Hemostemix Submits FDA Pre-IND Application for Multi-Indication Basket Trial of Autologous Stem Cell Therapy ACP-01
核心洞察
Hemostemix (搜索) filed FDA Pre-IND Application 1517 for a basket protocol Phase I clinical trial evaluating ACP-01 (VesCell (搜索)™) across seven ischemic and vascular conditions including vascular dementia (搜索), peripheral arterial disease (搜索), and heart failure.
The company plans to integrate real-world evidence from Florida's SB 1768 program, where ACP-01 is legally available, to support expedited regulatory pathways including potential RMAT designation.
ACP-01 has demonstrated safety in 498 treatments to date, with published results showing 0% mortality and 83% wound healing rates in chronic limb-threatening ischemia (搜索) patients compared to the typical 50% five-year mortality rate.
Hemostemix (搜索) Inc. has filed FDA Pre-IND Application 1517, marking a significant regulatory milestone for the autologous stem cell therapy company as it seeks to advance its lead therapy ACP-01 (VesCell (搜索)™) across multiple ischemic and vascular indications through a comprehensive basket protocol approach.
The Calgary-based company submitted the application on September 23, 2025, proposing a Phase I clinical trial designed to evaluate safety, feasibility, and early efficacy signals of ACP-01 in seven distinct patient populations: vascular dementia (搜索) (earliest medically validated subtype), peripheral arterial disease (搜索) (PAD), angina (搜索), ischemic cardiomyopathy (搜索), non-ischemic dilated cardiomyopathy (搜索), congestive heart failure (搜索) (CHF), and total body ischemia.
Strategic Regulatory Approach
The basket protocol represents a strategic shift that could accelerate ACP-01's development timeline by studying multiple indications under one unified trial design. This approach aims to maximize the breadth of ACP-01's therapeutic potential in ischemia-related diseases while generating pivotal safety and exploratory efficacy data across cardiology, neurology, and vascular medicine.
"This FDA filing is a transformational milestone for Hemostemix (搜索)," stated Thomas Smeenk, CEO of Hemostemix. "By proposing a basket trial that spans cardiology, vascular disease, and vascular dementia (搜索), we are unlocking ACP-01's full potential as a regenerative therapy."
Real-World Evidence Integration
A key component of Hemostemix (搜索)'s regulatory strategy involves integrating systematically collected real-world evidence from Florida, where ACP-01 is legally available under SB 1768. Patients treated in Florida will be followed prospectively and documented as if they were in a Phase I open-label clinical trial, with standardized endpoints and monitoring. This dataset will be positioned as supportive evidence for IND advancement and expedited regulatory designations such as RMAT (Regenerative Medicine Advanced Therapy).
Clinical Track Record
Hemostemix (搜索) has established a substantial clinical foundation for ACP-01, having completed seven clinical studies involving 318 subjects with results published in 11 peer-reviewed publications. The company reports 498 safe treatments completed to date, with clinically relevant efficacy demonstrated in no-option patients with severe angina (搜索), ischemic and non-ischemic dilated cardiomyopathy (搜索), and related co-morbidities.
Particularly notable are the results from the company's Phase II clinical trial for chronic limb-threatening ischemia (搜索) (CLTI), published in the Journal of Biomedical Research & Environmental Science. As reported to the 41st meeting of vascular surgeons by UBC and University of Toronto researchers, the treatment achieved 0% mortality, cessation of pain, and wound healing in 83% of patients followed for up to 4.5 years as a midpoint result. This compares favorably to the typical five-year mortality rate of 50% in the CLTI patient population.
Market Implications
The successful advancement of this basket protocol could position Hemostemix (搜索) to pursue multiple billion-dollar markets in parallel, significantly expanding the company's addressable market. Positive FDA feedback could shorten timelines to pivotal trials and eventual commercialization, potentially positioning Hemostemix as a first-mover in autologous stem cell therapies for ischemia and vascular dementia (搜索).
The filing represents Hemostemix (搜索)'s formal engagement with the FDA to align on the regulatory path for ACP-01 in multiple high-unmet-need conditions, with the potential for expedited pathways including Fast Track or Breakthrough Therapy eligibility.
Founded in 2003 and recognized as a World Economic Forum Technology Pioneer Award winner, Hemostemix (搜索) has developed and patented its autologous blood-based stem cell therapy platform. A recent peer-reviewed article in Cells (June 29, 2025) provides scientific foundation for how ACP-01 and NCP-01 may enhance brain-computer interface performance by reducing inflammation, fostering angiogenesis and synaptic plasticity, potentially extending implant longevity.