Stage (next event)
Catalyst Info & Data Links
TITLE: Setmelanotide for Alström syndrome - Phase 3 (Top-line Data)
ClinicalTrials.gov (NCT03746522): Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity
WHAT IS THE CATALYST EVENT?
WHEN WILL THE EVENT (OR DID THE EVENT) OCCUR?
Late Q4 2020/ Early Q1 2021
MECHANISM OF ACTION
Setmelanotide is an investigational, melanocortin-4 receptor (MC4R) agonist. The MC4R is part of the key biological pathway that independently regulates hunger, caloric intake, and energy expenditure. Variants in genes may impair the function of the MC4R pathway, potentially leading to hyperphagia and early-onset, severe obesity. Rhythm is currently developing setmelanotide as a targeted therapy to potentially restore the function of an impaired MC4R pathway and, in so doing, potentially reduce hunger and weight in patients with rare genetic disorders of obesity. Currently, no pharmacologic therapies exist to treat these conditions.
Updated by HC
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