XyloCor Therapeutics Announced Positive Results for XC001
XyloCor Therapeutics announced the completion of the Phase 2 portion of its Phase 1/2 clinical trial (EXACT) designed to assess the safety and preliminary evidence of efficacy of lead gene therapy candidate XC001 (encoberminogene rezmadenovec) in patients with refractory angina.
The EXACT clinical trial met both safety and efficacy objectives.
There were no safety issues related to drug product or unexpected serious adverse events related to XC001 administration. Six‑month data from 28 patients in the Phase 2 portion of the study showed improvements in several key efficacy measures, including reduction in ischemic burden.
From Duke University
Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study, said, “We are excited to see EXACT completing its 6‑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. 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.”
XC001 is a one‑time gene therapy designed to reduce ischemic burden by creating new blood vessels in the heart. In the Phase 2 portion of the EXACT trial, evidence of the drug’s mechanism of action was demonstrated by the reduction of ischemic burden measured by cardiac positron emission tomography (PET) imaging. The reduction in ischemic burden was accompanied by an improvement in total exercise duration, an important measure of exercise capacity.
Prior to treatment, almost all subjects had marked limitations on ordinary physical activity. Six months after treatment, nearly half of all subjects were able to conduct ordinary physical activity without causing angina. The data from the Phase 2 EXACT study are potentially meaningful for patients with refractory angina, which includes more than one million people in the United States, who have no treatment options.
From XyloCor Therapeutics
Al Gianchetti, President, and CEO of XyloCor Therapeutics, said, “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. 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.”
The recently completed Epicardial Delivery of the XC001 Gene Therapy for Refractory Angina Coronary Treatment EXACT)clinical trial was a Phase 1/2 multicenter, open‑label, single‑arm trial. Twelve subjects per dose cohort who have refractory angina were enrolled into four ascending dose groups, followed by an expansion phase of the trial in which additional subjects were enrolled at the highest tolerated dose (1 x 1011 vp, the highest tested dose). The investigational gene therapy is administered directly to the heart muscle through a mini‑thoracotomy by a cardiac surgeon.
Chronic Refractory Angina
In the United States, coronary artery disease is a leading cause of death and disability.
Chronic angina pectoris: This angina occurs as a result of atherosclerotic plaques that block the coronary arteries.
Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain.
XyloCor Therapeutics is a private, clinical‑stage biopharmaceutical company.
The firm develops potential gene therapies for patients with cardiovascular disease. Its lead product candidate, XC001 is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options.
XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks.
The company, which was co‑founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University.
For more information, visit www.xylocor.com.
Although XyloCor Therapeutics is a private firm, its cardiovascular programs are interesting to us for various reasons, including the firm’s solid science and its potential gene therapies for patients with cardiovascular disease. Impressive has been the recently completed Epicardial Delivery of XC001 Gene Therapy for Refractory Angina. Encouraging is EXACT Phase 1/2 multicenter clinical trial where twelve subjects having refractory angina were enrolled into, followed by an expansion phase of the trial in which additional subjects were enrolled at the highest tolerated tested dose.
More impressive, however, were the clinical trial results demonstrating that no serious adverse events have been related to the product and the improvements in exercise capacity and reductions in episodes of chest pain.
We like the fact that Cardiac imaging results provided mechanistic evidence supporting the therapeutic potential of XC001 in cardiovascular disease
Bottomline, our enthusiasm comes from the fact that no other product has reached these results and that XyloCor might save millions of patients from expected heart attacks.
There is still a possibility that one day this private firm might turn public, so investors can buy its stocks.
People can also invest in private firms. Those interested might call the firm’s Investor relations.
Corporate and Investor Relations
Brian Davis, [email protected] 610-541-2056
Media Contact Mike BeyerSam Brown Inc. Healthcare Communications [email protected] 312-961-2502