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news.US-based Cortice Biosciences has started patient enrolment in a Phase I clinical trial designed to assess the safety, tolerability and preliminary efficacy of its lead drug candidate, TPI 287, to treat patients with progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD).
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Both, PSP and CBD represent diseases of the central nervous system and are collectively called as tauopathies, and for which no FDA approved therapies exist.
The Phase I trial is being led by investigators at the University of California San Francisco (UCSF), with support from CBD Solutions, a non-profit organization dedicated to finding a cure for CBD.
Tauopathies, which are a class of neurodegenerative diseases characterized by the presence of neurofibrillary tangles in the brain, have overlapping and distinct pathological characteristics as well as symptoms that can initially present as Alzheimer’s and/or Parkinson’s diseases.
A common feature of tauopathies is dysfunctional tau protein, which is needed for the function of neuronal microtubules (MTs) required for trafficking of cellular cargo along axons.
The company said that TPI 287 may stabilize MTs, which contribute to neuronal cell death and progressive cognitive and motor defects associated with tauopathies, to compensate for dysfunctional tau.
According to Cortice, results from trials with TPI 287 and other MT stabilizing agents in animal models of tauopathies supports the rationale for evaluating the drug candidate as treatment for PSP and CBD.
CBD Solutions chief executive officer Samuel Svensson said CBD and PSP are rare disorders with a high unmet medical need.
"This collaborative effort is important progress and in line with our vision of bringing new therapeutic interventions that could slow down disease progression in these devastating diseases," Svensson said.