Our Pipeline

We are studying rare genetic disorders of obesity.

Setmelanotide


An investigational, melanocortin-4 receptor (MC4R) agonist in clinical development for the treatment of rare genetic disorders of obesity


Proof
of Concept

Phase 1

Phase 2

Phase 3
Setmelanotide
POMC Deficiency Obesity
LEPR Deficiency Obesity
Bardet-Biedl Syndrome
Alström Syndrome
MC4R Pathway Heterozygous Obesity
POMC Epigenetic Disorders
✔ Human Proof of Concept

For more information about Rhythm’s clinical trials, please visit clinicaltrials.gov

Pre-approval Access Policy

Rhythm’s lead product candidate, setmelanotide, is an MC4R agonist designed to restore impaired MC4R pathway function caused by genetic variants that occur upstream of the MC4R. Setmelanotide has the potential to affect weight loss and reduce hunger, and it is specifically in clinical development for individuals affected by rare genetic disorders of obesity.

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to setmelanotide for the treatment of obesity associated with genetic variants upstream of the MC4R in the MC4R pathway, which includes POMC deficiency obesity, LEPR deficiency obesity, Bardet-Biedl syndrome (BBS), and Alström syndrome. Also, the FDA has granted Orphan Drug Designation for setmelanotide for the treatment of POMC deficiency obesity, LEPR deficiency obesity and Bardet-Biedl syndrome. The European Medicines Agency (EMA) has also granted PRIority MEdicines (PRIME) designation for setmelanotide for the treatment of obesity and the control of hunger associated with deficiency disorders of the MC4R pathway. The EMA’s Committee for Orphan Medicinal Products issued a positive opinion recommending setmelanotide for designation as an orphan medicinal product for the treatment of patients with BBS.

Proof-of-concept has been established with setmelanotide in POMC deficiency obesity, LEPR deficiency obesity, BBS, and Alström syndrome, and Rhythm is currently evaluating setmelanotide in pivotal trials for these indications:

  • Rhythm’s pivotal, phase 3 studies of setmelanotide in POMC and LEPR deficiency obesity met their primary and key secondary endpoints with statistically significant and clinically meaningful results, and the company expects to submit its New Drug Application to the FDA in the fourth quarter of 2019 or first quarter of 2020
  • The Company expects to submit its Marketing Authorization Application to EMA following completion of the submission to the FDA.

RM-853 (preclinical)


An investigational ghrelin O-acyltransferase (GOAT) inhibitor

RM-853 is a potent, orally available GOAT inhibitor currently in preclinical development for Prader-Willi syndrome (PWS). In people living with PWS, levels of active ghrelin are elevated, contributing to insatiable hunger and severe obesity. RM-853 is designed to block GOAT, the key enzyme involved in the production of the active form of ghrelin, with the expected effect of lowering active ghrelin levels.