Rhythm Pharmaceuticals Presents Phase 3 Pediatrics Trial Data at Pediatric Endocrine Society Meeting

Rhythm Pharmaceuticals, Inc., a biopharmaceutical company specializing in rare neuroendocrine diseases, recently presented three significant presentations at The Pediatric Endocrine Society’s (PES) Annual Meeting held in Chicago, IL, from May 2-5, 2024. The company focused on the potential of its lead product, setmelanotide, in reducing obesity in young children with specific genetic deficiencies.

The highlight of the event was an oral presentation that detailed data from a 52-week, Phase 3 pediatric trial involving 12 patients aged between 2 and under 6 years old. These patients suffered from Bardet-Biedl syndrome (BBS) or obesity due to proopiomelanocortin (POMC) or leptin receptor (LEPR) deficiency. The trial demonstrated that setmelanotide achieved a significant mean reduction in BMI-Z score by 3.04 and a mean reduction in body mass index (BMI) of 18.4 percent. Dr. Megan Kelsey from Children’s Hospital Colorado, who presented these findings, emphasized the severe impact of these conditions on young patients and the promising potential of setmelanotide to bring about clinically significant weight loss at an early age.

In addition to the oral presentation, Rhythm Pharmaceuticals also showcased two poster presentations at the PES meeting. The first poster explored "Weight Reduction in Pediatric Patients With Hypothalamic Obesity Treated With a Therapy for 12 Months," while the second poster focused on the "Efficacy in Weight Reduction and Safety in Pediatric Age Groups With Rare Melanocortin-4 Receptor Pathway–Related Obesity Treated With a Therapy for 12 Months."

Rhythm Pharmaceuticals is aiming to expand the label for setmelanotide to include pediatric patients aged 2 to under 6 years. The company plans to complete the submission of a supplementary New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) within the first half of 2024.

Setmelanotide, marketed under the name IMCIVREE®, is an MC4R agonist approved for chronic weight management in adults and pediatric patients aged 6 and older who suffer from obesity due to specific genetic deficiencies, including POMC, proprotein convertase subtilisin/kexin type 1 (PCSK1), or LEPR deficiency. It is also approved for patients with Bardet-Biedl syndrome (BBS). In the European Union, this drug is authorized for treating obesity and controlling hunger associated with genetically confirmed BBS or biallelic POMC (including PCSK1) or LEPR deficiency in adults and children 6 years and older.

Rhythm Pharmaceuticals is committed to developing treatments that address rare, genetically-driven diseases. The company is also working on expanding the clinical applications of setmelanotide in other rare conditions and is advancing other investigational MC4R agonists, including LB54640 and RM-718, as well as a preclinical suite of small molecules aimed at treating congenital hyperinsulinism.

Setmelanotide has some limitations and is not indicated for treating obesity due to benign variants of POMC, PCSK1, or LEPR deficiencies or for general obesity types that are not related to these genetic conditions. Patients with serious hypersensitivity to setmelanotide or its components should not use this medication. Common side effects include skin pigmentation changes, injection site reactions, nausea, headache, and depression, among others. For pediatric use, regular assessment of growth and development is recommended to ensure the therapy does not adversely affect growth and maturation.

Overall, the presentations at PES 2024 highlighted the promising efficacy and safety profile of setmelanotide in treating young children with specific rare genetic obesity, underscoring Rhythm Pharmaceuticals' ongoing efforts to provide transformative treatments for patients with rare neuroendocrine diseases.