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

-- Oral presentation showcased previously disclosed data that demonstrate setmelanotide achieved 3.04 mean reduction in BMI-Z score and 18.4 percent mean reduction in BMI in 12 patients ages 2-<6yo with obesity due to POMC/LEPR deficiency or BBS --

-- Company remains on track to complete submission of sNDA to the FDA seeking a label expansion for pediatric patients in 1H 2024 --

-- Two additional posters presented at PES 2024 --

BOSTON, May 06, 2024 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, today announced that the Company delivered three presentations – one oral and two posters – at The Pediatric Endocrine Society’s (PES) Annual Meeting held May 2-5, 2024 in Chicago, IL.

In the oral presentation, Rhythm showcased data that was previously announced on December 6, 2023, from its 52-week, Phase 3 pediatrics trial in patients between the ages of 2 and younger than 6 years (N=12) with Bardet-Biedl syndrome (BBS) or obesity due to proopiomelanocortin (POMC) or leptin receptor (LEPR) deficiency. Treatment with setmelanotide achieved the primary endpoint with a 3.04 mean reduction in BMI-Z score (a measure of body mass index deviations from what is considered normal) and 18.4 percent mean reduction in body mass index (BMI).

“The hyperphagia and severe obesity of rare, genetically-caused melanocortin-4 receptor (MC4R) pathway diseases can present at a young age and have a significant impact on patients and their families,” said presenting author Megan Kelsey, M.D., Children’s Hospital Colorado. “These data demonstrate setmelanotide’s potential to achieve clinically significant weight loss in children ages 2 to younger than 6 years old, potentially allowing us to treat patients earlier in life.”

Rhythm remains on track to complete submission of a supplementary New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking a label expansion to treat pediatric patients between the ages of 2 and younger than 6 years in approved indications in the first half of 2024.

Rhythm also presented two additional poster presentations at PES 2024:

• “Weight Reduction in Pediatric Patients With Hypothalamic Obesity Treated With a Therapy for 12 Months”
• “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”

About Rhythm Pharmaceuticals