EMANATE: A Study of Setmelanotide in Patients With Specific Gene Variants in the MC4R Pathway

Summary

The protocol describes a randomized, double-blind, placebo-controlled trial with independent sub-studies of setmelanotide in patients with obesity and at least one of the specific gene variants in the Melanocortin-4 Receptor pathway: * POMC or PCSK1 (Sub-study 035a) * LEPR (Sub-study 035b) * SRC1 (Sub-study 035c) * SH2B1 (Sub-study 035d) The objectives and endpoints are identical for these sub-studies.

Timeline

Start

2021-12-10

Primary completion

2025-12-22

Completion

2026-12-31

Outcome

Missed primary endpoint

release “today announced the topline results from its EMANATE trial. The four substudies of this global, Phase 3 trial evaluating setmelanotide did not meet primary endpoints.” rhythmtx.com ↗

release “Phase 3 EMANATE trial. The trial did not achieve the primary endpoint in each of its four independent substudies” rhythmtx.com ↗

Publications

• Delplanque J, Le Collen L, Loiselle H, Leloire A, Toussaint B, Vaillant E, Charpentier G, Franc S, Balkau B, Marre M, Henriques E, Buse Falay E, Derhourhi M, Froguel P, Bonnefond A. Monoallelic pathogenic variants in LEPR do not cause obesity. Am J Hum Genet. 2024 Dec 5;111(12):2668-2674. doi: 10.1016/j.ajhg.2024.10.014. Epub 2024 Nov 18.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Setmelanotide	Protein / enzyme biologic	—	Subcutaneous

Indications