The scoop

Humacyte is a regenerative medicine company focused on vascular therapies. Yale University’s Laura Niklason cofounded Humacyte in 2004 to develop and commercialize technology to grow bioengineered blood vessels—and potentially other human tissues—in the laboratory.

Humacyte CEO Carrie Cox envisages a plethora of uses for the technology, but, in the near term, Humacyte’s main focus is on developing a bioengineered blood vessel for use as a conduit during hemodialysis. Patients with end-stage renal disease need to undergo dialysis multiple times a week to survive. The process needs a conduit to access the blood.

If a fistula cannot be used, an artificial vessel made of polytetrafluoroethylene (PTFE)—a material better known as Teflon—is often inserted to connect the artery and vein. This facilitates access to the blood but carries a risk of infection and clotting. These adverse events pose a risk to patients and lead to the failure and abandonment of grafts, driving up the cost of hemodialysis.

Phase 2 data suggest Humacyte’s technology can address some of these shortcomings. In two studies that enrolled 60 patients, Humacyte saw one infection over 82 patient-years of follow-up. While clotting occurred at a rate comparable to that seen in PTFE, in most cases it was possible to reopen the graft. After one year, 89% of grafts were accessible, more than the 55% to 65% seen in PTFE studies. This suggests the technology has economic and clinical benefits, although data from a trial with a control arm are needed to confirm the improvement over PTFE.

Humacyte saw no evidence of immune rejection in phase 2 trials. And, supported by early data, its ambitions extend beyond simply avoiding rejection.

“It appears that the tissue becomes incorporated into the patient and is recognized as their own tissue and becomes self. And, amazingly, there is evidence that there is healing taking place in the patient’s body with these tissues,” Cox said.

What makes Humacyte fierce

The long-term goals for Humacyte and the investors who backed a $150 million Series B round in 2015 go way beyond the hemodialysis program. The prospects for that product, while important to Humacyte and potentially hemodialysis patients, are dwarfed by the possible impact of the process behind its creation.

The process doesn’t use a patient’s own cells. Rather, Humacyte takes vascular smooth muscle cells and, through a production process involving bioreactors and decellularization, creates bioengineered blood vessels. CEO Carrie Cox thinks this puts Humacyte at the forefront of regenerative medicine.

“Some of the early work focused on growing patient’s tissues from their own cells. But we don’t need to do that, that’s a cumbersome, expensive, difficult step. We’ve learned how to grow tissues. These are stable tissues and they’re available at the time of need,” Cox said.

Humacyte envisages a future of off-the-shelf implants.

“The platform technology, once you learn how to grow a new human tissue that can be implanted into almost any patient or virtually any patient, that’s pretty astounding. I think the potential that it creates to go farther is certainly there and one that we’ll have fun exploring in the future,” Cox said.

Creating new vessels for coronary artery bypass graft (CABG) surgery is high on Humacyte’s to-do list. Beyond that, Cox and her team have an eye on addressing all vascular injuries and needs. The potential of the technology has attracted the Department of Defense, which is working with Humacyte to see if bioengineered blood vessels could be used to save the limbs of soldiers who suffer injuries as a result of improvised explosive devices (IEDs).

What to look for

Humacyte is in the middle of a phase 3 trial pitting its bioengineered blood vessels against expanded PTFE grafts. The trial is on its way to enrolling its target of 350 patients, putting it on track to deliver data in 2018. With FDA having awarded fast-track status to the program, it could be in line for priority review if the results impress.

In parallel to the advance of the lead program, Humacyte is working to unlock the potential of its platform, be that through using it to create vessels to address IED injuries or providing an off-the-shelf product for use in CABG surgery.

These vascular-focused efforts are keeping Humacyte occupied for now. But back in the laboratory of its founder, researchers are cooking up new use cases.

“Our founder, Laura Niklason, has also done some work in trachea and esophagus, which is something else that down the road we could also perhaps pursue,” Cox said. — Nick Paul Taylor, @FierceBiotech