XyloCor Therapeutics reported positive topline results Thursday from Phase II of the Phase I/II trial of its gene therapy for refractory angina.
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Only a few days ahead of American Heart Month, XyloCor Therapeutics reported positive topline results from Phase II of the Phase I/II trial of its gene therapy for refractory angina.
While gene therapy gets more attention for its potential in hemophilia, ocular diseases and neuromuscular indications like spinal muscular atrophy, it’s also making inroads in the cardiovascular space.
In Phase II of the EXACT trial, XyloCor’s lead gene therapy asset, XC001, met both safety and efficacy goals.
Six-month data from 28 patients with refractory angina showed the therapy hit several key metrics. This included a reduction of ischemic burden as measured by cardiac positron emission tomography imaging.
The patients also saw an improvement in exercise duration. Six months after administration of XC001, nearly all participants could conduct ordinary physical activity without experiencing angina, XyloCor noted in a Thursday press release.
Ischemia denotes a restriction in blood supply to any tissue or muscle group – including the heart. It causes a deficiency of oxygen that can lead to chest pains inherent in chronic angina. XC001 is a one-time gene therapy intended to reduce this ischemic burden by creating new blood vessels in the heart.
The results showed “intriguing benefits in these needy patients across a variety of objective and subjective measures,” said Thomas Povsic, M.D., Ph.D., professor of medicine, Duke University School of Medicine and EXACT national principal investigator.
Povsic said that there is “mechanistic evidence” demonstrating XC001 “is a scientifically-sound approach for achieving a biological effect.”
XyloCor reported no safety issues in the trial.
An Emerging Space
Tenaya Therapeutics is also pursuing gene therapy for cardiovascular indications.
The South San Francisco-based biotech is using adeno-associated virus 9 to deliver healthy copies of select genes to target cardiac tissue and express the protein of interest at high levels. TN-201 is in preclinical development for genetic hypertrophic cardiomyopathy and genetic arrhythmogenic RV cardiomyopathy.
Earlier in January, researchers from the University of Texas Southwestern Medical Center successfully used base editing technology to repair damaged heart tissue in mice.
The study, published in Science, described how the scientists modified an enzyme called the calcium calmodulin-dependent protein kinase IIδ, conferring “protection from ischemia/reperfusion injury” to cultured heart muscle cells.”
