Rhythm Pharmaceuticals Announces Publication of Results from Phase 2 Study of Setmelanotide for the Treatment of Hypothalamic Obesity in The Lancet Diabetes & Endocrinology - Seite 2

• 89% (16 of 18) of patients achieved the primary endpoint;
• 78% (14 of 18) of patients achieved a 10% or greater reduction in BMI at 16 weeks;
• Mean percent reduction in BMI was 15% from baseline;
• In pediatric patients (n=13), the mean (standard deviation [SD]) BMI Z score at Week 16 was 2.7 (1.3), a reduction of 1.3 (1.0) points from baseline; and
• Mean (SD) most hunger score at baseline was 6.6 (1.6), compared with 3.7 (2.5) at Week 16, for a reduction of –2.9 (2.3) points or 45% for patients ≥12 years of age (n=11).
  

The publication also includes preliminary data from Rhythm’s long-term extension of the Phase 2 study that were disclosed at ObesityWeek 2023. These data show patients with hypothalamic obesity (n=12) achieved mean BMI reduction of approximately 26% at one year on setmelanotide treatment.

Consistent with prior experience, setmelanotide was generally well tolerated. The most common adverse events (AEs) in the primary trial included nausea (61.1%), vomiting (33.3%), skin hyperpigmentation (33.3%), diarrhea (22.2%), and COVID-19 (22.2%). Two patients discontinued due to AEs and a third patient was non-compliant. There were no serious AEs, no AEs that led to study discontinuation during the trial, and no new safety concerns were observed during the long-term extension trial.

In January 2024, Rhythm announced the completion of screening for enrollment in the ongoing pivotal, Phase 3 clinical trial evaluating setmelanotide in patients with acquired hypothalamic obesity. The Company remains on track to obtain topline study results in the first half of 2025.

About Rhythm Pharmaceuticals
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. 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). 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. 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.