BOULDER, Colo.--(BUSINESS WIRE)--Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced the dosing of the first patient in the Phase 2 CIRRUS-HCM trial of EDG-7500. EDG-7500 is a novel oral, selective, cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with HCM and other diseases of diastolic dysfunction.

科罗拉多州博尔德（BUSINESS WIRE）--领先的肌肉疾病生物制药公司Edgewise Therapeutics，Inc.（纳斯达克：EWTX）今天宣布，在EDG-7500的第二阶段CIRRUS-HCM试验中，第一名患者的剂量。EDG-7500是一种新型的口服选择性心肌肌节调节剂，专门设计用于减缓早期收缩速度，并解决与HCM和其他舒张功能障碍疾病相关的心脏松弛受损问题。

The Phase 2 trial will assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of EDG-7500 in patients with obstructive HCM. Part A of the trial will evaluate single doses and Part B will evaluate multiple oral doses of EDG-7500 over 28 days..

2期试验将评估EDG-7500在阻塞性HCM患者中的安全性，耐受性，药代动力学（PK）和药效学。试验的A部分将评估单剂量，B部分将评估28天内多次口服EDG-7500的剂量。。

“Based on the strength of clinical and preclinical data to-date, we are advancing EDG-7500 into the Phase 2 CIRRUS-HCM trial in patients with obstructive HCM,” said Marc Semigran, M.D., Chief Development Officer, Edgewise Therapeutics. “We are encouraged by the effects of EDG-7500 observed in a translatable preclinical disease model of obstructive HCM.

Edgewise Therapeutics首席开发官Marc Semigran医学博士说：“根据迄今为止临床和临床前数据的优势，我们正在将EDG-7500推进梗阻性HCM患者的2期CIRRUS-HCM试验。”。“我们对在阻塞性HCM的可翻译临床前疾病模型中观察到的EDG-7500的作用感到鼓舞。

EDG-7500 demonstrated potent left ventricular (LV) outflow tract gradient relief while maintaining overall normal LV contractility. Importantly, in preclinical models of non-obstructive HCM, both acute and chronic administration of EDG-7500 has also been observed to be associated with significant improvements in ventricular filling and diastolic function.”.

EDG-7500表现出有效的左心室（LV）流出道梯度缓解，同时保持整体正常的LV收缩力。重要的是，在非阻塞性HCM的临床前模型中，EDG-7500的急性和慢性给药也被观察到与心室充盈和舒张功能的显着改善有关。”。

Kevin Koch, Ph.D., President and Chief Executive Officer, Edgewise Therapeutics added, “Advancing EDG-7500 into patients with obstructive HCM is an important milestone for Edgewise. EDG-7500 is representative of the robust discovery and development capabilities at Edgewise and our commitment to patients with serious muscle diseases.”.

Edgewise Therapeutics总裁兼首席执行官凯文·科赫博士补充道：“将EDG-7500推进梗阻性HCM患者是Edgewise的一个重要里程碑。EDG-7500代表了Edgewise强大的发现和开发能力以及我们对严重肌肉疾病患者的承诺。”。

The Company expects to report data from the single dose arm of this trial and a Phase 1 trial of EDG-7500 in healthy volunteers in the third quarter of 2024. Furthermore, the Company expects to initiate a 28-day trial in patients with obstructive and non-obstructive HCM in the second half of 2024. The Company also expects to begin an open-label extension trial of EDG-7500 in the fourth quarter of 2024..

该公司预计将在2024年第三季度报告该试验的单剂量组和EDG-7500在健康志愿者中的第一阶段试验的数据。此外，该公司预计在2024年下半年开始对阻塞性和非阻塞性HCM患者进行为期28天的试验。该公司还预计在2024年第四季度开始EDG-7500的开放标签扩展试验。。

About the Phase 2 CIRRUS-HCM Trial

关于第二阶段CIRRUS-HCM试验

CIRRUS-HCM is a multi-center, two-part, open-label trial of EDG-7500 in patients with obstructive HCM at up to 20 clinical sites in the U.S. The trial will evaluate the safety, tolerability, PK and pharmacodynamics of EDG-7500 in up to 30 adults with obstructive HCM. Participants enrolled in this trial will receive EDG-7500 as a single oral dose and may then receive multiple oral doses over 28 days.

CIRRUS-HCM是EDG-7500在美国多达20个临床部位对阻塞性HCM患者进行的多中心，两部分，开放标签试验。该试验将评估EDG-7500在多达30名阻塞性HCM成人中的安全性，耐受性，PK和药效学。参加该试验的参与者将以单次口服剂量接受EDG-7500，然后可能在28天内接受多次口服剂量。

To learn more about this trial (NCT06347159), go to clinicaltrials.gov..

要了解有关此试验的更多信息（NCT06347159），请访问clinicaltrials.gov。。

About Hypertrophic Cardiomyopathy

关于肥厚型心肌病

HCM is the most common form of genetic heart disease, affecting approximately one in 200-500 individuals. HCM is caused by abnormal proteins in the heart, including cardiac myosin, that lead to excessive cardiac contraction, referred to as hypercontractility. This disruption in cardiac muscle contractility leads to increased stress and thickening of the walls of the major pumping chamber of the heart, the LV.

HCM是遗传性心脏病的最常见形式，大约每200-500个人中就有一个人受到影响。HCM是由心脏异常蛋白质引起的，包括心肌肌球蛋白，导致心脏过度收缩，称为过度收缩。心肌收缩力的这种破坏导致心脏主要泵室LV的压力增加和壁增厚。

The LV becomes less compliant and therefore less able to fill with and pump blood resulting in a decrease in the LV chamber volume. Individuals with HCM can become extremely limited in their functional capacity and ability to perform the activities of daily living. In addition, these individuals are at increased risk of heart failure, stroke, atrial fibrillation, and sudden cardiac arrest.

LV变得不太顺应，因此不能填充和泵送血液，导致LV室体积减小。患有HCM的人的功能能力和进行日常生活活动的能力可能会受到极大限制。此外，这些人患心力衰竭，中风，心房颤动和心脏骤停的风险增加。

Despite advancements in the development of therapies to treat HCM, there is currently a significant unmet need for additional therapeutic approaches and options for patients..

尽管治疗HCM的疗法取得了进展，但目前对患者的其他治疗方法和选择的需求尚未得到满足。。

About EDG-7500 for Hypertrophic Cardiomyopathy

关于EDG-7500治疗肥厚型心肌病

EDG-7500 is a novel oral, selective, cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with HCM and other diseases of diastolic dysfunction. Preclinical data in models of both obstructive and non-obstructive HCM suggest the ability to drive a broadly effective clinical response at a low risk of decreasing left ventricular ejection fraction below normal at all doses tested.

EDG-7500是一种新型的口服选择性心肌肌节调节剂，专门设计用于减缓早期收缩速度，并解决与HCM和其他舒张功能障碍疾病相关的心脏松弛受损问题。阻塞性和非阻塞性HCM模型中的临床前数据表明，在所有测试剂量下，以较低的左心室射血分数降低至正常水平以下的风险驱动广泛有效的临床反应的能力。

Based on EDG-7500’s self-limiting effect on systolic contraction observed in preclinical models, the Company plans to investigate fixed-dose regimens of EDG-7500, thus potentially avoiding intensive safety monitoring of patients on current therapy..

基于在临床前模型中观察到的EDG-7500对收缩收缩的自限性作用，该公司计划研究EDG-7500的固定剂量方案，从而有可能避免对当前治疗的患者进行强化安全监测。。

The Company is advancing a randomized, placebo-controlled, single and multiple ascending dose Phase 1 trial (NCT06011317) evaluating safety, tolerability, PK and pharmacodynamics in healthy adults. The Company recently initiated the Phase 2 CIRRUS-HCM trial to evaluate the safety, tolerability, PK and pharmacodynamics of EDG-7500 in patients with obstructive HCM.

该公司正在推进一项随机，安慰剂对照，单次和多次递增剂量1期试验（NCT06011317），评估健康成年人的安全性，耐受性，PK和药效学。该公司最近启动了2期CIRRUS-HCM试验，以评估EDG-7500在阻塞性HCM患者中的安全性，耐受性，PK和药效学。

To learn more about this trial (NCT06347159), go to clinicaltrials.gov..

要了解有关此试验的更多信息（NCT06347159），请访问clinicaltrials.gov。。

About Edgewise Therapeutics

关于方丝疗法

Edgewise Therapeutics is a leading muscle disease biopharmaceutical company developing novel therapeutics for muscular dystrophies and serious cardiac conditions. The Company’s deep expertise in muscle physiology is driving a new generation of novel therapeutics. Sevasemten is an orally administered skeletal myosin inhibitor in late-stage clinical trials in Becker and Duchenne muscular dystrophies.

Edgewise Therapeutics是一家领先的肌肉疾病生物制药公司，正在开发针对肌肉营养不良和严重心脏病的新型疗法。该公司在肌肉生理学方面的深厚专业知识正在推动新一代新型疗法的产生。Sevasemten是Becker和Duchenne肌营养不良症晚期临床试验中口服的骨骼肌球蛋白抑制剂。

EDG-7500 is a novel cardiac sarcomere modulator for the treatment of hypertrophic cardiomyopathy and other diseases of diastolic dysfunction, currently in Phase 2 clinical development. The entire team at Edgewise is dedicated to our mission: changing the lives of patients and families affected by serious muscle diseases.

EDG-7500是一种新型心肌肌节调节剂，用于治疗肥厚型心肌病和其他舒张功能障碍疾病，目前处于2期临床开发阶段。Edgewise的整个团队致力于我们的使命：改变受严重肌肉疾病影响的患者和家庭的生活。