Rhythm, a biopharmaceutical company, and Actavis, a global pharmaceutical company, have initiated a phase IIb clinical trial assessing the efficacy and safety of relamorelin (RM-131), Rhythm's ghrelin agonist, for the treatment of gastroparesis in patients with type 1 and type 2 diabetes. Ghrelin is a peptide hormone produced in the stomach that stimulates gastrointestinal (GI) motility. Actavis has an exclusive option to acquire Rhythm's wholly owned subsidiary, Rhythm Pharmaceuticals, upon the completion of the phase IIb study.

"This is the second of two studies assessing the efficacy and safety of relamorelin for the treatment of diabetic gastroparesis," said Fred Fiedorek, M.D., chief medical officer of Rhythm. "This phase IIb study is designed to expand our understanding of RM-131's profile for treating diabetic patients with both moderate and severe gastroparesis, prior to the initiation of phase III registration studies."

The phase IIb trial is designed to evaluate the effect of relamorelin on signs and symptoms of gastroparesis, GI motility and safety in patients with diabetic gastroparesis. The randomized, double-blind, placebo-controlled study will evaluate the safety and efficacy of dosing regimens ranging from 10mcg to 100mcg administered twice daily over three months. The trial is expected to enroll approximately 400 patients with diabetic gastroparesis at clinical sites in the U.S. and Europe.

Rhythm previously completed two successful phase II clinical trials with relamorelin:

• In a phase II trial for diabetic gastroparesis, patients treated with relamorelin 10mcg twice daily demonstrated statistically significant improvements in gastric emptying and vomiting symptoms, and in a pre-specified subgroup also showed statistically significant improvements in the other symptoms of gastroparesis.
• In a separate phase IIa study for chronic constipation, patients treated with relamorelin 100 mcg once daily for two weeks demonstrated statistically significant improvements in spontaneous bowel movements and in lower GI transit. Relamorelin demonstrated a potent prokinetic effect on GI transit in both studies, as well as in phase I studies, and generally was well-tolerated.

"The need for better treatments for diabetic gastroparesis is pressing," said Keith Gottesdiener, M.D., CEO of Rhythm. "This is a debilitating condition that affects several million people in the U.S., with only one drug approved by the FDA in the past 30 years. Thus far, relamorelin has been shown to directly improve impaired gastric function in our clinical trials, and we are excited about its potential to significantly improve the health of people suffering from this disorder."

"The clinical results with relamorelin in the initial phase II trial in diabetic gastroparesis were precedent-setting," said David Nicholson, executive vice president of Global Brands R&D for Actavis. "Both Rhythm and Actavis are looking forward to the results from this important trial, and to the positive impact this drug may have on the lives of people with GI functional disorders."

Relamorelin is a phase II ghrelin agonist in development for the treatment of diabetic gastroparesis and GI functional disorders. Derived from the natural ghrelin sequence, relamorelin has been optimized to stimulate GI motility, with greater potency and enhanced stability and pharmacokinetics. Relamorelin has completed a phase II trial in diabetic gastroparesis and a phase IIa study in chronic constipation, and additional phase II trials are under way in lower GI functional disorders and diabetic gastroparesis. The FDA has granted Fast Track review status to relamorelin for the treatment of diabetic gastroparesis.