FDA Office of Orphan Products Development Awards Rhythm $1 Million Grant to Support Phase 2 Study of Setmelanotide in Prader-Willi Syndrome

BOSTON, Sept. 24, 2015 /PRNewswire/ -- Rhythm announced today the award of a $1 million research grant from the FDA Office of Orphan Products Development* to its subsidiary, Rhythm Metabolic, Inc., to support the ongoing Phase 2 clinical trial of setmelanotide, the company's novel peptide MC4 agonist, for the treatment of obesity and hyperphagia in Prader-Willi syndrome (PWS).

The ongoing Phase 2 clinical trial is designed to evaluate the effects of setmelanotide on weight reduction and PWS-specific food-related behavior in obese patients with PWS. This randomized, double-blind, placebo-controlled study is evaluating the safety and efficacy of setmelanotide administered once daily by subcutaneous injection for up to 10 weeks of treatment. The trial will enroll approximately 36 obese patients with PWS.

"We are extremely grateful for this grant from the FDA Office of Orphan Products Development and the important support of the Foundation for Prader-Willi Research in making it happen," said Keith Gottesdiener, MD, CEO of Rhythm. "This grant is meaningful and substantive to advance our ongoing setmelanotide program for the treatment of Prader-Willi."

"The Foundation for Prader-Willi Research is thrilled to have contributed to this amazing effort, bringing the patient voice to the proposal," stated Theresa Strong, Director of Research Programs for FPWR. "We are grateful for the work of Dr. Keith Gottesdiener, CEO of Rhythm and his team, as well as the investigators at the clinical trial sites -- Dr. Jennifer Miller and Elizabeth Roof -- and we look forward to seeing this drug evaluated in Prader-Willi syndrome."

About Setmelanotide (RM-493)

Setmelanotide is a potent, first-in-class MC4 agonist in development for the treatment of obesity caused by genetic deficiencies in the MC4 pathway, a key pathway in humans that regulates energy expenditure, homeostasis, and appetite. MC4's critical role in weight regulation was validated with the discovery that a mutation of the MC4 receptor gene results in early-onset and severe obesity, as do other genetic defects in the MC4 pathway. A Phase 2 trial is ongoing for the treatment of Prader-Willi syndrome (PWS), a rare genetic disorder that causes life-threatening obesity. Recent scientific evidence implicates defects in the MC4 pathway as the root cause of the weight and appetite abnormalities in PWS. A second Phase 2 trial is ongoing for the treatment of POMC deficiency obesity, a very rare, life-threatening genetic disorder. As in Prader-Willi syndrome, the obesity and appetite abnormalities in POMC deficiency patients is caused by a genetic defect in the MC4 pathway.

About Prader-Willi Syndrome

Prader-Willi Syndrome (PWS) is a rare genetic disease with a prevalence ranging from approximately one in 8,000 to one in 25,000 patients in the U.S. A hallmark of PWS is severe hyperphagia -- an overriding physiological drive to eat -- leading to severe obesity and other complications. Obesity is one of the greatest health threats to PWS patients, and hyperphagia impairs the ability of PWS patients to live independently, requiring costly and constant supervision to prevent overeating. Without supervision, these patients are likely to die prematurely as a result of choking, stomach rupture, or from complications caused by morbid obesity. Currently, there are no approved treatments for the obesity and hyperphagia associated with PWS.

About Rhythm (www.rhythmtx.com)

Rhythm is a biopharmaceutical company developing peptide therapeutics that address unmet needs in gastrointestinal diseases and obesity. Rhythm's subsidiary, Rhythm Pharmaceuticals, Inc., is developing the ghrelin peptide agonist, relamorelin (RM-131), for the treatment of diabetic gastroparesis and other gastrointestinal functional disorders. Rhythm's subsidiary, Rhythm Metabolic, Inc., is developing the MC4R peptide agonist, setmelanotide (RM-493), for obesity caused by genetic deficiencies in the MC4 pathway. The company is based in Boston, Massachusetts.

* This grant partially funds the initial proof-of-concept Phase 2 study in PWS; the remainder is funded by Rhythm. 

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Bart Henderson 
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