Our Pipeline

We are studying rare genetic disorders of obesity.

 

Rhythm’s Pipeline


Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist in late-stage clinical development for the treatment of rare genetic disorders of obesity. RM-853 is a GOAT inhibitor currently in preclinical development.

Disorder Early-Stage Development Phase 2 Phase 3 Registration PHASE
Setmelanotide Pivotal Studies

POMC deficiency obesity

Registration

LEPR deficiency obesity

Registration

Bardet-Biedl syndrome

PHASE 3

Alström syndrome

PHASE 3
Setmelanotide Basket Study

POMC or LEPR heterozygous deficiency obesity

PHASE 2

SRC1 deficiency obesity

PHASE 2

SH2B1 deficiency obesity

PHASE 2

MC4R deficiency obesity

PHASE 2

Smith-Magenis syndrome

PHASE 2

Additional disorders*

PHASE 2
RM-853

Prader-Willi syndrome**

Early Stage

 

Setmelanotide is an investigational, MC4R agonist in late-stage clinical development for the treatment of rare genetic disorders of obesity

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 receptor. Setmelanotide has the potential to reduce weight and reduce hunger. It is in clinical development for individuals living with rare genetic disorders of obesity.

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to setmelanotide for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin 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, BBS and Alström 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 European Commission has adopted the EMA’s Committee for Orphan Medicinal Products’ positive opinion and designated setmelanotide as an orphan medicinal product for the treatment of patients with POMC deficiency, LEPR deficiency, BBS and Alström syndrome.

Rhythm’s pivotal, phase 3 studies of setmelanotide in POMC and LEPR deficiency obesities met their primary and key secondary endpoints with statistically significant and clinically meaningful results. The Company submitted its New Drug Application to the FDA in the first quarter of 2020 and expects to submit its Marketing Authorization Application to EMA in the second quarter of 2020.

RM-853 (preclinical)


RM-853 is a pre-clinical 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.

 

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

For information about access to investigational therapeutics outside of clinical trials, please visit Policy on Expanded Access to lnvestigational Drugs

* Rhythm is currently assessing setmelanotide in additional disorders, including POMC epigenetic disorders LEP and CPE deficiency obesity, as part of investigator-initiated protocols within the basket study. Given the recent discovery of these rare disorders of the MC4R pathway, there is currently limited or no genetic sequencing or epidemiology data that defines prevalence. However, Rhythm believes that these are rare disorders which may be setmelanotide-responsive.

** Rhythm is currently assessing opportunities to further evaluate setmelanotide in Prader-Willi syndrome and plans to pursue these in parallel with the development of RM-853.