Rhythm Pharma’s drug for rare obesity meets late-stage trial goal
Rhythm Pharmaceuticals (RYTM.O), opens new tab said on Monday its drug Imcivree notably reduced weight in patients with a rare obesity disorder caused by damage to the brain, meeting the main goal of a late-stage trial.
Shares of the company rose 2.5% to $48.10 in premarket trading despite U.S. index futures being sharply lower.
The 120-person trial tested patients with acquired hypothalamic obesity, a condition caused by damage to the hypothalamus in the brain, often due to tumors, their treatment or other injuries.
The therapy, chemically known as setmelanotide, reduced weight by 16.5% as measured on the body mass index (BMI) after a year, compared to a 3.3% increase for patients on placebo, the company said in a statement.
Imcivree is Rhythm’s only approved treatment, and an expanded approval for the rare disorder could unlock a $2 billion-plus opportunity, according to Jefferies analysts. The drug brought in sales of $41.8 million in the fourth quarter of 2024.
Imcivree is currently approved to treat genetic obesity in adults and children aged two years and older.
The weight loss seen in the trial is “very strong, looks highly consistent and clears investor expectations, setting up a blockbuster expansion opportunity,” said Stifel analyst Paul Matteis.
The efficacy of the drug is “outstanding in a highly difficult-to-treat patient population,” Matteis added.
Rhythm estimates there are 5,000 to 10,000 people living with hypothalamic obesity in the U.S.
The therapy also met secondary goals such as improvements in daily maximal hunger score, a measure of the intensity of hunger, for patients aged 12 years or older.
The company expects to complete regulatory applications in the United States and Europe in the third quarter of this year.
