NORTH CHICAGO, Ill., July 17, 2015 /PRNewswire/ -- C2N Diagnostics and AbbVie (NYSE: ABBV) today announced that the U.S. Food and Drug Administration (FDA) has granted their investigational recombinant humanized anti-tau antibody, C2N-8E12 (ABBV-8E12), an orphan drug designation for the treatment of progressive supranuclear palsy (PSP). The companies also have begun a Phase 1 clinical study of C2N-8E12 in patients with PSP.
"The FDA's orphan drug designation recognizes the lack of treatment options for patients with PSP, a debilitating neurological disease, and is an important milestone in the development of potential therapies," stated Joel Braunstein, M.D., chief executive officer, C2N Diagnostics.
"We are encouraged by the preclinical data of anti-tau antibodies and are committed to exploring the potential of this class of molecules," said Jim Sullivan, Ph.D., vice president, pharmaceutical discovery, AbbVie.
C2N-8E12 is a humanized antibody targeting the tau protein found in neurofibrillary tangles in the brain of patients with tauopathies such as PSP and Alzheimer's disease (AD). The Phase 1 study is a randomized, double-blind, placebo-controlled, single ascending dose, multicenter study that will evaluate the safety, tolerability and pharmacokinetics of C2N-8E12 in an estimated 32 subjects with PSP (https://clinicaltrials.gov/ct2/show/NCT02494024).
About Orphan Drug Designation
The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.1
The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval for an investigational use. Sponsors must establish safety and efficacy of a compound in the treatment of a disease through adequate and well-controlled studies.
About Progressive Supranuclear Palsy
Progressive supranuclear palsy (also known as Steele-Richardson-Olszewski syndrome) is a progressive neurodegenerative disorder, with an estimated annual incidence of one per 100,000 people over the age of 60. Within the U.S., the disease affects approximately 20,000 individuals. The most common features of PSP are the presence of loss of balance leading to unexplained falls and, in later stages, blurred vision and problems controlling eye movement may occur. Other nonspecific symptoms of PSP, such as slowed movements or behavioral or cognitive changes, are similar to other brain disorders, particularly Parkinson's disease. For this reason, correct diagnosis of PSP is often delayed. The course of PSP is progressive and may predispose individuals to serious complications, such as choking, pneumonia, head injury and fractures caused by falls. Currently, there are no approved treatments for PSP. It is one of more than 20 different neurodegenerative disorders characterized by neurofibrillary degeneration and tau inclusions as a predominant central nervous system lesion. 2,3
AbbVie is a global, research-based biopharmaceutical company formed in 2013 following separation from Abbott Laboratories. The company's mission is to use its expertise, dedicated people and unique approach to innovation to develop and market advanced therapies that address some of the world's most complex and serious diseases. Together with its wholly-owned subsidiary, Pharmacyclics, AbbVie employs more than 28,000 people worldwide and markets medicines in more than 170 countries. For further information on the company and its people, portfolio and commitments, please visit www.abbvie.com. Follow @abbvie on Twitter or view careers on our Facebook or LinkedIn page.
About C2N Diagnostics
C2N Diagnostics, LLC (www.c2ndiagnostics.com) was formed by scientific co-founders Drs. David Holtzman and Randall Bateman of Washington University School of Medicine in St. Louis, MO and LifeTech Research, a technology research and venture development firm (www.lifetechresearch.com). C2N is commercializing a suite of biomarker assays and tools to enable drug discovery, clinical drug development at lower risk and cost, and early detection of debilitating neurodegenerative disorders. The company's products include the SILK-A?®, SILK-ApoE™, SISAQ-A?™, and SISAQ-Tau™ Assays, which rely upon stable isotope labeling and mass spectrometry for the measurement of the kinetics, or in vivo metabolism, and quantitation of brain derived proteins. Beyond Alzheimer's Disease, products are in development to target Parkinson's Disease, traumatic brain injury, schizophrenia and Amyotrophic Lateral Sclerosis, among other conditions. For additional information, please contact firstname.lastname@example.org or call 1-877-C2N-DIAG (1-877-226-3424).
Some statements in this news release may be forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions, among others, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those indicated in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry.
Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," in AbbVie's 2014 Annual Report on Form 10-K/A, which has been filed with the Securities and Exchange Commission. AbbVie undertakes no obligation to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law.
- Litvan I, Agid Y, Jankovic J, et al. Accuracy of clinical criteria for the diagnosis of progressive supranuclear palsy (Steele-Richardson-Olszewski syndrome). Neurology 1996;46:922-30.
- Williams DR, Lees AJ. Progressive supranuclear palsy: clinicopathological concepts and diagnostic challenges. Lancet Neurol 2009;8:270-79.
SOURCE AbbVie Inc.