Rhythm Pharmaceuticals Presents Data Demonstrating BMI Reduction in Patients with Hypothalamic Obesity Treated with Setmelanotide over Six Months at ENDO 2023 - Seite 2

Consistent with prior clinical experience in other rare MC4R pathway diseases, setmelanotide was observed to be generally well tolerated and no new safety concerns were observed in the long-term extension trial, as of the cutoff date Nov. 30, 2022.

Additional presentations
Rhythm and its collaborators presented three additional posters at ENDO 2023:

As presented in a poster titled, “Treatment History and Comorbidities Reported by Patients with Hypothalamic Obesity Treated with Setmelanotide in a Phase 2 Trial,” 78% of patients with hypothalamic obesity who had participated in that trial had attempted and failed lifestyle modifications, such as diet modification and calorie restriction, before enrolling in this trial. A total of nine patients had used and failed to achieve weight loss with pharmacotherapies, including seven who tried using multiple anti-obesity medications.

Data presented on a poster entitled, “Effect of Setmelanotide on Metabolic Parameters and Vital Signs in a Phase 2 Trial of Patients with Hypothalamic Obesity,” showed that most patients experienced reduction in waist circumference with favorable changes in body composition and no adverse changes in metabolic, glycemic, or vital sign parameters.

Also presented was, “Trial Design of a Double-blind, Randomized, Placebo-Controlled, Phase 3 Study of Setmelanotide in Patients with Hypothalamic Obesity.” This ongoing pivotal trial is designed to enroll 120 patients 4 years old or older randomized 2:1 to setmelanotide therapy or placebo for a total of 60 weeks, including up to eight weeks for dose titration. The primary endpoint is the percent change in BMI after approximately 52 weeks on a therapeutic regimen of setmelanotide versus placebo. Rhythm expects to complete patient enrollment in the first quarter of 2024.

All Rhythm’s presentations from ENDO will be available on the Publications and Presentations section of its website: https://www.rhythmtx.com/publications/.

About Hypothalamic Obesity
Hypothalamic obesity is a rare, acquired form of extreme obesity that occurs following damage to the hypothalamic region of the brain, which includes the MC4R pathway and is responsible for controlling physiological functions such as hunger and weight regulation. It most frequently follows the growth or surgical removal of craniopharyngioma, astrocytoma or other rare brain tumors. Patients experience rapid weight gain, a reduction in energy expenditure, and an increase in hunger in the first six to 12 months following tumor resection, and ultimately develop severe obesity. In addition, people living with hypothalamic obesity may also experience delayed puberty and infertility, decreased physical activity, excessive daytime sleepiness, attention hyperactivity disorder, seizures and psychiatric conditions. Based on an analysis of incidence rates and prevalence reports of certain brain tumor types, as well as survival and obesity rates tied to these brain tumor types, Rhythm estimates there are approximately 5,000-10,000 patients living with hypothalamic obesity in the U.S. with approximately 500 new cases each year. There are no FDA approved therapies for hypothalamic obesity.