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Penwest Pharmaceuticals Co. (PPCO)
Q4 2009 Earnings Call

March 4, 2010 11:00 am ET

(…) The first study which we initiated in December 2009 is in patients with Friedreich's Ataxia or FA. FA which afflicts one in 50,000 people in the US is a debilitating, life shortening, degenerative neuromuscular disorder. FA patients have gene mutations that limit the production of a protein call frataxin, an important protein that functions in the (Technical Difficulty) – and thus affects energy function. Onset of symptoms can vary from childhood to adulthood.

There are currently no approved treatments in the US or Europe.
The study with A0001 is being conducted at a single site of Children’s Hospital of Philadelphia. The Phase IIa clinical trial is a double-blind randomized placebo controlled trial that includes two dosage strength of A0001 and placebo. Penwest is targeting 30 patients to complete this study with a 2:1 randomization of drug to placebo. The patients will be dosed b.i.d. for 28 days. 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. This study is certainly screening patients and we expect data in the third quarter of this year. (...)

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