-- New data showed setmelanotide achieved consistent, meaningful weight reduction --
-- Multiple presentations delivered at the first-ever Joint Congress between the European Society for Paediatric Endocrinology and the European Society of Endocrinology (ESPE-ESE); 32nd annual European Congress on Obesity (ECO); and the 2025 annual meeting of the Pediatric Endocrine Society (PES 2025) --
BOSTON, May 14, 2025 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced the presentation of new, real-world data at two European congresses that show consistent improvements in body mass index (BMI), BMI-z, and hunger scores in a total of 35 patients with acquired or congenital hypothalamic obesity who were treated with setmelanotide for up to nine months.
Physicians from Sorbonne University in Paris delivered two oral presentations with data from patients in the early-access programs in France. At the first-ever Joint Congress between the European Society for Paediatric Endocrinology and the European Society of Endocrinology (ESPE-ESE), the presentation detailed real-world data from 30 patients with acquired hypothalamic obesity on setmelanotide therapy for up to nine months. For the second presentation at the 32nd annual European Congress on Obesity (ECO), they also shared data from five patients with congenital hypothalamic obesity, including four patients who reached six months on setmelanotide therapy.
“These real-world data show that patients with acquired or congenital hypothalamic obesity on setmelanotide treatment achieved clinically meaningful weight reductions and reductions in hunger beginning at three months that were sustained and deepened in those patients who reached six and nine months on therapy,” said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. “There is a significant unmet need for a treatment that can address the weight gain and overwhelming hunger that patients with either acquired or congenital hypothalamic obesity live with, and we believe these data – which are consistent with data from our Phase 2 and 3 clinical trials – are supportive of the potential efficacy MC4R agonists can achieve for these patients.”
Real-world setmelanotide weight outcomes in French patients with acquired or congenital hypothalamic obesity The presentations delivered at ESPE-ESE and ECO 2025 detailed clinical outcomes from 30 patients (10 patients younger than 18; 20 patients 18 or older) with acquired hypothalamic obesity who received setmelanotide treatment as part of the pre-approval, early-access program at 14 different treatment centers in France. Highlights include:
- Of the 20 adult patients with acquired hypothalamic obesity treated with setmelanotide therapy:
- Across all patients, there was an overall -11.9% mean change in body mass index (BMI) from baseline at month 3;
- Ten (n=10) who reached month 6 on therapy achieved -19.2% mean change in BMI; and
- Eight (n=8) who reached month 9 on therapy achieved -23.0% mean change in BMI; and
- Adult patients reported meaningful decreases in hunger scores after 3 and 6 months of treatment with setmelanotide.
- For pediatric patients with acquired hypothalamic obesity treated with setmelanotide, BMI z-score (a measure that represents standard deviations of a child’s BMI that corrects for age and sex) decreased from baseline at all timepoints analysed. A clinically meaningful reduction in BMI z-score is defined as a ≥0.2-point reduction. Data from these pediatric patients include:
- Ten (n=10) pediatric patients who reached month 3 on therapy achieved a mean BMI z-score decrease of 0.3 from baseline;
- Seven (n=7) pediatric patients who reached month 6 on therapy achieved a mean BMI z-score decrease of 0.4 decrease from baseline; and
- Two (n=2) pediatric patients who reached month 9 on therapy achieved a mean BMI z-score decrease of 0.4 from baseline.
The presentation at ECO 2025 also featured data from five patients (4 younger than 18; 1 older than 18) with congenital hypothalamic obesity. Highlights include:
- Four (n=4) pediatric patients who reached month 3 on setmelanotide therapy achieved a mean BMI z-score decrease of -0.2 from baseline; and
- Three (n=3) pediatric patients who reached month 6 on setmelanotide therapy achieved a mean BMI z-score decrease of -0.4 from baseline; and
- One adult patient with congenital hypothalamic obesity achieved a -14.8% BMI reduction baseline at month 6 of setmelanotide therapy.
Setmelanotide was generally well tolerated with the most frequent AEs being injection site reactions and skin hyperpigmentation. No new safety signals related to use of setmelanotide were observed, consistent with setmelanotide’s well-established and well-understood safety profile. For further information about setmelanotide’s safety profile, please see the information below under “WARNINGS AND PRECAUTIONS”.
Additional Congress PresentationsAdditional Rhythm presentations were also shared at ECO and ESPE/ESE. Two presentations will also be shared at the Pediatric Endocrinology Society (PES) annual meeting from May 15-18 in National Harbor, Maryland.
ECO 2025Rhythm and its collaborators also presented one encore oral presentation, three encore poster presentations and hosted one satellite symposium at ECO:
- “DAYBREAK Trial: Setmelanotide vs placebo in patients with Melanocortin-4 Receptor Pathway Variants”
- “Frequency of Bardet-Biedl syndrome variants in a population with early-onset obesity”
- “Weight loss at 18 months of setmelanotide in 2 to