July 11, 2014 12:22 PM ET


Company Overview of Rhythm Pharmaceuticals Inc.

Company Overview

Rhythm Pharmaceuticals Inc. develops peptide therapeutics for metabolic diseases. Its products include RM-131, a ghrelin peptide agonist for the treatment of gastrointestinal functional disorders; and RM-493, a melanocortin-4 receptor peptide therapeutic for the treatment of obesity and diabetes. The company was incorporated in 2008 and is based in Boston, Massachusetts.

29 Newbury Street

3rd Floor

Boston, MA 02116

United States

Founded in 2008





Key Executives for Rhythm Pharmaceuticals Inc.

Chief Executive Officer
Founder and Chief Development Officer
Age: 64
Chief Scientific Officer
Age: 59
Compensation as of Fiscal Year 2014.

Rhythm Pharmaceuticals Inc. Key Developments

Rhythm Pharmaceuticals Inc. Presents at BIO International Convention, Jun-25-2014 10:30 AM

Rhythm Pharmaceuticals Inc. Presents at BIO International Convention, Jun-25-2014 10:30 AM. Venue: San Diego Convention Center, San Diego, California, United States.

Rhythm Pharmaceuticals Inc. Initiates Clinical Trial of RM-493 for Obesity Caused by Genetic Variant

Rhythm Pharmaceuticals Inc. announced the initiation of the first of a series of clinical trials with RM-493, the company's novel melanocortin 4 receptor (MC4R) agonist, for the treatment of obesity in individuals with a genetic deficiency in the MC4R pathway. This RM-493 clinical trial is a new approach that targets the root cause of obesity in the MC4 heterozygote population. MC4R deficiency due to mutations in the MC4R gene has been identified as the most common monogenic cause of obesity. Up to 6% of people with a BMI >35 have MC4R deficiency, and this group has a higher predisposition than the general population for severe obesity and its complications, such as diabetes. MC4R deficiency also correlates with early-onset obesity, which lengthens exposure to the risk of obesity-associated diseases. Furthermore, MC4R deficiency may also undermine the beneficial effects of diet and exercise for weight loss, limiting treatment options for these individuals. This first MC4R deficiency trial is a Phase 1B study designed to evaluate the effect of RM-493 on weight loss and safety in obese patients with a loss-of-function variant of the MC4R gene. Patients will be treated for up to four weeks with RM-493. This study expands on Rhythm's ongoing Phase 2 clinical program for RM-493 for the treatment of obesity. RM-493 has demonstrated impressive efficacy on weight loss in both preclinical studies and early clinical trials in subjects with normal, fully functioning MC4R genes. This new clinical study a personalized medicine approach to the treatment of obesity in people with a poorly functioning MC4R gene and has the potential to restore function in this pathway and improve weight regulation. The company is excited about the potential to address this significant unmet need in metabolic disease. RM-493 is a small-peptide melanocortin 4 receptor (MC4R) agonist that is in clinical development for the treatment of obesity and diabetes. The MC4 receptor mediates a key pathway in humans that regulates energy homeostasis and food intake. The MC4R pathway is well validated in humans; loss-of-function mutations of MC4R are associated with obesity and have a reported prevalence of 4%-6% in severe obesity. In the MC4R heterozygote population, RM-493 may restore MC4R function by increasing activity in the one healthy copy of MC4R.

Rhythm Initiates Phase 2 Clinical Trial of Ghrelin Prokinetic RM-131 for Lower GI Functional Disorders

Rhythm announced the initiation of an additional Phase 2 clinical trial for RM-131, the company's novel ghrelin agonist, for the treatment of chronic constipation. Ghrelin is a peptide hormone produced in the stomach that stimulates gastrointestinal motility. This is the first of two studies planed to initiate this year to assess the efficacy and safety of RM-131 for the treatment of lower GI functional disorders. This Phase 2 trial is designed to evaluate the effects of RM-131 on symptoms and lower gastrointestinal (GI) function in patients with significant chronic constipation. The trial will assess both the efficacy and safety of RM-131 administered daily for two weeks and is expected to enroll approximately 48 patients. Rhythm has completed three Phase 1 clinical trials with RM-131 in both healthy volunteers and patients with Type 1 and Type 2 diabetes, and a Phase 2 clinical trial of RM-131 is currently under way for the treatment of diabetic gastroparesis. The Phase 1 human clinical trials demonstrated potent effects of RM-131 on both upper and lower GI function, increasing both gastric emptying and lower GI transit with good tolerability. In one study conducted in healthy volunteers, multiple daily subcutaneous doses of RM-131 reduced colonic transit time by up to 54%-- from approximately 42 hours in placebo subjects to approximately 18 hours.RM-131 is a small-peptide analog of ghrelin, a hormone produced in the stomach that stimulates gastrointestinal activity. Derived from the natural ghrelin sequence, RM-131 has been optimized to stimulate gastrointestinal motility, with greater potency and enhanced stability and pharmacokinetics. The U.S. Food and Drug Administration (FDA) has granted Fast Track review status to RM-131 for the treatment of diabetic gastroparesis. About Lower GI Functional Disorders: 15 million people in the U.S. seek medial care annually for chronic constipation and irritable bowel syndrome (IBS), and approximately 15% are refractory to existing therapies. These conditions are two of the most common GI motility disorders, with significant health consequences. There is a compelling need for new treatments for lower GI functional disorders, but there are no safe and effective prokinetic drugs currently approved for these conditions in the U.S.

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