30th January 2023

Gene therapy for angina shows promise in Phase 2 trial

U.S. biotech company XyloCor has announced positive safety and efficacy results from its latest trial of XC001, a novel gene therapy for patients with refractory angina.

XyloCor Therapeutics, a biotech company based in Pennsylvania, has completed the second phase of a clinical trial known as EXACT (Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment). This has provided safety and efficacy data for XC001 (encoberminogene rezmadenovec) – a potential gene therapy for patients with refractory angina.

The EXACT trial successfully achieved both safety and efficacy objectives. No safety issues are reported from the drug product or unexpected serious adverse events related to its administration. Six-month data from 28 patients in the study has demonstrated improvements in several areas of health, including a reduction in their ischemic burden.

"We are excited to see EXACT completing its six-month endpoint. 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, PhD, Professor of Medicine at Duke University in North Carolina and Principal Investigator for the EXACT study. "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."

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen, resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina. Many people lack treatment options and are frequently highly symptomatic, which severely impacts quality of life and may exacerbate comorbidities. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department due to patients' chest pain.

XC001 is a one-time gene therapy, administered directly to the heart muscle, and designed to create new blood vessels in the heart to bypass diseased areas and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving the patient's quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is also designed to avoid toxicity issues and other side effects observed with previous gene therapies.

In the Phase 2 EXACT trial, researchers observed the drug's mechanism of action using positron emission tomography (PET) imaging. They noted a reduction in ischemic burden alongside an improvement in total exercise duration. Prior to treatment, almost all subjects had marked limitations on ordinary physical activity. Six months after treatment, nearly half of all subjects had their physical activity levels restored to normal, without causing angina. The data from this study offers hope to patients with refractory angina, which includes more than a million people in the United States alone who currently have no treatment options.

"We are excited to share this positive topline data from the Phase 2 portion of the EXACT trial, reinforcing our confidence in XC001 as a novel therapeutic approach with the potential to address the significant unmet medical needs of people with refractory angina," said Al Gianchetti, President and CEO of XyloCor. "We now look forward to pursuing key upcoming milestones in XC001's continued development – including finalising our pivotal trial design through our ongoing discussions with the FDA and other regulatory authorities."

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