drugset / Press release

Results from XyloCor Therapeutics’ Phase 1 Portion of EXACT Trial of XC001 for Cardiovascular Disease Published in Circulation: Cardiovascular Interventions Wayne, PA, August 3, 2023 — XyloCor Therapeutics, Inc., a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, announced today that Circulation: Cardiovascular Interventions has published results from the Phase 1 portion of its Phase 1/2 clinical trial (EXACT) of lead gene therapy candidate XC001 (encoberminogene rezmadenovec) in patients with refractory angina. The findings from the Phase 1 dose escalation study, previously reported at the American Association for Thoracic Surgery (AATS) and the American Society of Gene and Cell Therapy (ASGCT) in May 2022, revealed that XC001 is well tolerated at all dose levels and provided justification to proceed to Phase 2 with the highest dose tested.

2023-08-03 · XyloCor Therapeutics, Inc. · original xylocor.com ↗

Results from XyloCor Therapeu3cs’ Phase 1 Por3on of EXACT Trial of XC001 for Cardiovascular Disease Published in Circula(on: Cardiovascular Interven(ons - Findings from the Phase 1 dose escala5on por5on of the EXACT trial of XC001 in refractory angina provided the dose selec5on and safety jus5fica5on for the recently completed Phase 1/2 study -XC001 is a one-5me gene therapy candidate designed to reduce ischemic burden by crea5ng new blood vessels in the heart through the local expression of mul5ple VEGF isoforms - XyloCor is moving forward with urgency to address poten5al of XC001 as transforma5ve therapy for pa5ents with ischemic heart disease with significant unmet need Wayne, PA, August 3, 2023 — XyloCor Therapeu/cs, Inc., a clinical-stage biopharmaceu/cal company developing novel gene therapies for cardiovascular disease, announced today that Circula(on: Cardiovascular Interven(ons has published results from the Phase 1 por/on of its Phase 1/2 clinical trial (EXACT) of lead gene therapy candidate XC001 (encoberminogene rezmadenovec) in pa/ents with refractory angina. The findings from the Phase 1 dose escala/on study, previously reported at the American Associa/on for Thoracic Surgery (AATS) and the American Society of Gene and Cell Therapy (ASGCT) in May 2022, revealed that XC001 is well tolerated at all dose levels and provided jus/fica/on to proceed to Phase 2 with the highest dose tested. “The results from the Phase 1 study provided the mechanis/c underpinning that was the catalyst for the successful comple/on of the Phase 2 EXACT trial,” said Thomas Povsic, M.D., Ph.D., lead author of the journal ar/cle, Professor of Medicine, Duke University School of Medicine and Na/onal Principal Inves/gator for the EXACT study. “Pa/ents with refractory angina are highly symptoma/c and have an exceedingly poor quality of life. With a robust body of posi/ve and sustained safety and efficacy out to 12 months from the EXACT trial, we believe that XC001 has the poten/al to fill the significant unmet need for this pa/ent popula/on who currently lack treatment op/ons.” "We are thrilled with the publica/on of these EXACT trial results in Circula(on: Cardiovascular Interven(ons, a highly-regarded and influen/al interna/onal journal for cardiovascular research," said Howard Di\rich, Chief Medical Officer of XyloCor . “We would like to acknowledge all of the authors for their contribu/ons in highligh/ng the promise of XC001 and thank pa/ents and their families for their par/cipa/on in the EXACT trial. Our team is singularly focused on con/nuing to unlock the transforma/ve poten/al of XC001 for improving outcomes in cardiovascular disease.” The Phase 1 por/on of the Phase 1/2 EXACT study was a first-in-human, mul/center, open-label, single- arm, dose-escala/on study to evaluate the safety, tolerability, and preliminary efficacy of increasing doses of XC001, and to establish the best dose to carry forward for addi/onal study in Phase 2. Twelve pa/ents were enrolled into four dosing cohorts. Notably, the study demonstrated that adenoviral vector doses higher than those used in previous studies were well tolerated and more robust efficacy was demonstrated at the higher doses. This established a dose of 1×1011 viral par/cles for future clinical research of XC001. The Circula/on: Cardiovascular Interven/ons full ar/cle is available at h\ps://www.ahajournals.org/doi/abs/10.1161/CIRCINTERVENTIONS.123.012997 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 grajing (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, Inc. 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 a\acks. 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 Rela3ons: A. Brian Davis, XyloCor Therapeu/cs, Inc. 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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