Results from XyloCor Therapeutics’ Phase 1 Portion of EXACT Trial of XC001 for Cardiovascular Disease Published in Circulation: Cardiovascular Interventions

  • Findings from the Phase 1 dose escalation portion of the EXACT trial of XC001 in refractory angina provided the dose selection and safety justification for the recently completed Phase 1/2 study

  • XC001 is a one-time gene therapy candidate designed to reduce ischemic burden by creating new blood vessels in the heart through the local expression of multiple VEGF isoforms

  • XyloCor is moving forward with urgency to address potential of XC001 as transformative therapy for patients with ischemic heart disease with significant unmet need

XyloCor治疗公司., 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. 这是一期剂量递增研究的结果, 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.

“The results from the Phase 1 study provided the mechanistic underpinning that was the catalyst for the successful completion of the Phase 2 EXACT trial,” 托马斯·波维西奇说.D., Ph.D., 期刊文章的主要作者, 医学教授, 杜克大学医学院和国家首席研究员的确切研究. 难治性心绞痛患者症状严重,生活质量极差. With a robust body of positive and sustained safety and efficacy out to 12 months from the EXACT trial, we believe that XC001 has the potential to fill the significant unmet need for this patient population who currently lack treatment options.”

"We are thrilled with the publication of these EXACT trial results in Circulation: Cardiovascular Interventions, 一个高度重视和有影响力的心血管研究国际期刊," XyloCor的首席医疗官Howard Dittrich说. “We would like to acknowledge all of the authors for their contributions in highlighting the promise of XC001 and thank patients and their families for their participation in the EXACT trial. Our team is singularly focused on continuing to unlock the transformative potential of XC001 for improving outcomes in cardiovascular disease.”

1/2期EXACT研究的1期部分是首次人体试验, 多中心, 非盲, 随访时间, 剂量递增研究来评估安全性, 耐受性, 增加XC001剂量的初步疗效, 并确定最佳剂量,以进行第二阶段的进一步研究. 12名患者被纳入4个给药队列. 值得注意的是, 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. 这为今后XC001的临床研究建立了1×1011病毒颗粒的剂量.



XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. 通过恢复血液流动, 与难治性心绞痛相关的胸痛可能减轻, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one-time, 当地政府. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.


The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial was a Phase 1/2 多中心, 非盲, 单组试验. 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 additional subjects were enrolled at the highest tolerated dose (1 x 1011 vp, 最高测试剂量). The investigational gene therapy is administered directly to the heart muscle through a mini-thoracotomy by a cardiac surgeon.


在美国,冠状动脉疾病是导致死亡和残疾的主要原因. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. 这通常是由于动脉粥样硬化斑块阻塞冠状动脉. 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). 这些患者目前没有治疗选择,而且通常症状严重, 这会严重影响他们的生活质量吗, 并可能加剧合并症,导致其健康状况进一步恶化. 顽固性心绞痛导致医疗资源的大量消耗, 包括因病人胸痛而去急诊室的次数.


XyloCor治疗公司. 是私人的, clinical-stage biopharmaceutical company developing potential best in-class gene therapies to transform outcomes for patients with cardiovascular disease. 公司的主要候选产品, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options. XyloCor有第二个临床前研究产品, XC002, 在发现阶段, 用于治疗因心脏病发作而造成心脏组织损伤的患者. 该公司是由罗纳德·克里斯托(Ronald Crystal), M.D.托德·罗森加特(Todd Rosengart).D.该公司拥有康奈尔大学的独家授权. 欲了解更多信息,请访问


A. Brian Davis, XyloCor治疗公司.


Mike Beyer, Sam Brown公司. 医疗通信