RHYTHM PHARMACEUTICALS, INC. (RYTM)

Rhythm Pharmaceuticals is a global, commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases, specifically hyperphagia and severe obesity caused by impaired signaling in the melanocortin-4 receptor (MC4R) pathway, a brain pathway that regulates hunger, caloric intake and energy expenditure. Its lead product, IMCIVREE (setmelanotide), an MC4R agonist for which it holds worldwide rights, was first approved in the United States in 2020 and is marketed in the United States, European Union, United Kingdom, Canada and other countries, with approvals, reimbursed access or named patient sales in more than 25 countries beyond the U.S. It is approved to reduce excess body weight and maintain weight reduction in patients aged 2 years and older with obesity due to Bardet-Biedl syndrome (BBS) or POMC, PCSK1 or LEPR deficiency, with the EC and MHRA authorizing similar uses and Health Canada approving it for patients aged 6 and older. The company's strategy centers on three underserved areas.

The first is hypothalamic dysfunction, where regulatory reviews of setmelanotide for acquired hypothalamic obesity are ongoing in the U.S. (FDA PDUFA goal date of March 20, 2026) and Europe, a Japanese cohort is being studied, and a substudy in congenital hypothalamic obesity is underway. The second is Prader-Willi syndrome, where setmelanotide is in an exploratory Phase 2 trial and RM-718 is in a Phase 1/2 trial. The third is rare genetic MC4R pathway diseases, where the Phase 3 EMANATE trial, with four substudies, and the completed Phase 2 DAYBREAK trial are evaluating additional genetically defined populations, including POMC/PCSK1 and LEPR heterozygous insufficiency, SRC1 deficiency and SH2B1 deficiency.

The pipeline also includes two earlier-stage MC4R agonists that are selective for MC4R and spare MC1R: bivamelagon, an oral small molecule with a Phase 3 hypothalamic obesity trial planned by year-end 2026, and RM-718, a weekly subcutaneous therapy. The company is also advancing preclinical candidates for congenital hyperinsulinism. It uses what it describes as the largest known obesity-focused DNA database, with approximately 120,000 sequencing samples, to support epidemiology, patient finding and genetic testing programs.

Field teams in the United States and Europe engage physicians treating severe obesity, and a team in Japan is preparing for potential registration and launch. As of February 1, 2026, the company had 414 employees, including 126 in 11 countries outside North America.