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Rhythm drug for genetic obesity disorders shows weight loss in midstage trials

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Rhythm drug for genetic obesity disorders shows weight loss in midstage trials

Rhythm Pharmaceuticals Inc. said its investigational drug setmelanotide helped patients with certain rare genetic disorders of obesity lose weight in midstage trials.

Setmelanotide works by activating the melanocortin 4 receptor, or MC4R, a protein that plays a role in regulating energy expenditure and appetite.

The phase 2 basket studies evaluated setmelanotide in patients with rare genetic obesity disorders such as Bardet-Biedl syndrome, or BBS; Alström syndrome; pro-opiomelanocortin, or POMC, and other MC4 heterozygous deficiency obesities; and proopiomelanocortin epigenetic disorders.

For the five patients with BBS, the drug helped them achieve an average of 18.5% weight loss and cut their average hunger score by 74.2% after 40 to 60 weeks of treatment. The company noted that one pediatric patient with type 1 diabetes had no body weight change but had an average hunger score decrease of 53.3% and improved average blood sugar level to 7.6% from 10.1%.

For the Alström syndrome patient group, one patient achieved 23.2% weight loss and had 45.5% reduction in hunger score after 38 weeks of treatment.

In the POMC and other MC4 heterozygous deficiency obesity group, two patients lost weight by 15.4% and 6.5%, respectively. The first patient's hunger score decreased by 80%, while the second patient was at 20%.

Furthermore, the patient with POMC epigenetic disorder showed a 60% decrease in hunger score in the titration phase and withdrew from the treatment after a four-week vacation. The patient has resumed treatment and lost weight by a total of 9.2% over the trial.

The Boston-based biopharmaceutical intends to hold discussions with the U.S. Food and Drug Administration regarding the final design, protocol and goals of a combined phase 3 study for setmelanotide in patients with BBS and Alström syndrome, which it plans to conduct by the end of 2018.