Phase 2 Begins in XC001 Trial for Refractory Angina


Previously, biopharmaceutical company XyloCor Therapeutics (“XyloCor”) performed the first portion (dose-escalation) of the Phase 1/2 EXACT clinical trial evaluating its investigational treatment XC001 (encoberminogene rezmadenovec) for patients with refractory angina. According to a news release, the Independent Data Monitoring Committee (IDMC) then allowed XyloCorto proceed to the Phase 2 part of the study. Within this study, 3 patients with refractory angina have been dosed at the highest dose. If effective, XC001 would offer a beneficial gene therapy solution for patients who are treatment-averse and for whom other treatments have not worked.


According to XyloCor, XC001 is:

designed to stimulate the formation of new coronary blood vessels to serve areas of the heart that are not receiving adequate blood supply. This treatment strategy is based on both pre-clinical and clinical evidence and should enable the patients to become less limited in their daily activities because of debilitating chest pain.

Through stimulating the formation of new coronary blood vessels, XC001 works to enhance blood flow. The treatment works by delivering a functional VEGF gene to the body and prompting angiogenesis, or the formation of blood vessels.

Now, researchers are evaluating the therapy within the Phase 1/2 EXACT clinical trial, which began last year. Altogether, 12 patients enrolled in the initial study, with 21 patients added in the expansion phase. For treatment, a cardiac surgeon performed a mini-thoracotomy and delivered treatment directly to the heart. Moving forward, XyloCor hopes to continue exploring XC001 as a treatment for chronic refractory angina in further clinical trials.

Refractory Angina

The University Pain Centre Maastricht (UPCM) describes chronic refractory angina, or refractory angina pectoris, as:

a sensation of pressure or pain in the chest that is the result of insufficient blood flow to the heart muscle and in which treatment with medication and heart surgery is either insufficient or no longer effective.

Typically, chronic refractory angina occurs when coronary arteries become narrowed (by up to 75%), causing the heart to both need more oxygen and to work harder. Risk factors for developing refractory angina include being obese, having high blood pressure or high cholesterol, smoking cigarettes, or having diabetes. Symptoms include:

  • Nausea and vomiting
  • Cold sweats
  • Severe pain often beneath the breastbone
    • Note: Pain may also radiate throughout other areas of the body, such as the arms, neck, jaw, or throat.
  • Shortness of breath
  • Extreme fatigue
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

Share this post

Share on facebook
Share on google
Share on twitter
Share on linkedin
Share on pinterest
Share on print
Share on email