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Rhythm Pharmaceuticals, Inc. (NASDAQ:RYTM) on Monday released topline results from the pivotal Phase 3 TRANSCEND trial evaluating setmelanotide, a melanocortin-4 receptor (MC4R) agonist, for the treatment of acquired hypothalamic obesity.

Acquired hypothalamic obesity is a rare, non-genetic form of obesity resulting from damage to the hypothalamus, the brain region controlling energy balance, often caused by tumors, surgery, or radiation, leading to rapid weight gain and metabolic complications.

The global trial met its primary endpoint with a statistically significant and clinically meaningful reduction in body mass index (BMI) with setmelanotide in adult and pediatric patients versus placebo.

Also Read: Rhythm Pharmaceuticals’ Obesity Treatments Target Less Crowded Space, Analyst Sees Long-Term Potential

Highlights from the topline data include:

• -19.8% placebo-adjusted difference in BMI reduction (N=120).

• The primary endpoint of mean BMI reduction was -16.5% from baseline for all patients on setmelanotide therapy (n=81) compared with +3.3% BMI change for patients on placebo (n=39) at 52 weeks.

• 80% of patients on setmelanotide achieved a BMI reduction of 5% or greater at 52 weeks.

• -19.2% placebo-adjusted BMI reduction was achieved in adult patients (n=49) and -20.2% in patients younger than 18 (n=71) at 52 weeks.

The company says the global Phase 3 TRANSCEND trial is believed to be the largest and longest placebo-controlled trial to evaluate therapy for patients with acquired hypothalamic obesity. The double-blinded, 52-week trial enrolled 120 patients.

Clinically meaningful improvements were observed across key secondary endpoints at week 52, including:

• 83% of patients on setmelanotide therapy achieved 5% or greater reduction in BMI (n=33 patients 18 or older) or BMI Z-score reduction of 0.2 or greater points (n=48 patients younger than 18).

• -1.4 placebo-adjusted mean change in the weekly average of the daily maximal hunger score for patients 12 years old or older (n=81) (p=0.003).

No new safety signals with setmelanotide were observed, in line with setmelanotide’s well-established and well-understood safety profile. Consistent with prior clinical experience, setmelanotide was generally well tolerated in the TRANSCEND study.

The most common treatment-emergent adverse events (affecting >20% of participants) were nausea, vomiting, diarrhea, injection site reaction, skin hyperpigmentation, and headache. No serious adverse events leading to study discontinuation were reported.

Price Action: RYTM stock is up 15.60% at $54.27 at the last check on Monday.