Rhythm Pharmaceuticals Presents Data from its Long-term Extension Study of Setmelanotide Showing Sustained and Deepened BMI Reduction in Patients with Hypothalamic Obesity at One Year at ObesityWeek 2023 - Seite 2

Hypothalamic Obesity Data at One Year
Twelve patients who enrolled in Rhythm’s open-label, 16-week Phase 2 trial and who also enrolled in the long-term extension trial and reached one year or more on setmelanotide were included in the one-year data analysis. In addition to the BMI and BMI Z score reductions provided above, highlights from the late-breaking poster, “Weight Reduction in Patients With Hypothalamic Obesity Treated with Setmelanotide for 12 Months” by lead author Christian L. Roth, M.D., Seattle Children’s Research Institute, include:

• Mean change of -1.1 in BMI Z score from baseline in pediatric patients (n=11) at one year on therapy;
• Three of 11 pediatric patients achieved normal weight at one year, as defined by the U.S. National Institutes of Health (NIH) and World Health Organization (WHO) (>5th to <85th BMI percentile);
• Eleven of 12 patients (91.7%) improved by one or more weight classes based on BMI or BMI percentile as defined by the NIH and WHO; and
• Body composition changes were favorable, with larger percent decreases in total fat mass compared with lean muscle mass.

There were no serious adverse events (AE), no AEs that led to study discontinuation during the trial, and no new safety concerns were observed.

Bardet-Biedl Syndrome Data at Three Years
Three years of treatment with setmelanotide showed sustained, meaningful benefit in weight-related measures in patients with BBS, as reported in a poster entitled, “3-Year Setmelanotide Weight Outcomes in Patients with Bardet-Biedl Syndrome and Obesity” by lead author Jack Yanovski, M.D., Ph.D., Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health.

POMC and LEPR Deficiency Data at Four Years
Long-term treatment with setmelanotide demonstrated sustained weight-related efficacy in pediatric and adult patients with obesity due to POMC or LEPR deficiency, as reported in the oral presentation, “Four-Year Setmelanotide Weight Outcomes of Patients with POMC and LEPR Deficiency Obesity” by lead author Wendy K. Chung, M.D., Ph.D., Division of Molecular Genetics, Department of Pediatrics, Columbia University, New York, NY.

Rhythm announced additional presentations including:

• In an oral presentation, “Impact of Setmelanotide on Metabolic Syndrome Risk in Patients with POMC and LEPR Deficiency” by lead author Martin Wabitsch, M.D., Ph.D., Department of Pediatrics and Adolescent Medicine, University of Ulm in Germany, data showed intervention with setmelanotide may reduce the risk of future metabolic syndrome, cardiovascular disease (CVD) and type 2 diabetes mellitus (T2DM) in patients with obesity due to POMC or LEPR deficiency obesity.
• In a poster titled, “Cardiac, Renal, and Endocrine/Diabetes Mellitus Outcomes in Children with Bardet-Biedl Syndrome” by lead author Jeremy Pomeroy, Ph.D., M.S., of the Marshfield Clinic Research Institute, researchers showed that the severity of obesity was associated with increased prevalence of cardiac, endocrine/diabetes, and renal outcomes early in life based on an analysis of 318 pediatric patients with BBS enrolled in the Clinical Registry Investigating BBS (CRIBBS). Researchers concluded that timely diagnosis and early implementation of hyperphagia and weight management strategies in pediatric patients with BBS may reduce the risk and burden associated with these comorbidities.
• In a poster titled, “Impact of Setmelanotide on Metabolic Syndrome Risk in Patients with Bardet-Biedl Syndrome” by lead author Andrea Haqq, M.D., M.H.S., Division of Pediatric Endocrinology at the University of Alberta, data showed that treatment response with one year of setmelanotide was associated with reductions in MetS-Z-BMI score in pediatric patients with BBS, which are associated with reduced risk of metabolic syndrome, CVD and T2DM.