PATTERSON, N.Y., Feb. 9, 2010 (GLOBE NEWSWIRE) -- Penwest Pharmaceuticals Co. (Nasdaq:PPCO) today announced that it has initiated a Phase IIa clinical trial for A0001 in patients with the A3243G mitochondrial DNA point mutation and evidence of impaired mitochondrial function. This point mutation is commonly associated with MELAS syndrome. The study is being conducted at the Newcastle upon Tyne Hospitals in Newcastle, England. Newcastle is a specialist neuroscience center in the United Kingdom that offers comprehensive services for both adults and children suspected of having a rare mitochondrial disorder.
The primary objective of this 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. The Phase IIa clinical trial is a double-blind, randomized, placebo-controlled trial. Penwest plans to enroll approximately 30 patients who meet the eligibility criteria 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.
Jennifer L. Good, Penwest's President and CEO, 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 a significant unmet medical need in these mitochondrial diseases, including MELAS, and we are hopeful that A0001 can provide an important treatment option for these patients."
About A0001
A0001, or alpha-tocopherolquinone, is a coenzyme Q 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.
About MELAS Syndrome
MELAS is a rare progressive neurodegenerative disorder that involves multiple system organs, including the CNS, skeletal muscle, eye, and cardiac muscle. MELAS is caused by mutations in the DNA in the mitochondria, with the majority of the patients with clinical characteristics of MELAS having a DNA point mutation at A3243G. In many patients with MELAS syndrome, presentation occurs with the first stroke-like episode, usually when an individual is between four and fifteen years of age. There is no known treatment for the underlying disease, which is progressive and fatal. MELAS is an acronym for mitochondrial myopathy (weakness of muscles throughout the body), encephalopathy (disease of the central nervous system), lactic acidosis (abnormal build-up of lactic acid, normally a waste product in the body) and stroke.
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, or alpha-tocopherolquinone, 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 our collaborators' product candidates under licensing collaborations.
Penwest Forward-Looking Statements
The matters discussed herein contain forward-looking statements for purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 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 the following: the timing of clinical trials, such as the Phase IIa clinical trial referenced above, and risks related to patient enrollment; 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 with respect to A0001; whether the results of clinical trials 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 Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 9, 2009, which risk factors are incorporated herein by reference.
The forward-looking statements contained in this press release speak only as of the date of the statements made. Penwest disclaims any intention or obligation to update any forward-looking statements, and these statements should not be relied upon as representing the Company's estimates or views as of any date subsequent to the date of this release.
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