XyloCor gene therapy hits goals, paving way for phase 3 in cardiovascular space with no current treatment

XyloCor Therapeutics’ lead candidate, a one-time gene therapy dubbed XC001, has met both safety and efficacy goals among 28 patients with refractory angina.

The results come from a phase 2 portion of the open-label, single-arm phase 1/2 study named EXACT. This portion of the trial assessed XC001’s safety and efficacy after six months among patients with the cardiovascular condition, which is caused by insufficient blood flow to the heart and currently has no available treatment options.

The gene therapy is designed to promote new blood vessels in the heart that will dodge diseased blood vessels and boost blood flow. The one-time, local administration of the gene therapy is aimed at achieving higher gene expression while minimizing systemic vector circulation and related side effects.

No serious adverse events or safety issues tied to XC001 or administration were reported, hitting the trial’s primary goal. The study also found patients showed improved exercise capacity. Before treatment, most of the patients had significant limitations on regular physical activity. Six months after treatment, nearly half of all patients could conduct ordinary physical activity without triggering angina.

The gene therapy also reduced episodes of chest pain, according to XyloCor’s news release, and the drug’s mechanism of action was demonstrated via reduction of ischemic burden measured with cardiac positron emission tomography imaging.  

“The trial met all of its safety and exploratory objectives, showing intriguing benefits in these needy patients across a variety of objective and subjective measures,” said Thomas Povsic, M.D., Ph.D., professor at the Duke University School of Medicine and national principal Investigator for EXACT. “The strong range of mechanistic evidence demonstrate that administration of XC001 is a scientifically‑sound approach for achieving a biological effect that has the potential to improve patients’ quality of life.”

The Pennsylvania biotech is now working to finalize design plans for a pivotal trial and continuing talks with the FDA and other regulators, according to XyloCor President and CEO Al Gianchetti.