CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK) (the Company), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today announced that the U.S.

马萨诸塞州剑桥市。-（商业新闻短讯）--Scholar Rock（纳斯达克：SRRK）（该公司）是一家晚期生物制药公司，专注于推进脊髓性肌萎缩症（SMA），心脏代谢紊乱和其他蛋白质生长因子起基础作用的严重疾病的创新治疗，今天宣布美国。

Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for its Phase 2 proof-of-concept trial of apitegromab to treat obesity in patients taking a GLP-1 receptor agonist (GLP-1 RA)..

美国食品和药物管理局（FDA）已批准该公司的研究性新药（IND）申请，该申请是针对阿匹曲单抗治疗服用GLP-1受体激动剂（GLP-1 RA）患者肥胖的2期概念验证试验。。

The Phase 2 trial is a randomized, double-blind, placebo-controlled, multi-center study to evaluate the effect of apitegromab, a highly selective myostatin inhibitor, to safely preserve lean muscle mass as an adjunctive therapy in overweight and obese adults who are taking a GLP-1 RA. Trial initiation is on track for mid-2024, and data from the apitegromab Phase 2 trial are expected in mid-2025.

2期临床试验是一项随机、双盲、安慰剂对照、多中心研究，旨在评估阿匹曲单抗（一种高度选择性的肌肉生长抑制素抑制剂）作为超重和肥胖成年人服用GLP-1 RA的辅助疗法安全保存瘦肌量的效果。2024年年中开始试验，预计2025年年中将有apitegromab 2期试验的数据。

In parallel, Scholar Rock is developing SRK-439, a novel investigational selective myostatin inhibitor, optimized for the treatment of obesity. The Company plans to file an IND for SRK-439 in 2025..

与此同时，Scholar Rock正在开发SRK-439，一种新型的研究性选择性肌肉生长抑制素抑制剂，用于治疗肥胖。该公司计划在2025年为SRK-439提交IND。。

“The FDA’s acceptance of our IND application to study apitegromab in obesity allows us to assess the effect of our highly selective myostatin inhibitor on preserving lean muscle mass, and safety and tolerability of our approach when combined with a GLP-1 RA. The IND builds on our encouraging apitegromab clinical and safety data to date and allows us to inform the development of our cardiometabolic program with SRK-439, a novel preclinical selective myostatin inhibitor optimized for development in cardiometabolic disorders,” said Jay Backstrom, M.D., MPH, President and Chief Executive Officer of Scholar Rock.

“美国食品和药物管理局（FDA）接受了我们的IND应用研究肥胖中的阿替戈马，这使我们能够评估我们的高选择性肌肉生长抑制素抑制剂对保留瘦肌肉质量的影响，以及与GLP-1 RA联合使用时我们方法的安全性和耐受性。IND建立在我们迄今为止令人鼓舞的阿替戈马临床和安全性数据的基础上，使我们能够为cardiometa的发展提供信息Scholar Rock总裁兼首席执行官Jay Backstrom医学博士说：“SRK-439是一种新型的临床前选择性肌肉生长抑制素抑制剂，可用于心脏代谢紊乱的发展。”。

“Maintaining lean mass during weight loss is important to overall metabolic health, and we look forward to initiating the Phase 2 proof-of-concept trial in apitegromab to validate our differentiated approach of selectively targeting only the pro- and latent forms of myostatin to retain muscle mass.”.

“在减肥过程中保持瘦体重对整体代谢健康很重要，我们期待着在apitegromab中启动第二阶段概念验证试验，以验证我们的差异化方法，即仅选择性靶向肌肉生长抑制素的前体和潜在形式以保留肌肉质量。”。

About SRK-439

关于SRK-439

SRK-439 is a novel, preclinical, investigational myostatin inhibitor that has high in vitro affinity for pro- and latent myostatin and maintains myostatin specificity (i.e., no GDF11 or Activin-A binding), and is initially being developed for the treatment of obesity. Based on preclinical data, SRK-439 has the potential to support healthier weight management by preserving lean mass and enhancing fat mass loss.

SRK-439是一种新型的临床前研究性肌生长抑制素抑制剂，对促肌生长抑制素和潜伏性肌生长抑制素具有较高的体外亲和力，并维持肌生长抑制素的特异性（即无GDF11或激活素a结合），最初正在开发用于治疗肥胖。根据临床前数据，SRK-439有可能通过保持瘦体重和增强脂肪量减少来支持更健康的体重管理。

The efficacy and safety of SRK-439 have not been established and SRK-439 has not been approved for any use by the FDA or any other regulatory agency..

SRK-439的有效性和安全性尚未确定，并且SRK-439尚未被FDA或任何其他监管机构批准用于任何用途。。

About Apitegromab

关于Apitegromab

Apitegromab is an investigational fully human monoclonal antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle. It is the first muscle-targeted treatment candidate to demonstrate clinical proof of concept in spinal muscular atrophy (SMA).

Apitegromab是一种通过选择性结合骨骼肌中肌生长抑制素的前体和潜伏形式来抑制肌生长抑制素活化的研究性全人单克隆抗体。它是第一个在脊髓性肌萎缩症（SMA）中证明临床概念验证的肌肉靶向治疗候选者。

Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. Scholar Rock believes that its highly selective targeting of pro- and latent forms of myostatin with apitegromab may lead to a clinically meaningful improvement in motor function in patients with SMA.

肌肉生长抑制素是生长因子TGFβ超家族的成员，主要由骨骼肌细胞表达，其基因的缺失与包括人类在内的多种动物的肌肉质量和力量增加有关。Scholar Rock认为，阿匹曲单抗对肌肉生长抑制素的前体和潜在形式的高度选择性靶向可能会导致SMA患者运动功能的临床意义改善。

The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. The efficacy and safety of apitegromab have not been established and apitegromab has not been approved for any use by the FDA or any other regulatory agency..

美国食品和药物管理局（FDA）已授予快速通道，孤儿药和罕见儿科疾病名称，欧洲药品管理局（EMA）已授予apitegromab优先药物（PRIME）和孤儿药品名称，用于治疗SMA。阿替格玛的有效性和安全性尚未确定，阿替格玛尚未被FDA或任何其他监管机构批准用于任何用途。。

About Scholar Rock

关于学者岩

Scholar Rock is a biopharmaceutical company that discovers, develops, and delivers life-changing therapies for people with serious diseases that have high unmet need. As a global leader in the biology of the transforming growth factor beta (TGFβ) superfamily of cell proteins and named for the visual resemblance of a scholar rock to protein structures, the clinical-stage company is focused on advancing innovative treatments where protein growth factors are fundamental.

Scholar Rock是一家生物制药公司，为严重疾病患者发现、开发和提供改变生活的疗法，这些患者的需求尚未得到满足。作为细胞蛋白转化生长因子β（TGFβ）超家族生物学的全球领导者，该公司以学者摇滚与蛋白质结构的视觉相似性而命名，临床阶段公司专注于推进蛋白质生长因子为基础的创新治疗。

Over the past decade, Scholar Rock has created a pipeline with the potential to advance the standard of care for neuromuscular disease, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted drugs can play a transformational role..

在过去的十年中，学者洛克（Scholar Rock）创建了一条管道，有可能提高神经肌肉疾病、心脏代谢紊乱、癌症和其他生长因子靶向药物可以发挥变革作用的疾病的护理标准。。

Scholar Rock is the only company to show clinical proof-of-concept for a muscle-targeted treatment in spinal muscular atrophy (SMA). This commitment to unlocking fundamentally different therapeutic approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity.

Scholar Rock是唯一一家为脊髓性肌萎缩症（SMA）的肌肉靶向治疗提供临床概念验证的公司。这种解锁根本不同的治疗方法的承诺是由专有平台的广泛应用所推动的，该平台开发了新型单克隆抗体，以非凡的选择性调节蛋白质生长因子。

By harnessing cutting-edge science in disease spaces that are historically under-addressed through traditional therapies, Scholar Rock works every day to create new possibilities for patients. Learn more about our approach at ScholarRock.com and follow @ScholarRock and on LinkedIn..

学者洛克（Scholar Rock）每天都在利用传统疗法无法解决的疾病领域的尖端科学，为患者创造新的可能性。在ScholarRock.com上了解更多有关我们的方法的信息，并关注@ScholarRock和LinkedIn。。

Availability of Other Information About Scholar Rock

学者岩其他信息的可用性

Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on Twitter and LinkedIn.

投资者和其他人应该注意，我们使用公司网站www.scholarrock.com与投资者和公众进行沟通，包括但不限于公司披露、投资者介绍和常见问题解答、证券交易委员会备案、新闻稿、公开电话会议记录和网络广播记录，以及推特和LinkedIn。

The information that we post on our website or on Twitter or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended..

我们在网站、推特或LinkedIn上发布的信息可能被视为重要信息。因此，我们鼓励投资者、媒体和其他有兴趣的人定期审查我们在那里发布的信息。我们网站或社交媒体的内容不应被视为通过引用纳入1933年《证券法》修订版下的任何备案中。。

Scholar Rock® is a registered trademark of Scholar Rock, Inc.

Scholar Rock®是Scholar Rock，Inc.的注册商标。

Forward-Looking Statements

前瞻性声明

This press release contains 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress, results and timing of its clinical trials for apitegromab and its preclinical programs, including SRK-439, regulatory feedback including with respect to the IND submitted in connection with the planned Phase 2 trial of apitegromab in combination with GLP-1 receptor agonists in obesity, and indication selection and development timing, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, expectations regarding the achievement of important milestones, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform.

本新闻稿包含1995年《私人证券诉讼改革法案》所指的“前瞻性声明”，包括但不限于关于Scholar Rock未来预期、计划和前景的声明，包括但不限于Scholar Rock对其增长、战略、进展，阿替格单抗临床试验的结果和时间安排及其临床前计划，包括SRK-439，监管反馈，包括与阿替格单抗联合GLP-1受体激动剂治疗肥胖症的计划2期试验相关的IND，以及适应症选择和开发时间，包括治疗潜力，其临床益处和安全性，对当前正在进行的临床前和临床试验的时机、成功和数据公布的期望，对重要里程碑的实现的期望，任何候选产品以与早期非临床、临床前或临床试验数据一致的方式在人体内表现的能力，以及其候选产品和专有平台的潜力。

The use of words such as “may,” “might,” “could,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements.

使用诸如“可能”、“可能”、“可能”、“将要”、“应该”、“期望”、“计划”、“预期”、“相信”、“估计”、“项目”、“打算”、“未来”、“潜力”或“继续”等词语以及其他类似表达旨在识别此类前瞻性陈述。所有此类前瞻性声明均基于管理层当前对未来事件的预期，并受到许多风险和不确定性的影响，这些风险和不确定性可能导致实际结果与此类前瞻性声明中规定或暗示的结果产生重大不利差异。

These risks and uncertainties include, without limitation, that preclinical and clinical data.

这些风险和不确定性包括但不限于临床前和临床数据。