There is a United States biotech company, XyloCor that is advancing its trials of SC001, a form of gene therapy for patients with refractory angina.

Phase 2 Trial of SC001

XyloCor is a biotech company in Pennsylvania, and it has just completed phase 2 of a clinical trial known as EXACT. That stands for Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment. That's a mouth full, but this has provided safety and efficacy data for XC001 (encoberminogene rezmadenovec), which is a gene therapy for patients with refractory angina

The EXACT trial has successfully achieved both safety and efficacy objectives, which means it has a chance to go to phase 3. There were no safety issues reported, or serious adverse effects related to it. There is six-month data from 28 patients in the study showing improvements in several areas of health, which includes a reduction in the ischemic burden.

Thomas Povsic who has a PhD and is a Professor of Medicine at Duke University and the Principle Investigator for this study says, "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"

Coronary artery disease is a big problem in the United States and often leads to death and disability. Chronic angina pectoris happens when the heart muscle does not get sufficient oxygen, which results in chest pain. Usually this is 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 the treatment options needed and are usually very symptomatic, severely impacting quality of life.

XC001 Genere Therapy

XC001 is a one and done only once therapy, which is 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, the drug was observed using positron emission tomography (PET) imaging. What was noted, is 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.

Al Gianchetti, the President and CEO of XyloCor had this to say, "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," and he also says, "We now look forward to pursuing key upcoming milestones in XC001's continued development – including finalizing our pivotal trial design through our ongoing discussions with the FDA and other regulatory authorities."

For more information, see this video from XyloCor: