1. Positive results from setmelanotide Phase 2 trial for PWS reported. 2. Seventeen patients remain on therapy, showing promising BMI reductions. 3. Phase 3 trial of setmelanotide to commence pending Phase 2 success. 4. Initiation of Phase 1 trial for RM-718 in PWS patients announced. 5. Upcoming conference call on December 11 to discuss trial updates.
FAQ
Why Bullish?
Positive clinical trial data generally boosts investor confidence, reminiscent of similar past trials in biotech leading to price increases.
How important is it?
The advancements in clinical trials may lead to FDA approval, significantly impacting RYTM's market position and financial outlook.
Why Long Term?
Future earnings could improve if Phase 3 trials succeed, similar to other successful biotech developments that resulted in sustained prices.
Rhythm Pharmaceuticals Reports Positive Preliminary Data from Phase 2 Trial for Setmelanotide in Prader-Willi Syndrome
BOSTON, Dec. 11, 2025 (GLOBE NEWSWIRE) — Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM) has announced promising preliminary results from its exploratory Phase 2 trial of setmelanotide in patients diagnosed with Prader-Willi syndrome (PWS). The company is looking to advance setmelanotide into a Phase 3 registrational trial pending the successful completion of this Phase 2 trial.
Key Trial Insights and Findings
Preliminary data reveal significant reductions in Body Mass Index (BMI) and hyperphagia in patients receiving setmelanotide. The trial enrolled 18 participants aged 6 to 65 years, with 17 remaining on active therapy. Notable insights include:
- Six (6) out of eight (8) patients reached Month 3 of therapy with measurable BMI reductions.
- Three (3) out of five (5) patients sustained through Month 6 showed reduced BMI, with some experiencing deeper declines.
- Six (6) out of seven (7) evaluable patients displayed significant decreases in Hyperphagia Questionnaire for Clinical Trials (HQ-CT) scores.
- Safety and tolerability results consistent with the established clinical profile of setmelanotide.
Supporting the Need for New Treatments
“There remains a profound unmet need in the PWS patient population,” stated Jennifer Miller, M.D., principal investigator for the trial. She highlighted that hyperphagia and severe obesity pose significant health challenges for those affected by PWS. The interim data provides insights into a possible treatment that addresses the unique requirements of PWS patients.
Future Directions for Rhythm Pharmaceuticals
Rhythm Pharmaceuticals is also moving ahead with a Part D arm in their Phase 1 study of the weekly MC4R agonist RM-718, which will enroll up to 20 patients with PWS. The first patient is expected to enter screening as early as December 2025.
“We are encouraged by these preliminary results, which support advancing setmelanotide into a registrational Phase 3 trial for PWS,” said David Meeker, M.D., CEO of Rhythm. He indicates a commitment to exploring the potential of MC4R agonism for patients who currently have few treatment options available.
Conference Call Details
Rhythm Pharmaceuticals will host a live conference call at 8:00 a.m. ET on December 11 to discuss these findings. The call will be available via the company’s Investor Relations website, and participants are encouraged to join 10 minutes early.
About Setmelanotide and Prader-Willi Syndrome
PWS is a rare genetic disorder characterized by numerous physical, mental, and behavioral challenges, primarily a relentless sense of hunger that often begins around age two. The condition affects approximately 400,000 people globally, with around 20,000 individuals in the U.S.
Rhythm’s lead drug, setmelanotide, is FDA-approved to manage severe obesity in patients with specific genetic conditions. Rhythm is actively pursuing additional clinical applications for setmelanotide, along with researching other MC4R agonists.
About Rhythm Pharmaceuticals
Rhythm Pharmaceuticals is dedicated to improving the quality of life for patients with rare neuroendocrine diseases. The company is advancing a portfolio that includes not only setmelanotide but also investigational compounds targeting congenital hyperinsulinism and other rare disorders.