공고 • May 05
Rhythm Pharmaceuticals Announces New Data Presentations for Setmelanotide in Pediatric Patients with Acquired Hypothalamic Obesity
Rhythm Pharmaceuticals, Inc. announced the presentation of new data to be delivered by Rhythm and its partners at The Pediatric Endocrine Society (PES) Annual Meeting, taking place April 30-May 3, 2026, in San Francisco. Long-Term Efficacy with Setmelanotide in Pediatric Patients with Acquired Hypothalamic Obesity Ashley Shoemaker, M.D., MSCI, Associate Professor of Pediatrics, Pediatric Endocrinology at Vanderbilt Health, presented a poster on Friday, May 1 with data on the continued efficacy and safety after 2.5 years of setmelanotide treatment in pediatric patients following their participation in the 16-week Phase 2 trial. Highlights from the presentation include: Pediatric patients with acquired hypothalamic obesity who received setmelanotide for up to 2.5 years achieved sustained reductions across multiple age-adjusted weight-related measures. Among pediatric participants who completed 2.5 years of treatment (n=10) as of Nov. 10, 2025, mean changes from baseline included: -16.4% change in BMI -1.6 change in BMI Z-score -34.2 percentage-point change in percent of the BMI 95th percentile (%BMI95) Adverse events observed were consistent with the previously reported safety profile of setmelanotide, with skin hyperpigmentation, nausea, vomiting, headache and injection site reaction being the most common. Weight Category Improvement Following Setmelanotide in Pediatric Patients with Acquired Hypothalamic Obesity Jennifer Miller, M.D., Professor of Pediatrics, Pediatric Endocrinology, University of Florida College of Medicine, presented data from the Phase 3 TRANSCEND trial in an oral presentation on Saturday, May 2 on the changes in weight category in the pediatric subpopulation of participants after 1 year of setmelanotide treatment. Highlights from the presentation include: At baseline, 59.7% of pediatric participants (40/67) had class II or III obesity; After 1 year of treatment, 71.1% of pediatric patients treated with setmelanotide (n=32) improved by =1 weight category, and 44.4% (n=20) improved by =2 weight categories, compared with 13.6% (n=3) improving one weight category and 0% improving two categories for placebo patients; Following 1 year of treatment, 44.4% of patients (n=20) were classified as healthy weight (n=10) or overweight (n=10), compared with 18.2% of placebo-treated patients (n=4) classified as overweight and none achieving healthy weight; Adverse events observed were consistent with the previously reported safety profile of setmelanotide. 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.
