Rhythm Pharmaceuticals, Inc. operates as a biopharmaceutical company. The company develops peptide therapeutics for the treatment of gastrointestinal (GI) diseases; and obesity, including obesity caused by genetic deficiencies in the MC4 pathway. Its portfolio includes relamorelin (RM-131), a ghrelin peptide agonist for the treatment of diabetic gastroparesis, a GI complication of diabetes and other GI functional disorders; and RM-493, a melanocortin-4 (MC4) receptor agonist for the treatment of obesity and obesity caused by genetic deficiencies in the MC4 pathway. Rhythm Pharmaceuticals, Inc. was founded in 2008 and is based in Boston, Massachusetts.
855 Boylston Street
Boston, MA 02116
Founded in 2008
Rhythm Pharmaceuticals, Inc. Presents Positive Data from Phase 1B Study of Setmelanotide for the Treatment of Genetic Obesity
Nov 6 15
Rhythm Pharmaceuticals, Inc. announced the results from a proof-of-concept, Phase 1b clinical trial assessing the safety and efficacy of setmelanotide (RM-493), the company's novel melanocortin-4 receptor (MC4R) agonist, in obese patients with a heterozygous genetic defect in MC4R. The trial demonstrated that after four weeks of treatment, setmelanotide reduced weight in patients with an MC4R heterozygous deficiency obesity, with good tolerability. The study results were featured in an oral presentation at the ObesityWeek 2015 conference in Los Angeles. Setmelanotide activates MC4R, which is part of the key pathway that can independently regulate energy homeostasis and appetite. The critical role of the MC4 pathway in weight regulation was validated with the discovery that single genetic defects along this pathway result in early onset and severe obesity. An expanding set of severe obesity genetic defects are now identified that involve genes in the pathway that are either upstream of MC4R—specifically, pro-opiomelanocortin (POMC) deficiency obesity, leptin receptor deficiency obesity, and likely Prader-Willi Syndrome (PWS)—or genes that are downstream of MC4R or that affect MC4R itself. In this pilot study, obese (BMI >/= 30kg/m2) patients with a heterozygous MC4R loss-of-function mutation were enrolled in a double-blind, placebo-controlled, randomized, parallel-group study for 4 weeks. Eight patients (six active, two placebo) received placebo or RM-493 at 0.01 mg/kg/day by continuous subcutaneous infusion. Key endpoints were safety, weight loss, waist circumference, and caloric intake. Setmelanotide was well tolerated over 4 weeks, with no serious adverse events or discontinuations. The most common side effects were headache and skin tanning, with the latter believed to be due to off-target activity at the related melanocortin-1 receptor. Setmelanotide demonstrated strong trends for placebo-subtracted weight loss (-2.62 kg; p=0.088); WC (-5.1 cm; p=0.188) and daily caloric intake (-351 kCal/day; p=not significant), without clinically important effects on heart rate or blood pressure.
Rhythm Pharmaceuticals Inc. Presents at BIO International Convention, Jun-25-2014 10:30 AM
Jun 17 14
Rhythm Pharmaceuticals Inc. Presents at BIO International Convention, Jun-25-2014 10:30 AM. Venue: San Diego Convention Center, San Diego, California, United States.