Hemostemix files FDA trial for ACP-01 stem therapy

Executive Summary

• Hemostemix Inc. has filed FDA Pre-IND Application 1517 seeking regulatory feedback on a proposed basket protocol for Phase I clinical trials of its lead therapy, ACP-01 (VesCell).
• The basket protocol aims to evaluate the safety, feasibility, and early efficacy signals of ACP-01 across multiple ischemic and vascular indications simultaneously, including vascular dementia, peripheral arterial disease (PAD), angina, and various forms of heart failure.
• The company plans to integrate real-world evidence (RWE) from Florida (under SB 1768) into the trial design to support expedited regulatory designations such as Regenerative Medicine Advanced Therapy (RMAT), fast-track, or breakthrough therapy status.

Key Details

• Regulatory Filing: Submission of FDA Pre-IND Application 1517 to initiate dialogue on the regulatory pathway for ACP-01.
• Proposed Trial Design: A unified basket protocol for a Phase I clinical trial designed to study multiple indications in parallel to accelerate development and maximize therapeutic potential.
• Target Indications:
  • Vascular dementia (described as the earliest medically validated subtype).
  • Peripheral arterial disease (PAD).
  • Angina.
  • Ischemic cardiomyopathy.
  • Non-ischemic dilated cardiomyopathy.
  • Congestive heart failure (CHF).
  • Total body ischemia.
• Real-World Evidence (RWE) Strategy: Hemostemix intends to present a plan to integrate systematically collected RWE from Florida, where ACP-01 is legally available under SB 1768. Patients will be followed prospectively with standardized endpoints and monitoring, treated as if in a Phase I open-label trial.
• Strategic Goals:
  • Secure FDA guidance on accelerated development pathways.
  • Pursue expedited designations: RMAT, Fast-Track, or Breakthrough Therapy.
  • Expand addressable market into multiple billion-dollar markets in cardiology, neurology, and vascular medicine.
• Company Background & Data:
  • 11 peer-reviewed publications demonstrating safety and efficacy.
  • 498 safe treatments completed to date.
  • Completed seven clinical studies involving 318 subjects.
  • Recent peer-reviewed article in Cells (June 29, 2025) discussing potential benefits for brain-computer interface performance via inflammation reduction and angiogenesis.
  • Phase II trial for chronic limb-threatening ischemia (CLTI) completed; results published in the Journal of Biomedical Research & Environmental Science.
  • Reported 0% mortality, cessation of pain, and 83% wound healing in CLTI patients followed for up to 4.5 years (vs. 50% five-year mortality in general population).

Notable Quotes

• "This FDA filing is a transformational milestone for Hemostemix. By proposing a basket trial that spans cardiology, vascular disease and vascular dementia, we are unlocking ACP-01's full potential as a regenerative therapy. Layering in real-world evidence from Florida strengthens our case for expedited pathways and positions Hemostemix to deliver shareholder value through accelerated regulatory approval." — Thomas Smeenk, Chief Executive Officer