This is my third Rhythm (NASDAQ: RYTM ) article, following 06/2025's "Rhythm Pharmaceuticals: Rare Genetic Obesity Pioneer With Excellent Prospects." In Excellent Prospects , I rated Rhythm as a "Buy." It has since enjoyed a tasty ~52% bump. In this article, I will assess its forward prospectsWriting under the pseudonym "out of ignorance", I very much regard investing as a learning process. Investing failures are tuition paid. Investing successes enter the trove of lessons learned. In my Seeking Alpha artic ...

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ConversationDavid ConnollyHead of Investor Relations & Corporate Communications Good morning, everybody. Thank you for joining us today. I'm Dave Connolly, IR here at Rhythm Pharmaceuticals. Before we begin the speaking program, I'll just remind you that this event may include remarks concerning future expectations, plans and prospects, which constitute forward-looking statements. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important f ...

ConversationDavid ConnollyHead of Investor Relations & Corporate Communications Good morning, everybody. Thank you for joining us today. I'm Dave Connolly, IR here at Rhythm Pharmaceuticals. Before we begin the speaking program, I'll just remind you that this event may include remarks concerning future expectations, plans and prospects, which constitute forward-looking statements. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important ...

Rhythm Pharmaceuticals (NasdaqGM:RYTM) Update / Briefing September 24, 2025 08:30 AM ET Company ParticipantsLewis Blevins - Endocrinologist, Professor of Medicine, and Medical DirectorCorinne Johnson - VPKaren Johnson - Registered NurseNone - Video NarratorAshley Shoemaker - Associate Professor of MedicineJennifer Lee - EVP of North AmericaDavid Meeker - Chairman, President, and CEOSeamus Fernandez - Senior Managing DirectorKris Jenner - PartnerEllen Horste - Equity Research AssociateConference Call Partici ...

Commercial Readiness for Acquired Hypothalamic Obesity September 24, 2025 ® © Rhythm® Pharmaceuticals, Inc. All rights reserved. Forward-looking Statements This presentation contains certain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, and that involve risks and uncertainties, including without limitation statements regarding the potential, safety, efficacy, and regulatory and clinical progress of our products and product candidates, including setmel ...

公司管线进展 - Terns Pharmaceuticals开发TERN-501用于代谢功能障碍相关脂肪性肝炎及GLP-1药物联合疗法[1] - TERN-701作为潜在癌症疗法处于1期研究阶段 预计第四季度公布数据[1] - Terns Pharmaceuticals近期启动TERN-601为期12周的2期减重临床试验 预计第四季度数据读出[3] 口服GLP-1竞争格局 - 口服药物生产成本低于皮下注射剂型[3] - 口服药物制造商有机会扩大生产并覆盖更多患者群体[2] - 基于生物技术公司口服GLP-1药物最新进展 Terns Pharmaceuticals面临激烈市场竞争[2] 肥胖治疗市场前景 - 抗肥胖领域仍处于发展初期 预计至少到2030年前保持快速增长[5] - 该领域将成为制药行业收入最大的治疗领域[5] - 成功药企将获得巨大财务收益并可能产生超越市场的回报[5] Rhythm Pharmaceuticals业务聚焦 - 公司专注于罕见病导致的肥胖治疗市场[7] - Imcivree于2020年获批用于特定蛋白质缺乏症患者体重管理[7] - 第二季度收入增长66.8%至4850万美元[7] Rhythm临床进展 - setmelanotide在下丘脑损伤所致肥胖患者中取得积极3期结果[8] - 已就该适应症提交监管申请 可能于年底前获得批准[8] - bivamelagon在2期下丘脑性肥胖研究中表现优异 计划明年启动3期试验[9] 目标市场规模 - 在美国、欧洲和日本市场目标患者群体达35,500人[10] - 目前尚无针对下丘脑性肥胖体重管理的获批疗法[10] - bivamelagon作为口服制剂较setmelanotide皮下注射具有额外优势[9] 投资风险因素 - Terns Pharmaceuticals股价表现高度依赖TERN-601中期研究进展[6] - 若试验失败可能导致股价大幅下跌[6] - Rhythm Pharmaceuticals面临临床或监管挫折风险[11]

PresentationMichael UlzEquity Analyst All right. Good afternoon, everyone, and thanks for joining us at the Morgan Stanley Global Healthcare Conference. I'm Mike Ulz, one the biotech analyst here. It's my pleasure to introduce David Meeker, President and CEO of Rhythm Pharmaceuticals. Before we get started, I just need to read a quick disclosure. For important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, pleas ...

PresentationMichael UlzEquity Analyst All right. Good afternoon, everyone, and thanks for joining us at the Morgan Stanley Global Healthcare Conference. I'm Mike Ulz, one the biotech analyst here. It's my pleasure to introduce David Meeker, President and CEO of Rhythm Pharmaceuticals. Before we get started, I just need to read a quick disclosure. For important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, pleas ...

公司及行业 * Rhythm Pharmaceuticals (RYTM) 专注于开发针对黑皮质素-4受体(MC4R)通路的疗法 该通路负责调节饱腹感信号和能量消耗[2] * 公司核心产品为IMCIVREE (setmelanotide) 用于治疗因MC4通路缺陷导致的罕见遗传性肥胖疾病[2][7] * 当前重点开发适应症包括获得性下丘脑性肥胖(HO) 并推进下一代MC4R激动剂(口服bivamelagon和每周注射剂RM-718)及Prader-Willi综合征的临床研究[6][36][47] 核心观点与论据 * **Bardet-Biedl综合征(BBS)市场机会稳固**: 美国患者数约4000-5000人 预计可获1000名用药患者 按净价30万美元计算 仅美国市场机会即达3亿美元 实际可能超过该预期[8] * **获得性下丘脑性肥胖(HO)市场潜力巨大**: 美国患者数估计为5000-10000人 近期分析更倾向于范围上限(近10000人) 日本患者数估计为5000-8000人 因肿瘤 prevalence较高[8][9][10][13] * **HO临床需求高度未满足**: 患者为"职业生涯中见过的最病重群体" 用药清单长达1-2页 生活质量受肥胖严重主导 现有治疗方案有限[16][17] * **TRANSCEND phase 3数据强劲**: 治疗组BMI降低16.5% 安慰剂组反而增加3.3% 安慰剂校正效应达19.8% 各年龄组(6-12,12-18,18+)反应一致(均19-20%) 显示长期患者仍可响应[19][20][21] * **与GLP-1联用显示协同潜力**: 试验中约30名患者使用GLP-1 既往使用GLP-1者响应与总体相似 同时使用GLP-1者BMI降低达25% 提示MC4通路校正后可恢复对减肥药响应[22][23][24] * **监管进展顺利**: HO适应症美国PDUFA日期定于2025年底 欧洲同步申报 预计2026年Q3获批 日本PMDA支持加入12名患者数据用于申报[9][25][26][34] * **下一代MC4R激动剂推进中**: 口服bivamelagon phase 2显示与setmelanotide相似疗效及明确剂量反应(200/400/600mg) 且色素沉着显著减少 phase 3计划2026年上半年启动 每周注射剂RM-718特异性更高 预计色素沉着问题更少[37][38][39][40][44][45] * **Prader-Willi综合征开发挑战与策略**: 生物学机制明确(MAGEL2/NDN基因影响MC4通路) 但患者行为噪声大 当前phase 2采用开放标签设计 剂量升至5mg 治疗期6个月 以体重减轻5%为成功标准[47][48][49][55] 其他重要内容 * **商业化准备就绪**: 销售团队扩建中 医学事务团队已转向HO支持 部分临床试验招募人员转岗疾病 awareness[28][31] * **投资者活动计划**: 将举办北美焦点会议 邀请专家分享HO见解及团队实地学习成果[28][29] * **日本市场特性**: 专家社区小而活跃 监管机构(PMDA)积极支持加速新药准入[14][15] * **专利保护延长**: 下一代项目将知识产权保护期延长至2040年以后[37] * **色素沉着问题改善**: setmelanotide因MC1激动导致普遍性色素沉着 bivamelagon仅偶发局部色素沉着(如痣、日光暴露区) 预计改善患者耐受性[39][40][42][43]