DANBURY, Conn., July 16, 2009 (GLOBE NEWSWIRE) -- Penwest Pharmaceuticals Co. (Nasdaq:PPCO) today announced the results of its Phase Ib clinical trial of A0001, a compound the Company is developing for the treatment of mitochondrial diseases. Drs. Amale Hawi, Senior Vice President, Pharmaceutical Development, and Thomas Sciascia, Senior Vice President & Chief Medical Officer, will be presenting these results at the Friedreich's Ataxia Therapeutics Meeting on Friday, July 17, 2009, in Philadelphia, PA.
In the Phase Ib trial, the drug was well tolerated by subjects, and no serious adverse events were reported. There was a dose-dependent increase in exposure approaching steady state within 2-4 days following repeat-dosing, and a maximum tolerated dose was established.
Based on these results, Penwest plans to advance A0001 into Phase IIa studies in patients with mitochondrial diseases. The Company intends to commence two Phase IIa trials - one focused on patients with Friedreich's Ataxia and the second focused on patients with the A3243G mitochondrial DNA point mutation associated with the "MELAS" syndrome - in the fourth quarter of 2009. The Phase IIa trials will be conducted with twice daily oral dosing. The Company expects data from both of these trials in the first half of 2010.
Jennifer L. Good, Penwest's President and CEO, said, "We are very pleased to have completed the Phase I safety work on A0001 so that we can advance this compound into patients. We believe A0001 is promising for patients who suffer from mitochondrial diseases and currently have very limited treatment options, and we are eager to begin to evaluate the clinical benefit in patients."
The Phase Ib trial was a single-blind, placebo-controlled, multiple ascending dose clinical trial in approximately 40 healthy male and female subjects for up to 14 days. The trial was designed to evaluate the safety and tolerability of A0001 and characterize the pharmacokinetic profile following repeat dosing.
About A0001
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 is developing A0001 under a collaboration and licensing agreement with Edison Pharmaceuticals, a privately held biopharmaceutical company headquartered in San Jose, CA.
About Mitochondrial Diseases
Mitochondrial diseases are devastating, potentially life-threatening illnesses for which there are no FDA-approved treatments. Penwest is focusing its development efforts on mitochondrial respiratory chain diseases, including, among other diseases, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), Friedreich's Ataxia, Leber's Hereditary Optic Neuropathy and coenzyme Q10 deficiency. These are orphan diseases that the Company believes currently affect approximately 10,000 to 35,000 patients per disease in the U.S. and Europe. A0001 has received orphan drug designation from the FDA for the treatment of inherited mitochondrial respiratory chain diseases.
About Penwest Pharmaceuticals
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, a coenzyme Q10 analog drug candidate for inherited mitochondrial respiratory chain diseases. Penwest is also applying its drug delivery technologies and drug formulation expertise to the formulation of our collaborators, product candidates under licensing collaborations.
Penwest Forward-Looking Statements
The matters discussed herein contain forward-looking statements that involve risks and uncertainties, which may cause the actual results in future periods to be materially different from any future performance suggested herein. For this purpose, any statements contained herein that are not statements of historical fact may be deemed to be forward-looking statements. Without limiting the foregoing, the words, "believes," "anticipates," "plans," "expects," "intends," "potential," "appears," "estimates," "projects," "targets," "may," "could," and similar expressions are intended to identify forward-looking statements. Important factors that could cause results to differ materially include: risks relating to the commercial success of Opana ER, including our reliance on Endo Pharmaceuticals Inc. for the commercial success of Opana ER and risks of generic competition; the need for capital; regulatory risks relating to drugs in development, including the timing and outcome of regulatory submissions and regulatory actions; uncertainty of success of collaborations; the timing of clinical trials; whether the results of clinical trials such as the results of the Phase Ib trial referred to above will be indicative of the results of future clinical trials and will warrant further clinical trials, warrant submission of an application for regulatory approval of, or warrant the regulatory approval of, the product that is the subject of the trial; whether the patents and patent applications owned by us will protect the Company's products and technology; actual and potential competition; and other risks as set forth under the caption Risk Factors in Penwest's Annual Report on Form 10-Q filed with the Securities and Exchange Commission on May 11, 2009, which risk factors are incorporated herein by reference.
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