CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), 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 it has completed enrollment in the Phase 2 EMBRAZE trial designed to show proof-of-concept of apitegromab, an investigational selective myostatin inhibitor antibody, to safely preserve lean muscle mass in individuals on GLP-1 receptor agonist (GLP-1 RA) therapy for obesity.

马萨诸塞州剑桥市。-（商业新闻短讯）--Scholar Rock（纳斯达克：SRRK）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，今天宣布已完成EMBRAZE第二阶段试验的注册，该试验旨在证明阿匹曲单抗（一种研究性选择性肌肉生长抑制素抑制剂抗体）的概念验证，以安全地保留GLP-1受体激动剂（GLP-1 RA）治疗肥胖个体的瘦肌肉质量。

Results from this trial are expected in the second quarter of 2025 and will inform the development of SRK-439, a novel, investigational selective myostatin inhibitor, which the Company plans to advance specifically for the treatment of cardiometabolic disorders, including obesity..

该试验的结果预计将于2025年第二季度公布，并将为SRK-439的开发提供信息，SRK-439是一种新型的研究性选择性肌肉生长抑制素抑制剂，该公司计划专门用于治疗包括肥胖在内的心脏代谢紊乱。。

EMBRAZE is a randomized, double-blind, placebo-controlled, Phase 2 trial evaluating the efficacy, safety and pharmacokinetics of apitegromab in adults who are overweight or obese without diabetes and taking a GLP-1 RA. The trial fully enrolled participants aged 18-65 to assess the primary endpoint of change in lean mass by DEXA scan at 24 weeks.

EMBRAZE是一项随机、双盲、安慰剂对照的2期临床试验，旨在评估阿哌曲单抗在超重或肥胖且无糖尿病且服用GLP-1 RA的成年人中的疗效、安全性和药代动力学。该试验充分招募了18-65岁的参与者，以通过DEXA扫描在24周时评估瘦体重变化的主要终点。

Initiation of enrollment was announced in May 2024..

2024年5月宣布开始招生。。

“We believe the rapid enrollment of the EMBRAZE trial speaks to the unmet need to preserve lean muscle mass for patients experiencing significant weight loss on GLP-1 therapies. As a leader in myostatin inhibition, Scholar Rock is uniquely positioned to play a meaningful role in the cardiometabolic space.

“我们认为，EMBRAZE试验的快速入选表明，对于GLP-1治疗体重明显减轻的患者，保持瘦肌肉质量的需求尚未得到满足。作为肌肉生长抑制素抑制的领导者，Scholar Rock在心脏代谢领域发挥着独特的作用。

Preserving muscle has the potential to improve the quality of weight loss as well as attenuate the weight regain often observed when people come off GLP-1 therapy,” said Jing Marantz, M.D., Ph.D., Chief Medical Officer at Scholar Rock. “We anticipate reporting results from EMBRAZE in the second quarter of 2025 and plan to use the data to further inform Scholar Rock’s development of SRK-439, which was designed specifically for the treatment of obesity.”.

Scholar Rock首席医学官医学博士Jing Marantz说：“保留肌肉有可能提高减肥质量，并减弱人们在停止GLP-1治疗后经常观察到的体重恢复。”。“我们预计在2025年第二季度报告EMBRAZE的结果，并计划利用这些数据进一步为Scholar Rock开发专门用于治疗肥胖症的SRK-439提供信息。”。

The Company also announced today that it will present new preclinical SRK-439 data in a poster presentation entitled, “SRK-439 Selectively Inhibits Myostatin to Promote Healthy Body Composition During Metformin Therapy” at The Obesity Society’s Annual Meeting at ObesityWeek®, November 3-6, in San Antonio, Texas.

该公司今天还宣布，将在11月3日至6日于德克萨斯州圣安东尼奥举行的肥胖协会年会上，在题为“SRK-439选择性抑制肌肉生长抑制素以促进二甲双胍治疗期间健康的身体成分”的海报展示中展示新的临床前SRK-439数据。

Preclinical data presented earlier showed that SRK-439 maintained lean mass and improved fat mass loss when used in combination with a GLP-1 RA in diet-induced obesity (DIO) mice. Preclinical data also supported the potential for SRK-439 to increase lean mass and attenuate fat mass regain, thus contributing to a favorable body composition following withdrawal from GLP-1 RA treatment.

早期提供的临床前数据显示，在饮食诱导的肥胖（DIO）小鼠中，SRK-439与GLP-1 RA联合使用可维持瘦体重并改善脂肪量损失。临床前数据也支持SRK-439增加瘦体重和减少脂肪量恢复的潜力，从而在退出GLP-1 RA治疗后有助于形成有利的身体成分。

The Company plans to file an IND for SRK-439 in 2025..

该公司计划在2025年为SRK-439提交IND。。

Apitegromab is being developed as potentially the first muscle-targeted therapy for the treatment of spinal muscular atrophy (SMA) and is the only muscle-targeted therapy to show clinical proof-of-concept in SMA. The Company is on track to report topline data in the fourth quarter of 2024 from the pivotal Phase 3 SAPPHIRE clinical trial in patients with SMA.

阿匹曲单抗可能是治疗脊髓性肌萎缩症（SMA）的第一种肌肉靶向疗法，也是唯一一种在SMA中显示临床概念验证的肌肉靶向疗法。该公司有望在2024年第四季度报告关键的3期SAPPHIRE SMA患者临床试验的topline数据。

If the trial is successful and apitegromab is approved by the U.S. Food and Drug Administration (FDA), the Company expects to initiate a commercial product launch in 2025..

。。

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 the Phase 3 SAPPHIRE Trial in Spinal Muscular Atrophy

关于脊髓性肌萎缩症的SAPPHIRE 3期试验

SAPPHIRE is an ongoing randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 SMA who are receiving SMN-targeted therapy (either nusinersen or risdiplam). SAPPHIRE targeted enrolling approximately 156 patients aged 2-12 years old in the main efficacy population.

SAPPHIRE是一项正在进行的随机，双盲，安慰剂对照的3期临床试验，评估阿匹曲单抗在接受SMN靶向治疗（nusinersen或risdiplam）的2型和3型SMA非卧床患者中的安全性和有效性。SAPPHIRE计划在主要疗效人群中招募约156名2-12岁的患者。

These patients were randomized 1:1:1 to receive for 12 months either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks. An exploratory population that targeted enrolling up to 48 patients aged 13-21 years old will also separately be evaluated. These patients were randomized 2:1 to receive either apitegromab 20 mg/kg or placebo.

这些患者以1:1:1的比例随机分配，每4周静脉注射一次apitegromab 10 mg/kg，apitegromab 20 mg/kg或安慰剂12个月。还将分别评估旨在招募多达48名13-21岁患者的探索性人群。这些患者以2:1的比例随机接受apitegromab 20 mg/kg或安慰剂。

For more information about SAPPHIRE, visit www.clinicaltrials.gov. Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established..

有关SAPPHIRE的更多信息，请访问www.clinicaltrials.gov。美国FDA或任何其他卫生部门尚未批准Apitegromab用于任何用途，其安全性和有效性尚未确定。。

About the Phase 2 EMBRAZE Trial in Obesity

关于肥胖的2期EMBRAZE试验

EMBRAZE is a randomized, double-blind, placebo-controlled, Phase 2 proof-of-concept trial evaluating the efficacy, safety and pharmacokinetics of apitegromab in adults with a body mass index (BMI) of >27 (overweight) or a BMI of >30 (obese) and taking a GLP-1 RA (tirzepatide or semaglutide). The target enrollment of EMBRAZE is 100 subjects aged 18-65 who are overweight or obese without diabetes.

EMBRAZE是一项随机，双盲，安慰剂对照的2期概念验证试验，评估阿哌曲单抗在体重指数（BMI）>27（超重）或BMI>30（肥胖）且服用GLP-1 RA（tirzepatide或semaglutide）的成年人中的疗效，安全性和药代动力学。EMBRAZE的目标招募对象是100名年龄在18-65岁之间的超重或肥胖且无糖尿病的受试者。

As part of the study design, the treatment period is 24 weeks, and all subjects will receive a GLP-1 RA. In addition, all subjects will be randomized 1:1 to receive either apitegromab or placebo by intravenous (IV) infusion every four weeks during the 24-week treatment period. The primary endpoint is change from baseline at Week 24 in lean mass assessed by dual-energy X-ray absorptiometry.

。此外，在24周的治疗期间，所有受试者将以1:1的比例随机接受apitegromab或安慰剂，每四周静脉（IV）输注一次。主要终点是通过双能X射线吸收测定法评估的瘦体重从第24周的基线变化。

Secondary endpoints include additional weight loss measures, safety and tolerability, and pharmacokinetic outcomes. Exploratory endpoints at Weeks 24 and 32 include cardiometabolic parameters (e.g. HbA1c), body composition, and physical function..

次要终点包括额外的减肥措施，安全性和耐受性以及药代动力学结果。第24周和第32周的探索性终点包括心脏代谢参数（例如HbA1c），身体成分和身体机能。。

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 cardiometabolic disorders, including obesity.

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），最初被开发用于治疗心脏代谢紊乱，包括肥胖。

Based on preclinical data, SRK-439 has the potential to support healthier weight management by preserving lean mass during weight loss. 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..

根据临床前数据，SRK-439有可能通过在减肥过程中保持瘦体重来支持更健康的体重管理。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 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，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利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 and timing of its clinical trials for apitegromab and its preclinical programs, including SRK-439, 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未来的期望、计划和前景的声明，包括但不限于Scholar Rock对其阿替格洛单抗及其临床前项目（包括SRK-439）临床试验的增长、战略、进展和时机的期望，以及适应症的选择和开发时机，包括其治疗潜力、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、成功和数据公告的期望，对重要里程碑成就的期望，任何候选产品以与早期非临床前、临床前一致的方式在人体中表现的能力临床或临床试验数据，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,” “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”、“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, including the results from the Phase 2 clinical trial of apitegromab are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same pr.

这些风险和不确定性包括但不限于临床前和临床数据，包括阿哌曲单抗2期临床试验的结果，不能预测，可能与未来或正在进行的临床试验产生的数据不一致或更有利相同的pr。