— FDA Grants Fast Track Status for Diabetic Gastroparesis —
BOSTON, Oct. 22, 2012 /PRNewswire/ -- Rhythm announced today the results of two Phase 1 clinical trials of RM-131, the company's novel ghrelin agonist for the treatment of diabetic gastroparesis, which demonstrated that RM-131 greatly accelerates gastric emptying in healthy subjects with good tolerability. The findings were presented at the American College of Gastroenterology 2012 Annual Scientific Meeting (ACG 2012) in Las Vegas on October 22, 2012. In addition, Rhythm announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track review status to RM-131 for the treatment of diabetic gastroparesis.
The presentation at ACG 2012 highlights RM-131's strong prokinetic activity in both single- and multiple-dose studies and its potential as a pan-gastrointestinal (GI) prokinetic for the treatment of upper and lower GI functional disorders, including diabetic gastroparesis. Clinical highlights include:
- RM-131 was shown to be effective in accelerating gastric emptying in healthy volunteers. After single daily subcutaneous administration of RM-131, gastric emptying time was significantly reduced by up to 55% in the single ascending dose study, and by up to 50% in the multiple ascending dose study.
- RM-131 was shown to be effective in accelerating lower colonic motility in healthy volunteers. After multiple daily subcutaneous doses of RM-131, colonic transit time was reduced by up to 54% (from ~42 hours in placebo subjects to ~18 hours).
- RM-131 was generally well tolerated, with no serious adverse events.
"These studies demonstrate substantial prokinetic effects of RM-131 on both gastric emptying and lower GI transit," said Lee M. Kaplan, MD, PhD, Associate Professor of Medicine at Harvard Medical School, Director of the Obesity, Metabolism, & Nutrition Institute at the Massachusetts General Hospital, and an author on the study. "Defects in gastric emptying underlie several functional GI disorders, including gastroparesis. Our treatment options for these common and debilitating diseases are currently very limited."
These clinical results are reported from two Phase 1 randomized, double-blind, placebo-controlled studies in healthy volunteers that evaluated the pharmacodynamic profile of RM-131: A single ascending dose (SAD) study that evaluated doses of 3 to 2,400 mcg administered once daily by subcutaneous injection, and a multiple ascending dose (MAD) study of doses from 10 to 300 mcg administered subcutaneously once or twice daily for 14 days. The two trials also assessed safety and tolerability.
Rhythm recently completed a Phase 1b clinical trial with RM-131 in patients with Type 2 diabetes that also demonstrated a potent prokinetic effect, reducing gastric emptying time by 66% after a single subcutaneous administration, with the greatest improvement observed in patients with the most abnormal gastric emptying. Together, these three Phase 1 studies provide the foundation for the company's ongoing Phase 2 clinical program.
"We are pleased with the positive Phase 1 studies for RM-131, and we also greatly appreciate receiving the Fast Track designation for diabetic gastroparesis," said Keith Gottesdiener, MD, CEO of Rhythm. "We look forward to continuing to work closely with the FDA as we advance the ongoing Phase 2 clinical trial of RM-131 for this disease."
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.
Gastroparesis affects a significant number of the 24 million diabetics in the U.S., with a prevalence of delayed gastric emptying of up to 30% among Type 1 and Type 2 diabetics. Gastroparesis symptoms include chronic nausea, vomiting, and malnutrition and results in a significant rate of hospitalization. This condition also often undermines measures to manage hyperglycemia.
About Rhythm (www.rhythmtx.com)
Rhythm is a biotechnology company developing peptide therapeutics that address unmet needs in metabolic diseases. Rhythm is developing the ghrelin peptide agonist, RM-131, for the treatment of diabetic gastroparesis and other GI functional disorders, and the MC4R peptide agonist, RM-493, for obesity and diabetes. Rhythm investors include MPM Capital, New Enterprise Associates, Third Rock Ventures, and Ipsen. The company is based in Boston, Massachusetts.