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更新于：2026-05-13

Apitegromab

更新于：2026-05-13

概要

概要

基本信息

药物类型

单克隆抗体

别名

Immunoglobulin g4 (234-proline), anti-(human progrowth differentiation factor 8) (human monoclonal srk-015 .gamma.4-chain), disulfide with human monoclonal srk-015 .lambda.-chain, dimer、SRK 015、SRK-015

靶点

MSTN

作用方式

抑制剂

作用机制

MSTN 抑制剂(生长分化因子-8 抑制剂)

治疗领域

神经系统疾病其他疾病遗传病与畸形+ [2]

在研适应症

脊髓性肌萎缩萎缩青少年脊髓性肌萎缩症+ [3]

非在研适应症

肌肉生长抑制素相关的肌肉肥大

原研机构

Scholar Rock, Inc.

在研机构

Scholar Rock, Inc.

非在研机构-

权益机构-

最高研发阶段申请上市

首次获批日期-

最高研发阶段(中国)-

特殊审评优先审评 (美国)、孤儿药 (美国)、罕见儿科疾病 (美国)、孤儿药 (欧盟)、优先药物（PRIME） (欧盟)、快速通道 (美国)

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结构/序列

Sequence Code 10153537H

来源: *****

Sequence Code 10153552L

来源: *****

关联

项与 Apitegromab 相关的临床试验

NCT07435129

/ Not yet recruiting临床2期

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter, 52-Week Study Evaluating the Efficacy and Safety of Apitegromab in Participants With Facioscapulohumeral Muscular Dystrophy (FORGE)

A randomized Phase 2 study to evaluate the efficacy and safety of apitegromab as a monotherapy in participant with FSHD

开始日期2026-08-01

申办/合作机构

Scholar Rock, Inc.

NCT07047144

/ Recruiting临床2期

A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)

This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score <55.

开始日期2025-09-15

申办/合作机构

Scholar Rock, Inc.

NCT06445075

/ Completed临床2期

A Phase 2 Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Apitegromab in Overweight and Obese Adult Subjects

A phase 2 study to evaluate the effects of apitegromab as an adjunctive therapy to GLP-1 receptor agonist therapy in subjects with overweight or obesity

开始日期2024-05-21

申办/合作机构

Scholar Rock, Inc.

100 项与 Apitegromab 相关的临床结果

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100 项与 Apitegromab 相关的转化医学

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100 项与 Apitegromab 相关的专利（医药）

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项与 Apitegromab 相关的文献（医药）

2026-02-01·DIABETES OBESITY & METABOLISM

Pharmacological intervention: Challenges and promising outcomes for fat loss and preservation of lean body mass in the treatment of overweight and type 2 diabetes

Review

作者: Aimelet, Viktor ; Holst, Jens Juul

Abstract:

Treatment with GLP‐1 receptor agonists (GLP‐1 RAs) is effective in reducing body weight in individuals with overweight and type 2 diabetes (T2D). However, measurements indicate that a considerable portion of the weight loss derives from fat‐free mass (FFM), including skeletal muscle, which may compromise metabolic health and physical function. We aimed to evaluate the evidence for the ability of pharmacological interventions to preserve or increase lean body mass (LBM) during weight loss with GLP‐1 RAs, assess their clinical potential and limitations, and identify knowledge gaps requiring further research. A literature review was conducted using PubMed, JAMA, Wiley, ResearchGate, The Royal Danish Library, and ClinicalTrials.gov. Included were preclinical and clinical studies on compounds with documented anabolic effects and established safety profiles. The primary outcomes assessed were changes in LBM, fat mass (FM), physical function, and adverse events. Activin II receptor inhibition with bimagrumab demonstrated significant preservation and increases in LBM, along with FM reduction, in both preclinical and phase 2 studies in individuals with overweight and T2D. Similar effects were observed for myostatin and activin A inhibitors (trevogrumab, garetosmab), latent myostatin inhibitors (apitegromab, SRK‐439), and Selective Androgen Receptor Modulators (enobosarm). Notably, enobosarm also improved physical function. Adverse events were generally mild and reversible; however, long‐term data remain limited. Pharmacological adjunct therapies show potential for improving body composition and physical function during GLP‐1 RA‐induced weight loss. Preliminary findings are promising, but larger, controlled trials are necessary to confirm efficacy and safety before clinical implementation can be considered.

2026-01-02·Expert Review of Neurotherapeutics

A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophy

Article

作者: Butterfield, Russell J. ; Krueger, Jena ; Servais, Laurent ; Crawford, Thomas O. ; Finkel, Richard S. ; Rossello, Jose ; Sansone, Valeria A. ; Kuntz, Nancy ; Kölbel, Heike ; Gomez Garcia de la Banda, Marta ; Yao, Bert ; Mercuri, Eugenio ; Marantz, Jing L. ; Song, Guochen ; van der Pol, W. Ludo ; Cances, Claude ; Pechmann, Astrid ; De Waele, Liesbeth ; Batley, Kaitlin ; Zhao, Guolin ; Mazzone, Elena Stacy ; Dunaway Young, Sally ; Seferian, Andreea M. ; Darras, Basil T. ; Tirucherai, Giridhar S.

2025-09-01·LANCET NEUROLOGY

Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial

Article

作者: Masson, Riccardo ; Nance, Jessica ; Sansone, Valeria A ; Krueger, Jena ; Bruno, Claudio ; Castellano, Inmaculada Pitarch ; Seferian, Andreea M ; Mathews, Katherine ; Servais, Laurent ; Marantz, Jing L ; Rossello, Jose ; Kuntz, Nancy ; Mazurkiewicz-Beldzinska, Maria ; Kölbel, Heike ; Perlman, Seth Javier ; Finkel, Richard S ; Proud, Crystal ; Deconinck, Nicolas ; Young, Sally Dunaway ; Song, Guochen ; van der Pol, W Ludo ; Cances, Claude ; Ramos-Platt, Leigh Marie ; Blaschek, Astrid ; Fryer, Robert ; Nazario, Adelie Navas ; Holzwarth, Dorothea ; Bernes, Saunder ; Childs, Anne-Marie ; Tesi-Rocha, Carolina ; Daron, Aurore ; Baranello, Giovanni ; Tirucherai, Giridhar S ; Messina, Sonia ; Yum, Sabrina ; Crawford, Thomas O ; Butterfield, Russell J ; Kwon, Jennifer ; de la Banda, Marta Gomez Garcia ; Davion, Jean-Baptiste B. ; Statland, Jeffrey M. ; Yao, Bert ; Richardson, Randal ; Mercuri, Eugenio ; Zaidman, Craig ; Pechmann, Astrid ; Parsons, Julie ; De Waele, Liesbeth ; Batley, Kaitlin ; Mazzone, Elena Stacy ; Zhao, Guolin ; Phan, Han ; Darras, Basil T ; Nascimento Osorio, Andres ; Connolly, Anne M. ; Steinborn, Barbara ; Kotulska-Jozwiak, Katarzyna ; Cartwright, Michael ; Rady, Chamindra Laverty

BACKGROUND:

Approved spinal muscular atrophy therapies greatly improve clinical outcomes; however, substantial motor function deficits persist. Apitegromab, a fully human monoclonal antibody, selectively inhibits myostatin activation, improving muscle function. We aimed to assess the safety and efficacy of apitegromab in patients with nonambulatory type 2 or type 3 spinal muscular atrophy receiving nusinersen or risdiplam.

METHODS:

SAPPHIRE, a double-blind, placebo-controlled, phase 3 trial, was done in 48 hospitals in Belgium, France, Germany, Italy, Poland, Spain, the Netherlands, the UK, and the USA. Eligible participants were aged 2-21 years, had genetically documented SMN-deficient nonambulatory type 2 or type 3 spinal muscular atrophy, an estimated life expectancy greater than 2 years, Hammersmith Functional Motor Scale-Expanded (HFMSE) scores 10-45, and had received at least 10 months' nusinersen or at least 6 months' risdiplam therapy at screening. Participants aged 2-12 years were randomly assigned 1:1:1 to receive apitegromab 20 mg/kg, apitegromab 10 mg/kg, or placebo every 4 weeks; participants aged 13-21 years were randomly assigned 2:1 to receive apitegromab 20 mg/kg or placebo every 4 weeks. All participants, parents or caregivers, investigators, and site personnel were unaware of the treatment assignment. The primary endpoint, change from baseline in HFMSE at 12 months, was assessed in participants aged 2-12 years who received at least one dose of apitegromab or placebo and had at least one post-baseline evaluable HFMSE assessment (modified intention-to-treat set). Comparisons of the combined apitegromab dose (20 mg/kg and 10 mg/kg) versus placebo and the 20 mg/kg dose versus placebo were done with a mixed-effects model with repeated measurement. Safety was analysed in all participants who received at least one dose of apitegromab or placebo through evaluation of adverse events, physical examinations, vital signs and cardiac assessments, laboratory evaluations, and concomitant medications. SAPPHIRE is registered with ClinicalTrials.gov, NCT05156320, and is completed.

FINDINGS:

From March 28, 2022, to Sept 4, 2024, we enrolled 188 patients (156 in the population aged 2-12 years and 32 in the population aged 13-21 years); of whom 128 participants received apitegromab and 60 participants received placebo. At 12 months, least squares mean difference in HFMSE change from baseline was 1·8 (95% CI 0·30 to 3·32, p=0·019) points for participants aged 2-12 years receiving apitegromab versus placebo (least squares mean 0·6 vs -1·2). Least squares mean difference in HFMSE change from baseline was 1·4 (95% CI -0·34 to 3·13; p=0·11) for apitegromab 20 mg/kg versus placebo (least squares mean 0·2 vs -1·2). The incidence and severity of adverse events were similar between apitegromab and placebo, and consistent with spinal muscular atrophy and background spinal muscular atrophy therapy. The most frequently reported adverse events were pyrexia (apitegromab, 33 [26%] of 128 vs placebo, 17 [28%] of 60), nasopharyngitis (32 [25%] vs 14 [23%]), cough (30 [23%] vs 12 [20%]), vomiting (29 [23%] vs ten [17%]), upper respiratory tract infection (28 [22%] vs 18 [30%]), and headache (27 [21%] vs 12 [20%]). No patients discontinued due to adverse events.

INTERPRETATION:

Participants in the apitegromab treatment groups (combined 20 mg/kg and 10 mg/kg dose) achieved statistically significant improvements in motor function compared with placebo; however, the least squares mean difference was not significant between apitegromab 20 mg/kg and placebo. Overall, SAPPHIRE results build on findings from the phase 2 TOPAZ trial, showing improved motor function with a generally well tolerated safety profile, supporting the use of muscle-targeting therapy for spinal muscular atrophy.

FUNDING:

Scholar Rock.

161

项与 Apitegromab 相关的新闻（医药）

2026-05-11

·BioSpace

Published FDA rejections point to manufacturing, data gaps as key stumbling blocks

iStock, patpitchaya The FDA’s decision last year to make complete response letters public provides new insight into why therapies sometimes fail to get the regulatory greenlight. Analysts say the information could help sponsors refine their regulatory strategies. More than half of FDA rejections come down to concerns with manufacturing, according to a recent analysis of complete response letters made public by the agency. Meanwhile, 41% of rejections were due to poor product quality, including impurities or failed stability testing. This breakdown, from Jefferies analysts, was made possible by an initiative the FDA launched last year to publish complete response letters (CRLs) “promptly after they are issued to sponsors.” While the CRLs are often heavily redacted and their publication delayed due to legal review following rejections, analysts have hailed the initiative as a positive step toward greater visibility into its decisions. The agency released an initial trove of complete response letters (CRLs) last July, making publicly available the reasoning behind its rejection of more than 200 drug applications. Since then, the agency’s repository of CRLs—which it uploads onto openFDA —has grown to nearly 350 as of publication. FDA FDA Rejections: We Read 200 CRLs So You Don’t Have To A journey through the FDA’s newly released complete response letters gave glimpses into the journeys to market for Eli Lilly’s Alzheimer’s antibody Kisunla, Sarepta’s DMD gene therapy Vyondys 53 and Gilead’s HIV drug Sunlenca. July 11, 2025 · 5 min read · Annalee Armstrong Read more “We appreciate the increased transparency,” Jefferies analyst Andrew Tsai told BioSpace in an email, noting that making the contents of rejection letters available to the public—and shareholders—has made companies more accountable. It also helps to clear up communication inconsistencies. In an interview with BioSpace earlier this month, TD Cowen analyst Ritu Baral recalled one case in which an unnamed company told its investors that the FDA’s reasoning for rejecting its drug application was unclear. Once the agency made the CRLs public, however, Baral was able to see that the agency had been clear and consistent across its various communications with the company. The second CRL for Replimune’s RP1 also pointed to differing perspectives from the FDA and the company regarding previous agreements. While Replimune CEO Sushil Patel contended after the product’s first rejection in July 2025 that there was alignment on the design of the confirmatory study, the agency maintained in its second CRL , issued in April, that its advice “has remained consistent as evidenced by our communications dating back to March 2021 and subsequent interactions.” “There is proof with these letters that company/management commentary have differed from what actually happened,” Tsai said. Of course, the FDA’s CRL archive is imperfect, and it has its fair share of critics. In addition to delays and redactions—which Harpreet Singh, chief medical officer at Precision for Medicine said is an inherent limitation to transparency. BioSpace ‘s own review has found that the trove isn’t user-friendly. The files are dumped in a zipped folder for the reader to sort through, often one at a time. The new publication policy has also sparked concerns over commercial sensitivity and the impact on emerging biotechs, “whose valuations can suffer from public criticisms,” said Singh, a former division director of oncology at the FDA. Indeed, last month, another unnamed pharmaceutical company filed a citizen’s petition asking the agency to reform the new policy. Before releasing a CRL, the FDA should “first inform the manufacturer that it intends to do so,” and give the drug sponsor the opportunity to contest the publication within 10 days, according to the petition. Still, Singh said, the FDA’s CRL initiative “has significantly improved sponsor accountability and investor communication,” while also reducing the ambiguity around the agency’s decisions. FDA FDA’s CRL transparency policy is boosting biopharma accountability Since the FDA began publishing its rejections of drug approval filings in July last year, companies have become more forthcoming about the details of agency decisions in their own disclosures, according to biopharma and regulatory analysts. April 13, 2026 · 5 min read · Nick Paul Taylor Read more More importantly, she said, the CRL archive serves as an educational resource for sponsors, allowing them to learn from the missteps of their peers and potentially setting them up for more successful reviews. Common pitfalls revealed The rejection letters published so far have imparted a couple of key lessons that could help sponsors refine their regulatory strategies. “The majority—and perhaps the vast majority—of CRLs boil down to two main issues,” Mizuho analyst Graig Suvannavejh told BioSpace via email. The first are rejections brought about by manufacturing issues, and the second is deficiencies with the sponsors’ data packages, failing to conclusively demonstrate that products are worthy of approval. Over at Jefferies, Tsai and his team conducted a quantitative assessment of the regulator’s repository of rejection letters. Their analysis showed that indeed, more than half of CRLs cited concerns with manufacturing, primarily linked to facilities that fail FDA inspections. Relatedly, 41% of rejections were due to poor product quality, including impurities or failed stability testing. A recent rejection for a pre-filled syringe of Regeneron’s high-dose Eylea falls into this group. In its rejection letter, issued last October, the FDA pointed to violations at a production facility run by Novo Nordisk. An inspection from June 2025 to July 2025 revealed contamination with “atypical extrinsic particles”—the agency’s language for unwanted impurities in the plant, such as cat hair and pests. The Indiana-based plant also “failed to determine a root cause of the contamination, assess the potential impact to the rest of the lot, or evaluate whether similar issues may have occurred in upstream batches,” according to the inspection report. Issues at the same plant led to the rejection of Scholar Rock’s spinal muscular atrophy therapy in September 2025. Manufacturing Violations at Novo’s Indiana Plant ‘Unacceptable,’ FDA Says Slapped with the most severe post-inspection classification, the state of Novo Nordisk’s Indiana manufacturing site could pose a problem for clients, including Regeneron and Scholar Rock. October 14, 2025 · 2 min read · Tristan Manalac Read more Both Regeneron and Scholar Rock have since found ways to move past these manufacturing hiccups. Regeneron found an alternative filler and resubmitted its package for the pre-filled syringe, and had a PDUFA date in April. Scholar Rock has also refiled its application for apitegromab and has been granted an action date in September. Aside from manufacturing issues, insufficient clinical data also emerged as a key stumbling block for many drug sponsors, with Jefferies’ analysis showing that 27% of CRLs noted the need for more data. These requests can range from additional subgroup or pharmacodynamic analyses using existing data to the need for another Phase 3 study, according to Tsai. Such was the case in the FDA’s recent rejection of Replimune’s melanoma drug RP1—the second time the viral immunotherapeutic has failed to achieve approval. In its latest CRL, the agency deemed the company’s data package “insufficient to conclude substantial evidence of effectiveness” of RP1, particularly pointing to Replimune’s use of a single-arm trial. Complete response letters Second rejection for tumor destroyer puts Replimune on a ‘challenging path’ Replimune’s CEO Sushil Patel has already warned that the biotech will need to cut staff and substantially scale back its U.S. manufacturing operations. April 13, 2026 · 2 min read · Tristan Manalac Read more REGENXBIO also faced clinical roadblocks for its Hunter syndrome gene therapy RGX-121, which the FDA refused to approve in March. The rejection, according to the CRL, was driven by a series of study design problems, including the company’s use of external controls and its enrollment criteria, which the regulator said raised “uncertainty that the enrolled population is representative of the target population.” Both REGENXBIO and Replimune ran into what Mizuho’s Suvannavejh called the “worst-case scenario” for drug sponsors, in which the FDA “will simply ask for the conduct of a brand-new clinical trial altogether.” While these types of denials are typically catastrophic for a company’s shares—Replimune plunged 20% in the aftermath of its rejection, while REGENXBIO fell 12%—Suvannavejh said there’s often just no way around them. “The FDA needs to feel highly confident that any new drug that gets approved provides an overall benefit/risk pro is net positive for patients,” he said. Setting up for success On a positive note, the FDA’s trove of CRLs holds up a mirror to the industry, allowing companies to see the blind spots in their regulatory processes, experts who spoke with BioSpace agreed. For instance, Jefferies’ analysis suggests that most “sponsors are seemingly more prepared when it comes to the clinical data package side of things vs CMC/manufacturing,” Tsai said. Accordingly, he said that running multiple successful pre-approval inspections is of “key importance” for most drug sponsors, helping to minimize the likelihood of rejection. Contracting the services of former FDA reviewers could be helpful in this regard, Tsai suggested. Singh concurred, saying sponsors “should prioritize facility inspections for reinforcing robust quality management systems,” using “validated analytical methods and strong stability data” to back their manufacturing packages. Companies should also have independent assessors review their applications to determine gaps and other weaknesses well ahead of submission, she said. This advice is especially important for first-time or otherwise inexperienced drugmakers, according to Singh. These companies should “lean on external expertise,” she said, in addition to comprehensively planning their regulatory activities and meetings and keeping strict timelines. On the clinical front, both Singh and Tsai agreed that being on the same page with the FDA from the outset and keeping the agency abreast with a company’s development strategy and managing the regulator’s expectations are critical to avoid unexpected rejections. “Proactive, consistent FDA engagement throughout development is crucial,” Singh said. “Mapping out interactions, scheduling meetings, documenting feedback, and addressing concerns before filing reduces CRL rates.” These recommendations point to how industry can leverage the FDA’s new CRL publication policy to “learn from common failure patterns and strengthen their regulatory strategies for greater approval success,” Singh said. For Suvannavejh, however, the FDA’s publication of CRLs goes beyond how it can help sponsors, and indeed, even beyond the biopharma sphere. “This is much more about restoring credibility to an agency whose reputation both in the U.S. and abroad has been significantly damaged over the past two years,” he said. .responsive-container { display: flex; flex-wrap: wrap; border: 1px solid #ededed; font-family: Helvetica, Arial, Sans-Serif; padding: 20px 20px 10px 20px; } .column-left { flex: 1; max-width: 45%; padding: 20px 20px 10px 20px; } .column-right { flex: 2; padding: 20px 20px 10px 20px; } @media (max-width: 768px) { .column-left, .column-right { max-width: 100%; flex: 100%; padding: 10px 0; } } Subscribe to ClinicaSpace! 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基因疗法

2026-05-09

·LONGPORT

Scholar Rock 第一季度财报电话会议亮点

Scholar Rock（纳斯达克股票代码：SRRK）宣布，FDA 已接受其重新提交的生物制品许可申请（Biologics License Application），该申请针对治疗脊髓性肌萎缩症的药物 apitegromab，PDUFA 行动日期定于 2026 年 9 月 30 日。首席执行官 David Hallal 指出，申请中包括了两个制造地点以供批准。该公司正在为在获得批准后立即推出 apitegromab 做准备，商业准备工作正在进行中。此外，Scholar Rock 正在推进其欧洲监管流程，并在美国扩展其患者服务项目，以支持 SMA 治疗中心和患者 3 只被低估的股票在本财报季有反弹潜力 Scholar Rock NASDAQ: SRRK 表示，美国食品药品监督管理局已接受其重新提交的生物制品许可申请（BLA）以审查 apitegromab，这是一种针对脊髓性肌萎缩症儿童和成人的研究性治疗，并设定了 2026 年 9 月 30 日的 PDUFA 行动日期。董事长兼首席执行官 David Hallal 表示，接受的申请包括两个填充和封闭设施：Catalent 在印第安纳州的工厂和第二个美国本土设施。他表示，这为公司提供了 “两个独立的 apitegromab 批准路径”。此更新是在去年九月发出的完整回应信之后，Scholar Rock 表示该信件仅与对 Novo Nordisk 所拥有和运营的 Catalent 印第安纳州填充和封闭设施的例行一般现场检查的观察有关。Hallal 表示，FDA 此后已完成对该设施的未通知重新检查，并且根据 FDA 指南，机构有最多 90 天的时间来对该地点进行分类。 Hallal 表示，Scholar Rock 在三月底重新提交了 BLA，“与 FDA 完全一致”，以包括两个生产地点。公司表示，FDA 审查和潜在批准所需的 apitegromab 药物产品已从第二个设施提交，并预计在第三季度初可从该设施获得商业供应，提前于 9 月 30 日的行动日期。公司表示推出可能在 PDUFA 日期之前进行 Scholar Rock 高管们反复表示，公司准备在获得批准后立即推出 apitegromab，包括在正式的 PDUFA 日期之前。对此，Hallal 在回答分析师提问时表示，涉及制造问题的 II 类重新提交有时可以在截止日期之前获得行动。 “我们只知道我们需要做好准备，因为很多时候 II 类重新提交和行动可以在该 PDUFA 日期之前由机构采取，” Hallal 说。研发总裁 Akshay Vaishnaw 表示，公司预计 apitegromab 将在第三季度末之前获得针对儿童和成人 SMA 的批准，可能由一个或两个填充和封闭设施支持。 Vaishnaw 还讨论了欧洲的监管流程，表示公司的市场授权申请正在通过 EMA 审查。他表示，Scholar Rock 和 EMA 原定举行口头解释会议，但在双方提前达成一致后，该会议不再需要。公司仍然预计在年中接近 CHMP 意见。 Vaishnaw 指出，欧洲的批准还需要 FDA 对 Catalent 印第安纳州设施的批准。Hallal 表示，欧洲监管机构已被告知 FDA 过程，并在时间上 “非常灵活”。商业准备专注于 SMA 治疗中心和支付方首席运营官 Keith Woods 表示，Scholar Rock 的美国商业团队正在为 “在获得批准后立即” 推出做准备，这可能在 9 月 30 日之前的任何时间发生。他表示，肌肉力量和运动功能仍然是近十年来 SMN 目标治疗引入后未满足的主要需求，引用公司参考的数据，95% 的患者仍然经历持续和渐进的肌肉萎缩。 Woods 表示，Cure SMA 分享的数据表明，约三分之一的美国 SMA 患者接受了两种或更多 SMN 目标治疗，无论是顺序还是组合。他表示，公司将此视为未满足需求的证据，以及 apitegromab 作为肌肉靶向治疗的机会。公司表示，其美国现场团队已接触到约 140 个 SMA 治疗中心、2600 名开处方医生和多学科护理团队。Woods 表示，Scholar Rock 正在准备其患者服务项目 Scholar Rock Supports，以为患者、护理人员和提供者提供个性化支持。在问答环节中，Woods 表示，公司已扩大了护理地点规划，包括通过合作伙伴接入美国超过 10,000 名家庭输液护士。他还表示，Scholar Rock 已扩大其专科药房网络，以便患者不必为不同的 SMA 治疗使用多个专科药房。在欧洲，Woods 表示，Scholar Rock 已在瑞士建立了区域总部，并在德国设立了当地领导团队，预计在 EMA 批准后推出。他表示，公司已在德国招聘了医疗和商业现场团队，并正在招募患者参与同情用药计划。管道包括 SMA 婴儿、FSHD 和皮下项目除了待处理的 SMA 申请外，Vaishnaw 表示，Scholar Rock 正在继续招募和给患者施药于 II 期 OPAL 试验，该试验评估 apitegromab 在 2 岁以下婴儿和幼儿中的效果。该研究包括接受 SMN1 目标基因治疗或正在接受 SMN2 目标治疗的参与者。公司还准备启动一项随机、双盲、安慰剂对照的 II 期试验，名为 FORGE，针对面肩肱肌萎缩症（FSHD）。Vaishnaw 表示，该研究预计将很快开始招募，计划包括 60 名患者。他将 FSHD 描述为一种罕见的神经肌肉疾病，在美国和欧洲有超过 30,000 名确诊患者，且没有批准的治疗方案。 Vaishnaw 表示，Scholar Rock 根据未满足的需求、FLExDUX4 小鼠模型的前临床数据以及先前的随机研究表明，肌肉质量的增加可能与 FSHD 的功能益处相关，优先考虑 FSHD。 Scholar Rock 还在推进 apitegromab 的皮下制剂。Vaishnaw 表示，1 期数据显示，1 月份呈现出良好的生物利用度和与静脉给药相当的药效学特征。公司计划在 apitegromab 获得批准后，稍后与美国和欧洲监管机构进行接洽。公司还讨论了 SRK-439，在准备好的发言中被描述为一种新型高效能抗肌肉抑制素抗体，目前处于第一阶段。Vaishnaw 表示，健康志愿者研究中的给药正在进行中，预计今年晚些时候会有初步数据。第一季度现金状况增强首席财务官 Vikas Sinha 表示，Scholar Rock 在 2026 年第一季度结束时拥有 4.8 亿美元的现金及现金等价物和可交易证券。总额包括公司在 3 月份从现有债务融资中提取的 1 亿美元和本季度通过市场股权计划获得的 9800 万美元净现金收益。 Sinha 表示，第一季度的运营费用为 1.02 亿美元，扣除基于股票的补偿后，运营费用为 8400 万美元。他表示，Scholar Rock 在 2026 年的财务优先事项仍然集中在支持商业发布准备、资助研发项目以及评估加强资产负债表的机会。在 FDA 批准 apitegromab 后，Sinha 表示 Scholar Rock 将有机会从现有债务融资中提取额外的 1.5 亿美元。公司还计划变现优先审查凭证，以进一步增强其资产负债表。 Hallal 在电话会议结束时表示，Scholar Rock 专注于为生活在脊髓性肌萎缩症（SMA）中的儿童和成人提供其所描述的首个肌肉靶向疗法，同时建立更广泛的神经肌肉疾病管线。关于 Scholar Rock NASDAQ: SRRK Scholar Rock 是一家临床阶段的生物技术公司，专注于发现和开发选择性靶向参与疾病过程的生长因子的蛋白质治疗。公司的研究平台旨在以高特异性调节内源性生长因子的激活和信号传导，旨在恢复一系列疾病的正常生物功能。Scholar Rock 的方法以其强调工程化抗体和生物制剂与生长因子前体或潜在复合物的相互作用而非活性形式而有所区别，可能提供更好的安全性和有效性。公司的主导项目 SRK-015（appercept）是一种针对潜在肌肉抑制素前蛋白激活的研究性单克隆抗体，正在评估用于治疗脊髓性肌萎缩症（SMA）。进一步阅读我们更看好的五只股票比 Scholar Rock 更好此即时新闻提醒由叙事科学技术和 MarketBeat 的金融数据生成，以便为读者提供最快的报道和公正的覆盖。请将任何问题或评论发送至 contact@marketbeat.com。你现在应该投资 1000 美元在 Scholar Rock 吗？在你考虑投资 Scholar Rock 之前，你需要听听这个。 MarketBeat 每天跟踪华尔街顶级评级和表现最佳的研究分析师及其推荐给客户的股票。MarketBeat 已识别出五只顶级分析师正在悄悄建议客户现在购买的股票，以便在更广泛的市场注意到之前……而 Scholar Rock 并不在名单上。虽然 Scholar Rock 目前在分析师中获得了买入评级，但顶级分析师认为这五只股票更值得购买。在这里查看这五只股票 3 只被低估的股票在本财报季有反弹潜力 Scholar Rock NASDAQ: SRRK 表示，美国食品药品监督管理局已接受其重新提交的生物制品许可申请（BLA）以审查 apitegromab，这是一种针对脊髓性肌萎缩症儿童和成人的研究性治疗，并设定了 2026 年 9 月 30 日的 PDUFA 行动日期。董事长兼首席执行官 David Hallal 表示，接受的申请包括两个填充和封闭设施：Catalent 在印第安纳州的工厂和第二个美国本土设施。他表示，这为公司提供了 “两个独立的 apitegromab 批准路径”。此更新是在去年九月发出的完整回应信之后，Scholar Rock 表示该信件仅与对 Novo Nordisk 所拥有和运营的 Catalent 印第安纳州填充和封闭设施的例行一般现场检查的观察有关。Hallal 表示，FDA 此后已完成对该设施的未通知重新检查，并且根据 FDA 指南，机构有最多 90 天的时间来对该地点进行分类。 Hallal 表示，Scholar Rock 在三月底重新提交了 BLA，“与 FDA 完全一致”，以包括两个生产地点。公司表示，FDA 审查和潜在批准所需的 apitegromab 药物产品已从第二个设施提交，并预计在第三季度初可从该设施获得商业供应，提前于 9 月 30 日的行动日期。公司表示推出可能在 PDUFA 日期之前进行 Scholar Rock 高管们反复表示，公司准备在获得批准后立即推出 apitegromab，包括在正式的 PDUFA 日期之前。对此，Hallal 在回答分析师提问时表示，涉及制造问题的 II 类重新提交有时可以在截止日期之前获得行动。 “我们只知道我们需要做好准备，因为很多时候 II 类重新提交和行动可以在该 PDUFA 日期之前由机构采取，” Hallal 说。研发总裁 Akshay Vaishnaw 表示，公司预计 apitegromab 将在第三季度末之前获得针对儿童和成人 SMA 的批准，可能由一个或两个填充和封闭设施支持。 Vaishnaw 还讨论了欧洲的监管流程，表示公司的市场授权申请正在通过 EMA 审查。他表示，Scholar Rock 和 EMA 原定举行口头解释会议，但在双方提前达成一致后，该会议不再需要。公司仍然预计在年中接近 CHMP 意见。 Vaishnaw 指出，欧洲的批准还需要 FDA 对 Catalent 印第安纳州设施的批准。Hallal 表示，欧洲监管机构已被告知 FDA 过程，并在时间上 “非常灵活”。商业准备专注于 SMA 治疗中心和支付方首席运营官 Keith Woods 表示，Scholar Rock 的美国商业团队正在为 “在获得批准后立即” 推出做准备，这可能在 9 月 30 日之前的任何时间发生。他表示，肌肉力量和运动功能仍然是近十年来 SMN 目标治疗引入后未满足的主要需求，引用公司参考的数据，95% 的患者仍然经历持续和渐进的肌肉萎缩。 Woods 表示，Cure SMA 分享的数据表明，约三分之一的美国 SMA 患者接受了两种或更多 SMN 目标治疗，无论是顺序还是组合。他表示，公司将此视为未满足需求的证据，以及 apitegromab 作为肌肉靶向治疗的机会。公司表示，其美国现场团队已接触到约 140 个 SMA 治疗中心、2600 名开处方医生和多学科护理团队。Woods 表示，Scholar Rock 正在准备其患者服务项目 Scholar Rock Supports，以为患者、护理人员和提供者提供个性化支持。在问答环节中，Woods 表示，公司已扩大了护理地点规划，包括通过合作伙伴接入美国超过 10,000 名家庭输液护士。他还表示，Scholar Rock 已扩大其专科药房网络，以便患者不必为不同的 SMA 治疗使用多个专科药房。在欧洲，Woods 表示，Scholar Rock 已在瑞士建立了区域总部，并在德国设立了当地领导团队，预计在 EMA 批准后推出。他表示，公司已在德国招聘了医疗和商业现场团队，并正在招募患者参与同情用药计划。管道包括 SMA 婴儿、FSHD 和皮下项目除了待处理的 SMA 申请外，Vaishnaw 表示，Scholar Rock 正在继续招募和给患者施药于 II 期 OPAL 试验，该试验评估 apitegromab 在 2 岁以下婴儿和幼儿中的效果。该研究包括接受 SMN1 目标基因治疗或正在接受 SMN2 目标治疗的参与者。公司还准备启动一项随机、双盲、安慰剂对照的 II 期试验，名为 FORGE，针对面肩肱肌萎缩症（FSHD）。Vaishnaw 表示，该研究预计将很快开始招募，计划包括 60 名患者。他将 FSHD 描述为一种罕见的神经肌肉疾病，在美国和欧洲有超过 30,000 名确诊患者，且没有批准的治疗方案。 Vaishnaw 表示，Scholar Rock 根据未满足的需求、FLExDUX4 小鼠模型的前临床数据以及先前的随机研究表明，肌肉质量的增加可能与 FSHD 的功能益处相关，优先考虑 FSHD。 Scholar Rock 还在推进 apitegromab 的皮下制剂。Vaishnaw 表示，1 期数据显示，1 月份呈现出良好的生物利用度和与静脉给药相当的药效学特征。公司计划在 apitegromab 获得批准后，稍后与美国和欧洲监管机构进行接洽。公司还讨论了 SRK-439，在准备好的发言中被描述为一种新型高效能抗肌肉抑制素抗体，目前处于第一阶段。Vaishnaw 表示，健康志愿者研究中的给药正在进行中，预计今年晚些时候会有初步数据。第一季度现金状况增强首席财务官 Vikas Sinha 表示，Scholar Rock 在 2026 年第一季度结束时拥有 4.8 亿美元的现金及现金等价物和可交易证券。总额包括公司在 3 月份从现有债务融资中提取的 1 亿美元和本季度通过市场股权计划获得的 9800 万美元净现金收益。 Sinha 表示，第一季度的运营费用为 1.02 亿美元，扣除基于股票的补偿后，运营费用为 8400 万美元。他表示，Scholar Rock 在 2026 年的财务优先事项仍然集中在支持商业发布准备、资助研发项目以及评估加强资产负债表的机会。在 FDA 批准 apitegromab 后，Sinha 表示 Scholar Rock 将有机会从现有债务融资中提取额外的 1.5 亿美元。公司还计划变现优先审查凭证，以进一步增强其资产负债表。 Hallal 在电话会议结束时表示，Scholar Rock 专注于为生活在脊髓性肌萎缩症（SMA）中的儿童和成人提供其所描述的首个肌肉靶向疗法，同时建立更广泛的神经肌肉疾病管线。关于 Scholar Rock NASDAQ: SRRK Scholar Rock 是一家临床阶段的生物技术公司，专注于发现和开发选择性靶向参与疾病过程的生长因子的蛋白质治疗。公司的研究平台旨在以高特异性调节内源性生长因子的激活和信号传导，旨在恢复一系列疾病的正常生物功能。Scholar Rock 的方法以其强调工程化抗体和生物制剂与生长因子前体或潜在复合物的相互作用而非活性形式而有所区别，可能提供更好的安全性和有效性。公司的主导项目 SRK-015（appercept）是一种针对潜在肌肉抑制素前蛋白激活的研究性单克隆抗体，正在评估用于治疗脊髓性肌萎缩症（SMA）。进一步阅读我们更看好的五只股票比 Scholar Rock 更好此即时新闻提醒由叙事科学技术和 MarketBeat 的金融数据生成，以便为读者提供最快的报道和公正的覆盖。请将任何问题或评论发送至 contact@marketbeat.com。你现在应该投资 1000 美元在 Scholar Rock 吗？在你考虑投资 Scholar Rock 之前，你需要听听这个。 MarketBeat 每天跟踪华尔街顶级评级和表现最佳的研究分析师及其推荐给客户的股票。MarketBeat 已识别出五只顶级分析师正在悄悄建议客户现在购买的股票，以便在更广泛的市场注意到之前……而 Scholar Rock 并不在名单上。虽然 Scholar Rock 目前在分析师中获得了买入评级，但顶级分析师认为这五只股票更值得购买。在这里查看这五只股票

2026-05-07

·Business Wire

Scholar Rock Reports First Quarter 2026 Financial Results and Recent Business Highlights

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company dedicated to improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular diseases by applying its leading platform in myostatin biology to advance musculoskeletal health, today reported financial results for the first quarter ended March 31, 2026, and provided an update on recent company developments. “With the FDA’s acceptance of our apitegromab BLA, we have achieved another critical milestone as we work with urgency to deliver on our mission to bring the world’s first muscle-targeted treatment to the SMA community,” said David L. Hallal, Chairman and Chief Executive Officer of Scholar Rock. “We are grateful for the FDA’s continued high level of engagement, and we are pleased that important progress continues to be made at both of our fill-finish facilities. Our U.S. commercial team stands ready to launch apitegromab on or at any time prior to the September 30th PDUFA date.” Mr. Hallal continued, “Our balance sheet is strong, our clinical-stage pipeline continues to advance, and we are poised, now more than ever, to usher in the next phase of innovation for patients with SMA.” Business Highlights and Upcoming Milestones Apitegromab Apitegromab is an investigational fully human monoclonal antibody designed to inhibit myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. It is the first and only muscle-targeted therapeutic candidate in SMA to demonstrate a statistically significant and clinically meaningful benefit in a pivotal Phase 3 clinical trial (SAPPHIRE). SMA Program Apitegromab BLA accepted by FDA with September 30, 2026 PDUFA action date. The accepted BLA includes two fill-finish facilities, Catalent Indiana and a second U.S.-based fill-finish facility. FDA completed reinspection of Catalent Indiana. Following the FDA’s acceptance of the apitegromab BLA, the Agency completed reinspection of Catalent Indiana. In accordance with FDA guidelines, classification of the facility is anticipated within 90 days following reinspection. Significant progress continues at second fill-finish facility. Apitegromab commercial supply from this facility is expected to be available early in the third quarter of 2026. Preparations ongoing for U.S. commercial launch. The Commercial team continues to expand its reach and engagement with key stakeholders, including a significant presence at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which was held March 8 – 11, 2026 in Orlando, FL and at the upcoming 2026 Annual SMA Conference, which is being held June 25 – 28, 2026 in Orlando, FL. The U.S. commercial team is prepared to launch apitegromab immediately upon FDA approval. European Medicines Agency (EMA) regulatory review ongoing. A Committee for Medicinal Products for Human Use (CHMP) opinion for the apitegromab Marketing Authorisation Application (MAA) is anticipated near mid-2026. The Scholar Rock team in Europe continues to engage with key stakeholders on SMA disease awareness and education initiatives, including at the 5 th International Scientific Congress on SMA, which was held March 11 – 14, 2026 in Budapest, Hungary. The Company is planning for an apitegromab launch in Europe in the second half of 2026, beginning with Germany. Enrollment progressing in Phase 2 OPAL clinical trial . Patients continue to be enrolled and dosed in the Phase 2 OPAL study (NCT07047144). The trial is evaluating apitegromab in infants and toddlers with SMA under two years of age who have received an approved SMN1-targeted gene therapy or who are receiving ongoing treatment with an approved SMN2-targeted therapy. Subcutaneous apitegromab development continues to progress. Scholar Rock is advancing a subcutaneous formulation of apitegromab intended to provide optionality for patients as a small volume, self- or caregiver-administered anti-myostatin antibody suitable for an autoinjector. A Phase 1 study in healthy volunteers has been completed. Further development activities are ongoing, including anticipated FDA and EMA regulatory engagements following apitegromab approvals. FSHD Program Phase 2 FORGE trial on track for initiation in mid-2026. Scholar Rock is developing apitegromab for the treatment of people with facioscapulohumeral muscular dystrophy (FSHD). FSHD is a rare, progressive neuromuscular disease characterized by muscle atrophy and functional decline, affecting approximately 30,000 individuals across the U.S. and Europe. Initiation of a Phase 2 randomized, double-blind, placebo-controlled trial, called FORGE, is expected in mid-2026. SRK-439 SRK-439 is a novel, investigational, subcutaneously administered myostatin inhibitor that binds to pro- and latent myostatin with high affinity and selectivity (i.e., no GDF11 or Activin A binding). Based on preclinical data, SRK-439 has the potential to potently inhibit myostatin and increase muscle mass. Phase 1 healthy volunteer study ongoing. A Phase 1 study evaluating SRK-439 in healthy volunteers is underway, with topline data expected in the second half of 2026. First Quarter 2026 Financial Results Scholar Rock reported a net loss of $105.5 million, including stock-based compensation of $18.2 million, for the quarter ended March 31, 2026, compared to a net loss of $74.7 million, including stock-based compensation of $13.4 million, for the quarter ended March 31, 2025. Net loss per common share was $0.83 for the quarter ended March 31, 2026, compared to $0.67 per common share for the quarter ended March 31, 2025. The Company did not record any revenue for the quarters ended March 31, 2026 and 2025. Research and development expense was $51.8 million, including $6.5 million in stock-based compensation, for the quarter ended March 31, 2026, compared to $48.7 million, including $4.0 million in stock-based compensation, for the quarter ended March 31, 2025. General and administrative expense was $50.2 million, including $11.7 million in stock-based compensation, for the quarter ended March 31, 2026, compared to $28.4 million, including $9.4 million in stock-based compensation, for the quarter ended March 31, 2025. As of March 31, 2026, Scholar Rock had cash, cash equivalents, and marketable securities of $479.9 million. This reflects a drawdown of $100.0 million from the Company’s debt facility and net cash proceeds of $98.0 million from the Company’s at-the-market (ATM) program. Conference Call Information Scholar Rock will host a conference call and webcast today, Thursday, May 7, at 8:00 a.m. ET to review its first quarter 2026 financial results and discuss recent business updates. To access the live audio webcast, please go to “Events and Presentations” in the Investors section of the Scholar Rock website at http://investors.scholarrock.com. To participate via telephone, please register in advance here. Upon registration, all telephone participants will receive a confirmation email detailing how to join the conference call. A replay of the webcast will be available on the Company’s website for approximately 90 days. About Scholar Rock Scholar Rock is a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for children and adults with spinal muscular atrophy (SMA) and other rare, severe and debilitating neuromuscular diseases. As a global leader in myostatin biology, a field focused on proteins that regulate muscle mass, the biopharmaceutical company is named for the visual resemblance of a scholar rock to protein structures. Our commitment to unlock fundamentally different treatment 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 works every day to create new possibilities for patients through its highly innovative anti-myostatin program, including opportunities in additional rare neuromuscular diseases. Learn more at ScholarRock.com and follow @ScholarRock on X and on LinkedIn. Scholar Rock® is a registered trademark of Scholar Rock, Inc. Availability of Other Information About Scholar Rock 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 X (formerly known as Twitter) and LinkedIn. The information that we post on our website or on X 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. Forward-Looking Statements 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 and development programs for apitegromab, including its subcutaneous formulation, SRK-439 and its preclinical programs, and indication selection and development timing, including the timing of any regulatory submissions, decisions and anticipated approvals, the therapeutic potential, clinical benefits and safety of any product candidates, its ability to address the observations identified in the complete response letter, expectations regarding actions by the FDA after its reinspection of the Catalent Indiana facility; the expected timing and outcome of FDA review of the accepted BLA for apitegromab, including the September 30, 2026 PDUFA action date; expectations regarding the availability and timing of commercial supply of apitegromab from Catalent Indiana and a second U.S.-based fill-finish facility, including expected supply from the second fill-finish facility; expectations regarding commercial launch timing, and 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, the potential of its product candidates and proprietary platform. 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. These risks and uncertainties include, without limitation, whether preclinical and clinical data, including the results from the Phase 3 SAPPHIRE trial and any results from ongoing or future clinical trials, including the Phase 2 OPAL clinical trial, the Phase 2 FORGE trial and the Phase 1 clinical trial of SRK-439, will be sufficient to support regulatory approval or further development; that preclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, data from any ongoing or future trials of apitegromab or data for SRK-439, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidates; whether the FDA will accept the remediations to the Catalent Indiana fill finish facility in response to the FDA Observations, whether the updated BLA will be sufficient to support regulatory approval, Scholar Rock’s ability to manage expenses or provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; and Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2026, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law. 2026 2025 $ 51,814 $ 48,678 50,202 28,412 102,016 77,090 (102,016 ) (77,090 ) (3,494 ) 2,367 $ (105,510 ) $ (74,723 ) $ (0.83 ) $ (0.67 ) 127,277,144 111,838,272 Scholar Rock Holding Corporation Condensed Consolidated Balance Sheets (unaudited) (in thousands) $ 479,944 $ 367,563 35,340 17,584 515,284 385,147 19,970 19,125 $ 535,254 $ 404,272 $ 59,643 $ 55,419 199,598 103,365 259,241 158,784 276,013 245,488 $ 535,254 $ 404,272

临床1期临床2期财报临床3期上市批准

100 项与 Apitegromab 相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
-	-	-	DB16096

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适应症	最高研发状态	国家/地区	公司	日期
脊髓性肌萎缩	申请上市	美国	Scholar Rock, Inc.	2025-01-30
萎缩	临床3期	美国	Scholar Rock, Inc.	2022-04-14
萎缩	临床3期	比利时	Scholar Rock, Inc.	2022-04-14
萎缩	临床3期	法国	Scholar Rock, Inc.	2022-04-14
萎缩	临床3期	德国	Scholar Rock, Inc.	2022-04-14
萎缩	临床3期	意大利	Scholar Rock, Inc.	2022-04-14
萎缩	临床3期	荷兰	Scholar Rock, Inc.	2022-04-14
萎缩	临床3期	波兰	Scholar Rock, Inc.	2022-04-14
萎缩	临床3期	西班牙	Scholar Rock, Inc.	2022-04-14
萎缩	临床3期	英国	Scholar Rock, Inc.	2022-04-14

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT06445075 (EMBRAZE, Company_Website) 人工标引	临床2期	体重下降	100	ApitegromabApitegromab 10mg/kg + Tirzepatide	遞顧壓築鏇簾廠糧膚艱(鑰糧醖選襯製齋窪蓋積) = 艱獵糧遞餘構鏇選選築顧繭夢繭膚繭築餘蓋鹽 (鹽衊糧選觸鬱製襯製襯, 1.25) 达到更多	积极	2025-06-18
				Placebo + Tirzepatide	遞顧壓築鏇簾廠糧膚艱(鑰糧醖選襯製齋窪蓋積) = 壓製齋獵鹽廠醖網憲廠顧繭夢繭膚繭築餘蓋鹽 (鹽衊糧選觸鬱製襯製襯, 1.14) 达到更多
NCT05156320 (SAPPHIRE, MDA2025)	临床3期	脊髓性肌萎缩	188	Apitegromab	蓋齋醖遞網淵鑰網餘夢(範鬱廠餘蓋餘製願膚簾): Difference (Mean) = 1.8, P-Value = 0.0192 更多	积极	2025-03-16
				Placebo
NCT05156320 (SAPPHIRE, PipelineReview) 人工标引	临床3期	脊髓性肌萎缩	188	Apitegromab (2-12 years + 10 mg/kg and 20 mg/kg)	憲範製壓鹹窪鑰願衊蓋(選膚範壓鏇簾鏇鹽繭鑰): Difference = 1.8, P-Value = 0.0192 达到	积极	2024-10-08
				Placebo (2-12 years)
NCT03921528 (TOPAZ \| TOPAZ Study, CTgov)	临床2期	青少年脊髓性肌萎缩症 \| 萎缩 \| II型脊髓性肌萎缩	58	SRK-015	顧網鑰鹽獵鬱衊簾艱繭(廠艱淵糧積襯夢蓋廠獵) = 鬱鏇齋範觸顧獵範膚齋衊築顧壓鹹鹽蓋網鬱顧 (選觸鹹觸艱選鬱積積憲, 3.5) 更多	-	2022-11-17
NCT03921528 (TOPAZ, AAN2022)	临床2期	脊髓性肌萎缩 serum latent myostatin	-	Apitegromab 2mg/kg	窪顧鑰鏇衊鬱憲範艱範(簾壓壓蓋餘遞鏇鬱醖網) = 鬱餘襯獵夢製鑰範醖襯獵製蓋蓋網範觸膚鏇襯 (簾淵醖構襯顧範艱憲獵 )	积极	2022-05-03
				Apitegromab 20mg/kg	窪顧鑰鏇衊鬱憲範艱範(簾壓壓蓋餘遞鏇鬱醖網) = 觸積窪鹹觸觸願齋繭觸獵製蓋蓋網範觸膚鏇襯 (簾淵醖構襯顧範艱憲獵 ) 更多
TOPAZ (EAN2021)	临床2期	脊髓性肌萎缩	-	Apitegromab 20mg/kg monotherapy	鏇鬱鏇積壓醖憲窪顧選(鬱築壓鑰窪膚繭鏇廠夢) = 膚繭製遞艱衊膚窪構顧夢觸網觸繭築鹽夢製膚 (醖鹽壓顧襯餘範積鹽選, -1.1 ~ 2.2)	积极	2021-06-18
				Apitegromab 20mg/kg as adjunctive treatment to nusinersen	鏇鬱鏇積壓醖憲窪顧選(鬱築壓鑰窪膚繭鏇廠夢) = 顧獵鑰選鏇簾顧齋觸積夢觸網觸繭築鹽夢製膚 (醖鹽壓顧襯餘範積鹽選, 0.1 ~ 2.7)