Hemostemix applies for vascular dementia study approval

Executive Summary

• Hemostemix Inc. filed a 102‑page Institutional Review Board (IRB) application to commence a Phase 1, multicenter, open‑label study of its autologous stem cell therapy ACP‑01 for vascular cognitive impairment and dementia (VCID/VaD).
• The trial will enroll 20–100 adults (age 50‑100), delivering ACP‑01 intrathecally with an optional second dose at three months and follow‑up to 12 months.
• Primary endpoints focus on safety and feasibility; secondary endpoints include cognitive, EEG, MRI, and quality‑of‑life measures, marking the first CNS application of ACP‑01.

Key Details

• Study Title: Phase 1 Study – Treatment of Vascular Cognitive Impairment and Dementia with Angiogenic Cell Precursors (ACP‑01).
• Objective: Evaluate safety, feasibility, and preliminary efficacy of intrathecal ACP‑01 in patients with VCID or vascular dementia.
• Design: Multicenter, open‑label, non‑randomized; 20–100 participants; ages 50‑100.
• Intervention: Autologous ACP‑01 administered via lumbar puncture (intrathecal); optional second dose at month 3.
• Follow‑up Schedule: Assessments at 3, 6, and 12 months post‑treatment.
• Primary Endpoints: Safety (incidence & tolerability of adverse events) and feasibility of intrathecal delivery.
• Secondary Endpoints:
• Cognitive function (MoCA, MCI screen).
• Executive function & quality of life (SF‑36, Barthel Index).
• EEG‑based brain network analytic (BNA) patterns.
• MRI‑assessed brain perfusion and volume changes.
• Exploratory Endpoints: Biomarkers (IL‑6, CRP, neurofilament light chain), correlation of EEG & cognitive improvements, dose‑response between one vs. two ACP‑01 administrations.
• Scientific Rationale: ACP‑01 expresses CXCR4 for homing to ischemic brain tissue; releases VEGF, angiogenin, IL‑8, and activates NF‑κB pathways linked to synaptic plasticity and memory consolidation.
• Delivery Mechanism: Direct intrathecal injection bypasses the blood‑brain barrier, enhancing CNS exposure compared with prior intravenous stem‑cell trials.
• Safety Oversight: Procedures performed by licensed neurosurgeons in outpatient settings; monitored by an independent Data Safety Monitoring Board (DSMB).
• Executive Quote: “Filing our vascular dementia Phase 1 protocol represents a major step forward… This next trial extends our platform to the brain where vascular repair may restore blood flow, memory and cognition itself,” – Thomas Smeenk, President & CEO.

Notable Quotes

"We have already demonstrated the safety and efficacy of ACP‑01 in the heart and limb. This next trial extends our platform to the brain where vascular repair may restore blood flow, memory and cognition itself." – Thomas Smeek, President & CEO, Hemostemix Inc.