Our Pipeline
“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.
