XyloCor Therapeutics, Inc., a company specializing in the development of gene therapies for cardiovascular diseases, announced that the Phase 1 findings of its clinical trial (EXACT) for XC001 (encoberminogene rezmadenovec) have been published in the journal Circulation: Cardiovascular Interventions. The trial results are promising, revealing that XC001 is well-tolerated at all dose levels for patients with refractory angina, paving the way for the Phase 2 study with the highest tested dose.

Phase 1 Results for XC001 Gene Therapy

The results from the Phase 1 dose escalation study were first reported at the American Association for Thoracic Surgery (AATS) and the American Society of Gene and Cell Therapy (ASGCT) in May 2022. Twelve patients were part of the multicenter, open-label, single-arm study, which focused on evaluating the safety, tolerability, and preliminary efficacy of XC001. The study confirmed that adenoviral vector doses higher than those previously used were well-tolerated, demonstrating more robust efficacy at the higher doses. This established a dose of 1×10^11 viral particles for future clinical research on XC001.

“We are thrilled with the publication of these EXACT trial results in Circulation: Cardiovascular Interventions,” said Howard Dittrich, Chief Medical Officer of XyloCor Therapeutics. “Our team is singularly focused on continuing to unlock the transformative potential of XC001 for improving outcomes in cardiovascular disease.”

Design and Purpose of XC001

XC001 is designed to promote the growth of new blood vessels in the heart to bypass diseased vessels, improving blood flow and potentially reducing chest pain related to refractory angina. This could enhance patients’ quality of life by allowing them to engage in daily physical activities without pain. Unlike other gene therapies, XC001 aims to minimize toxicity by employing a one-time, local administration strategy. This approach permits higher gene expression in the heart while reducing systemic vector circulation and associated side effects.

Details of the EXACT Study

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial comprised both Phase 1 and 2 in a multicenter, open-label, single-arm design. Twelve subjects were enrolled into four ascending dose groups, with the highest tolerated dose (1 x 10^11 vp) chosen for further research. The investigational gene therapy is directly administered to the heart muscle by a cardiac surgeon.

  Cardiovascular Diseases, encoberminogene rezmadenovec, Howard Dittrich, refractory angina, USA, XC001, XyloCor Therapeutics