Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM) has announced preliminary unaudited net revenues from global sales of IMCIVREE® (setmelanotide) and provided updates on its clinical development pipeline. The company anticipates continued growth, driven by its focus on rare neuroendocrine diseases.
Financial Highlights
For the fourth quarter of 2024, Rhythm Pharmaceuticals expects net revenues from global IMCIVREE® sales to be approximately $42 million, marking a 26% increase from the previous quarter. Full-year 2024 net revenues are projected to reach approximately $130 million, compared to $77.4 million in 2023. U.S. sales contributed approximately 74% of the fourth-quarter revenues and 73% of the full-year revenues. The company plans to report its full financial results in late February 2025.
Setmelanotide Development
Acquired Hypothalamic Obesity (HO): Rhythm remains on track to report topline data from its global Phase 3 trial evaluating setmelanotide in acquired HO in the first half of 2025. Enrollment is complete in the supplemental Japanese cohort, which will support a regulatory submission in Japan.
Congenital HO: The company anticipates enrolling the first patients with congenital HO in a 34-week substudy of the ongoing global Phase 3 trial in the first quarter of 2025. This substudy is independent of the pivotal Phase 3 trial in acquired hypothalamic obesity.
Genetically Caused MC4R Pathway Diseases: Enrollment is complete in the Phase 3 EMANATE trial, which includes substudies for SH2B1 (n=121), POMC and/or PCSK1 (n=79), SRC1 (n=73), and LEPR (n=23) deficiencies. Topline data from the EMANATE trial is expected in the first half of 2026. The primary endpoint for each substudy is the difference in mean percent change in BMI from baseline to 52 weeks in setmelanotide arm compared to placebo arm.
Prader-Willi Syndrome (PWS): Rhythm plans to initiate a new, 26-week, open-label Phase 2 trial evaluating setmelanotide for the treatment of PWS in Q1 2025. The trial will enroll up to 20 patients aged 6 to 65 years, with dose escalation up to 5 mg/day. The primary endpoints are safety and tolerability, with secondary endpoints assessing weight, hyperphagia, behavior, and pharmacokinetics. PWS affects an estimated 400,000 people worldwide and 20,000 in the United States. Currently, there are no approved therapies that effectively reduce hyperphagia or address low energy expenditure in PWS patients.
Other Pipeline Programs
Bivamelagon (LB54640): Rhythm is on track to complete enrollment in the Phase 2 trial evaluating bivamelagon, an oral MC4R agonist, in acquired HO in the first quarter of 2025.
RM-718: Following acceptance of a protocol amendment, Rhythm expects to begin dosing patients with acquired HO in Part C of the Phase 1 trial evaluating RM-718, a weekly MC4R agonist, in the first quarter of 2025. The company plans to enroll up to 30 patients with acquired hypothalamic obesity for 16 weeks in Part C of this Phase 1 trial.
David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm, stated, “Rhythm enters 2025 poised for the next level of growth... We are set to begin our congenital HO substudy with the potential to further expand the opportunity related to injury to and or failure of the hypothalamus to develop. These indications represent significant unmet medical needs and potentially transformative opportunities for Rhythm.”
