RESEARCH TRIANGLE PARK, N.C. — Humacyte, an innovator in biotechnology and regenerative medicine, announced today that the U.S. Food and Drug Administration (FDA) has granted HUMACYL®, its investigational human acellular vessel (HAV), the Regenerative Medicine Advanced Therapy (RMAT) designation. This designation means that the FDA will help facilitate the efficient development and expedited review of the HAV for vascular access to patients in need of life-sustaining hemodialysis. Carved from the model of other FDA expedited programs such as Breakthrough Therapy, the RMAT designation is a new expedited program for regenerative medicine products.

Since receiving the FDA’s Fast Track Designation in 2014, Humacyte has continued to develop clinical evidence indicating that HUMACYL has the potential to address unmet medical needs for hemodialysis patients by decreasing vascular access complications, that in turn may result in more favorable morbidity and mortality outcomes. More than 400,000 people with end-stage renal disease (ESRD) undergo hemodialysis procedures to eliminate deadly toxins from blood when their kidneys fail. Alternative methods are needed to establish durable long-term vascular access for dialysis with infection rates, and other complications as low as possible, to help patients obtain better health care.

Humacyte is currently undertaking a Phase III clinical trial of HUMACYL compared to an expanded polytetrafluoroethylene (ePTFE) graft in 350 patients with kidney failure who are not candidates for fistula placement. The trial involves approximately 35 sites in the U.S., Europe and Israel. The Phase III study’s primary objective is to compare the durable use (secondary patency) of the HAV with that of the ePTFE graft during its use as a conduit for hemodialysis. The company published Phase II results in May 2016, showing HUMACYL may have the potential for long-term use and safety in patients suffering from ESRD who require renal replacement therapy and are not candidates for fistula.

“Being one of the first companies to receive the Regenerative Medicine Advanced Therapy Designation from the FDA is an honor and a testament to the significant potential for HUMACYL to address a great unmet medical need for patients who are undergoing dialysis,” said Carrie S. Cox, Chairman and Chief Executive Officer of Humacyte. “We look forward to continuing to work closely with the FDA to facilitate the development and expedited review of HUMACYL with the goal of bringing this novel vascular access product to patients requiring hemodialysis. We applaud the efforts of the FDA in expanding support to advanced therapeutic products through this great new program.”

The RMAT designation provides for additional resources from the FDA, in an effort to expedite the review pathway. The designation makes a product eligible for the same actions to expedite the development and review of a marketing application that are available to drugs that receive breakthrough therapy designation, including timely advice and interactive communications with FDA, as well as proactive and collaborative involvement by senior FDA managers and experienced review and regulatory health project management staff. A product designated as an RMAT also may be eligible for other FDA expedited programs, such as Priority Review. FDA also may conduct a rolling review of products in its expedited programs, reviewing portions of a marketing application before the complete application is submitted.

The FDA’s RMAT designation was a primary component of the 21st Century Cures Act, signed into law by former President Barack Obama in December 2016 and championed by former Vice President Joe Biden. According to this legislation, eligible drugs include regenerative medicine therapy, further defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or a combination product using such therapies or products. Further, eligible drugs are those intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and present preliminary clinical evidence indicating that the drug has the potential to address unmet medical needs for such disease or condition. Humacyte was granted the RMAT designation through HUMACYL meeting these criteria for the creation of vascular access in performing hemodialysis.

Humacyte is also developing and seeking regulatory approval for additional HAV applications, and recently announced the commencement of a U.S. Phase II clinical trial of the bioengineered vessel as a bypass graft in patients with peripheral arterial disease (PAD).