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Rhythm Pharmaceuticals Presents Positive Interim Six-month Data from Phase 2 Trial of Setmelanotide in Patients with Prader-Willi Syndrome (PWS) at ENDO 2026

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-- Patients with PWS treated with setmelanotide therapy (N=17) achieved clinically meaningful BMI or BMI z-score reductions, reductions in fat mass with preservation of lean mass, and improvements in hyperphagia and anxiety measures --

-- Positive results reinforce rationale for Phase 3 development of MC4R agonism in PWS --

-- Company to hold conference call on Saturday, June 13, at 8 a.m. CT, 9 a.m. ET --

BOSTON, June 13, 2026 (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 preliminary data from a Phase 2 trial evaluating setmelanotide in patients with Prader-Willi syndrome (PWS) delivered during the Endocrine Society’s Annual Meeting (ENDO 2026) in Chicago.

“Patients and families living with PWS face severe hyperphagia and obesity due to underlying MC4R pathway dysfunction and have limited effective treatment options. These results show that MC4R agonism has the potential to deliver sustained and durable improvements in outcomes across BMI, hyperphagia scores, body composition, and food-related behaviors and anxiety,” said Jennifer Miller, M.D., University of Florida Division of Endocrinology, Department of Pediatrics in the College of Medicine, the principal investigator for this Phase 2 trial. “Importantly, such reductions in HQ-CT score and anxiety, as well as weight reduction, have the potential to ease the burden not only on patients, but also on their caregivers who manage the daily challenges of this disease.”

Rhythm enrolled 18 patients with PWS aged 6-23 years old with a BMI ≥30 kg/m² for patients ≥18 years of age or BMI ≥95th percentile for age and sex for patients younger than 18 in this Phase 2 trial. The 52-week trial remains ongoing, and 17 patients remain on active therapy as of June 12, 2026.

Results from the six-month analysis demonstrate that treatment with setmelanotide was associated with improvements across multiple clinically relevant endpoints, as of a data cut off date of May 7, 2026. Highlights include:

Consistent BMI reductions in pediatric and adult patients at Month 6:
- -3.06% mean reduction in BMI (N=17 pts);
- -3.11% mean reduction in BMI in adult patients (n=10); with six achieving >2.5% BMI reduction, and four achieving >4% BMI reduction;
- −3.00% mean reduction in BMI in pediatric patients (n=7);
- −0.35 mean reduction in BMI z-score from baseline in pediatric patients (n=7);
- Five (5) of seven pediatric patients achieved clinically meaningful BMI z-score reduction >0.2;
Setmelanotide achieved preservation of lean mass and reductions in fat mass across 16 patients with data available from DEXA scans:
- +0.74% mean gain in lean mass and -4.19% mean loss in fat mass across 16 patients;
- Six (6) of nine adult patients achieved >5% reduction in fat mass;
- Five (5) of seven pediatric patients gained ≥2.95% in lean mass;
Clinically meaningful improvement in hyperphagia score observed in patients with moderate to severe hyperphagia, defined as a ≥7-point reduction in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score
- Eight (8) of 10 patients who entered trial with moderate to severe hyperphagia (>13 at baseline) achieved clinically meaningful improvement of 7 points or better.
Improvement in PWS Anxiousness and Distress Behaviors Questionnaire (PADQ) which measures anxiousness, emotional distress, and behavioral dysregulation.
- Of the 15 patients who had a baseline score >11, 10 patients achieved clinically meaningful improvement of ≥11 points; and
Safety and tolerability results have been consistent with the well-established profile observed with setmelanotide.