The primary objective of the study is to investigate whether treatment with A0001 has a discernible impact on various functional, biochemical and subject/clinician-rated scales relevant in the treatment of FA.
Reportedly, the Phase IIa clinical trial is a double-blind, randomised, placebo-controlled trial that includes a high and low dose of A0001 and a placebo. Penwest plans to enroll approximately 42 patients with a 2:1 randomisation of drug to placebo. The patients will be dosed for 28 days. The company expects data from the trial in the third quarter of this year.
The company said that Friedreich’s Ataxia Research Alliance (FARA) is helping to enroll patients for the study by utilising its patient registry.
Ronald Bartek, president and co-founder at FARA, said: “The Friedreich’s ataxia community is excited that A0001 is ready to be dosed in our patients. We believe the science behind the molecule is very interesting and that A0001 could be an important therapy for FA patients. FARA has been helping support the development of A0001 for a number of years and is continuing to assist its development partners at Penwest by working with the patient community to ensure the trial enrolls rapidly.”
Jennifer Good, president and CEO of Penwest, said: “We are very pleased to be advancing A0001 into a proof of concept trial in patients. There is an unmet medical need in Friedreich’s ataxia and we are hopeful that A0001 can provide an important treatment option for these patients.”