AbbVie and C2N Diagnostics have received orphan drug designation from the US Food and Drug Administration (FDA) for their investigational recombinant humanised anti-tau antibody, C2N-8E12 (ABBV-8E12), to treat progressive supranuclear palsy (PSP).
C2N-8E12 is a humanised antibody that targets the tau protein observed in neurofibrillary tangles in the brain of patients with tauopathies such as PSP and Alzheimer’s disease (AD).
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PSP is a progressive neurodegenerative, with annual incidence of one in 100,000 people over the age of 60.
Its most common features are the presence of loss of balance leading to unexplained falls, and in later stages, it may cause blurred vision and problems controlling eye movement.
C2N Diagnostics CEO Joel Braunstein said: "The FDA’s orphan drug designation recognises the lack of treatment options for patients with PSP, a debilitating neurological disease, and is an important milestone in the development of potential therapies."
Both firms have started Phase I clinical trial of C2N-8E12 in patients with PSP.
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By GlobalDataThe study is a randomised, double-blind, placebo-controlled, single ascending dose and multicentre trial, which will assess the safety, tolerability and pharmacokinetics of C2N-8E12 in an estimated 32 subjects with PSP.
AbbVie pharmaceutical discovery vice-president Dr Jim Sullivan said: "We are encouraged by the preclinical data of anti-tau antibodies and are committed to exploring the potential of this class of molecules."
Image: Autopsy specimen in Steele-Rirchardson-Olzewski syndrome (progressive supranuclear palsy). Photo: courtesy of Jensflorian.
