Our Pipeline

We are studying rare genetic diseases of obesity.

“We know that all obesity is not the same. There are many genes involved in the MC4R pathway associated with severe obesity and insatiable hunger, and we are continuing efforts to explore the effect of these genes on obesity as we uncover the potential of setmelanotide or other investigational products for the treatment of rare genetic diseases of obesity”

– Linda Shapiro, M.D., Ph.D.,  Chief Medical Officer

Rhythm’s Pipeline

Setmelanotide is a melanocortin-4 receptor (MC4R) agonist designed to address the underlying cause of obesities driven by genetic variants that impair function of the MC4R pathway.

IMCIVREE (setmelanotide) was approved in 2020 by the U.S. Food and Drug Administration for chronic weight management in adult and pediatric patients 6 years old and older with obesity due to POMC, PCSK1 or LEPR deficiency. In 2021, it was authorised by the European Commission and Great Britain’s MHRA for the treatment of obesity and the control of hunger associated with genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above. For more information, including important safety information, visit IMCIVREE.com

We are advancing setmelanotide in several additional trials. Here is our investigational pipeline:

Phase 2 Phase 3 Regulatory Submission PHASE
Setmelanotide (daily)

Bardet-Biedl and Alström syndromes

REGULATORY SUBMISSION

EMANATE Trial

Five independent, genetically-defined sub-studies

PHASE 3

Pediatrics* Trial

Open-label children 2- to 6-years old

PHASE 3

Exploratory Basket Study

Ongoing study MC4R rescuable

PHASE 2

Hypothalamic Obesity

Exploratory, open-label

PHASE 2

DAYBREAK Trial

31 additional genes

PHASE 2
Weekly formulation

Switch Trial

Double-blind trial in patients** previously in open-label extension study of daily setmelanotide.

PHASE 3

De novo (planned for 2H2022)

Double-blind, placebo-controlled study in patients with BBS

PHASE 3

*Enrolled in the pediatrics trial are patients with obesity due to biallelic POMC, PCSK1 or LEPR deficiency or a clinical diagnosis of BBS with genetic confirmation.

**Enrolled in the switch trial are patients with obesity due to biallelic or heterozygous POMC, PCSK1 or LEPR deficiency or a clinical diagnosis of BBS with genetic confirmation.