae of ischemic stroke (cerebral infarction).
The Phase I clinical study in China of hNPC01 for the treatment of chronic motor dysfunction caused by 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 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 efficacy.Based on the preliminary safety and efficacy data from 20 clinical cases of ischemic stroke, and given that patients with hemorrhagic and ischemic stroke share similar pathologies and equally urgent needs during the chronic motor impairment phase (from 6 months to over 1 year post-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.With the successful IND approval for the hemorrhagic stroke indication, hNPC01 has become the only forebrain neural cell product globally to receive U.S. FDA clinical trial approval for both major subtypes of stroke—ischemic and hemorrhagic. Meanwhile, this indication expansion strategy facilitates more efficient acceleration of clinical development for hemorrhagic stroke by building upon previously explored therapeutic regimens, patient populations, and dosing in ischemic stroke trials, thereby striving to promptly meet the urgent need of patients with neurological injury for affordable innovative cell therapies.References:
Public Information
Scan the QR code to join the groupMastering CAR-T therapy, stem cell therapy, and geneTherapy,Comprehensive Information on Oncolytic Viruses and Nucleic Acid Therapeutics
