Hemostemix gets FDA support for basket protocol

Executive Summary

• Hemostemix Inc. successfully completed its pre-IND meeting with the FDA regarding its investigational therapy, ACP-01.
• The FDA provided positive guidance, accepting the company's proposed "basket protocol" approach for evaluating ACP-01 across multiple ischemic and vascular-related conditions, subject to specific design criteria.
• The company also received FDA guidance on its plan to collect Real-World Evidence (RWE) from jurisdictions including Florida, Canada, and The Bahamas, supporting the upcoming IND submission.

Key Details

• Pre-IND Meeting Outcome: The company met with the FDA to discuss the development plan for ACP-01.
• Basket Protocol Approval: The FDA indicated that the basket protocol approach is acceptable. This design allows for the evaluation of ACP-01 across multiple conditions sharing common underlying pathophysiology.
• Conditions Targeted: The basket protocol covers multiple ischemic and vascular-related conditions.
• FDA Conditions: Acceptance is subject to the inclusion of clearly defined eligibility criteria, appropriate handling of patient heterogeneity, and rigorous clinical and statistical design.
• Real-World Evidence (RWE) Guidance: The FDA supported the company's plan to capture RWE for ACP-01 in Florida, Canada, and The Bahamas.
• RWE Standards: The guidance confirms that the proposed data collection structure is consistent with FDA publications regarding patient privacy, safety, and data integrity.
• Next Steps: Hemostemix is incorporating FDA feedback into its Investigational New Drug (IND) submission, data models, and statistical analyses.
• IND Submission Components: The upcoming IND filing will include product comparison validation studies.
• Company Background: Hemostemix is an autologous stem cell therapy platform company founded in 2003. It has developed VesCell (ACP-01), a blood-based stem cell therapy.
• Clinical History: The company has completed seven clinical studies involving 318 subjects, with results published in 11 peer-reviewed publications.
• Previous Trial Data: A Phase II clinical trial for chronic limb-threatening ischemia (CLTI) was completed. Reported results included a 0% mortality rate (compared to a 50% five-year mortality rate in the general CLTI population), cessation of pain, and wound healing in 83% of patients followed for up to 4.5 years.

Notable Quotes

• "The FDA's guidance supports Hemostemix's basket protocol strategy, and collection of RWE, enabling the company to advance across multiple ischemic diseases," stated Thomas Smeenk, chief executive officer. "We are incorporating the FDA's feedback in our IND submission, data models and statistical analyses of patients treated with ACP-01."