Our Focus

“As we advance our understanding of the underlying biology of the MC4R pathway, our clinical development programs are designed to evaluate and bring a potential new treatment option to individuals living with severe obesity and hyperphagia.”

– Murray Stewart, MD, Chief Medical Officer

Rare genetic diseases of obesity may be caused by an impaired melanocortin-4 receptor (MC4R) pathway resulting from genetic variants. Setmelanotide, Rhythm’s investigational drug which is an MC4R agonist, has the potential to restore the function of an impaired MC4R pathway, and in doing so, help to reduce hunger and body weight in individuals living with these diseases. Rhythm is currently focused on investigating the efficacy and safety of setmelanotide in the treatment of several rare genetic diseases.

Clinically, these diseases share the hallmark characteristics of early-onset severe obesity and insatiable hunger (hyperphagia).

Setmelanotide Phase 3 Pivotal Studies

Setmelanotide Phase 2 Basket Study

* Calculations regarding non-syndromic disorder assume a U.S. population of 327 million, of which 1.7% have early-onset, severe obesity (Hales et al in JAMA – April 2018: Trends Obesity and Severe Obesity Prevalence in US Youth and Adults by Sex and Age, 2007-2008 to 2015-2016);

**Estimated prevalence of U.S. individuals with rescuable variants of the MC4R.

Rhythm’s Uncovering Rare Obesity® Program is the only no-cost, comprehensive genetic testing program for rare genetic diseases of obesity.
Learn more →

For definitions to select clinical terms within this site, please visit glossary

For more information on Rhythm’s publications and presentations, please visit publications