Rhythm Pharmaceuticals to Announce Preliminary Data from Exploratory Phase 2 Trial Evaluating Setmelanotide in Prader-Willi Syndrome

Core Viewpoint - Rhythm Pharmaceuticals is set to disclose preliminary data from its exploratory Phase 2 trial of setmelanotide in patients with Prader-Willi syndrome (PWS) during a live conference call on December 11, 2025 [1] Company Overview - Rhythm Pharmaceuticals is a commercial-stage biopharmaceutical company focused on 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 syndromes [4][5] - Setmelanotide is also authorized by the European Commission and the UK's Medicines & Healthcare Products Regulatory Agency for similar indications [4][6] - Rhythm is advancing a clinical development program for setmelanotide in other rare diseases and is exploring investigational MC4R agonists and small molecules for congenital hyperinsulinism [4] Conference Call Details - The live conference call and webcast will take place at 8:00 a.m. ET on December 11, 2025, with participants encouraged to join ten minutes early [2] - A webcast of the call will be available on the Rhythm Pharmaceuticals website and archived for 30 days [3] Indications and Usage - In the U.S., setmelanotide is indicated for patients with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS), POMC, PCSK1, or LEPR deficiency [5] - In the EU and UK, it is indicated for obesity and hunger control associated with genetically confirmed BBS or loss-of-function biallelic POMC, including PCSK1 and LEPR deficiencies [6] Limitations and Contraindications - Setmelanotide is not indicated for patients with benign or likely benign variants of POMC, PCSK1, or LEPR deficiency, nor for other types of obesity unrelated to these conditions [7][8] Warnings and Precautions - Adverse reactions include skin hyperpigmentation, sexual arousal disturbances, and potential for depression and suicidal ideation [9][10][11] - Serious hypersensitivity reactions have been reported, necessitating caution in prescribing [12] Adverse Reactions - Common adverse reactions (≥20% incidence) include skin hyperpigmentation, injection site reactions, nausea, headache, diarrhea, abdominal pain, vomiting, depression, and spontaneous penile erection [15]