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news.Penwest Pharmaceuticals (Penwest) has initiated A0001 Phase IIa clinical trial in patients with the A3243G mitochondrial DNA point mutation and evidence of impaired mitochondrial function. The point mutation is commonly associated with Melas syndrome.
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A0001 or alpha-tocopherolquinone, is a coenzyme Q analog demonstrated to improve mitochondrial function in-vitro. The study is being conducted at the Newcastle upon Tyne Hospitals in Newcastle, England.
The primary objective of the study is to investigate whether treatment with A0001 has a discernible impact in the treatment of these patients using metabolic imaging, a number of functional assessments, biochemical measures and patient/clinician-rated scales as compared to placebo.
Reportedly, the Phase IIa clinical trial is a double-blind, randomised, placebo-controlled trial. Penwest plans to enroll approximately 30 patients who meet the eligibility criteria with a 2:1 randomisation of drug to placebo. The patients will be dosed for 28 days. The company expects data from this trial in the third quarter of this year.
Jennifer Good, president and CEO of Penwest, said: “We are very pleased to commence the second proof of concept trial for A0001 in patients. We look forward to evaluating the clinical data from the two on-going Phase IIa trials to determine if there are meaningful signs of efficacy from the drug. There is an unmet medical need in these mitochondrial diseases, including melas, and we are hopeful that A0001 can provide an important treatment option for these patients.”