Rare Genetic Diseases Of Obesity-Focused Rhythm Pharma's Bardet-Biedl Syndrome Drug Meets Primary Goal In Pediatric Trial

During the R&D event for investors, Rhythm Pharmaceuticals Inc RYTM released new topline data from its Phase 3 pediatrics trial evaluating one year of setmelanotide in patients aged two and below six years with Bardet-Biedl syndrome (BBS) or proopiomelanocortin (POMC) or leptin receptor (LEPR) deficiency obesity.

Data highlights:

• 83.3% of all patients (10 of 12) achieved ≥ 0.2 reduction in BMI-Z score from baseline to week 52;
• 18.4% mean reduction from baseline in BMI at week 52 (N=12);
• 3.04 mean reduction from baseline in BMI-Z score at week 52 (N=12).

Rhythm also submitted a Type II variation application to the European Medicines Agency seeking regulatory approval and authorization for setmelanotide to treat obesity and control of hunger in pediatric patients.

The company anticipates submitting a supplementary New Drug Application to the FDA in 1H of 2024, seeking a similar label expansion.

Rhythm presented data from several pre-clinical studies showing that RM-718, a new, weekly, MC4R-specific agonist, demonstrated the potential to reduce body weight and hunger with a favorable safety profile. The company anticipates beginning Phase 1 in-human trials in 1H of 2024.

Price Action: RYTM shares are up 4.65% at $38.52 on the last check Wednesday.