Pipex Pharmaceuticals Files New Drug Application (NDA) With FDA to Market COPREXA for the Treatment of Initially
ANN ARBOR, MI, Nov 29 (MARKET WIRE) -- Pipex Pharmaceuticals, Inc. (AMEX: PP), a specialty pharmaceutical company developing innovative late-stage drug candidates for the treatment of neurologic and fibrotic diseases, announced today that it has filed a New Drug Application (NDA) with the FDA for its lead drug candidate, COPREXA (oral tetrathiomolybdate), for the treatment of initially presenting neurologicWilson's disease.
"This NDA filing represents a transforming event for Pipex as well as an important potential new treatment option for initially presenting neurologic Wilson's disease patients. In addition to making this potential new treatment option available to initially presenting neurologic Wilson's disease patients,we look forward to increasing awareness of this debilitating disease that is all too often misdiagnosed for years or, if diagnosed at all, is diagnosed too late to save these patients' lives," said Steve H. Kanzer, CPA, Esq., Pipex's Chairman & CEO. "I would like to personally thank our internal regulatory, analytical, clinical, manufacturing teams, as well as our external consultantsand advisors, all of whom cooperated in the preparation of this important NDA filing."
"Following the completion of our two Pre-NDA meetings for COPREXA duringAugust and September of this year, we are pleased to have met our previous guidance to file this application by the end of November," said Dr. Charles L. Bisgaier, President of Pipex. Dr. Bisgaier went on to say, "We are gratified to bring COPREXA one step closer to these neurologic Wilson's disease patients."
"As a clinician and inventor of COPREXA, I was extremely pleased with the timing of the COPREXA NDA submission," said Dr. George Brewer, ProfessorEmeritus at the University of Michigan, and Chairman of Pipex's COPREXA Scientific Advisory Board. "As a clinician with considerable experience in the treatment and management of Wilson's disease patients, I believe that COPREXA will fulfill an important long standing unmet need for the treatment of initially presenting neurologic Wilson's disease, a serious debilitating andlife threatening disease which is not adequately addressed by existing therapies."
About COPREXA(TM)
COPREXA(TM) is an oral, small-molecule, anti-copper agent that is highly specific for the reduction of free copper in serum, the most toxic form of copper in the body, and is thus suited for the treatment of central nervoussystem (CNS) diseases in which abnormal serum and CNS copper homeostasisare implicated. COPREXA has completed two clinical trials in initially presenting neurologic Wilson's disease patients, the results of which have been previously published(1)(2). Pipex is also developing COPREXA for fibrotic disorders based upon the rationale that the fibrotic disease process isdependent upon the availability of free copper in the body. COPREXA has demonstrated the ability to inhibit fibrosis in a number of well established animal models through the sequestration of available copper and inhibition of key fibrotric cytokines, including secreted protein acid rich in cysteine (SPARC), NFKB,TGF-B, FGF-2, IL-1, IL-6, IL-8, and connective tissue growth factor (CTGF). COPREXA has been designated as an "Orphan Drug" by the FDA for the treatment of initiallypresenting neurologic Wilson's Disease.
COPREXA has completed a 20-patient, one year, open label, phase I/IIclinical trial for the treatment of refractory idiopathic pulmonary fibrosis (IPF), afatal respiratory disease. The results of this trial were presented in May of this year at the annual meeting of the American Thoracic Society (ATS). Pipex is planning to initiate a phase III clinical trial of COPREXA in IPF and, if successful, Pipex may be permitted to file a supplemental NDA for COPREXA for this additional indication. COPREXA is also in a phase II clinical trial for the treatment of primary biliary cirrhosis (PBC), a fibrotic disease of the liver.
About Wilson's Disease
Wilson's disease is an autosomal recessive genetic disease attributable to mutations of the ATP7B gene. Worldwide, it is estimated that there are between 10 million and 30 million carriers of the heterozygous mutated gene. These mutations lead to an inability to properly clear excess free copper from the body via the liver into the bile and stool. As a result, copper accumulates in the liver and elevated levels of toxic free copper enter the systemic circulation, cross the blood brain barrier, and enter the cerebral spinal fluid
(CSF) and brain. These increased levels of free copper cause significant neurologic damage, resulting in tremors, impaired speech, impaired coordination, and Parkinson's-like dystonia.
Psychiatric symptoms of neurologically-presenting Wilson's patients will generally precede neurologic symptoms by months or years and may include loss of emotional control, temper tantrums, emotional outbursts, bouts of crying, severe depression, suicidal ideation, loss of inhibitions, delusions,hallucinations and loss of ability to focus on tasks. Neurologic symptoms later develop as aresult of neurodegeneration in the basal ganglia of the brain and include impaired speech, tremor, dystonia, incoordination and dysphasia. Crippling movement disorders may ultimately occur. Without proper treatment, Wilson's disease is usually fatal by the age of 30. However, if treatment is begun early enough, symptomaticrecovery is usually complete and a life of normal length and qualitycan beexpected. For more information about Wilson's disease, please visit or contact the Wilson's Disease Association at www.wilsonsdisease.org.
About Pipex Pharmaceuticals, Inc.
Pipex Pharmaceuticals, Inc. ("Pipex") is a specialty pharmaceutical companythat is developing proprietary, late-stage drug candidates for thetreatment of neurologic and fibrotic diseases. Pipex's strategy is to exclusively in-license proprietary, clinical-stage drug candidates and complete the further clinical testing, manufacturing and regulatory requirements sufficientto seek marketing authorizations via the filing of New Drug Applications (NDAs) with the FDA in the US and Marketing Application Authorizations (MAAs) with the European Medicines Evaluation Agency (EMEA). For further information pleasevisit www.pipexinc.com.
This press release contains forward-looking statements, within the meaningof Section 21E of the Securities Exchange Act of 1934, that reflect Pipex Pharmaceuticals, Inc. and subsidiaries ("we" or "our") current expectationsabout its future results, performance, prospects and opportunities,including statements regarding the potential use of COPREXA as well as the prospects for regulatory filings in the treatment of neurologic Wilson's disease, includingthat our NDA will be accepted for filing by the FDA and/or that the FDA will agree with our analysis of data supporting the safety, clinical efficacy, manufacturing, stability and other regulatory requirements necessary for COPREXAto be approved for use in neurologically presenting Wilson's disease or that evenif approved for initial indication, that we will be able to conduct and complete necessary initial and registration clinical trials required to support and receive FDA approval for a Supplemental New Drug Application to market COPREXA for the treatment of other disease indications, such as, idiopathic pulmonaryfibrosis, Alzheimer's disease and Huntington's disease, for example. Wherepossible, the Company has tried to identify these forward-lookingstatements by using words such as "anticipates," "believes," "intends," or similar expressions. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements, including risks set forth in our filings with the Securities and Exchange Commission. We cannot assure you that we will be able to successfully develop or commercialize products based on our technologies, including COPREXA(TM), TRIMESTA(TM), zinc-monocysteine, SOLOVAX(TM), EFFIRMA(TM) or Anti-CD4 802-2,particularly in light of the significant uncertainty inherent in developing, manufacturing and conducting preclinical and clinical trials of new pharmaceuticals, and obtaining regulatory approvals, that our technologies will prove to be safeand effective, that our cash expenditures will not exceed projected levels,that we will be able to obtain future financing or funds when needed,that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that we will be able to successfully obtain any further grants and awards, maintain our existing grants which are subject to performance, that we will be able to patent, register or protect our technology from challenge and products from competition or maintain or expand our license agreements with our current licensors, or that our business strategy will be successful. All forward-looking statements made in this press release are made as of the date hereof, and the Company assumes no obligation to update the forward-looking statements included in this news release whether as a result of new information, future events, or otherwise, other than as required by law.
(1) Brewer, G.J., et al., Treatment of Wilson disease with ammonium tetrathiomolybdate: III. Initial therapy in a total of 55 neurologicallyaffected patients and follow-up with zinc therapy. Arch Neurol. 2003 Mar; 60(3):379-85.
(2) Brewer, G.J., Askari, F., Lorincz, M.T., Carlson, M., Schilsky, M., Kluin, K.J., Hedera, P., Moretti, P., Fink, J.K., Tankanow, R., et al.
2006. Treatment of Wilson disease with ammonium tetrathiomolybdate: IV. Comparison of tetrathiomolybdate and trientine in a double-blind study of treatment of the neurologic presentation of Wilson disease. Arch Neurol 63:521-527. For Further Information Contact: Steve H. Kanzer, CPA, Esq. Chairman and Chief Executive Officer
(734) 332-7800 Thomas Redington (Investor Relations) Redington, Inc.
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