Rhythm Pharmaceuticals Announces Completion of Screening for Enrollment in Setmelanotide Phase 3 Hypothalamic Obesity Trial and Additional Updates

-- Phase 3 hypothalamic obesity top-line data expected in 1H2025 --

-- Positive reimbursement decision achieved for IMCIVREE® (setmelanotide) to treat patients with Bardet-Biedl syndrome and POMC/LEPR deficiencies in Spain --

-- IND application for RM-718 accepted by the FDA --

BOSTON, Jan. 04, 2024 -- Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients and their families living with rare neuroendocrine diseases, provided a business update and announced it has completed screening for enrollment in the pivotal, Phase 3 clinical trial evaluating setmelanotide in patients with acquired hypothalamic obesity.

Rhythm closed screening and anticipates overenrolling the ongoing phase 3 hypothalamic obesity trial with more than 140 patients consented and going through active screening and baseline assessments. In this trial, patients with acquired hypothalamic obesity aged 4 years or older will be randomized 2:1 to setmelanotide therapy or placebo for a total of 60 weeks, including up to eight weeks for dose titration. The Company expects to obtain top-line study results in H1 2025.

“2023 was a truly transformational year for Rhythm, as we made excellent progress with the execution of our IMCIVREE® (setmelanotide) commercial plan while also advancing important development efforts to expand the number of patients living with hyperphagia and severe obesity that could potentially benefit from this therapy,” said David Meeker, M.D., Chair, President and Chief Executive Officer of Rhythm. “We believe the rapid over enrollment in less than one year and execution of our Phase 3 hypothalamic obesity trial is a strong indicator of the unmet need for an effective therapy for patients who have no approved therapeutic options and the community’s excitement around setmelanotide’s potential to make a meaningful difference in their lives.”... Rhythm Pharmaceuticals' Press Release -

Rhythm Pharmaceuticals : RM-493 Reverses Obesity and Insulin Resistance in Obese Primates

June 21, 2010— Rhythm, a biotechnology company developing peptide therapeutics for metabolic diseases, announced a study of obese primates treated with RM-493 showing a reversal of obesity and insulin resistance, and improvement in both heart rate and blood pressure. RM-493 is a novel peptide agonist targeting the melanocortin 4 receptor (MC4R) that is a clinical candidate for the treatment of obesity and diabetes. The study, “A Novel MC4R Agonist Reduces Body Weight and Improves Glucose Homeostasis in High Fat Diet-Induced Obese Rhesus Macaques,” was presented at the 92nd annual meeting of the Endocrine Society (ENDO 2010) in San Diego.

“The goal of this study was to determine the effects of RM-493 on food intake, weight, and cardiovascular function, as well as to evaluate safety with long-term treatment,”... Rhythm's Press Release -

Ipsen Grants Rhythm Exclusive Worldwide License for Two Programs in the Field of Metabolic Disorders

March 12, 2010— Ipsen (Euronext: FR0010259150; IPN), a global biotechnology specialty care group, and Rhythm Pharmaceuticals (Rhythm), a biotechnology company developing peptide therapeutics for metabolic diseases, announced that they have concluded a license agreement for Ipsen’s proprietary peptide therapeutics targeting obesity, metabolic diseases, and gastrointestinal disorders. Under the terms of the agreement, Ipsen has granted Rhythm an exclusive worldwide license for research, development, and commercialization of its melanocortin and ghrelin programs originating from Ipsen research.


“The agreement with Rhythm is a clear catalyst for the development of proprietary molecules from Ipsen in promising indications within the area of metabolic diseases, which are outside of our core strategic focus... Rhythm Pharmaceuticals' Press Release - [PDF] Ipsen's Press Release -