hNPC01 / Hopstem Biotech - LARVOL DELTA

Hopstem Biotechnology announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application and Fast Track designations for hNPC01…for the treatment of chronic motor dysfunction due to Hemorrhagic Stroke and Traumatic Brain Injury (Business Insider) - "The encouraging safety and efficacy findings from Hopstem’s Phase I clinical studies of hNPC01 in patients with chronic motor dysfunction due to ischemic stroke include: At 12 months, patients in the target subgroup achieved a mean improvement of 16 points on the Fugl-Meyer Motor Scale (FMMS) , with nearly 80% achieving clinically significant improvement (≥10-point gain)."

Fast track • IND • P1 data • CNS Disorders • Ischemic stroke

Hopstem Biotechnology…has secured the first Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA for its proprietary human forebrain neural progenitor cell injection, hNPC01 (Yahoo Finance) - "The candidate has displayed potential for treating persistent motor dysfunction due to basal ganglia ischemic stroke in phase I clinical study....The agency will grant a dedicated RMAT comprehensive consultation meeting to accelerate the formulation of streamlined clinical development and commercial manufacturing roadmaps, enabling Hopstem to efficiently compile critical evidence for regulatory approval. This milestone establishes hNPC01 as the world’s first forebrain neural progenitor cell therapy to receive FDA RMAT designation."

FDA event • Ischemic stroke

The Phase 1 Study of induced Pluripotent Stem Cell Derived Forebrain Neural Progenitor Cells (hNPC01) Treating Chronic Ischemic Stroke (ISC 2026) - "A total of 23 participants 6 to 60 months post ischemic stroke with modest to severe motor dysfunctions were enrolled in 3 dose cohorts, received either 1.5×107, 3.0×107, or 6.0×107 hNPC01 cells via intracerebral stereotactic injection, with 1-2 months of cyclosporine immunosuppression.To date, except 3 lost-to-follows, 20 participants have reached 12-months observation endpoint, and 5 of them had their 18-months visit with further motor improvement. In addition, 100% of the 4 participants in IIT study with language deficits or dysarthria improved 1 point in their corresponding section of NIHSS. The imaging evidence supported graft survival (infarcts filling, increased new tissue volume, white matter regeneration, and enhanced PET metabolism) and neurological functional reconstruction, up to 12 or 18-months in responders.The finding that hNPC01 induced continuous FMMS improvements through 12 months or 18 months without reaching a plateau, is consistent..."

P1 data • Cardiovascular • Ischemic stroke

Phase IIa Clinical Study of Human Neural Precursor Cell (hNPC) Transplantation for Ischemic Brain Injury ——A Phase IIa Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of hNPC01 Injection Derived from Human Induced Pluripotent Stem Cells in Improving Hemiplegic Sequelae of Ischemic Stroke (ChiCTR) - P2 | N=21 | Not yet recruiting | Sponsor: Neurosurgery Department of Seventh Medical Center, Senior Department of Neurosurgery, the First Medical Center of PLA Gene; PLA General Hospital

New P2 trial • Cardiovascular • CNS Disorders • Ischemic stroke • Transplantation • Vascular Neurology

hNPC01-ISCHEMIC: Clinical Trial of Human Neural Precursor Cell (hNPC) Transplantation for Ischemic Brain Injury(——A Clinical Trial Evaluating the Safety, Tolerability, and Efficacy of hNPC01 Injection Derived from Human Induced Pluripotent Stem Cells in Improving Hemiplegic Sequelae of Ischemic Stroke)(Phase I) (ChiCTR) - P1 | N=9 | Completed | Sponsor: Neurosurgery Department of Seventh Medical Center, Senior Department of Neurosurgery, the First Medical Center of PLA General Hospital; PLA General Ho

New P1 trial • Cardiovascular • CNS Disorders • Ischemic stroke • Transplantation • Vascular Neurology

The Preclinical and First-in-Human Study of iPSC-Derived Human Forebrain Neural Progenitor Cell Injection-hNPC01 in Treating Chronic Ischemic Stroke (ASGCT 2024) - "The current observation of first dose cohorts showed good safety and tolerance of hNPC01, as well as sign of motor function improvement in volunteer subjects with ischemic stroke onset 17 to 40 months prior to the enrollment. It is expected that the 12 month safety observation and exploration of evaluation tools will provide more valuable information for the future development of iPSC cell therapy for stroke or traumatic brain injury caused brain injury patients with chronic symptoms."

Late-breaking abstract • P1 data • Preclinical • Cardiovascular • CNS Disorders • Ischemic stroke • Oncology • Vascular Neurology

The Preclinical and First-in-Human Study of iPSC-Derived Human Forebrain Neural Progenitor Cell Injection-hNPC01 in Treating Chronic Ischemic Stroke (ASGCT 2024) - "The current observation of first dose cohorts showed good safety and tolerance of hNPC01, as well as sign of motor function improvement in volunteer subjects with ischemic stroke onset 17 to 40 months prior to the enrollment. It is expected that the 12 month safety observation and exploration of evaluation tools will provide more valuable information for the future development of iPSC cell therapy for stroke or traumatic brain injury caused brain injury patients with chronic symptoms."

P1 data • Preclinical • Cardiovascular • CNS Disorders • Ischemic stroke • Oncology • Vascular Neurology

A Safety and Tolerability Study of Human Forebrain Neural Progenitor Cells Injection (hNPC01) in Subjects With Chronic Ischemic Stroke (clinicaltrials.gov) - P1 | N=21 | Recruiting | Sponsor: Hopstem Biotechnology Inc.

New P1 trial • Cardiovascular • Ischemic stroke