Penwest Provides Results Of Phase Ib Trial Of A0001

Penwest has provided the results of its phase Ib clinical trial of A0001, for the treatment of mitochondrial diseases.

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.

Based on these results, Penwest plans to advance A0001 into phase IIa studies in patients with mitochondrial diseases. The company plans 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.

Moreover, 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 Good, president and CEO of Penwest, 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.