XC001 demonstrated durable improvements across multiple efficacy measures 12 months after treatment, 强调其科学合理的方法来实现生物效应和改善心绞痛症状
患者表现出持续12个月的运动能力改善和胸痛发作的减少
来自EXACT试验的强有力的机制证据强调了XC001治疗心血管疾病的巨大潜力
XyloCor Therapeutics, 一个临床阶段的生物制药公司开发新的基因治疗心血管疾病, 今天，该公司报告了其1/2期临床试验(EXACT)的2期数据，该试验旨在评估先导基因治疗候选药物XC001 (encoberminogene rezmadenovec)对难治性心绞痛患者的安全性，并提供初步证据.
At the 12-month mark in the extension period of the trial, XC001 demonstrated durable improvements across multiple efficacy measures, 包括持续改善总运动时间和减少缺血性负担和缺血性症状.
Earlier this year, XyloCor报告了为期6个月的2期临床试验的阳性结果. New results at 12 months highlight significant, clinically-meaningful impacts that are now sustained out to 12 months, 这表明XC001有潜力成为一种新的治疗方法，以满足难治性心绞痛的重大医疗需求.
“The durability and, in the case of exercise time, 12个月后观察到的持续改善标志着一种可持续的活动，这是心血管疾病基因治疗进步中令人兴奋的一步,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “这些12个月的数据建立在试验3个月和6个月时取得的积极结果的基础上. In total, EXACT研究的结果为推动XC001下一阶段的开发提供了强有力的机制证据, suggesting that a single treatment may have long-term benefit.”
XC001是一种一次性基因治疗候选药物，旨在通过在心脏中创建新血管来减轻缺血负担. 在EXACT试验2期6个月的初步研究期之后，进行了为期12个月的随访. At 12 months, 患者表现出持续和持续的总运动时间(TED)比基线增加, representing a significant and clinically meaningful change. In addition, there was a sustained and robust decrease in episodes of chest pain (angina) and nitroglycerin use. 12个月时的心脏成像为实现生物学效应的潜在作用机制提供了额外的证据, confirmed by a sustained reduction in ischemic burden observed over time.
“With the 12-month results from our EXACT trial, XyloCor继续在实现心血管疾病患者基因治疗的承诺方面发挥主导作用,” said Al Gianchetti, President and CEO of XyloCor. “这些结果进一步增强了我们的信心，我们正走在改变心血管疾病结果的正确道路上.”
About XC001
XC001旨在促进心脏新生血管，绕过病变血管，改善血液流动. By restoring blood flow, chest pain associated with refractory angina may decrease, 有可能改善病人的生活质量，使他们能够参与日常的体育活动，否则会引起疼痛. XC001旨在通过一次性策略避免与其他基因疗法观察到的毒性问题, local administration. 这种方法允许XC001在心脏中实现更高的基因表达，同时最大限度地减少全身载体循环和相关副作用.
About the EXACT Study
心外膜递送XC001基因治疗难治性心绞痛冠状动脉治疗(EXACT)临床试验是1/2期多中心试验, open-label, single-arm trial. 12名难治性心绞痛患者(每剂量组3名)被分为4个递增剂量组, 随后是试验的扩展阶段，其中以最高耐受剂量(1 x 1011vp)招募更多受试者, the highest tested dose). 研究基因疗法是由心脏外科医生通过小型开胸手术直接施用于心肌.
About Chronic Refractory Angina
In the United States, coronary artery disease is a leading cause of death and disability. 慢性心绞痛发生时，心脏肌肉没有得到足够的氧气，导致胸痛. This is usually due to atherosclerotic plaques that block the coronary arteries. 难治性心绞痛是一个日益严重的问题，发生在慢性心绞痛患者中，尽管有最佳的药物治疗，但仍有症状，并且不再符合经皮冠状动脉介入治疗(PCI)和冠状动脉旁路移植术(CABG)等机械干预的条件。. 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.
About XyloCor
XyloCor Therapeutics is a private, 临床阶段的生物制药公司开发潜在的同类最佳基因疗法，以改变心血管疾病患者的预后. The Company’s lead product candidate, XC001, 是否在临床开发中研究对没有治疗选择的难治性心绞痛患者的使用. 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