Rhythm Pharmaceuticals Presents Data Demonstrating BMI Reduction in Patients with Hypothalamic Obesity Treated with Setmelanotide over Six Months at ENDO 2023

-- Mean BMI reduction of 21.0% from baseline observed in 13 patients at six months, showing progression from 16.8% mean BMI reduction at 16 weeks --

-- Phase 3 trial evaluating setmelanotide in acquired hypothalamic obesity ongoing; completion of enrollment expected 1Q 2024 --

BOSTON, June 17, 2023 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases, today announced data that showed meaningful weight loss was sustained and progressed in patients with hypothalamic obesity treated with setmelanotide for six months as part of the long-term extension of its Phase 2 trial.   

“Hypothalamic obesity is a challenging disease to manage with patients generally refractory to standard treatments for obesity. The setmelanotide results to date have been remarkable with setmelanotide demonstrating sustained and deepening reductions in multiple body mass index (BMI) measures at six months of treatment, showing progressive and consistent improvement over previously reported 16-week BMI reduction data,” said Christian Roth, M.D., Seattle Children’s Research Institute and Division of Endocrinology, Department of Pediatrics, University of Washington, who presented these data during The Endocrine Society Annual Meeting & Expo (ENDO 2023) being held June 15-18 in Chicago. “This impressive response adds to the evidence suggesting setmelanotide may provide a meaningful clinical benefit for patients with this disease who currently have no approved therapeutic options.”

Rhythm enrolled 18 patients in its open-label, 16-week Phase 2 trial designed to evaluate setmelanotide in patients with acquired hypothalamic obesity. Thirteen of those patients¹ who enrolled in the long-term extension trial reached a total of six months or more on setmelanotide therapy, as of the data cutoff date of Nov. 30, 2022. Highlights from the data presented show:

• 21.0 mean percent reduction in BMI at month 6 from baseline, which progressed from 16.8 mean percent reduction in BMI at week 16 across these 13 patients;
• 10 of 13 (76.9%) patients achieved 10% BMI reduction or greater at month 6 and all 13 patients achieved 5% BMI reduction or greater;
• 9.2 kg (23.9%) and 3.0 kg (6.3%) mean decreases observed in fat mass and lean muscle mass, respectively, at week 16 in pediatric patients (n=11); and
• 1.7 point mean decrease from baseline in BMI-Z score from baseline in patients younger than 18 (n=11).

In addition, Rhythm presented data demonstrating that all patients achieved improvement in severity of their obesity, and 10 of 13 patients achieved an improvement in weight classification by one or more class, as defined by the U.S. National Institutes of Health and the World Health Organization, which characterize obesity classes based on BMI². Among pediatric patients, there was a 34.3 percentage point decrease in the 95^th percentile at month 6, which corresponds to an average move from morbid obesity to mild obesity.