Setmelanotide Advancement In Prader-Willi Syndrome: Rhythm Pharmaceuticals' Latest Clinical Data And Market Impact

Clinical Trial Update And Disease Background

Rhythm Pharmaceuticals (RYTM) is set to unveil early-stage findings from its Phase 2 exploratory trial investigating Setmelanotide’s efficacy in treating Prader-Willi syndrome, with the presentation occurring via live webcast on December 11, 2025.

Prader-Willi syndrome adalah a rare inherited genetic condition marked by pathological hunger sensations (hyperphagia), severe weight accumulation, and dysfunction in multiple endocrine systems. The condition presents significant clinical challenges, as available therapeutic interventions remain limited and patients frequently experience dangerous health complications stemming from progressive weight gain that cannot be effectively controlled through conventional means.

Setmelanotide: Current Approvals And Pipeline Progress

Setmelanotide, administered as a once-daily subcutaneous injection, has already received regulatory clearance in the United States and more than 15 additional countries for obesity management and hunger suppression linked to Bardet-Biedl syndrome or genetic deficiencies in POMC, PCSK1, or LEPR. The medication is sold globally under the brand designation IMCIVREE.

The pharmaceutical firm submitted a supplemental new drug application (sNDA) seeking FDA approval for Setmelanotide in acquired hypothalamic obesity, with regulatory decision anticipated by March 20, 2025. Beyond this indication, Rhythm’s broader development portfolio encompasses multiple MC4R receptor agonists in various stages of advancement, including Bivamelagon—a next-generation investigational compound—and RM-718, a differentiated therapeutic candidate. The company is also pursuing preclinical research into small-molecule treatments for congenital hyperinsulinism. RM-718 is presently undergoing Phase I evaluation, while Bivamelagon’s pivotal Phase III trial is anticipated to initiate in 2026 following regulatory alignment discussions with U.S. and European authorities.

Financial Performance And Stock Trajectory

Third-quarter 2025 financial results demonstrated robust commercial traction, with Rhythm recording $51.3 million in global net product revenue specifically from IMCIVREE sales, reflecting substantial year-on-year expansion. Domestic U.S. market sales increased 19% sequentially, reaching $38.2 million, though international revenue experienced fluctuations due to ordering patterns. As of September 30, 2025, the company maintained $416.1 million in liquid assets—comprising cash, cash equivalents, and short-term investments—providing substantial financial runway for continued research advancement.

RYTM shares opened at approximately $48 on April 7, 2025, when initially highlighted. Throughout the preceding 12 months, the stock has ranged between $45.90 and $116.00. The most recent close registered $104.77, representing a 4.03% daily increase, with after-hours trading activity pushing the price higher to $108.80—an additional 3.85% gain.