Rhythm Pharmaceuticals published data Monday showing Imcivree (setmelanotide) improved quality of life for patients with obesity related to Bardet-Biedl Syndrome.
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Monday, Rhythm Pharmaceuticals announced the publication of Phase III data analysis that showed Imcivree (setmelanotide) improved quality of life for patients with obesity related to Bardet-Biedl Syndrome.
The Boston-based biopharma shared the analysis in the Orphanet Journal of Rare Diseases.
BBS is a rare genetic disease characterized by symptoms including early-onset obesity, visual impairments, renal disease and hyperphagia - an extreme, unsatisfied hunger. This insatiable hunger is particularly disruptive to the quality of life of patients and their families.
Imcivree re-establishes the melanocortin-4 receptor (MC4R) pathway, which plays a vital role in regulating the appetite. Trial results showed the drug meaningfully reduced BMI, improved body weight measures, lowered total cholesterol and reduced obsessive focus on food.
In the newly published 52-week analysis, nine patients under 18 showed an 11.2-point jump on the Pediatric Quality of Life Inventory measure. The 11 adult patients achieved an increase of 12 points from baseline on the Impact of Weight on Quality-of-Life Questionnaire. In adults, the score increases significantly correlated with changes in body weight and BMI.
David Connolly, head of investor relations and corporate communications at Rhythm told BioSpace the trial was the largest ever conducted in BBS.
These results were also presented at the Pediatric Endocrine Society (PES) 2022 Virtual Annual Meeting in May 2022.
Tim Ogden, president of the BBS Foundation, told BioSpace the “very positive clinical and quality of life data on Imcivree/setmelanotide is a huge encouragement to the community.”
“We consistently hear from people with BBS who experience hyperphagia that managing it is even more difficult than managing vision loss,” he said.
In a prepared statement, David Meeker, president and CEO of Rhythm, said the company is “focused on…delivering a precision medicine that can restore normal pathway signaling.”
Stamped by the FDA in June 2022 for weight management in patients with BBS 6 years and older, Imcivree is the first drug specifically approved for chronic weight management in this population.
Rhythm is studying setmelanotide in additional indications. Topline data from a Phase II trial in hypothalamic obesity showed 14 of the 18 patients lost more than 10% of their BMI in 16 weeks. The company expects to initiate a Phase III trial in 120 hypothalamic obesity patients for whom “nothing exists and nothing works,” as nothing else targets the MC4R pathway, Connolly said.
“Those patients are waiting for a therapy,” he said. “This is really a big game changer for us.”
