Plans to Advance A0001 Into Phase IIa Trials in Patients in Second Half of 2009
DANBURY, Conn., July 16, 2009 (GLOBE NEWSWIRE) -- Penwest Pharmaceuticals Co. (Nasdaq:PPCO) today announced the results of its Phase Ib clinical trial of A0001, a compound the Company is developing for the treatment of mitochondrial diseases. Drs. Amale Hawi, Senior Vice President, Pharmaceutical Development, and Thomas Sciascia, Senior Vice President & Chief Medical Officer, will be presenting these results at the Friedreich's Ataxia Therapeutics Meeting on Friday, July 17, 2009, in Philadelphia, PA.
In the Phase Ib trial, the drug was well tolerated by subjects, and no serious adverse events were reported. There was a dose-dependent increase in exposure approaching steady state within 2-4 days following repeat-dosing, and a maximum tolerated dose was established.
Based on these results, Penwest plans to advance A0001 into Phase IIa studies in patients with mitochondrial diseases. The Company intends to commence two Phase IIa trials - one focused on patients with Friedreich's Ataxia and the second focused on patients with the A3243G mitochondrial DNA point mutation associated with the "MELAS" syndrome - in the fourth quarter of 2009. The Phase IIa trials will be conducted with twice daily oral dosing. The Company expects data from both of these trials in the first half of 2010.
Jennifer L. Good, Penwest's President and CEO, said, "We are very pleased to have completed the Phase I safety work on A0001 so that we can advance this compound into patients. We believe A0001 is promising for patients who suffer from mitochondrial diseases and currently have very limited treatment options, and we are eager to begin to evaluate the clinical benefit in patients."
The Phase Ib trial was a single-blind, placebo-controlled, multiple ascending dose clinical trial in approximately 40 healthy male and female subjects for up to 14 days. The trial was designed to evaluate the safety and tolerability of A0001 and characterize the pharmacokinetic profile following repeat dosing.
About A0001
A0001, or alpha-tocopherol quinone, is a coenzyme Q10 analog demonstrated to improve mitochondrial function in-vitro. Penwest believes that impairment of mitochondrial function is a key component of the diseases that it plans to target with A0001, and that enhancing mitochondrial function may provide substantial clinical benefit to patients. The Company is developing A0001 under a collaboration and licensing agreement with Edison Pharmaceuticals, a privately held biopharmaceutical company headquartered in San Jose, CA.
About Mitochondrial Diseases
Mitochondrial diseases are devastating, potentially life-threatening illnesses for which there are no FDA-approved treatments.
