Rhythm Pharmaceuticals Announces Licensing Agreement with Takeda for the Development and Commercialization of Preclinical Treatment for Prader-Willi Syndrome
-- Oral Ghrelin O-Acyltransferase (GOAT) Inhibitor Complements and Expands Efforts in Prader-Willi Syndrome --
-- In Preclinical Models, Prevented Body Weight Gain and Reduced Fat Mass --
-- Rhythm Expects to File Investigational New Drug Application in First Quarter of 2020 --
Ghrelin is an orexigenic peptide, secreted by the stomach and proximal small intestine in response to a negative energy balance. Ghrelin plays a key physiological role in stimulating appetite and promoting food intake, thereby maintaining overall energy balance. In people living with PWS, levels of active ghrelin are elevated, contributing to an unrelenting hunger, known as hyperphagia, which leads to severe obesity.
“The in-licensing of
In preclinical research,
Under the terms of the agreement, Rhythm will assume sole responsibility for the global product development and commercialization of
“Rhythm aims to develop meaningful drug candidates for people living with metabolic disorders,” said
Rhythm’s lead product candidate, setmelanotide, is an investigational, first-in-class melanocortin-4 receptor (MC4R) agonist. The Company is currently focusing setmelanotide clinical development on six rare genetic disorders of obesity: pro-opiomelanocortin (POMC) deficiency obesity, leptin receptor (LEPR) deficiency obesity, Bardet-Biedl syndrome (BBS), Alström syndrome, POMC heterozygous deficiency obesity, and POMC epigenetic disorders. The Company previously completed a Phase 2 trial of setmelanotide in PWS. In this trial, setmelanotide showed modest effects on reducing hyperphagia, but had no effect on weight across the study population, though there was some evidence of clinically-meaningful weight loss in the subset of patients treated at the highest dose level for the longest duration. Rhythm is currently assessing opportunities to further evaluate setmelanotide in PWS and plans to pursue these in parallel with the development of
About Prader-Willi Syndrome
Prader-Willi Syndrome (PWS) is the most common known genetic cause of life-threatening obesity in children. Prevalence estimates have ranges from one in 8,000 to one in 25,000, with at least 8,000 people in the U.S. diagnosed with PWS. A hallmark of PWS is severe hyperphagia, an overriding physiological drive to eat, leading to severe obesity and other complications. For PWS patients, hyperphagia and obesity are the greatest threats to their health. There is currently no approved treatment for the obesity and hyperphagia associated with PWS.
Rhythm is a biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare genetic disorders of obesity. Rhythm is currently evaluating the efficacy and safety of setmelanotide, the Company’s first-in-class melanocortin-4 receptor (MC4R) agonist, in Phase 3 studies in patients with pro-opiomelanocortin (POMC) deficiency obesity (which includes deficiencies in both the POMC and PCSK1 genes) and leptin receptor (LEPR) deficiency obesity. Rhythm also supports
This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties, including statements regarding the potential efficacy of
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