The Michael J. Fox Foundation for Parkinson’s Research (MJFF) gave Rhythm a $1.35 million research grant to help fund a Phase II trial of RM-131, the company’s ghrelin agonist, for the treatment of refractory constipation in patients with Parkinson’s disease. The study expands an ongoing Phase II program for RM-131 for the treatment of both upper and lower gastrointestinal (GI) functional disorders which Rhythm initiated earlier this year.
The MJFF-funded trial will assess the efficacy and safety of RM-131 administered daily for two weeks. Approximately 56 patients are expected to be enrolled. Rhythm is conducting the study in partnership with the Parkinson’s Study Group and Ronald Pfeiffer, M.D., University of Tennessee.
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, according to the company, which is also evaluating RM-131 for the treatment of diabetic gastroparesis in type 1 diabetic patients. In May, Rhythm reported that RM-131 significantly accelerates early gastric emptying and reduces upper GI symptoms in type 1 diabetic patients with diabetic gastroparesis.
In 2010, Rhythm obtained an exclusive license to Ipsen’s ghrelin programs. As part of that agreement, Ipsen could receive payments of up to U.S. $80 million upon the achievement of certain milestones as well as royalties on future sales of the products. In addition, Ipsen acquired 17% equity in Rhythm.