BOSTON, July 08, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced the appointment of Alastair “Al” Garfield, Ph.D.

波士顿，2024年7月8日（环球通讯社）--商业阶段的生物制药公司Ryth Pharmaceuticals，Inc.（纳斯达克：RYTM），专注于改变患有罕见神经内分泌疾病的患者及其家属的生活，今天宣布任命阿拉斯泰尔·加菲尔德博士。

as Chief Scientific Officer, effective July 1..

作为首席科学官，于7月1日生效。。

“We are excited to welcome Al back to Rhythm as we continue to leverage our understanding of the melanocortin-4 receptor (MC4R) pathway to bring potential therapies to patients and their families,” said David Meeker, M.D., Rhythm Chairman, Chief Executive Officer and President. “Al’s leadership and established scientific expertise and experience in the brain’s regulation of appetite and body weight were instrumental in defining the Rhythm of today, and we are genuinely thrilled he chose to rejoin Rhythm to help us achieve our vision for the future of MC4R therapeutics.”.

“随着我们继续利用对黑皮质素-4受体（MC4R）途径的理解，为患者及其家属带来潜在的治疗方法，我们很高兴欢迎Al回到节奏上来，”节奏主席、首席执行官兼总裁大卫·米克（DavidMeeker）医学博士说。“Al在大脑调节食欲和体重方面的领导才能和成熟的科学专业知识和经验有助于确定今天的节奏，我们真的很高兴他选择重新加入节奏，以帮助我们实现对MC4R疗法未来的愿景。”。

Dr. Garfield said, “After being part of the team to develop the first generation of MC4R agonists and seeing the transformational promise this precision medicine approach has delivered to patients and families living with hyperphagia and severe obesity, I am excited about the potential for additional opportunities for Rhythm.

加菲尔德博士说：“作为开发第一代MC4R激动剂团队的一员，看到这种精准医学方法为患有食欲亢进和严重肥胖症的患者和家庭带来了变革性的希望，我对额外的节奏机会的潜力感到兴奋。

Decades of research interest in the MC4R pathway tell us that this robustly validated target has broad clinical relevance, and as the industry leader in this mechanism, I believe Rhythm is uniquely positioned to deliver on this potential.”.

数十年来对MC4R途径的研究兴趣告诉我们，这一经过强有力验证的目标具有广泛的临床相关性，作为该机制的行业领导者，我相信节奏在发挥这一潜力方面具有独特的优势。”。

Dr. Garfield rejoins Rhythm after serving as Senior Vice President and Head of Translational Sciences and Strategy at Rectify Pharma, where he helped deliver first-in-class therapeutics for rare disease. Prior to joining Rectify, Dr. Garfield was Senior Vice President and Head of Translational Research at Rhythm.

加菲尔德博士在担任Rective Pharma的高级副总裁兼转化科学和战略主管后，重新加入了Rhythm，他在那里帮助提供了一流的罕见疾病治疗方法。在加入Rective之前，加菲尔德博士是Rhythm的高级副总裁兼转化研究负责人。

He also held roles at Pfizer, where he was a lab head in the Cardiovascular Metabolic Disease Research Unit and led novel obesity target identification..

他还曾在辉瑞（Pfizer）任职，在那里他是心血管代谢疾病研究部门的实验室负责人，并领导了新的肥胖目标识别。。

Dr. Garfield’s holds a first-class degree in molecular and cellular biology and a Ph.D. in molecular genetics from the University of Bath, UK. His postdoctoral fellowships at the University of Cardiff, UK and University of Cambridge, UK focused on neural pathways and genes underlying mammalian energy balance.

加菲尔德博士拥有英国巴斯大学分子与细胞生物学一级学位和分子遗传学博士学位。他在英国加的夫大学和英国剑桥大学获得博士后奖学金，重点研究哺乳动物能量平衡的神经通路和基因。

As an assistant professor at the University of Edinburgh, UK and visiting professor Harvard Medical School, Dr. Garfield’s independent research expanded the field’s understanding of the hypothalamic melanocrotinergic networks regulating metabolism, body weight and appetite. His research has been published in leading scientific journals including Nature, Nature Neuroscience, Neuron, and Cell Metabolism.

作为英国爱丁堡大学助理教授和哈佛医学院客座教授，加菲尔德博士的独立研究扩展了该领域对调节新陈代谢、体重和食欲的下丘脑黑色素能网络的理解。他的研究发表在包括《自然》、《自然神经科学》、《神经元和细胞代谢》在内的主要科学期刊上。

Dr Garfield has authored numerous original research and review articles on the MC4R pathway, including “Melanocortin-4 receptor-regulated energy homeostasis,” which was published in Nature Neuroscience in 2016 (https://pubmed.ncbi.nlm.nih.gov/26814590/)..

加菲尔德博士撰写了许多关于MC4R途径的原创研究和评论文章，包括2016年发表在《自然神经科学》上的“黑皮质素-4受体调节的能量稳态”(https://pubmed.ncbi.nlm.nih.gov/26814590/)。。

About Rhythm Pharmaceuticals

关于节奏制药

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.

节奏是一家商业阶段的生物制药公司，致力于改变患有罕见神经内分泌疾病的患者及其家人的生活。节奏的主要资产IMCIVREE®（setmelanotide）是一种MC4R激动剂，旨在治疗食欲亢进和严重肥胖，已获得美国批准。

Food and Drug Administration (FDA) for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1) or leptin receptor (LEPR) deficiency confirmed by genetic testing, or patients with a clinical diagnosis of Bardet-Biedl syndrome (BBS).

美国食品和药物管理局（FDA）对6岁及以上的成人和儿科患者进行慢性体重管理，这些患者因促黑素皮质激素（POMC），前蛋白转化酶枯草杆菌蛋白酶/kexin 1型（PCSK1）或瘦素受体（LEPR）缺陷而患有单基因或综合征性肥胖，或经基因检测证实患有Bardet-Biedl综合征（BBS）的临床诊断患者。

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 6 years of age and above.

欧盟委员会（EC）和英国药品和保健品管理局（MHRA）均已授权setmelanotide用于治疗肥胖和控制与基因确认的BBS或基因确认的功能丧失的双等位基因POMC相关的饥饿，包括PCSK1，成人和6岁及以上儿童的缺乏症或双等位基因LEPR缺乏症。

Additionally, Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases, as well as investigational MC4R agonists LB54640 and RM-718, and a preclinical suite of small molecules for the treatment of congenital hyperinsulinism. Rhythm’s headquarters is in Boston, MA..

此外，Rthym正在推进其他罕见疾病中setmelanotide的广泛临床开发计划，以及研究性MC4R激动剂LB54640和RM-718，以及一套用于治疗先天性高胰岛素血症的临床前小分子。节奏的总部位于马萨诸塞州波士顿。。

Setmelanotide Indication

Setmelanotide适应症

In the United States, setmelanotide is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1 or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance (VUS) or BBS..

在美国，setmelanotide适用于6岁及以上因POMC，PCSK1或LEPR缺乏而患有单基因或综合征性肥胖的成人和儿科患者的慢性体重管理，这是通过FDA批准的测试确定的，该测试证明了POMC，PCSK1或LEPR基因的变异，这些变异被解释为致病性，可能致病性或不确定意义（VUS）或BBS。。

In the European Union, 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 6 years of age and above. In Europe, setmelanotide should be prescribed and supervised by a physician with expertise in obesity with underlying genetic etiology..

在欧盟，setmelanotide用于治疗肥胖和控制与遗传证实的BBS或功能丧失的双等位基因POMC相关的饥饿，包括PCSK1，成人和6岁及以上儿童的缺乏症或双等位基因LEPR缺乏症。在欧洲，setmelanotide应由具有潜在遗传病因学肥胖专业知识的医生开具并监督。。

Limitations of Use

使用限制

Setmelanotide is not indicated for the treatment of patients with the following conditions as setmelanotide would not be expected to be effective:

Setmelanotide不适用于以下情况的患者，因为Setmelanotide预计不会有效：

Obesity due to suspected POMC, PCSK1 or LEPR deficiency with POMC, PCSK1 or LEPR variants classified as benign or likely benign

由于疑似POMC、PCSK1或LEPR缺乏而导致的肥胖，POMC、PCSK1或LEPR变异被归类为良性或可能良性

Other types of obesity not related to POMC, PCSK1 or LEPR deficiency, or BBS, including obesity associated with other genetic syndromes and general (polygenic) obesity.

与POMC，PCSK1或LEPR缺乏症或BBS无关的其他类型的肥胖，包括与其他遗传综合征相关的肥胖和一般（多基因）肥胖。

Contraindication

禁忌症

Prior serious hypersensitivity to setmelanotide or any of the excipients in IMCIVREE. Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported.

先前对setmelanotide或IMCIVREE中的任何赋形剂产生严重超敏反应。已经报道了严重的超敏反应（例如过敏反应）。

WARNINGS AND PRECAUTIONS

警告和注意事项

Skin Pigmentation and Darkening of Pre-Existing Nevi: Generalized increased skin pigmentation and darkening of pre-existing nevi have occurred because of its pharmacologic effect. Full body skin examinations prior to initiation and periodically during treatment should be conducted to monitor pre-existing and new pigmentary lesions..

皮肤色素沉着和预先存在的痣变黑：由于其药理作用，皮肤色素沉着普遍增加，预先存在的痣变黑。应在开始之前和治疗期间定期进行全身皮肤检查，以监测先前存在的和新的色素性病变。。

Heart rate and blood pressure monitoring: In Europe, heart rate and blood pressure should be monitored as part of standard clinical practice at each medical visit (at least every 6 months) for patients treated with setmelanotide.

心率和血压监测：在欧洲，对于接受setmelanotide治疗的患者，应在每次就诊时（至少每6个月）监测心率和血压，作为标准临床实践的一部分。

Disturbance in Sexual Arousal: Spontaneous penile erections in males and sexual adverse reactions in females have occurred. Patients who have an erection lasting longer than 4 hours should seek emergency medical attention.

性唤起障碍：男性发生自发性阴茎勃起，女性发生性不良反应。勃起持续时间超过4小时的患者应寻求紧急医疗护理。

Depression and Suicidal Ideation: Depression and suicidal ideation have occurred. Patients should be monitored for new onset or worsening depression or suicidal thoughts or behaviors. Consideration should be given to discontinuing setmelanotide if patients experience suicidal thoughts or behaviors, or clinically significant or persistent depression symptoms occur..

抑郁症和自杀念头：抑郁症和自杀念头已经发生。应监测患者新发或恶化的抑郁症或自杀念头或行为。如果患者出现自杀念头或行为，或出现临床上显着或持续的抑郁症状，应考虑停用塞特黑肽。。

Hypersensitivity Reactions: Serious hypersensitivity reactions (e.g., anaphylaxis) have been reported. If suspected, advise patients to promptly seek medical attention and discontinue setmelanotide.

超敏反应：据报道有严重的超敏反应（例如过敏反应）。如果怀疑，建议患者立即就医并停用塞特黑肽。

Pediatric Population: The prescribing physician should periodically assess response to setmelanotide therapy. In growing children, the impact of weight loss on growth and maturation should be evaluated. In Europe, the prescribing physician should monitor growth (height and weight) using age- and sex-appropriate growth curves..

儿科人群：处方医生应定期评估对setmelanotide治疗的反应。在成长中的儿童中，应评估体重减轻对生长和成熟的影响。在欧洲，处方医生应该使用适合年龄和性别的生长曲线来监测生长（身高和体重）。。

Risk of Serious Adverse Reactions Due to Benzyl Alcohol Preservative in Neonates and Low Birth Weight Infants: Setmelanotide is not approved for use in neonates or infants. Serious and fatal adverse reactions including “gasping syndrome” can occur in neonates and low birth weight infants treated with benzyl alcohol-preserved drugs..

苯甲醇防腐剂对新生儿和低出生体重儿产生严重不良反应的风险：Setmelanotide不被批准用于新生儿或婴儿。使用苯甲醇保存药物治疗的新生儿和低出生体重儿可能会出现严重致命的不良反应，包括“喘息综合征”。。

ADVERSE REACTIONS

不良反应

Most common adverse reactions (incidence ≥20%) included skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection.

最常见的不良反应（发生率≥20%）包括皮肤色素沉着过度，注射部位反应，恶心，头痛，腹泻，腹痛，呕吐，抑郁和自发性阴茎勃起。

USE IN SPECIFIC POPULATIONS

在特定人群中使用

Lactation: Not recommended when breastfeeding.

哺乳期：不建议母乳喂养。

To report SUSPECTED ADVERSE REACTIONS, contact Rhythm Pharmaceuticals at +1 (833) 789-6337 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. See section 4.8 of the Summary of Product Characteristics for information on reporting suspected adverse reactions in Europe.

要报告可疑的不良反应，请致电+1（833）789-6337联系Rthym Pharmaceuticals或致电1-800-FDA-1088或www.FDA.gov/medwatch联系FDA。有关报告欧洲疑似不良反应的信息，请参阅产品特征摘要第4.8节。

Please see the full Prescribing Information for additional Important Safety Information.

有关其他重要安全信息，请参阅完整的处方信息。

Forward-looking Statements

前瞻性声明

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 potential, safety, efficacy, regulatory and clinical progress, or benefits of any of our products or product candidates, including potential MC4R therapeutics; our potential market position and opportunities; updates to our executive team; and the timing of any of the foregoing.

本新闻稿包含1995年《私人证券诉讼改革法案》所指的前瞻性声明。本新闻稿中所有与历史事实无关的声明均应视为前瞻性声明，包括但不限于关于我们任何产品或候选产品（包括潜在的MC4R治疗剂）的潜在性，安全性，有效性，监管和临床进展或益处的声明；我们潜在的市场地位和机会；我们执行团队的最新情况；以及上述任何一项的时间安排。

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, our ability to successfully commercialize setmelanotide and develop and commercialize additional potential MC4R therapeutics, 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 discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the three months ended March 31, 2024 and our other filings with the Securities and Exchange Commission.

使用“期望”、“预期”、“相信”、“可能”、“将会”等词语和类似术语的陈述也是前瞻性陈述。此类声明存在许多风险和不确定性，包括但不限于我们招收患者参加临床试验的能力、临床试验的设计和结果、竞争的影响、获得或获得必要监管批准的能力、与数据分析和报告相关的风险、我们成功将塞特黑肽商业化并开发和商业化其他潜在MC4R疗法的能力、我们的流动性和费用、我们留住关键员工和顾问的能力以及吸引、留住和激励合格人员的能力、一般经济状况，以及我们截至2024年3月31日的三个月10-Q季报中“风险因素”标题下讨论的其他重要因素，以及我们向证券交易委员会。

Except as required by law, we undertake no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring .

除法律要求外，我们没有义务对本版本中包含的前瞻性声明进行任何修订，也没有义务对其进行更新以反映发生的事件或情况。

Corporate Contact:

公司联系人：

David Connolly

大卫·康诺利

Head of Investor Relations and Corporate Communications

投资者关系和企业沟通主管

Rhythm Pharmaceuticals, Inc.

节奏制药公司。

857-264-4280

857-264-4280

dconnolly@rhythmtx.com

dconnolly@rhythmtx.com

Media Contact:

媒体联系人：

Adam Daley

亚当·戴利

Berry & Company Public Relations

Berry&Company公共关系

212-253-8881

212-253-8881

adaley@berrypr.com

adaley@berrypr.com