Scholar Rock contends its experimental drug for spinal muscular atrophy can achieve what none of the currently available therapies can do to treat this rare disease. After positive data from a pivotal study sent the biotech’s shares skyward, Scholar Rock has raised $300 million from a stock offering that will be applied toward regulatory submissions and launch preparation of what could become its first commercialized product..

。在一项关键研究的积极数据使该生物技术公司的股票飙升后，Scholar Rock通过股票发行筹集了3亿美元，将用于监管提交和启动可能成为其第一个商业化产品的准备工作。。

The stock sale follows the company’s Phase 3 data readout earlier this week showing its drug, apitegromab, met the goal of showing muscle function improvement in spinal muscular atrophy (SMA). The Cambridge, Massachusetts-based biotech is planning regulatory submissions to the FDA and the European Medicines Agency early next year..

本周早些时候，该公司公布了第三阶段数据，显示其药物阿匹曲单抗达到了显示脊髓性肌萎缩症（SMA）肌肉功能改善的目标。。。

SMA stems from mutations to the gene that codes for survival motor neuron (SMN), a protein key to keeping motor neurons healthy. Patients who have the rare inherited disease develop progressively worsening muscle weakness. Few therapies are available for SMA. Biogen was first with Spinraza, an antisense oligonucleotide intended to increase production of functional SMN protein.

SMA源于编码存活运动神经元（SMN）的基因突变，SMN是保持运动神经元健康的关键蛋白质。患有罕见遗传病的患者肌肉无力逐渐恶化。很少有治疗SMA的方法。Biogen首先使用Spinraza，这是一种反义寡核苷酸，旨在增加功能性SMN蛋白的产生。

Novartis offers a one-time treatment with Zolgensma, a gene therapy that replaces the defective gene with a functioning one. Evrysdi, a drug developed by PTC Therapeutics and commercialized by Roche, offers another approach. This oral small molecule is designed to get the SMN2 gene to produce more SMN protein..

诺华提供Zolgensma一次性治疗，Zolgensma是一种用功能基因替代缺陷基因的基因疗法。Evrysdi是一种由PTC Therapeutics开发并由罗氏商业化的药物，它提供了另一种方法。这种口服小分子旨在获得SMN2基因以产生更多的SMN蛋白。。

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Scholar Rock takes a different and perhaps complementary approach to available SMA therapies. Treatments that target SMN prevent further degeneration of motor neurons, but they do not directly affect muscle atrophy, the company said in an investor presentation. Apitegromab is a monoclonal antibody designed to block activation of myostatin, a protein in skeletal muscle that inhibits muscle growth.

Scholar Rock对可用的SMA疗法采取了不同且可能互补的方法。该公司在投资者介绍中表示，针对SMN的治疗可以防止运动神经元进一步退化，但不会直接影响肌肉萎缩。Apitegromab是一种单克隆抗体，旨在阻断肌肉生长抑制素的激活，肌肉生长抑制素是骨骼肌中抑制肌肉生长的蛋白质。

It’s administered as an intravenous infusion every four weeks. The placebo-controlled Phase 3 test evaluated the study drug in patients who were already taking the standard chronic SMA therapies Spinraza or Evrysdi. This trial enrolled 188 SMA patients ages 2 to 21..

每四周静脉注射一次。安慰剂对照的3期试验评估了已经服用标准慢性SMA疗法Spinraza或Evrysdi的患者的研究药物。该试验招募了188名2至21岁的SMA患者。。

In preliminary results reported Monday, Scholar Rock said the drug achieved statistically significant and clinically meaningful improvement at 12 months, measured according to a scale used to assess muscle function in SMA patients (a higher score indicates better muscle function). The company added that 30% of patients who received the study drug showed a 3 point or greater improvement in score compared to 12.5% of patients in the placebo group.

在周一公布的初步结果中，学者洛克（Scholar Rock）表示，根据用于评估SMA患者肌肉功能的量表（分数越高表示肌肉功能越好），该药物在12个月时取得了统计学上显着且具有临床意义的改善。。

Early motor function improvement was observed at eight weeks and that benefit expanded at week 52, as measured according to the rating scale. The drug was well tolerated and no serious adverse events were reported..

。该药物耐受性良好，未报告严重不良事件。。

“The results clearly demonstrate robust and clinically meaningful improvement in motor function in patients with SMA,” Scholar Rock CEO Jay Backstrom said in a prepared statement. “At Scholar Rock, we are working with urgency to deliver the potentially transformative benefits of apitegromab to children and adults with SMA in the U.S., Europe, and around the world.”.

Scholar Rock首席执行官Jay Backstrom在一份准备好的声明中表示：“研究结果清楚地表明，SMA患者的运动功能得到了有力且具有临床意义的改善。”。“在Scholar Rock，我们正在紧急工作，为美国，欧洲和世界各地患有SMA的儿童和成人提供apitegromab潜在的变革性益处。”。

The apitegromab results will be presented on Friday during the Annual Congress of the World Muscle Society meeting in Prague. In the investor presentation, Scholar Rock claimed its drug is the first and only muscle-targeted therapy to show both clinically meaningful and statistically significant functional improvement in SMA.

apitegromab的研究结果将于周五在布拉格举行的世界肌肉协会年会上公布。在投资者介绍中，Scholar Rock声称其药物是第一种也是唯一一种在SMA中显示出临床意义和统计学显着功能改善的肌肉靶向疗法。

The company added that apitegromab is the first and only anti-myostatin therapy to show muscle improvement in a Phase 3 study. Biohaven might have something to say about that soon. That company’s experimental SMA therapy taldefgrobep alfa, an inhibitor of both myostatin and the activin A pathway, is expected to post Phase 3 data by the end of this year..

该公司补充说，apitegromab是第一个也是唯一一个在3期研究中显示肌肉改善的抗肌肉生长抑制素疗法。Biohaven可能很快就会有话要说。该公司的实验性SMA疗法taldefgrobep alfa是一种肌肉生长抑制素和激活素A途径的抑制剂，预计将于今年年底发布第三阶段数据。。

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Accelerating Claim Processing: Strategies to Shorten the Life of a Claim

加速索赔处理：缩短索赔寿命的策略

These strategies and practices can significantly shorten the life cycle of claims, leading to quicker resolutions and improved financial outcomes.

这些战略和做法可以大大缩短索赔的生命周期，从而更快地解决问题，改善财务成果。

By Greenway Health

绿道健康

Shares of Scholar Rock soared more than 300% after the biotech’s data release, and the company seized on the opportunity to raise money. Late Tuesday, Scholar Rock priced an offering of more than 10.2 million shares at $28.25 each. The offering includes pre-funded warrants to purchase 353,983 shares at the public offering price.

Scholar Rock的股价在生物技术公司发布数据后飙升了300%以上，该公司抓住机会筹集资金。周二晚些时候，学者摇滚（Scholar Rock）将1020多万股股票的发行定价为每股28.25美元。本次发行包括以公开发行价格购买353983股股票的预支认股权证。

Scholar Rock has also granted underwriters a 30-day option to purchase up to 1.5 million shares of stock at the public offering price, which could raise $42 million more..

Scholar Rock还授予承销商30天的期权，以公开发行价格购买至多150万股股票，这可能会再筹集4200万美元。。

Scholar Rock said regulatory submissions for apitegromab in the U.S. and Europe are planned for the first quarter of next year, setting the stage for a potential fourth quarter 2025 commercial launch. Scholar Rock sees additional potential applications for apitegromab. The company is evaluating the drug as a way to help preserve muscle in patients taking a GLP-1 drug for weight loss.

学者洛克（Scholar Rock）表示，计划于明年第一季度在美国和欧洲提交apitegromab的监管报告，为2025年第四季度可能的商业推出奠定基础。Scholar Rock发现了apitegromab的其他潜在应用。该公司正在评估该药物，以帮助服用GLP-1减肥药的患者保持肌肉。

Muscle loss is a known side effect of the GLP-1 drug class, leading a growing number of companies to try and address the complication. Late last month, BioAge Labs’ IPO raised more than $200 million to continue clinical development of an oral small molecule intended to preserve muscle mass. A Phase 2 study is underway evaluating the BioAge drug in combination with Eli Lilly obesity drug Zepbound; preliminary results are expected in the third quarter of 2025.

肌肉损失是GLP-1类药物的已知副作用，导致越来越多的公司尝试解决这种并发症。上个月底，BioAge实验室首次公开募股筹集了2亿多美元，以继续开发一种旨在保持肌肉质量的口服小分子。正在进行一项2期研究，评估BioAge药物与礼来肥胖症药物Zepbound的联合应用；初步结果预计将在2025年第三季度公布。

By then, Scholar Rock should already have topline results for the obesity study, which on track to post data in the second quarter of 2025..

到那时，学者洛克（Scholar Rock）应该已经有了肥胖研究的最终结果，该研究有望在2025年第二季度发布数据。。

In the prospectus, Scholar Rock said proceeds from the offering combined with its existing capital will be used to support commercialization of apitegromab and to advance other programs. The company estimates that the stock offering provides enough capital to fund operations into the fourth quarter of 2026..

Scholar Rock在招股说明书中表示，此次发行的收益加上其现有资本将用于支持apitegromab的商业化以及推进其他项目。该公司估计，此次股票发行为2026年第四季度的运营提供了足够的资金。。

Image: koto_feja, Getty Images

图片来源：koto_feja，盖蒂图片社

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Clinical Trials

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rare disease

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spinal muscular atrophy

脊髓性肌萎缩症

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