– hNPC01 is the FIRST iPSC cell therapy product received FDA IND clearance for stroke, and the FIRST pluripotent stem cell-derived forebrain progenitor cell product entering registered clinical trial in global.

– Company plans to initiate the US Phase I/IIa clinical trial in the first quarter of 2025.

– The on-going registered phase I trial of hNPC01 in same indication in China has suggested preliminary good safety profile and sign of efficacy in 4 chronic stroke patients.

Hopstem Biotechnology, an innovative biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies for CNS diseases, today announced that the Company has been notified by the U.S. Food and Drug Administration (FDA) that the Company’s Phase 1/2a clinical trial may proceed to assess hNPC01 in patients with chronic ischemic stroke who have stable hemiplegia sequalae. This represents the first indication FDA Investigational New Drug (IND) application clearance for hNPC01, also a first-in-global pluripotent stem cell-derived forebrain progenitor cell product entering registered clinical trial.

There are currently more than 100 million chronic stroke patients in global suffering from hemiplegia, and an increase of more than 12 million each year, with 22% experienced stroke at age of 19-49, and 67% at age under 70 (WSO Global Stroke Fact Sheet 2022). Tissue plasminogen activator for ischemic stroke and neuroprotective drugs require to be administrated in a short time window at acute stage of stroke to reduce neural injury. However, there is no effective treatment to chronic stroke patients after 6 months of stroke onset and continuous rehabilitation, and it is hard for traditional drug therapy to target hemiplegia symptoms at this stage by mechanism of action.

The human forebrain progenitor cell injection hNPC01 manufactured from GMP induced-pluripotent stem cell (iPSC) line and cell bank, is an allogeneic, off-the-shelf cell therapy product that is composed of more than 95% cortical fate progenitor cells and neurons, and less than 5% same lineage brain cells. hNPC01 has shown to be able to differentiate into all six layers of functional cortical neurons and glia cells both in vitro and in vivo (animal models), and these progeny neurons were able to form new circuitry with animal’s different brain regions, and to promote motor function recovery after stroke in animal ischemic stroke models. In an on-going registered phase I trial in same indication in China, hNPC01 has demonstrated preliminary good safety profile and sign of efficacy in 4 chronic stroke patients.

The single-center Phase 1/2a clinical trial in US is designed to evaluate the safety, tolerability, and clinical response of hNPC01 in ischemic stroke patients with moderate to severe hemiplegia motor symptoms who experienced stroke 6 to 60 months ago. This is a randomized controlled dose-escalation study in which subjects will receive a single dose of hNPC01 injection via intracerebral injection surgery. The Company plans to initiate the trial in the first quarter of 2025, with initial data expected by the end of 2025.