Rhythm Reports Positive Data from Long-term Extension Study of Obesity Therapy

Rare Daily Staff

Rhythm Pharmaceuticals announced several data presentations showing that setmelanotide therapy resulted in sustained and deepened weight loss in patients with severe obesity caused by rare MC4R pathway diseases during The Obesity Society’s Annual Meeting at ObesityWeek.

Highlights included results from Rhythm’s open-label, long-term extension studies evaluating setmelanotide in individuals with hypothalamic obesity, Bardet-Biedl syndrome (BBS), or obesity due to POMC or LEPR deficiency, as of a data cutoff date of June 13, 2023, which showed 25.5 percent reduction in mean body mass index (BMI) from baseline in patients with hypothalamic obesity (n=12) at one year; 16.0 percent reduction in mean BMI in adult patients with Bardet-Biedl syndrome (BBS) at three years; 0.7 reduction in mean BMI Z score in pediatric patients younger than 18 with BBS at three years; 20.3 percent reduction in mean BMI in adult patients with obesity due to POMC or LEPR deficiency at four years; and 1.2 reduction in mean BMI Z score in pediatric patients younger than 18 due to POMC or LEPR deficiency at four years.

“We believe the one-year BMI reduction of more than 25 percent in patients with hypothalamic obesity – where individuals trended towards normal body weight – is particularly encouraging as it shows continued improvement over our previously reported 16-week and six-month data,” said David Meeker, chair, president, and CEO of Rhythm. “The hypothalamic obesity data affirm our confidence as we advance our pivotal, phase 3 trial and work to bring setmelanotide to patients and families facing this challenging disease for which there are no approved, effective therapies.”

In the United States, setmelanotide is indicated for chronic weight management in adult and pediatric patients 6 years of age and older with monogenic or syndromic obesity due to POMC, PCSK1 or LEPR deficiency as determined by an FDA-approved test demonstrating variants in POMC, PCSK1 or LEPR genes that are interpreted as pathogenic, likely pathogenic, or of uncertain significance and BBS.

In the European Union, setmelanotide is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed Bardet-Biedl syndrome (BBS) or genetically confirmed loss-of-function biallelic POMC, including PCSK1, deficiency or biallelic LEPR deficiency in adults and children 6 years of age and above.

In Canada, setmelanotide is indicated for the treatment of obesity due to Bardet-Biedl syndrome (BBS) or genetically-confirmed biallelic POMC, PCSK1, or LEPR deficiency due to variants interpreted as pathogenic, likely pathogenic, or of uncertain significance in adults and children 6 years of age and above.

Photo: David Meeker, chair, president, and CEO of Rhythm