-- Phase 3 trial evaluating setmelanotide in pediatric patients ages 2-<6yo (N=12) over 52 weeks achieved primary endpoint -- -- Setmelanotide achieved 3.04 mean reduction in BMI-Z score and 18.4 percent mean reduction in BMI in patients ages 2-<6yo with obesity due to POMC/LEPR deficiency or BBS -- -- RM-718 showed potential to reduce body weight and hyperphagia with no off-target cardiovascular effects and no hyperpigmentation observed in pre-clinical studies; first in-human studies anticipated to begin in the first half of 2024 -- -- Responder rates of 25 to 56 percent demonstrated in six genetic cohorts of Phase 2 DAYBREAK trial of setmelanotide -- -- Company to host R&D webcast today at 8:00 a.m.

--第三阶段试验评估了52周内2至6岁（N=12）儿童患者的setmelanotide达到了主要终点--setmelanotide在2至6岁因POMC/LEPR缺乏或BBS肥胖的患者中BMI-Z评分平均降低了3.04，BMI平均降低了18.4%--RM-718显示出减轻体重和食欲亢进的潜力，没有脱靶的心血管疾病在临床前研究中未观察到效果和色素沉着过度；预计将于2024年上半年开始的首次人体研究--setmelanotide第二阶段黎明试验的六个基因队列显示，应答率为25%至56%--该公司将于今天上午8:00主持研发网络广播。

ET-- BOSTON, Dec. 06, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today hosted an R&D event for investors and analysts, “Rhythm Update on MC4R Pathway Programs.” During the event, the Company announced new topline data from its ongoing Phase 3, open-label pediatrics trial evaluating one year of setmelanotide therapy in patients between the ages of 2 and younger than 6 years with Bardet-Biedl syndrome (BBS) or proopiomelanocortin (POMC) or leptin receptor (LEPR) deficiency obesity and new preliminary data from the first stage of its exploratory Phase 2 DAYBREAK trial.

ET——波士顿，2023年12月6日（环球通讯社）——节奏制药公司（Nasdaq:RYTM），一家全球商业阶段的生物制药公司，专注于改变由罕见的黑皮质素-4受体（MC4R）途径疾病引起的食欲亢进和严重肥胖患者及其家属的生活，今天为投资者和分析师举办了一场研发活动，“MC4R路径计划的节奏更新”。在活动期间，该公司宣布了正在进行的第三阶段的新topline数据，开放标签儿科试验评估了2岁至6岁以下患有Bardet-Biedl综合征（BBS）或proopiomelanocortin（POMC）或瘦素受体（LEPR）缺乏症肥胖的患者一年的setmelanotide治疗，以及其探索性2期DAYBREAK试验第一阶段的新初步数据。

Rhythm also presented pre-clinical data on its new drug candidate, RM-718, which is designed to be a more selective MC4R agonist with weekly administration, and its plans for Phase 1 clinical development. The Company hosted Amy Wood, Executive Director and Co-Fou.

Rhythm还介绍了其新药候选药物RM-718的临床前数据，RM-718被设计为每周给药的更具选择性的MC4R激动剂，以及其1期临床开发计划。该公司接待了执行董事兼联合创始人艾米·伍德（AmyWood）。