Rhythm Pharmaceuticals, Inc.

We are a global, commercial-stage biopharmaceutical company focused on rare neuroendocrine diseases. We develop melanocortin-4 receptor (MC4R) agonists, which are precision medicines for hyperphagia and severe obesity caused by rare MC4R pathway diseases. These diseases include acquired or congenital hypothalamic obesity, Bardet-Biedl syndrome (BBS), and Prader-Willi syndrome (PWS). The MC4R pathway in the brain regulates hunger, caloric intake, and energy expenditure, impacting body weight. Our lead product, IMCIVREE (setmelanotide), is an MC4R agonist for which we hold worldwide rights. It is marketed in the United States, European Union (EU), United Kingdom, Canada, and other countries and regions for certain rare MC4R pathway diseases, including BBS. We are also developing two earlier-stage investigational MC4R agonists with global rights: bivamelagon (formerly LB54640), an oral small molecule, and RM-718, designed for weekly subcutaneous administration. These are designed to be highly selective for MC4R and are not expected to cause hyperpigmentation. We anticipate launching IMCIVREE in the United States in 2026 for acquired hypothalamic obesity, a rare disease caused by hypothalamic injury that may impair MC4R pathway signaling. There are approximately 10,000 patients in the United States living with acquired hypothalamic obesity, with an annual incidence of 500 new cases. Similar prevalence and incidence are observed in Europe, and there are between 5,000 and 8,000 patients in Japan.