Rhythm Pharma Strikes Chord As Early PWS Trial Results Show Promise

Rhythm Pharmaceuticals Inc. (NASDAQ:RYTM) stock is higher on Thursday, after the company shared preliminary results from its exploratory Phase 2 trial of setmelanotide in patients with Prader-Willi syndrome (PWS).

PWS is a rare genetic disorder characterized by muscle weakness and feeding difficulties in infancy. An insatiable hunger and tendency toward severe obesity starting in childhood could follow.

PWS also involves delayed development, intellectual disability, behavioral issues, and physical traits, such as short stature.

Rhythm enrolled 18 patients. The 52-week trial remains ongoing.

Setmelanotide therapy demonstrated potential therapeutic benefit with BMI and hyperphagia (extreme hunger) reductions in patients with PWS at Month 3 (n=8) and Month 6 (n=5).

Also Read: Acadia Pharma Faces Trial Setback As Prader-Willi Syndrome Trial Falls Short

Highlights from preliminary results, as of a cut-off date of Nov. 14, include:

• 6 of 8 patients who reached Month 3 of setmelanotide therapy achieved BMI reductions from baseline.
• 3 of 5 patients who reached Month 6 of setmelanotide therapy achieved reductions in BMI, with two seeing deeper reductions versus Month 3 and one unchanged.
• 6 of 7 evaluable patients who reached Month 3 of setmelanotide therapy achieved a meaningful reduction in the Hyperphagia Questionnaire.
• 17 of the 18 patients enrolled remain on active setmelanotide therapy, and Safety and tolerability results have been consistent with setmelanotide’s well-established clinical profile.

The company also announced plans to advance setmelanotide into a Phase 3 registrational trial in PWS. But that depends on the successful completion of this Phase 2 trial.

Rhythm also announced that it has initiated a Part D arm in the Phase 1 trial of MC4R agonist RM-718. It will enroll up to 20 patients with PWS. Rhythm anticipates screening the first patient for this 26-week open-label trial of RM-718 in December 2025.

In March, the U.S. Food and Drug Administration (FDA) approved Soleno Therapeutics Inc.’s (NASDAQ:SLNO) Vykat XR (diazoxide choline) extended-release tablets for hyperphagia in adults and children with PWS.

Price Action: RYTM stock is up 14.24% to $119.69 as of Thursday’s close.

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