Core Insights - Rhythm Pharmaceuticals presented four data presentations at ObesityWeek® 2025, showcasing the efficacy of setmelanotide in treating acquired hypothalamic obesity [1][2][5] Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, with its lead product, setmelanotide (IMCIVREE®), approved for treating obesity related to specific genetic conditions [6][9][10] Clinical Data Highlights - Setmelanotide demonstrated a mean placebo-adjusted BMI reduction of 27.1% in patients receiving concomitant GLP-1 therapy and a 19.0% reduction in patients treated with setmelanotide alone [8][7] - Significant improvements in cardiometabolic health were observed in patients treated with setmelanotide, including better blood pressure and lipid levels [3][7] - Patient-reported outcomes indicated meaningful changes in hunger, weight, energy levels, and physical activity among participants in the TRANSCEND trial [2][3] Research Presentations - Dr. Christian Roth presented findings on the robust efficacy of setmelanotide in reducing BMI compared to previous anti-obesity treatments [2] - A poster by Dr. Jennifer Miller highlighted significant improvements in cardiometabolic parameters associated with setmelanotide treatment [3] - Dr. Roohi Kharofa's research focused on hyperphagia and genetic variants in early-onset obesity, involving data from 212 participants [4]

Core Viewpoint - Rhythm Pharmaceuticals has announced an extension of the FDA review period for the supplemental New Drug Application (sNDA) for IMCIVREE® (setmelanotide) by three months, moving the PDUFA goal date from December 20, 2025, to March 20, 2026 [1][2] Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, with its lead product IMCIVREE® (setmelanotide) aimed at treating acquired hypothalamic obesity [1][6] - Setmelanotide is already approved by the FDA for reducing excess body weight in patients aged 2 years and older with specific genetic obesity syndromes [6][7] Regulatory Update - The FDA requested additional sensitivity analyses of clinical efficacy data from Rhythm's Phase 3 pivotal trial, which has been classified as a 'major amendment' allowing for an extended review period [2] - No new safety or manufacturing data was requested by the FDA, indicating that the focus remains on the efficacy data [2] Management Commentary - David Meeker, CEO of Rhythm, expressed confidence in the product profile of setmelanotide and the ongoing collaboration with the FDA to bring the treatment to patients who currently lack approved options [3] Conference Call Information - Rhythm Pharmaceuticals will hold a conference call and webcast to discuss the update at 8:00 a.m. ET, with registration recommended ten minutes prior to the start [4][5]

Core Insights - Rhythm Pharmaceuticals has entered into Product Listing Agreements for IMCIVREE in several Canadian provinces, allowing public reimbursement for eligible patients with Bardet-Biedl syndrome and obesity [1][2][3] - The approval of IMCIVREE by Health Canada in May 2023 and its inclusion in the National Strategy for Drugs for Rare Diseases is a significant advancement for patients [2][4] - The agreements highlight the recognition of the burden of obesity associated with Bardet-Biedl syndrome and the company's commitment to equitable access for all patients in Canada [3][4] Company Overview - Rhythm Pharmaceuticals is a biopharmaceutical company focused on rare neuroendocrine diseases, with its lead product IMCIVREE (setmelanotide) approved for weight management in patients with specific genetic conditions [4][5] - IMCIVREE is indicated for weight management in patients aged 6 years and older with obesity due to Bardet-Biedl syndrome and other genetic deficiencies [5][6] - The company is also advancing a clinical development program for additional rare diseases and investigational MC4R agonists [4][5] Product Information - IMCIVREE is an MC4R agonist that has received regulatory approvals in Canada, the U.S., and Europe for treating obesity related to Bardet-Biedl syndrome and other genetic conditions [4][6][8] - The product is administered via subcutaneous injection and is indicated for patients with genetically confirmed conditions [5][6] - The company emphasizes the importance of physician supervision for prescribing IMCIVREE in the EU and UK [8]

Core Viewpoint - Rhythm Pharmaceuticals, Inc. is set to report its third quarter 2025 financial results and provide a corporate update on November 4, 2025, at 8:00 a.m. ET [1] Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases [3] - The company's lead product, IMCIVREE® (setmelanotide), is an MC4R agonist approved by the FDA for treating obesity related to specific genetic conditions [3][4] - Setmelanotide is indicated for patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or deficiencies in POMC, PCSK1, or LEPR [4][5] Upcoming Events - Rhythm will host a live conference call and webcast for its financial results on November 4, 2025 [1] - The company will also participate in two fireside chats on November 12, 2025, at the Guggenheim Healthcare Innovation Conference and the Stifel Healthcare Conference [7] Product Indications - In the U.S., setmelanotide is indicated for long-term weight reduction in patients with specific genetic obesity conditions [4] - In the EU and UK, it is indicated for obesity treatment and hunger control associated with genetically confirmed BBS or related deficiencies [5] Limitations and Contraindications - Setmelanotide is not indicated for obesity due to benign variants of POMC, PCSK1, or LEPR [6][8] - Serious hypersensitivity reactions have been reported, contraindicating its use in patients with prior serious hypersensitivity to the drug [9] Warnings and Precautions - Patients may experience disturbances in sexual arousal, depression, and hypersensitivity reactions [10][11][12] - Skin hyperpigmentation and the development of new melanocytic nevi have been observed, necessitating regular skin examinations [13] Adverse Reactions - Common adverse reactions (≥20% incidence) include skin hyperpigmentation, injection site reactions, nausea, headache, and depression [15]

Core Viewpoint - Rhythm Pharmaceuticals, Inc. is set to report its second quarter 2025 financial results and provide a corporate update on August 5, 2025, highlighting its focus on rare neuroendocrine diseases and the development of its lead asset, setmelanotide [1][4]. Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with rare neuroendocrine diseases [4]. - The company's lead product, IMCIVREE® (setmelanotide), is an MC4R agonist approved by the FDA for reducing excess body weight and maintaining weight reduction in patients aged 2 years and older with specific genetic obesity conditions [4][5]. - Setmelanotide is also authorized by the European Commission and the UK's MHRA for similar indications in adults and children [4][6]. Upcoming Events - Rhythm Pharmaceuticals will host a live conference call and webcast on August 5, 2025, at 8:00 a.m. ET to discuss its financial results [1]. - Alastair Garfield, Chief Scientific Officer, and David Connolly, Head of Investor Relations, will participate in a fireside chat at the Canaccord Genuity 45th Annual Growth Conference on August 13, 2025, at 12:30 p.m. ET [2]. Product Indications - In the U.S., setmelanotide is indicated for patients with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or deficiencies in POMC, PCSK1, or LEPR [5]. - In the EU and UK, it is indicated for obesity treatment and hunger control associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1 and LEPR deficiencies [6]. Clinical Development - Rhythm is advancing a broad clinical development program for setmelanotide in other rare diseases and is also exploring investigational MC4R agonists, bivamelagon and RM-718, along with a preclinical suite of small molecules for congenital hyperinsulinism [4].

Core Insights - Rhythm Pharmaceuticals is set to disclose topline results from a Phase 2 trial evaluating bivamelagon for acquired hypothalamic obesity on July 9, 2025 [1][2] Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases [4] - The company's lead asset, IMCIVREE® (setmelanotide), is approved for treating obesity related to specific genetic conditions [4][5] - Rhythm is advancing a clinical development program for setmelanotide and other investigational MC4R agonists, including bivamelagon and RM-718 [4] Bivamelagon Phase 2 Trial Details - The Phase 2 trial is a randomized, placebo-controlled, double-blind study involving 28 patients aged 12 and older [3] - Patients received daily oral doses of bivamelagon (200 mg, 400 mg, or 600 mg) or placebo for 14 weeks [3] - The primary endpoint is the change in body mass index after 14 weeks of treatment, with an option for patients to continue therapy for up to 52 weeks [3]

Core Insights - Rhythm Pharmaceuticals, Inc. announced the acceptance of three late-breaking abstracts for presentation at The Endocrine Society's Annual Meeting (ENDO 2025) scheduled for July 12-15 in San Francisco, CA [1] Group 1: Clinical Trials and Presentations - Dr. Susan Phillips will present data from the pivotal Phase 3 TRANSCEND trial evaluating setmelanotide for acquired hypothalamic obesity, marking it as the largest randomized, placebo-controlled trial in this area to date [2] - A poster presentation will be given by Dr. Christian Roth detailing exit interviews from 30 patients or caregivers in the US who participated in the Phase 3 TRANSCEND trial [2] - Dr. Vidhu Thaker will present results from a 14-week Phase 2 trial of bivamelagon, an oral MC4R agonist, in participants with acquired hypothalamic obesity [2] Group 2: Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, with its lead asset, IMCIVREE® (setmelanotide), approved by the FDA for treating obesity related to specific genetic conditions [3][4] - Setmelanotide is indicated for reducing excess body weight and maintaining weight reduction in patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome or POMC, PCSK1, or LEPR deficiencies [4][5][6] - The company is advancing a broad clinical development program for setmelanotide and other investigational MC4R agonists, including bivamelagon and RM-718, as well as a preclinical suite for congenital hyperinsulinism [3]