New data from long-term extension trial evaluating setmelanotide in Bardet-Biedl Syndrome is presented at at ENDO 2022.-Rhythm Pharmaceuticals, Inc.

Rhythm and its collaborators delivered these data in poster presentations at the Endocrine Society Annual Meeting & Expo (ENDO), being held June 11-14, 2022 in Atlanta.

Bardet-Biedl Syndrome; A total of 42 patients with BBS who were treated with setmelanotide in Rhythm’s Phase II or Phase III trial continued into the LTE. As of the data cutoff, 30 and 19 of these patients had received at least 18 and 24 months of setmelanotide therapy, respectively. The data were presented in a poster, “Long-term Efficacy of Setmelanotide in Patients with Bardet-Biedl Syndrome,” by Jesús Argente, M.D., Ph.D., Universidad Autónoma de Madrid in Spain.

Highlights include: i. Mean (SD) percent change in BMI across all patients was -9.5% (10.5%; n=30) and -14.3% (11.6%; n=19) at 18 and 24 months, respectively; ii. Patients 18 years old or older achieved a percent change in weight of -8.6% (10.3%; n=15) and -14.9% (10.4%; n=6) at 18 and 24 months of treatment, respectively; iii. Six of 15 patients (40%) and five of six patients (83.3%) achieved greater than 10% weight reduction at 18 and 24 months of treatment, respectively; iv. Patients younger than 18 years old achieved a mean (SD) change in BMI Z score of -0.83 (0.50; n=13) and -0.72 (0.54; n=12) at 18 and 24 months of treatment, respectively; v. All patients younger than 18 years of age had a BMI Z score reduction of greater than 0.2 points. Twelve of 13 patients (92.3%) and 9 of 12 patients (75%) younger than 18 years of age achieved BMI Z score reductions of -0.3 points at 18 and 24 months of treatment, respectively; and vi. As of the data cutoff, a total of 38 patients were receiving ongoing treatment in the LTE trial; four patients discontinued treatment, one due to an adverse event that was not related to treatment.