Cortice begins patient enrolment in Phase I trial of TPI 287 to treat rare tauopathy diseases

8th July 2014 (Last Updated July 8th, 2014 18:30)

US-based Cortice Biosciences has initiated enrolling patients in a Phase I trial of its lead drug candidate, TPI 287, for treatment of patients with progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD).

US-based Cortice Biosciences has initiated enrolling patients in a Phase I trial of its lead drug candidate, TPI 287, for treatment of patients with progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD).

The trial is designed to evaluate the safety, tolerability and preliminary efficacy of TPI 287 in these patients with PSP and CBD, which represent diseases of the central nervous system (CNS).

Collectively, PSP and CBD are known as tauopathies, which are a class of neurodegenerative diseases characterised by the presence of neurofibrillary tangles in the brain.

Tauopathies have overlapping and distinct pathological characteristics as well as symptoms that can initially present as Alzheimer's or Parkinson's diseases.

"This collaborative effort is important progress and in line with our vision of bringing new therapeutic interventions that could slow down progression in these devastating disease."

The company said that currently there are no FDA approved therapies for PSP and CBD.

Investigators at the University of California San Francisco (UCSF) are leading the Phase I trial of TPI 287 with support provided from CBD Solutions, a non-profit organisation dedicated to finding a cure for CBD.

One of the common features of tauopathies is dysfunctional tau protein, which is necessary for the function of neuronal microtubules (MTs) required for trafficking of cellular cargo along axons.

According to the company, MT defects contribute to neuronal cell death and progressive cognitive and motor defects associated with tauopathies.

A novel taxoid that penetrates the brain, TPI 287 is expected to stabilise MTs to compensate for dysfunctional tau.

CBD Solutions chief executive officer Samuel Svensson said: "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."

The company said that results from trials with TPI 287 and other MT stabilising agents in animal models of tauopathies supports the rationale for assessing the drug candidate as treatment for PSP and CBD.