Setmelanotide for the Treatment of Leptin Receptor (LEPR) Deficiency Obesity

Phase 3 Completed 15 enrolled Rhythm Pharmaceuticals, Inc.

NaSingle-groupOpen-labelTreatment

Summary

To demonstrate statistically significant and clinically meaningful effects of setmelanotide on percent body weight change in participants with LEPR deficiency obesity due to rare bi-allelic or loss-of function mutations at the end of 1 year of treatment.

Timeline

Start: 2018-01-30
Primary completion: 2020-09-25
Completion: 2020-09-25

Outcome

Met primary endpoint

release “LEPR Primary: Proportion of Participants Achieving at Least 10% Change in Body Weight i 45.5% (p=0.0001)” rhythmtx.com ↗

release “Phase 3 clinical trials evaluating setmelanotide for the treatment of POMC and LEPR deficiency obesities. Both trials met their primary endpoints” rhythmtx.com ↗

release “53.3% of patients in the LEPR trial (8/15; p<0.0001) achieved ≥10% weight loss from baseline at 52 weeks” rhythmtx.com ↗

Publications

Clement K, van den Akker E, Argente J, Bahm A, Chung WK, Connors H, De Waele K, Farooqi IS, Gonneau-Lejeune J, Gordon G, Kohlsdorf K, Poitou C, Puder L, Swain J, Stewart M, Yuan G, Wabitsch M, Kuhnen P; Setmelanotide POMC and LEPR Phase 3 Trial Investigators. Efficacy and safety of setmelanotide, an MC4R agonist, in individuals with severe obesity due to LEPR or POMC deficiency: single-arm, open-label, multicentre, phase 3 trials. Lancet Diabetes Endocrinol. 2020 Dec;8(12):960-970. doi: 10.1016/S2213-8587(20)30364-8. Epub 2020 Oct 30.

Drugs

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