Rhythm Pharma To Present Preliminary Phase 2 Data Of Setmelanotide In Prader-Willi Syndrome Today

(RTTNews) - Rhythm Pharmaceuticals, Inc. (RYTM) will disclose preliminary data from its exploratory Phase 2 trial of Setmelanotide in patients with Prader-Willi syndrome during a live webcast scheduled for December 11, 2025.

Prader-Willi syndrome (PWS) is a rare genetic disorder characterized by insatiable hunger (hyperphagia), severe obesity, and multiple endocrine abnormalities. Current treatment options are limited, and patients often struggle with life-threatening complications related to uncontrolled weight gain.

Setmelanotide, a once-daily injection, is already approved for the treatment of obesity and to help control hunger associated with Bardet-Biedl syndrome or biallelic POMC, PCSK1, or LEPR deficiency, and is marketed under the brand name IMCIVREE. The drug is approved in the U.S. and over 15 other countries.

Rhythm has also filed a supplemental new drug application (sNDA) for Setmelanotide in acquired hypothalamic obesity, with the FDA decision due on March 20, 2025.

Beyond PWS, Rhythm continues to advance a broad pipeline of MC4R agonists, including Bivamelagon, an investigational next-generation therapy, and RM-718, a differentiated candidate in development, as well as preclinical small molecules for congenital hyperinsulinism.

RM-718 is currently being evaluated in a phase I trial, while a pivotal phase III trial for Bivamelagon is planned to begin in 2026, pending alignment with U.S. and European regulatory agencies.

Financial Numbers

Rhythm reported third-quarter 2025 net product revenue of $51.3 million from global sales of IMCIVREE, representing strong year-over-year growth. U.S. sales rose 19% sequentially to $38.2 million, while ex-U.S. revenue was impacted by ordering variability.

The company ended September 30, 2025, with cash, cash equivalents, and short-term investments totaling $416.1 million, providing a strong cash runway to support development programs.

When we alerted readers to RYTM on April 7, 2025, it was trading around $48.

RYTM has traded in the range of $45.90 to $116.00 over the past year. The stock closed yesterday at $104.77, up 4.03%, and rose further to $108.80 in after-hours trading, up 3.85%.