XyloCor wraps up PhII for heart disease gene therapy, plans for pivotal trial

XyloCor Therapeutics says patients with heart disease who got its gene therapy could exercise for longer and had fewer chest pain attacks. The biotech announced it completed a Phase I/II trial of the gene therapy Thursday morning, and plans to move forward with a pivotal trial.

In the Phase II portion of the trial, 28 patients with angina (or chest pain) caused by coronary artery disease and who had no other treatment options were enrolled and were given the highest tested dose from the first part of the trial. Patients were followed for six months.

In terms of secondary efficacy endpoints, patients saw improvements on an exercise test, which challenged how long they could walk on a treadmill. They also had fewer chest pain attacks compared to baseline, and XyloCor said that nearly half of the 28 patients could do ordinary physical activity without chest pain at six months.

For the key secondary endpoints, changes were statistically significant, CEO Al Gianchetti told Endpoints News. 

There were no serious adverse events related to the adenovirus vector or drug, though there were some adverse events related to the administration procedure, “but none were unexpected,” Gianchetti said. The gene therapy was injected directly to the heart by surgeons.

Gianchetti noted that by targeting directly to the heart, they could give “relatively low doses” of the viral-based therapy. Gianchetti declined to get into the specifics on both the efficacy and safety data, noting that XyloCor plans to present the results at a scientific meeting.

Ronald Crystal

XyloCor was founded by Weill Cornell gene therapy professor Ronald Crystal and Baylor College of Medicine heart surgery professor Todd Rosengart, who once was a professor at Weill. The therapy, licensed from Weill, delivers the gene for VEGF, an essential growth factor for blood vessel cells.

The goal is that by delivering the gene to the heart, the treatment can promote new blood vessel growth, where blood can flow through the heart instead of the diseased vessels.

Others are also trying to use genetic medicine to tackle heart disease, which is the leading cause of death in the US. Tenaya Therapeutics is working on a number of programs for genetic heart conditions as well as heart failure, though it has yet to put these candidates in the clinic.

And Verve Therapeutics is using base editing to permanently turn off a gene in the liver that encodes for bad cholesterol. While it’s testing the therapy first in patients who have familial hypercholesterolemia, a rare genetic disease, it hopes to one day use it for patients with atherosclerotic cardiovascular disease.

Gianchetti joined as XyloCor’s first CEO in 2016. Based just outside Philadelphia in Wayne, PA, XyloCor raised a Series A extension of $22.6 million in 2021. And moving forward with the new data in hand, Gianchetti said XyloCor needs to raise more capital as well, though he did not specify how.

therapeutics
gene therapy
biotech