Scholar Rock, Inc.

1. 钟南山院士揭示慢阻肺和肺癌危害大，两项检查有助早发现 钟南山院士在首届中国呼吸健康大会上表示，慢阻肺和肺癌危害大，早期筛查是关键。他提倡将肺功能检测纳入常规体检，并利用低剂量螺旋CT结合AI技术提高肺癌早期诊断率，以降低死亡率。同时，强调提高基层医生对呼吸系统疾病早筛早治的认知及水平。 2. 全球首个“O+Y”双免疫抗癌疗法在中国获批，疾病进展或死亡风险大幅降低79% 全球首个“O+Y”双免疫疗法在中国获批，用于MSI-H/dMMR结直肠癌一线治疗，可降低79%的疾病进展或死亡风险，该适应症全球首次获批。 3. “减肥神药”引发肌肉流失，再生元制药等生物技术公司研发新疗法 “减肥神药”导致肌肉流失问题受关注，再生元等生物技术公司研发新药以减少肌肉流失，礼来、Biohaven和Scholar Rock等公司也在开发相关药物，临床试验正在进行。 4. 康方生物再融资近20亿港元，意在推动全球临床开发 康方生物通过配售融资19.4亿港元，用于核心产品全球临床开发等，此次配售为其历史最高单价和总额。 5. 出资1亿元购买“北京大兴”土地使用权 锦波生物出资1亿元购买北京大兴土地使用权，用于人源化胶原蛋白数据库与产品开发。该公司是重组人源化胶原蛋白领军企业，已完成多型基础研究，并有产品获批上市。同时，锦波生物专注功能蛋白研究与产业化，拥有多项国家发明专利。 6. 太美医疗科技再跌超11%，上市5日股价接近腰斩，公司三年累亏超12亿元 太美医疗科技港股股价大跌，上市5日跌近50%，三年亏损超12亿元，但营收稳步增长，是中国医药及医疗器械数字化解决方案供货龙头。 本文仅用于学术分享，转载请注明出处。若有侵权，请联系微信：bioonSir 删除或修改！

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。 2024年的最后一个季度将有多款候选药物的临床数据揭晓，针对适应症包括肥胖症、抑郁症、阿尔茨海默症、疼痛管理、肌萎缩症、精神分裂症和多发性硬化症。药物模态则涵盖小分子、单抗、蛋白、CAR-T、多肽，以及一种非常特殊的抗体-多肽偶联物。 安进Peptibody减肥药MariTIDE 在百舸争流的肥胖症开发领域中，安进的候选药MariTide（Maridebart Cafraglutide）非常显眼。这是因为它非常特殊的抗体-多肽偶联物（也称作Peptibody）和别具一格的作用机制。MariTide分子是将两个GLP-1受体激动剂多肽偶联到一个抗GIP（Glucose-dependent Insulinotropic Peptide，葡萄糖依赖性促胰岛素多肽，亦称“抑胃肽”）抗体之上，组成一个三部分缀合的抗体多肽偶联物。其中GLP-1受体激动剂负责激活GLP-1受体，刺激胰岛素分泌，而抗GIP抗体负责下调GIP的分泌，两股力量协同在一起，实现可控的减肥目标（图1）。 图1. MariTide分子结构以及作用机理示意图。（图片来源：Amgen） MariTide将在今年年底之前公布II期临床结果，在比较和评估3 种选定剂量的MariTide与安慰剂相比在第52 周诱导和维持体重减轻的效果，对象包括超重或肥胖但不患有糖尿病的参与者（队列A）和超重或肥胖且患有2 型糖尿病的参与者（队列B）。整个行业对此项研究结果高度关注。 诺和诺德新重磅减肥药CagriSema 诺和诺德将在今年年底前公布其减肥候选药物CagriSema 的III期研究结果。CagriSema是 GLP-1药物Semaglutide（即Ozempic和 Wegovy中的活性成分）和cagrilintide（胰淀素类似物）的组合。今年即将公布的III期研究是2.4 mg Semaglutide和2.4 mg cagrilintide剂量的疗法结果。除此之外，诺和诺德还在今年夏天开始了一项新的CagriSema III期试验，测试降低的剂量，即1.0 mg segmaglutide/1.0 mg cagrilintide和1.7 mg semaglutide/1.7 mg cagrilintide的减肥效果，给药频率为每周两次。新III期研究的结果估计将于2026年公布。 艾伯维精神分裂症药物emraclidine 百时美施贵宝的Cobenfy今年九月份成为了首个获得FDA批准的靶向大脑毒蕈碱受体的精神分裂症药物。与此同时，艾伯维正在积极开发一种同类型的竞争药物，名为emraclidine。该候选药是艾伯维去年87亿美元 收购Cerevel Therapeutics带来的，目前正在对精神分裂症和阿尔茨海默病精神病进行测试。同刚刚获批的Cobenfy一样，emraclidine也是选择性M4毒蕈碱受体正变构调节剂。与主要针对多巴胺受体拮抗作用的传统抗精神病药物不同，emraclidine调节毒蕈碱通路，可能提供抗精神病作用且副作用更少。其精神分裂症关键临床试验结果将在今年年底前完成。 脊髓性肌萎缩症单抗候选药apitegromab Scholar Rock 正在开发一种名为apitegromab的单抗药物，阻断肌生长抑制素（myostatin）的前体蛋白形式，肌生长抑制素是一种阻碍肌肉生长的蛋白质，限制骨骼肌细胞的增殖和分化，以防止肌肉过度生长。Apitegromab主要适应症是脊髓性肌萎缩症（SMA, spinal muscular atrophy）。Apitegromab的一项3 期安慰剂对照研究评估apitegromab 对2型和 3 型 无法独立行走的SMA患者的效果，该研究将于本季度公布结果。 脊髓性肌萎缩症 (SMA)是一种影响运动神经元（控制随意肌运动的神经细胞）的疾病。这些细胞位于脊髓中。由于肌肉无法对来自神经的信号作出反应，它们会因不活动而萎缩（变弱和萎缩）。每6000 名婴儿中就有一名患有SMA。它是影响幼儿的最普遍的遗传性疾病之一，也是婴儿死亡的主要原因。 Biohaven脊髓性肌萎缩症药物BHV-2000 Biohaven Pharmaceuticals 也在开发一种肌生长抑制素阻断药物taldefgrobep alfa（又名BHV-2000）。Taldefgrobep alfa (BHV-2000) 是一种完全人类重组蛋白，专门用于抑制肌生长抑制素和激活素A 的信号传导，这两种物质都是肌肉和脂肪组织的关键调节剂。Taldefgrobep可与肌生长抑制素结合，并充当激活素2b 受体拮抗剂。Taldefgrobep的新型作用机制有可能显著减少脂肪量、增加瘦肉量并改善多种代谢参数。Taldefgrobep alfa的III期结果将于年底前公布。 Vertex非阿片类止痛药期待突破 Vertex Pharmaceuticals 的下一个重大增长点可能是非阿片类止痛药suzetrigine（又名VX-548）。FDA正在审查suzetrigine用于急性疼痛的疗效，预计将于明年1 月 30日做出决定。在此之前，Vertex将在 12月公布一项针对慢性疼痛的II期研究结果，该研究针对腰骶神经根病（俗称坐骨神经痛）。 止痛药领域是相当多非阿片类药物的滑铁卢，suzetrigine如今有可能扭转这种局面。Suzetrigine的最大机遇可能在慢性疼痛，阿片类药物的效果较差，且风险较大。Vertex表示，suzetrigine的研究取得了明显的成功，它的安全性和有效性使其既可用于治疗手术性急性疼痛，也可用于治疗非手术性急性疼痛。市场分析师认为，如果获得批准，suzetrigine可能会成为一剂重磅炸弹，普遍估计到2030 年销售额可能达到15亿美元。但人们普遍认为，慢性疼痛市场更大，仅下背部疼痛就是全球致残的主要原因。鉴于过去的证据表明阿片类药物在这种情况下效果较差，并且伴有便秘、嗜睡、心率减慢和呼吸减慢等风险，suzetrigine将在这一领域超于阿片类止痛片。 ALTO重度抑郁症口服药II期读数 Cobenfy的批准为精神疾病领域的药物开发带来了新气象。Alto Neuroscience的口服重度抑郁症(MDD) 候选药ALTO-100的安慰剂对照IIb研究也将迎来关键读数。 ALTO-100，以前称为NSI-189 (最初由NeuralStem开发)是一种海马体神经发生刺激剂和间接脑源性神经营养因子(BDNF) 调节剂，目前正在开发用于治疗重度抑郁症、双相抑郁症和创伤后应激障碍(PTSD)，还可能用于治疗认知障碍和神经退行性疾病感兴趣。。ALTO-100是2021年Alto Neuroscience以490万美元的价格从NeuralStem合并后的公司Palisade Bio购买的资产。 阿尔茨海默病新机制单抗药物AL002 II期结果即将出炉 Alector 的阿尔茨海默病候选药AL002即将揭晓安慰剂对照的II期研究结果。AL002是Alector与艾伯维联合开发的。AL002是一种人源化单克隆IgG1 抗体，靶向髓系细胞2 上表达的触发受体(TREM2)，这是散发性阿尔茨海默病的重要遗传风险因素。TREM2是一种跨膜受体，在先天免疫细胞亚群上表达，并选择性地在构成大脑免疫系统的小胶质细胞上表达。TREM2功能降低可能导致阿尔茨海默病和其他形式的痴呆症。AL002通过激活 TREM2信号传导来抵消这种功能下降，从而提高细胞存活率和增殖率以及小胶质细胞活性，并减缓痴呆症的进展。小胶质细胞（microglia）是中枢神经系统中的主要免疫细胞，类似于外周免疫系统中的巨噬细胞。它们在大脑和脊髓中发挥着多种重要的免疫和支持功能。 Cassava争议药物III期结果即将问世 最近令Cassava Sciences陷入丑闻漩涡的阿尔茨海默病候选药simufilam的一项III期研究也将于今年12月完成。 Cassava Sciences两位前高管日前因涉及Simufilam的误导性声明，遭到了美国证券交易委员会（SEC）的指控。SEC指控Cassava前CEO Remi Barbier和前神经科学高级副总裁Lindsay Burns在2020年9月有关Simufilam的中期试验中作出多项误导性声明。他们删减了临床试验中40%受试者的数据，从而使得药物的效果看起来更好。公司和高管发布了关于2期试验的误导性结果，声称Simufilam对改善阿尔茨海默症患者的认知功能有效，实际上，完整数据表明药物并未改善患者的“情景记忆”。对此，Cassava同意支付4000万美元民事罚款，而Barbier和Burns分别支付17.5万和8.5万美元罚款，并接受董事和高管职位的禁令。与其合作的City University of New York教授Hoau-Yan Wang支付5万美元罚款。 该事件进一步加剧了要求暂停Simufilam晚期试验的呼声。专家们质疑该药物的疗效，并发现相关研究存在数据操纵的证据。在这种情况下，simufilam于12月完成的一项III期试验就格外令人关注。 多发性骨髓瘤CAR-T疗法anito-cel ArCellx 与吉利德共同开发的靶向BMCA的CAR-T疗法anito-cel，针对多发性骨髓瘤，将于11 月初在美国血液学会年会公布其II期临床iMMagine-1结果。Anito-cel已获得FDA的快速通道、孤儿药和再生医学先进疗法称号。去年，FDA在一名参与者死亡后部分暂停了iMMagine-1试验，但几个月后，试验修改被解除，旨在预防和管理不良事件的风险。如果anito-cel能够在多发性骨髓瘤领域获得最终的成功，它将对BMS的CAR-T疗法Abecma发起挑战。 Ref. Karlsson, C. D. Novo Nordisk initiates new phase 3 trial with Wegovy successor. Med Watch. 18. 09. 2024. Incorvaia, D. AbbVie's dopamine-focused Parkinson's treatment reduces disease burden in phase 3 trial. Fierce Biotech. 26. 09. 2024. Wosen, J. Vertex pain drug results met with measured optimism — and questions — by observers and experts. STAT. 30. 01. 2023. Alector Announces Completion of Enrollment in the INVOKE-2 Phase 2 Clinical Trial of AL002, a TREM2 Monoclonal Antibody, in Individuals With Early Alzheimer’s Disease. Alector Press Release. 07. 09. 2023. Piller, C. Company misled investors on possible Alzheimer’s drug, SEC charges. Science. 30. 09. 2024. Wexler, M. CAR T-cell therapy anito-cel set for Phase 3 trial in multiple myeloma. Myeloma Research News. 14. 05. 2024. Feuerstein, A. The biotech scorecard for the fourth quarter: 14 stock-moving events to watch. STAT. 02. 10. 2024. -END- 药事纵横投稿须知：稿费已上调，欢迎投稿 各位朋友好，觉得本文对您有帮助，请随手点一下下方的在看，以便让你的朋友也能看到哦。

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. 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. 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. 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. sponsored content Upcoming Webinar: Battle of the Budget: Employers vs. Rising Healthcare Costs Gain valuable knowledge and practical insights on how employers are addressing an increasingly complex healthcare budget landscape. By Personify Health 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. 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. 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. 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. “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.” 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. 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. Sponsored Post Get Ready for HLTH 2024: A Preview Guide HLTH 2024, scheduled for October 20-23 at the Venetian Expo Center in Las Vegas, will highlight new programming, a physicians roundtable, generative AI, the shifting landscapes of telemedicine, retail health, women's health, and lots more. By Stephanie Baum 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 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 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. 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. 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. 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. Image: koto_feja, Getty Images