drugset / Press release

XyloCor Therapeutics Reports Sustained Results in 12‑Month Extension of Phase 2 EXACT Clinical Trial of XC001 Novel Gene Therapy for Refractory Angina Wayne, PA, July 18, 2023 — XyloCor Therapeutics, a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today reported positive 12‑month data from the Phase 2 portion of its Phase 1/2 clinical trial (EXACT) designed to assess the safety and provide preliminary evidence of efficacy of lead gene therapy candidate XC001 (encoberminogene rezmadenovec) in patients with refractory angina. At the 12‑month mark in the extension period of the trial, XC001 demonstrated durable improvements across multiple efficacy measures, including continued improvement in total exercise duration and reductions in ischemic burden and ischemic symptoms. Earlier this year, XyloCor reported positive results from the primary study period for the Phase 2 portion EXACT trial at six months. New results at 12 months highlight significant, clinically‑meaningful impacts that are now sustained out to 12 months, pointing to the potential of XC001 as a novel therapeutic approach for the significant unmet medical need in refractory angina.

2023-07-18 · XyloCor Therapeutics, Inc. · original xylocor.com ↗

XyloCor Therapeu.cs Reports Sustained Results in 12-Month Extension of Phase 2 EXACT Clinical Trial of XC001 Novel Gene Therapy for Refractory Angina -XC001 demonstrated durable improvements across mul7ple efficacy measures 12 months a<er treatment, underscoring its scien7fically-sound approach to achieve biological effect and improve angina symptoms -Pa7ents showed con7nued improvements in exercise capacity and reduc7ons in episodes of chest pain that were sustained to 12 months -Robust body of mechanis7c evidence from EXACT trial highlights significant poten7al of XC001 in cardiovascular disease Wayne, PA, July 18, 2023 — XyloCor Therapeu.cs, a clinical-stage biopharmaceu.cal company developing novel gene therapies for cardiovascular disease, today reported posi.ve 12-month data from the Phase 2 por.on of its Phase 1/2 clinical trial (EXACT) designed to assess the safety and provide preliminary evidence of efficacy of lead gene therapy candidate XC001 (encoberminogene rezmadenovec) in pa.ents with refractory angina. At the 12-month mark in the extension period of the trial, XC001 demonstrated durable improvements across mul.ple efficacy measures, including con.nued improvement in total exercise dura.on and reduc.ons in ischemic burden and ischemic symptoms. Earlier this year, XyloCor reported posi.ve results from the primary study period for the Phase 2 por.on EXACT trial at six months. New results at 12 months highlight significant, clinically-meaningful impacts that are now sustained out to 12 months, poin.ng to the poten.al of XC001 as a novel therapeu.c approach for the significant unmet medical need in refractory angina. “The durability and, in the case of exercise .me, con.nued improvements observed at 12 months signals a sustainable ac.vity which is an exci.ng step forward in the advancement of gene therapy for cardiovascular disease,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and Na.onal Principal Inves.gator for the EXACT study. “These 12-month data build upon the posi.ve results achieved at the 3- and 6-month marks of the trial. In total, the outcomes of the EXACT study form a robust body of mechanis.c evidence to propel the next stage of XC001’s development, sugges.ng that a single treatment may have long-term benefit.” XC001 is a one-.me gene therapy candidate designed to reduce ischemic burden by crea.ng new blood vessels in the heart. The six-month primary study period in the Phase 2 por.on of the EXACT trial was followed by a month 12 follow up period. At 12 months, pa.ents demonstrated sustained and con.nued increases in total exercise dura.on (TED) over baseline, represen.ng a significant and clinically meaningful change. In addi.on, there was a sustained and robust decrease in episodes of chest pain (angina) and nitroglycerin use. Cardiac imaging at 12 months provided addi.onal evidence of the poten.al mechanism of ac.on to achieve a biological effect, confirmed by a sustained reduc.on in ischemic burden observed over .me. “With the 12-month results from our EXACT trial, XyloCor con.nues to take a lead role in fulfilling the promise of gene therapy for people with cardiovascular disease,” said Al GiancheZ, President and CEO of XyloCor . "These results further enhance our confidence that we are on the right path for transforming outcomes in cardiovascular disease.” About XC001 XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow , chest pain associated with refractory angina may decrease, poten.ally improving pa.ents’ quality of life by enabling them to engage in daily physical ac.vi.es that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one-.me, local administra.on. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circula.on and associated side effects. About the EXACT Study The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial was a Phase 1/2 mul.center, open-label, single-arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, followed by an expansion phase of the trial in which addi.onal subjects were enrolled at the highest tolerated dose (1 x 1011 vp, the highest tested dose). The inves.ga.onal gene therapy is administered directly to the heart muscle through a mini-thoracotomy by a cardiac surgeon. About Chronic Refractory Angina In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resul.ng in chest pain. This is usually due to atherosclero.c plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in pa.ents with chronic angina who are symptoma.c despite op.mal medical therapy and are no longer eligible for mechanical interven.ons like percutaneous coronary interven.on (PCI) and coronary artery bypass gracing (CABG). These pa.ents currently have no treatment op.ons and are frequently highly symptoma.c, which severely impacts their quality of life, and may exacerbate comorbidi.es and cause further deteriora.on of their health status. Refractory angina results in significant consump.on of healthcare resources, including visits to the emergency department as a result of pa.ents’ chest pain. About XyloCor XyloCor Therapeu.cs is a private, clinical-stage biopharmaceu.cal company developing poten.al best in- class gene therapies to transform outcomes for pa.ents with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to inves.gate use for pa.ents with refractory angina for whom there are no treatment op.ons. XyloCor has a second preclinical inves.ga.onal product, XC002, in discovery stage, being developed for the treatment of pa.ents with cardiac .ssue damage from heart adacks. 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 informa.on, visit www.xylocor .com. Corporate and Investor Rela.ons: A. Brian Davis, XyloCor Therapeu.cs brian.davis@xylocor .com 610-541-2056 Media Contact: Mike Beyer, Sam Brown Inc. Healthcare Communica.ons [email protected] 312-961-2502

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