Penwest Pharmaceuticals Co. recently announced that it initiated a Phase IIa clinical trial for A0001 in December and that the screening of patients is underway. The study is being conducted at The Children's Hospital of Philadelphia in patients with Friedreich's Ataxia (FA).

The primary objective of this 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. The Phase IIa clinical trial is a double-blind, randomized, 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 randomization 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. The Friedreich's Ataxia Research Alliance (FARA) is helping to enroll patients for this study by utilizing its patient registry.

“The Friedreich's ataxia community is excited that A0001 is ready to be dosed in our patients,” said Ronald J. Bartek, FARA's President and Co-Founder. “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.”

“We are very pleased to be advancing A0001 into a proof-of-concept trial in patients,” added Jennifer L. Good, Penwest's President and CEO. “There is a significant unmet medical need in Friedreich's Ataxia, and we are hopeful that A0001 can provide an important treatment option for these patients."

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 exclusively licensed A0001 from Edison Pharmaceuticals, a privately held biopharmaceutical company headquartered in San Jose, CA.

Friedreich's Ataxia is a debilitating, life-shortening, degenerative neuromuscular disorder. About one in 50,000 people in the US have FA. Onset of symptoms can vary from childhood to adulthood. FA patients have gene mutations that limit the production of a protein called frataxin. Frataxin is known to be an important protein that functions in the mitochondria of the cell. Frataxin helps to move iron and is involved with the formation of iron-sulfur clusters, which are necessary components in the function of the mitochondria and thus energy production. It is also known that specific nerve cells degenerate in people with FA, and this is directly manifested in the symptoms of the disease.

Penwest is a drug development company focused on identifying and developing products that address unmet medical needs, primarily for rare disorders of the nervous system. Penwest is currently developing A0001, or alpha tocopherol quinone, a coenzyme Q10 analog demonstrated to improve mitochondrial function in vitro. Penwest is also applying its drug delivery technologies and drug formulation expertise to the formulation of its collaborators' product candidates under licensing collaborations.