Rhythm Pharmaceuticals
Rhythm Pharmaceuticals focuses on transforming the lives of patients with hyperphagia and severe obesity caused by rare MC4R pathway diseases through the development of targeted therapeutics.
Company Overview
Rhythm Pharmaceuticals is a biotechnology company focused on transforming the lives of patients and their families affected by hyperphagia and severe obesity caused by rare melanocortin-4 receptor (MC4R) pathway diseases. The company develops therapeutics targeting the MC4R pathway for treating rare genetic diseases of obesity, including Proopiomelanocortin (POMC) Deficiency Obesity, Leptin Receptor (LEPR) Deficiency Obesity, Bardet-Biedl Syndrome (BBS), and several others.
FDA Approved Product: IMCIVREE (Setmelanotide)
IMCIVREE (setmelanotide) is approved by the U.S. FDA for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1, or LEPR deficiency, or Bardet-Biedl syndrome (BBS). It is also authorized by the European Commission and the UK's Medicines & Healthcare Products Regulatory Agency (MHRA) for the treatment of obesity and control of hunger associated with genetically confirmed BBS or other specific genetic deficiencies in both adults and children.
Clinical Trials and Research Programs
Rhythm Pharmaceuticals conducts extensive clinical trials to evaluate its therapies. Notable trials include the Phase 3 EMANATE trial and the Phase 2 DAYBREAK trial, both focused on assessing setmelanotide in people with obesity and specific genetic variants relevant to the MC4R pathway. The company is also advancing setmelanotide in additional trials, including a Phase 3 trial in pediatric patients and another evaluating a once-weekly formulation. Additionally, Rhythm has a translational research program supported by the largest known DNA database focused on obesity.
Future Developments: RM-718 and LB54640
Rhythm Pharmaceuticals is developing RM-718, an investigational, weekly MC4R-specific agonist, and LB54640, an investigational oral small molecule MC4R agonist. These developments aim to provide new therapeutic options for patients with rare genetic diseases of obesity. The progress in these areas reflects the company's commitment to transforming the treatment landscape for obesity caused by genetic factors.
Educational and Genetic Testing Programs
Rhythm Pharmaceuticals operates the GOLD Academy, a program designed to educate healthcare providers about rare genetic causes of obesity and the significance of the MC4R pathway. The company also has a comprehensive genetic testing program called Uncovering Rare Obesity, which aims to help identify rare genetic diseases of obesity. These initiatives emphasize the company's dedication to enhancing the understanding and identification of rare genetic obesity conditions.