XyloCor Begins EXACT-2 Trial of Catheter-Based XC001 Gene Therapy to Treat CAD

July 25, 2025—XyloCor Therapeutics, a biopharmaceutical company developing therapies for cardiovascular disease, recently announced the first patient has been enrolled in the EXACT-2 trial. The phase 2b, multicenter, randomized, double-blind study is evaluating the company’s catheter-based gene therapy candidate XC001 (encoberminogene rezmadenovec) in patients with coronary artery disease and refractory angina.

EXACT-2, which is composed of 100 patients with refractory angina, will measure the safety and efficacy of one-time gene therapy with XC001 delivered using the company’s Extroducer infusion catheter system. Extroducer is an endocardial delivery catheter designed to inject therapies directly into the heart in a simple injection procedure in the cardiac catheterization lab, stated XyloCor.

According to the company, XC001 is an adenoviral vector-based gene therapy encoding for vascular endothelial growth factor. It further noted that the percutaneous administration approach—injecting directly into the heart muscle—allows XC001 to achieve higher gene expression levels locally in the heart while minimizing systemic vector circulation and associated side effects. The first patient in the study was treated by Timothy Henry, MD, at The Christ Hospital Health Network in Cincinnati, Ohio.

“After seeing the convincing results from the EXACT-1 trial, we were eager to participate in EXACT-2,” commented Dr. Henry in the press release. “We believe that XC001 delivered through the Extroducer infusion catheter system will maintain the accuracy of delivery of XC001 to the heart and improve the safety over the surgical administration approach used in EXACT-1.”

XyloCor reported that the phase 1/2 EXACT-1 trial results supported the potential of XC001 to reduce cardiac ischemia, reduce anginal symptoms, and improve the quality‑of‑life for cardiac patients who have no other treatment options. The results demonstrated the potential for XC001 to be safely administered and achieve durable clinical improvements, including increases in exercise duration, decrease in ischemic burden as measured by positron emission tomography imaging, and a reduction in angina frequency.

In EXACT-1, 93% of patients in the trial entered with chest pain so severe that it markedly limited daily activities; whereas at 6 months, 43% reported no chest pain with ordinary activities and no serious adverse events related to the study drug, reported the company.

XyloCor also advised it is initiating a second clinical trial this year—a double-blind phase 2 trial of XC001 as an adjunctive treatment to coronary artery bypass graft surgery.