WAYNE, Pa.--(BUSINESS WIRE)--XyloCor Therapeutics, Inc., a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today presented final results from the Phase 2 portion of its Phase 1/2 clinical trial (EXACT) of its lead gene therapy candidate XC001 (encoberminogene rezmadenovec) for refractory angina at the Society for Cardiovascular Angiography & Interventions (SCAI) 2024 Scientific Sessions, May 2-4, 2024 in Long Beach, CA.

宾夕法尼亚州韦恩（商业新闻短讯）--XyloCor Therapeutics，Inc.，一家开发心血管疾病新型基因疗法的临床阶段生物制药公司，今天在2024年5月2日至4日于加利福尼亚州长滩举行的心血管造影与干预学会（SCAI）2024科学会议上，介绍了其主要基因治疗候选药物XC001（encoberminogene rezmadenovec）治疗难治性心绞痛的1/2期临床试验（EXACT）第2阶段的最终结果。

These encouraging results supporting XC001’s safety and efficacy potential are being simultaneously published in Circulation: Cardiovascular Interventions..

这些支持XC001安全性和有效性潜力的令人鼓舞的结果同时发表在《循环：心血管干预》上。。

The EXACT trial assessed the use of one-time gene therapy with XC001 as a new therapeutic approach in refractory angina – a debilitating and chronic condition that impacts over one million people in the United States and is growing in prevalence. XC001 is designed to reduce ischemic burden by creating new blood vessels in the heart through the local expression of multiple vascular endothelial growth factor (VEGF) isoforms.

EXACT试验评估了使用XC001一次性基因治疗作为难治性心绞痛的新治疗方法，难治性心绞痛是一种衰弱的慢性疾病，影响美国100多万人，并且患病率正在上升。XC001旨在通过多种血管内皮生长因子（VEGF）亚型的局部表达在心脏中产生新血管来减轻缺血负担。

In the Phase 2 portion of the EXACT trial, 32 patients with class II-IV angina were dosed with the maximal dose of XC001 through minimally-invasive transepicardial delivery (direct administration to the heart)..

在EXACTE试验的第2阶段，32名II-IV级心绞痛患者通过微创经心尖部给药（直接给予心脏）服用最大剂量的XC001。。

“The results of the EXACT trial suggest that angiogenic gene therapy with XC001 has the potential to improve cardiovascular outcomes for refractory angina patients without revascularization or other treatment options,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and Kenta Nakamura, M.D., Assistant Professor at the University of Washington, lead authors of the EXACT study results.

“EXACT试验的结果表明，对于无血运重建或其他治疗选择的难治性心绞痛患者，使用XC001进行血管生成基因治疗有可能改善心血管预后，”杜克大学医学院医学教授Thomas Povsic博士和华盛顿大学助理教授Kenta Nakamura医学博士说，他们是EXACT研究结果的主要作者。

“There is significant need for novel therapies for this serious and disabling condition and we hope that the clinically meaningful evidence emerging from the EXACT trial is the catalyst for continued development that will further validate the potential of this innovative gene therapy for patients.”.

“对于这种严重且致残的疾病，迫切需要新的治疗方法，我们希望从EXACT试验中获得的具有临床意义的证据是持续发展的催化剂，这将进一步验证这种创新基因治疗对患者的潜力。”。

The Phase 2 results validated the transformative disease modifying potential of XC001 to reduce ischemia and improve the quality-of-life for cardiac patients who have no treatment options. The results demonstrated that treatment with XC001 can be safely administered and achieve durable clinical improvements including: increases in exercise duration, decrease in ischemic burden as measured by Positron Emission Tomography (PET) imaging, and a reduction in angina frequency.

第二阶段的结果验证了XC001的转化性疾病改善潜力，以减少缺血并改善没有治疗选择的心脏病患者的生活质量。结果表明，XC001治疗可以安全地进行，并实现持久的临床改善，包括：运动持续时间增加，正电子发射断层扫描（PET）成像测量的缺血负荷减少，以及心绞痛频率降低。

Notably, 93% of patients in the trial entered the trial with chest paint so severe that it markedly limited daily activities and six months after treatment 43% of patients had no chest pain with ordinary activities. VEGF gene therapy with XC001 was well tolerated in the patient population and there were no serious adverse events related to the drug or unexpected serious adverse events related to XC001 administration..

值得注意的是，93%的试验患者进入试验时胸部彩绘严重，明显限制了日常活动，治疗6个月后，43%的患者没有胸痛，活动正常。XC001的VEGF基因治疗在患者人群中耐受性良好，没有与药物相关的严重不良事件或与XC001给药相关的意外严重不良事件。。

“The results from the EXACT trial represent a promising moment for people with refractory angina and the cardiovascular community as we drive forward toward our goal to deliver a long overdue new treatment option,” said Al Gianchetti, President and CEO of XyloCor. “We are preparing our next clinical trial to advance the development of XC001 and further unlock its transformative medical potential for patients and their families.”.

XyloCor总裁兼首席执行官Al-Gianchetti说：“随着我们朝着提供一种早该实现的新治疗选择的目标迈进，EXACT试验的结果对难治性心绞痛患者和心血管社区来说是一个充满希望的时刻。”。“我们正在准备下一个临床试验，以推进XC001的发展，并进一步释放其对患者及其家属的变革性医疗潜力。”。

Details regarding the SCAI 2024 scientific session is as follows:

有关SCAI 2024科学会议的详细信息如下：

Title: VEGF Gene Therapy Improves Exercise Time, Ischemia, and Symptoms in Patients with Refractory Angina: Results of the Phase II EXACT Trial

标题：VEGF基因治疗可改善难治性心绞痛患者的运动时间，缺血和症状：II期EXACT试验的结果

Lead Presenter: Kenta Nakamura, M.D., Associate Professor at the University of Washington

主持人：华盛顿大学副教授Kenta Nakamura医学博士

Date and Time: Thursday, May 2, 2024; 9:31-9:38 AM PT

日期和时间：2024年5月2日，星期四；9： PT上午31-9:38

Location: Long Beach Convention Center, 104A, First Level

地点：长滩会议中心，104A，一楼

An additional press release from SCAI on the Phase 2 EXACT Trial results and poster session is available at https://scai.org/gene-therapy-treatment-increasing-bodys-signal-new-blood-vessel-growth-shows-promise.

SCAI关于第二阶段确切试验结果和海报会议的其他新闻稿可在https://scai.org/gene-therapy-treatment-increasing-bodys-signal-new-blood-vessel-growth-shows-promise.

The Circulation: Cardiovascular Interventions full article titled “Angiogenic Gene Therapy for Refractory Angina: Results of the EXACT Phase 2 Trial” is available at https://www.ahajournals.org/doi/epdf/10.1161/CIRCINTERVENTIONS.124.014054.

循环：心血管干预题为“难治性心绞痛的血管生成基因治疗：精确2期试验的结果”的全文可在https://www.ahajournals.org/doi/epdf/10.1161/CIRCINTERVENTIONS.124.014054.

About XC001

关于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, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain.

XC001旨在促进心脏中的新血管，从而绕过患病血管并改善血流。通过恢复血流，与难治性心绞痛相关的胸痛可能会减轻，从而使患者能够进行日常体育活动，否则会引起疼痛，从而可能改善患者的生活质量。

XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one‑time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects..

XC001旨在通过一次性局部给药的策略避免其他基因疗法观察到的毒性问题。这种方法允许XC001在心脏中实现更高的基因表达，同时最大程度地减少体细胞循环和相关的副作用。。

About the EXACT Study

关于确切的研究

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial was a Phase 1/2 multicenter, 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 additional subjects were enrolled at the highest tolerated dose (1 x 1011 vp, the highest tested dose).

XC001基因治疗难治性心绞痛冠状动脉治疗（EXACT）临床试验的心外膜递送是一项1/2期多中心，开放标签，单臂试验。将12名患有难治性心绞痛的受试者（每个剂量组n=3）纳入四个递增剂量组，然后进行试验的扩展阶段，其中以最高耐受剂量（1×1011 vp，最高测试剂量）招募其他受试者。

In the EXACT trial, this investigational gene therapy was administered directly to the heart muscle through a mini‑thoracotomy by a cardiac surgeon..

在EXACT试验中，这项研究性基因治疗是由心脏外科医生通过小切口直接对心肌进行的。。

About Chronic Refractory Angina

关于慢性难治性心绞痛

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 resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. 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).

在美国，冠状动脉疾病是导致死亡和残疾的主要原因。当心肌没有获得足够的氧气导致胸痛时，就会发生慢性心绞痛。这通常是由于动脉粥样硬化斑块阻塞冠状动脉。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，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..

这些患者目前没有治疗选择，并且经常出现高度症状，这严重影响了他们的生活质量，并可能加剧合并症并导致其健康状况进一步恶化。难治性心绞痛导致医疗资源的大量消耗，包括因患者胸痛而去急诊室就诊。。

About XyloCor

关于XyloCor

XyloCor Therapeutics, Inc. is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options.

XyloCor Therapeutics，Inc.是一家私营的临床阶段生物制药公司，开发潜在的一流基因疗法，以改变心血管疾病患者的预后。该公司的主要候选产品XC001正在临床开发中，以研究对难治性心绞痛患者的使用情况，这些患者没有治疗选择。

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.

XyloCor在发现阶段有第二种临床前研究产品XC002，用于治疗心脏病发作引起的心脏组织损伤患者。该公司由医学博士罗纳德·克里斯托（RonaldCrystal）和医学博士托德·罗森加特（ToddRosengart）共同创立，拥有康奈尔大学（CornellUniversity）的独家许可证。

For more information, visit www.xylocor.com..

有关更多信息，请访问www.xylocor.com。。