Setmelanotide Acetate

本月多项重磅数据公布，从ELCC到NEJM，创新疗法密集释放积极信号，全球研发格局加速演进2026年3月，全球医药研发领域持续保持高活跃度。从欧洲肺癌大会（ELCC）到阿尔茨海默病与帕金森病国际会议（AD/PD™ 2026），从《新英格兰医学杂志》到《柳叶刀》，肿瘤双抗、PROTAC蛋白降解剂、CAR-T细胞疗法、AI手术机器人等多项前沿技术接连释放重磅信号。同时，中国企业在创新药研发与BD交易上继续展现出强劲的国际竞争力。01 肿瘤领域：双抗药物领跑，CAR-T与ADC多点开花本月，肿瘤治疗领域最受关注的当属双抗类药物在肺癌一线治疗中的突出表现。康方生物依沃西数据再升级：在ELCC 2026上，康方生物公布了PD-1/VEGF双抗依沃西的HARMONi-2研究生活质量分析数据。结果显示，依沃西不仅显著延长了无进展生存期（11.14个月 vs 5.82个月），在患者的整体健康状况、躯体功能以及咳嗽、呼吸困难等肺癌典型症状的控制上，也全面优于帕博利珠单抗。这意味着该药物有望成为PD-L1阳性非小细胞肺癌一线治疗的新标准。BioNTech双抗与ADC管线密集亮相：BioNTech在ELCC上披露了其PD-L1×VEGF-A双抗Pumitamig的多项数据。该药物在非小细胞肺癌多个亚型（包括EGFR突变患者）及小细胞肺癌一线治疗中均展现出积极疗效，安全性可控。数据显示，在EGFR突变NSCLC患者中，Pumitamig联合化疗展现出具有临床意义的生存获益。此外，其HER3靶向ADC（BNT326/YL202）在非小细胞肺癌中的首次临床数据也同步公布，在既往接受过标准治疗后进展的晚期或转移性NSCLC患者中展现出抗肿瘤活性和良好的安全性。同时，BioNTech的另一款CTLA-4抗体Gotistobart在2线及以上鳞状NSCLC患者中，与标准化疗相比显示出54%的死亡风险降低，具有临床意义的抗肿瘤活性。Elicera Therapeutics CAR-T疗法ELC-301表现惊艳：Elicera Therapeutics公布了其iTANK平台武装的CD20靶向CAR-T疗法ELC-301在复发/难治性B细胞淋巴瘤患者中的I/IIa期CARMA研究数据。结果显示，在最高剂量组（队列3）治疗的前两名患者中，均达到完全代谢缓解（CMR），且未观察到剂量限制性毒性。截至目前，8名接受治疗的患者中，6名在第1个月达到CMR，疾病控制率达100%，总缓解率为7/8，最佳缓解持续时间长达至少12个月。原位转分化技术迈入临床：纽伦捷生物的NRG-103注射液针对胶质母细胞瘤的注册临床一期已在武汉启动。该技术通过将残余肿瘤细胞“转化”为正常细胞，从根源上降低耐药与复发风险，是全球首个实现该技术肿瘤基因疗法获批中美双IND的企业。TGCT新药III期结果登上《柳叶刀》：和誉医药宣布，其CSF-1R抑制剂Pimicotinib治疗腱鞘巨细胞瘤（TGCT）的全球III期MANEUVER研究结果发表于国际顶级医学期刊《柳叶刀》。数据显示，该药物在TGCT患者中取得了显著的疗效和良好的安全性。该药已于2025年12月在中国获批上市，美国NDA正在审评中。其他临床进展：翰森制药的GLP-1/GIP双激动剂奥莱泊肽在肥胖治疗的III期临床中显示出积极疗效。02 神经退行性疾病领域：PROTAC与基因疗法齐头并进本月，神经领域迎来多项重磅进展，多款First-in-class药物释放关键临床数据。Arvinas PROTAC降解剂ARV-102数据惊艳：Arvinas在AD/PD™ 2026会议上公布了其口服PROTAC降解剂ARV-102的I期临床数据。该药物靶向LRRK2蛋白——帕金森病的关键致病靶点。结果显示，在帕金森病患者中，ARV-102实现了脑脊液中LRRK2蛋白超过50%的降解，且剂量依赖性良好。更重要的是，该药物同时降低了与神经炎症相关的生物标志物（如CD68、GPNMB）。ARV-102总体安全耐受性良好，未报告严重不良事件。基于此积极数据，Arvinas计划于2026年第二季度启动进行性核上性麻痹（PSP）的Ib期临床试验。罗氏BTK抑制剂fenebrutinib III期临床成功：罗氏宣布其口服、脑渗透性BTK抑制剂fenebrutinib在复发性多发性硬化症（RMS）的III期FENhance 1研究中达到主要终点。数据显示，fenebrutinib相比特立氟胺使年复发率降低51%，与此前公布的FENhance 2研究结果（降低59%）一致。两项研究结果合计相当于每17年发生一次复发。fenebrutinib有望成为首个同时适用于RMS和原发性进行性多发性硬化症（PPMS）的高效口服治疗药物。AC Immune TDP-43 PET示踪剂首次人体成像：AC Immune在AD/PD™ 2026上展示了其TDP-43 PET示踪剂ACI-19626的I期临床数据。这是全球首次实现在活体人脑中检测TDP-43病理的影像学证据，有望为ALS、FTD等多种TDP-43蛋白病提供精准诊断工具。齐鲁制药阿尔茨海默病1类新药获批临床：3月13日，CDE官网显示，齐鲁制药自主研发的1类新药QLH2405注射液获得临床试验默示许可，拟定适应症为阿尔茨海默病源性轻度认知障碍和轻度阿尔茨海默病。跃赛生物iPSC来源癫痫细胞疗法获FDA IND批准：3月12日，跃赛生物自主研发的针对癫痫的iPSC（诱导多能干细胞）来源异体细胞药物UX-GIP001注射液获得美国FDA IND批准。这是全球首款进入临床阶段的iPSC来源异体癫痫细胞治疗药物。拜耳基因疗法管线更新：拜耳公布了其临床开发管线进展，其中包括两款针对帕金森病的基因疗法——Bemdaneprocel（细胞疗法）已进入III期临床，AB-1005（AAV基因疗法）处于II期临床阶段。03 自身免疫疾病领域：大额BD交易频现，新药数据登顶NEJM本月自身免疫疾病领域同样动作频频，罗氏重磅III期数据登上NEJM。罗氏Gazyva治疗狼疮III期数据登顶NEJM：罗氏宣布，其Type II抗CD20单抗Gazyva（obinutuzumab）治疗系统性红斑狼疮（SLE）的III期ALLEGORY研究结果发表于《新英格兰医学杂志》。数据显示，超过四分之三（76.7%）接受Gazyva联合标准治疗的患者在52周时达到SRI-4应答（评估疾病严重程度和症状的指标），而安慰剂组为53.5%。Gazyva在包括BICLA应答、糖皮质激素减量等所有关键次要终点上均显著优于安慰剂。如果获批，Gazyva将成为首个用于SLE的Type II抗CD20疗法。德琪医药与优时比达成全球许可协议：德琪医药将其临床前阶段的CD19/CD3双抗ATG-201授权给优时比（UCB），用于治疗B细胞相关的自身免疫性疾病。根据协议，德琪医药将获得8000万美元首付款及近期里程碑付款，以及超11亿美元的后续里程碑付款。宜明昂科红斑狼疮新药获批临床：宜明昂科宣布，其全球首创的CD47×CD20双靶点分子IMM0306的皮下注射剂型（IMM0306S）获中国NMPA批准临床，用于治疗系统性红斑狼疮。相较于静脉输注，皮下制剂可降低峰浓度、减少输注反应，显著提升患者用药便利性。益方生物TYK2抑制剂获FDA批准II期临床：益方生物自主研发的TYK2抑制剂D-2570获美国FDA批准，开展治疗银屑病的II期临床试验。此前该药物在中国完成的银屑病II期临床试验已显示出显著疗效和良好安全性。深信生物mRNA疗法IND获批：深信生物宣布其mRNA候选药物IN026获美国FDA批准临床试验，该药物针对难治性痛风，是mRNA技术在慢性代谢疾病领域的重要探索。恒瑞医药海曲泊帕新增SAA适应症：恒瑞医药宣布，其自主研发的1类创新药海曲泊帕乙醇胺片获得国家药品监督管理局批准，新增“联合免疫抑制治疗适用于15岁及以上初治重型再生障碍性贫血（SAA）患者”的适应症。04 代谢与心血管疾病领域：GLP-1新星崛起，PCSK9抑制剂数据将亮相诺和诺德/联邦制药UBT251 II期临床成功：诺和诺德与联邦制药联合开发的GLP-1/GIP/胰高血糖素三靶点激动剂UBT251在中国2型糖尿病患者中的II期临床试验达到主要终点。数据显示，治疗24周后，UBT251最高可使HbA1c降低2.16%，体重降低9.8%，疗效优于司美格鲁肽1mg组（HbA1c降幅1.77%，体重降幅4.8%）和安慰剂组。基于此结果，诺和诺德计划于2026年下半年启动全球II期临床试验。默沙东口服PCSK9抑制剂III期数据即将亮相ACC.26：默沙东宣布，将在美国心脏病学会（ACC.26）年会上公布其口服PCSK9抑制剂enlicitide的III期CORALreef AddOn试验结果。该药物有望成为首个获批的口服PCSK9抑制剂，为他汀类药物治疗后血脂仍不达标的患者提供新的治疗选择。此外，默沙东还将公布sotatercept在CpcPH-HFpEF（联合性毛细血管前和后毛细血管性肺动脉高压合并射血分数保留的心力衰竭）患者中的II期CADENCE试验数据。Rhythm Pharmaceuticals setmelanotide III期研究未达主要终点：Rhythm Pharmaceuticals公布了setmelanotide在MC4R通路罕见遗传性肥胖患者中的III期EMANATE研究结果。四个遗传亚组研究均未达到预设的主要终点。然而，事后分析显示，在POMC/PCSK1杂合变异和SRC1基因变异患者中，setmelanotide在52周时实现了具有统计学意义的BMI降低。05 新兴技术与行业趋势：AI手术机器人落地，全球研发格局重塑在技术与模式创新层面，本月也传来多项值得关注的信号。国内首款胰腺疾病智能体与手术机器人发布：北京大学第三医院在中关村论坛上发布了全国首个覆盖胰腺疾病全病程的“胰腺疾病诊疗智能体”，以及全球首台实现椎板自主切削操作的“脊柱椎板减压手术机器人”。这表明中国在医疗AI与高端手术机器人领域的自主创新能力正在加速落地。全球研发格局加速重构：数据显示，中国目前占全球创新药候选药物的20%以上，中国启动的临床试验数量已超过美国，主要由I期和I/II期项目驱动。全球早期药物研发格局正从美国主导转向以美国和中国为中心的“双中心”结构。体内CAR-T技术取得突破：加州大学旧金山分校在《Nature》上发表研究，开发了一种双载体系统，可在体内直接生成CAR-T细胞，为人源化小鼠模型中的B细胞发育不全、血液系统恶性肿瘤和实体瘤提供了治疗水平的CAR-T细胞。这一技术有望大幅降低CAR-T疗法的生产成本和制备时间。国家药监局批准罗赛促红素α注射液上市：国家药监局批准广东三生制药有限公司申报的罗赛促红素α注射液（商品名：新比澳）上市，用于治疗因慢性肾脏病引起的贫血且正在接受促红细胞生成素治疗的血液透析患者。06 FDA审评动态与行业新闻3月迎来多项FDA审评关键日期，包括：首个用于治疗丁肝的药物bulevirtide、首个用于先天性耳聋的基因疗法DB-OTO、以及首个每周一次的胰岛素icodec（Awiqli）等。siRNA疗法获快速通道资格：舶望制药宣布其在研siRNA疗法BW-20805获得美国FDA授予快速通道资格，用于治疗遗传性血管性水肿（HAE）。该药物靶向前激肽释放酶（PKK）。---结语2026年3月的全球医药研发，既是数据的集中披露期，也是多项前沿技术从概念走向临床的里程碑时刻。双抗药物在肿瘤与自免领域的持续拓展、PROTAC等新技术在神经退行性疾病中的首次临床验证、CAR-T细胞疗法在淋巴瘤中的持续突破、GLP-1三靶点激动剂在代谢疾病中的惊艳数据、AI与机器人的深度融合、以及中国在全球研发网络中角色的持续强化，共同构成了本月行业发展的多条主线。未来几个月，随着更多临床数据与BD交易的落地，全球医药创新格局或将迎来新一轮洗牌。---参考资料：Roche官网、Arvinas官网、BioNTech官网、Merck官网、Novo Nordisk官网、Rhythm Pharmaceuticals官网、Elicera Therapeutics官网、和誉医药官网、齐鲁制药、跃赛生物、恒瑞医药、美迪西、中国食品药品网等

-- European Commission decision anticipated in second quarter of 2026 -- BOSTON, March 26, 2026 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending to expand the current marketing authorization for IMCIVREE® (setmelanotide) to include the treatment of obesity and the control of hunger in adults and children 4 years of age and above with acquired hypothalamic obesity (HO) due to hypothalamic injury or impairment. “The positive CHMP opinion with a broad label following closely upon last week's FDA approval validates the quality of the data which supports our filings in acquired HO and further confirms our global approach to drug development,” said David Meeker, M.D., Chairman, President and Chief Executive Officer of Rhythm. “Acquired HO represents a significant unmet medical need across geographies and a unique opportunity for Rhythm which we are well positioned to execute on.” “Acquired hypothalamic obesity is a devastating neuroendocrine disease characterized by reduced energy expenditure, hyperphagia (pathological insatiable hunger) and accelerated, sustained weight gain resulting from physical injury or structural abnormality of the hypothalamus. This disease condition is driven by impairment of MC4R pathway signaling and other hypothalamic functional damage, making it exceptionally difficult to manage with existing approaches,” said Professor Hanneke van Santen, M.D., PhD., professor of pediatric endocrinology at the Prinses Máxima Center and Wilhelmina Children’s Hospital, Utrecht, The Netherlands. “There is a clear and urgent need for a precision medicine that targets the root cause of the disease and offer patients a meaningful path forward.” The CHMP opinion will now be reviewed by the European Commission (EC), which has the authority to grant and expand marketing authorizations for medicinal products in the European Union. A final decision is anticipated in the second quarter of 2026. About Acquired Hypothalamic Obesity Acquired hypothalamic obesity is a rare neuro endocrine disease characterized by accelerated and sustained weight gain caused by hypothalamic injury or impairment. Hypothalamic injury or impairment may lead to decreased alpha-melanocyte-stimulating hormone (α-MSH) production and disruption of MC4R pathway signaling. The MC4R pathway is responsible for regulating energy balance and body weight. Acquired hypothalamic obesity most frequently follows the growth or treatment of craniopharyngioma, astrocytoma or other hypothalamic-pituitary tumors. Additional causes of injury or dysfunction may include traumatic brain injury, stroke, inflammation, or anatomic abnormalities. Due to impairment of the MC4R pathway, patients experience accelerated and sustained weight gain, often accompanied by hyperphagia and/or decreased energy expenditure. Rhythm estimates there are approximately 10,000 people living with acquired hypothalamic obesity in the U.S. and approximately 10,000 people living with acquired hypothalamic obesity in the EU. About Rhythm Pharmaceuticals Rhythm is a commercial-stage biopharmaceutical company committed to transforming the lives of patients and their families living with rare neuroendocrine diseases. Rhythm’s lead asset, IMCIVREE® (setmelanotide), an MC4R agonist designed to treat hyperphagia and severe obesity, is approved by the U.S. Food and Drug Administration (FDA) to reduce excess body weight and maintain reduction long term in adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity, adult and pediatric patients 2 years of age and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or genetically confirmed pro-opiomelanocortin (POMC), including proprotein convertase subtilisin/kexin type 1 (PCSK1), deficiency or leptin receptor (LEPR) deficiency. Both the European Commission (EC) and the UK’s Medicines & Healthcare Products Regulatory Agency (MHRA) have authorized setmelanotide for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists bivamelagon and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA. Setmelanotide Indication In the United States, setmelanotide is indicated to reduce excess body weight and maintain weight reduction long term in adults and pediatric patients aged 4 years and older with acquired hypothalamic obesity, in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS). In the European Union and the United Kingdom, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 2 years of age and above. In the European Union and the United Kingdom, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology. Limitations of Use Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective: Obesity due to suspected POMC, PCSK1, or LEPR deficiency with POMC, PCSK1, or LEPR variants classified as benign or likely benign Other types of obesity not related to acquired HO, BBS, or POMC, PCSK1 or LEPR deficiency, including obesity associated with other genetic syndromes and general (polygenic) obesity. Important Safety Information CONTRAINDICATIONS Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. WARNINGS AND PRECAUTIONS Disturbance in Sexual Arousal: Spontaneous penile erections and increased frequency of penile erections in males have occurred. Inform patients that these events may occur and instruct patients who have an erection lasting longer than 4 hours to seek emergency medical attention. Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Monitor patients for new onset or worsening depression or suicidal thoughts or behaviors. Consider discontinuing IMCIVREE if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur. Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue IMCIVREE. Skin Hyperpigmentation, Darkening of Pre-existing Nevi, and Development of New Melanocytic Nevi: Generalized or focal increases in skin pigmentation occurred in the majority of IMCIVREE-treated patients. IMCIVREE may also cause development of new melanocytic nevi or darkening of pre-existing nevi. Perform a full body skin examination prior to initiation and periodically during treatment to monitor pre-existing and new pigmented lesions. Acute Adrenal Insufficiency with Acquired HO: Patients with acquired HO and secondary adrenal insufficiency reported serious adverse reactions related to acute adrenal insufficiency in 5% of IMCIVREE-treated patients and no placebo-treated patients. In patients with secondary adrenal insufficiency, monitor for clinical signs of acute adrenal insufficiency. Sodium Imbalance in Patients with Acquired HO and Central Diabetes Insipidus: Patients with acquired HO and concomitant central diabetes insipidus (DI)/arginine vasopressin (AVP) deficiency reported hyponatremia in 6% of IMCIVREE-treated patients and 2% of placebo-treated patients and hypernatremia in 5% of IMCIVREE-treated patients and 4% of placebo-treated patients. Monitor serum sodium levels with changes in fluid intake and hydration status. Adjust the doses of concomitant therapies for DI/AVP deficiency as needed. ADVERSE REACTIONS Most common adverse reactions (incidence ≥20% in at least 1 indication) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection. USE IN SPECIFIC POPULATIONS Treatment with IMCIVREE is not recommended when breastfeeding. Discontinue IMCIVREE when pregnancy is recognized unless the benefits of therapy outweigh the potential risks to the fetus. To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe. Please see the full Prescribing Information for additional Important Safety Information. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the safety, efficacy, potential benefits of, and clinical design or progress of any of our products or product candidates at any dosage or in any indication; the approval and use of IMCIVREE in patients with acquired hypothalamic obesity and its availability for patients; the commercial growth of IMCIVREE; our expectations surrounding potential regulatory submissions, progress, or approvals and timing thereof for any of our product candidates, including a potential marketing authorization in the European Union as well as the Company’s engagement with Japan’s Pharmaceuticals and Medical Devices Agency and plans to seek authorization for setmelanotide to treat acquired hypothalamic obesity in Japan; the timing of review and final decision by the EC on expanding marketing authorization for IMCIVREE in the EU; the estimated market size and addressable population for our drug products, including IMCIVREE for the treatment of hypothalamic obesity in the United States, the European Union and Japan; the future announcement of data from our ongoing clinical trials, including the Japanese cohort of our Phase 3 trial evaluating setmelanotide for patients with acquired hypothalamic obesity, the substudy evaluating setmelanotide for patients with congenital hypothalamic obesity; Part C of the Phase 1 trial evaluating RM-718, and the open-label Phase 2 trial evaluating setmelanotide in patients with Prader-Willi syndrome; the ongoing enrollment in and potential progress or outcomes of our clinical trials; the presentation of the full data from the TRANSCEND study at an upcoming medical meeting; and the content, date and timing of any of the foregoing. Statements using words such as “expect”, “anticipate”, “believe”, “may”, “will” and similar terms are also forward-looking statements. Such statements are subject to numerous risks and uncertainties, including, but not limited to, our ability to enroll patients in clinical trials, the design and outcome of clinical trials, the impact of competition, the ability to achieve or obtain necessary regulatory approvals, risks associated with data analysis and reporting, unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives, risks associated with the laws and regulations governing our international operations and the costs of any related compliance programs, our ability to successfully commercialize setmelanotide, our liquidity and expenses, our ability to retain our key employees and consultants, and to attract, retain and motivate qualified personnel, and general economic conditions, and the other important factors, including those discussed under the caption “Risk Factors” in Rhythm’s Annual Report on Form 10-K for the year ended December 31, 2025 and our other filings with the Securities and Exchange Commission. Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this press release or to update them to reflect events or circumstances occurring after the date of this press release, whether as a result of new information, future developments or otherwise. Corporate Contact: David Connolly Head of Investor Relations and Corporate Communications Rhythm Pharmaceuticals, Inc. dconnolly@rhythmtx.com Media Contact: Layne Cosgrove Real Chemistry llitsinger@realchemistry.com