Penwest Initiates Dosing in Phase Ia Mitochondrial Disease Trial
Penwest Pharmaceuticals has initiated dosing in a Phase Ia clinical trial of A0001, a compound the company is developing for the treatment of symptoms associated with mitochondrial diseases.
The Phase Ia trial is a placebo-controlled single ascending dose clinical trial. The trial is designed to evaluate the safety and tolerability of A0001 at various doses and to collect pharmacokinetic data.
The trial is also designed to determine if there is a maximum tolerated dose of the drug. The company plans to enroll a total of 60 healthy volunteers in the trial.
Penwest anticipates that further development of A0001 would involve a Phase Ib multiple ascending dose trial in healthy volunteers for which dosing could begin in early 2009, followed by a Phase IIa proof of concept trial in patients with mitochondrial diseases.
Jennifer Good, president and CEO of Penwest, said: “This compound is important to patients who suffer from mitochondrial diseases and currently have very limited treatment options. We are eager to complete the safety work for this compound so that we can take the important next step of beginning to evaluate clinical benefit in patients.”