Our Pipeline

We are studying rare genetic disorders of obesity.

“We know that there are many more genes involved in the MC4R pathway, and we have the capability to explore the effect of these genes on obesity as we uncover the potential of setmelanotide or other products for the treatment of rare genetic disorders of obesity.”

– Murray Stewart, MD, Chief Medical Officer

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 or LEPR heterozygous 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

Preclinical

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, oral ghrelin o-acyltransferase inhibitor currently in pre-clinical development. We are assessing the safety and therapeutic potential of RM-853 as well as opportunities to identify populations that may benefit from a mechanistically rationalized therapeutic approach. RM-853 is designed to block GOAT, the key enzyme involved in the production of the active form of ghrelin, with the potential 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.