DANBURY, Conn., Feb. 25, 2009 (GLOBE NEWSWIRE) -- Penwest Pharmaceuticals Co. (Nasdaq:PPCO) today announced that it has begun dosing in a Phase Ib clinical trial of A0001, a compound the Company is developing for the treatment of mitochondrial diseases.
Mitochondrial diseases are devastating, potentially life-threatening illnesses for which there are no FDA-approved treatments. Penwest is currently focusing its development efforts on mitochondrial respiratory chain diseases, including, among other diseases, MELAS, Friedreich's Ataxia, Leber's Hereditary Optic Neuropathy and COQ10 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.
The Phase Ib trial is a single-blind, placebo-controlled, multiple ascending dose clinical trial in healthy subjects. The trial is designed to evaluate the safety and tolerability of A0001 and characterize the pharmacokinetic profile following repeat dosing. Penwest plans to enroll a total of 30 healthy volunteers in the trial. The Company expects to announce data from this trial in the second quarter of 2009.
Following completion of this safety trial, Penwest expects to conduct a Phase IIa trial in patients with mitochondrial respiratory chain disease in the second half of 2009. In parallel with the Phase Ib trial, the Company has initiated long-term animal toxicology studies to support the clinical program.
David Lynch, M.D., Ph.D., of The Children's Hospital of Philadelphia, said, "Mitochondrial diseases today represent a significant unmet medical need. Currently, there are very limited treatment options, and no FDA-approved therapeutics. This study will continue the process to determine whether A0001 may be therapeutic in certain patients with mitochondrial diseases."
Jennifer L. Good, Penwest's President and CEO, said, "We are very pleased to advance A0001 into the next phase of its development. We are now planning our Phase II clinical program to evaluate the biological activity of the compound, which will be conducted in the second half of this year."
About A0001
A0001 is a coenzyme Q10 analog demonstrated in-vitro to improve mitochondrial function. 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
A number of diseases have been associated with the failure of mitochondrial function. Mitochondria are specialized organelles present within every cell of the body, except red blood cells, and are responsible for creating more than 90% of the energy needed by the body's cells to sustain life and support growth. When they fail, the cell is unable to meet its energy needs to carry out the processes of life. As a result, cell injury and even cell death follow.
It is now commonly believed that diseases related to mitochondrial pathology are greatly under-diagnosed and the true prevalence is difficult to determine. Research has consistently produced data that shows mitochondrial dysfunction may be present in many very common illnesses and chronic conditions of adulthood. These conditions include Alzheimer's Dementia, Parkinson's disease, Amyotrophic Lateral Sclerosis (ALS), Huntington's, diabetes, hypertension, heart disease, osteoporosis, cancer and even the aging process itself. Furthermore, autoimmune diseases such as multiple sclerosis, lupus and rheumatoid arthritis also appear to have mitochondria pathology. While it is unclear that mitochondrial defects cause these problems, it is clear that mitochondria function is measurably disturbed in each of these diseases.
About Penwest Pharmaceuticals
Penwest is a drug development company focused on identifying, developing and commercializing products that address unmet medical needs, primarily for disorders of the nervous system. Penwest is currently applying its drug development and drug delivery expertise to the development of products that are in various stages of development, and that it intends to commercialize independently or through third party alliances.
Penwest Forward-Looking Statement
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," 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 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 10, 2008, 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.
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