Setmelanotide Successfully Clears Phase 3 Of Clinical Trial

Release Date: 25-Sep-2019

Setmelanotide is a peptide drug, which is product of Rhythm pharmaceuticals. They developed this drug to reduce the weight gain and hunger in genetic obesity disorders. This drug has hit the goals of two late-stage clinical trials, and the company now plans to file for FDA approvals late this year or in early 2020.


The Rhythm pharmaceutical announced preliminary Phase 3 data for its drug, setmelanotide, in two rare, inherited obesity disorder pro-opiomelancortin (POMC) deficiency and leptin receptor (LEPR) deficiency. In both diseases, the Boston Company said its drug hit the main goals of weight reduction. Reduction in weight and hunger takes place was confirmed in this study.


This drug targets the melancortin 4 receptor, known as MC4. It is a protein, that plays an important role in the regulation of apetite and hunger. In the case of rare genetic obesity disorders such as POMC and LEPR, this protein is unable to work properly which results in the unstable hunger. As a result, overeating takes place, which develops into severe obesity.


Setmelanotide is an injectable peptide drug, which ismainly developed to boost the activity of MC4. The study of Rhythm involves the patient with more than the age of 6 years. The testing was performed in open-label and single-arm study model.the duration of study was about 1 year. The main goal of both one-year studies was achieving 10 percent weight loss compared to historical measures of this patient population. Secondary goals included reductions in body weight and hunger measured according to a hunger rating scale.


Side effects of this drug include some hypersensitive reactions at the injection site, as well as nausea and vomiting. The withdrawal symptoms involve an experience of mild hypereosinophilia. It is a blood disorder characterized by high numbers of white blood cells.


Rhythm is going to publish the data of Phase III of clinical trial in the end of this year. At that point, the company will be preparing to file a “rolling” submission to the FDA for both POMC and LEPR. This makes the process of approval faster and this drug is expected to get an approval in 2020.


According to the Keith Gottesdiener, CEO of Rhythm pharmaceuticals, even though some patients did not hit the weight-loss goal established for the studies, their reduction in hunger is meaningful. He also added that the company has spoken with regulators about the importance of that measure in the lives of patients.

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