Cortice Announces Enrollment of a Phase 1 Clinical Trial Evaluating TPI 287 in Rare Tauopathy Diseases

Orphan Drug Designation Granted for Treatment of Progressive Supranuclear Palsy

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| Source: Cortice Biosciences

NEW YORK, July 7, 2014 (GLOBE NEWSWIRE) -- Cortice Biosciences announced today that enrollment has begun in a Phase 1 trial designed to evaluate safety, tolerability and preliminary efficacy of the company's lead drug candidate, TPI 287, for treatment of patients with progressive supranuclear palsy (PSP) and corticobasal degeneration (CBD) (clinicaltrials.gov/NCT02133846). PSP and CBD represent diseases of the central nervous system collectively known as tauopathies, and for which no FDA approved therapies exist. This trial is being led by investigators at the University of California San Francisco (UCSF), and with generous support from CBD Solutions, a non-profit organization dedicated to finding a cure for CBD.

Tauopathies are a class of neurodegenerative diseases characterized by the presence of neurofibrillary tangles in the brain. All have overlapping and distinct pathological characteristics, and have symptoms that can initially present as Alzheimer's and/or Parkinson's diseases. A common feature of tauopathies is dysfunctional tau protein, which is normally required for the function of neuronal microtubules (MTs) essential for trafficking of cellular cargo along axons. MT defects contribute to neuronal cell death and progressive cognitive and motor defects associated with tauopathies. 

As a novel taxoid that penetrates the brain, TPI 287 may stabilize MTs to compensate for dysfunctional tau. Results from studies with TPI 287 and other MT stabilizing agents in animal models of tauopathies supports the rationale for evaluating TPI 287 as treatment for PSP and CBD.

"CBD and PSP are rare disorders with a high unmet medical need," said Samuel Svensson, Ph.D, Chief Executive Officer of CBD Solutions. "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 same UCSF investigators are also conducting a Phase 1 study designed to assess the safety and preliminary efficacy of TPI 287 as a potential treatment for Alzheimer's disease (clinicaltrials.gov/NCT01966666). 

Cortice was recently notified that TPI 287 has been granted orphan drug designation by the FDA Office of Orphan Products Development for the treatment of PSP. This designation is given to novel drugs for the treatment of rare diseases or conditions that affect fewer than 200,000 patients in the United States. Provisions include a seven-year period of U.S. marketing exclusivity to TPI 287, as well as tax credits for clinical research costs, clinical research trial design assistance, and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

About TPI 287

TPI 287 is a taxoid that binds to and stabilizes the assembly of microtubules similarly to the taxanes, paclitaxel and docetaxel. Compared with these other taxanes, TPI 287 has the distinct advantage of being able to readily penetrate the blood-brain barrier. Microtubule stabilization by TPI 287 in the brain may have potential as treatment of neurodegenerative diseases known as tauopathies that are associated with dysfunctional tau protein and defective microtubule architecture. As an anti-cancer agent designed to inhibit cellular division and growth, TPI 287 is also being evaluated for the treatment of glioblastoma, secondary brain metastases, and pediatric neuroblastoma.

About tauopathies

Tauopathies are a collection of over twenty different neurodegenerative diseases characterized by the presence of neurofibrillary tangles (NFTs) in the brain. NFTs are composed of dysfunctional tau protein whose presence has been correlated with disease severity and progression of cognitive and motor defects. Tau dysfunction has been associated with defective microtubule architecture in the axons of affected neurons, which is a major cause of impaired neuronal function. Examples include the rare diseases progressive supranuclear palsy and corticobasal degeneration, and much larger indications such as Alzheimer's disease. More information can be found at www.psp.org, www.theaftd.org, and www.alzfdn.org.

About CBD Solutions

CBD Solutions, based in Stockholm, Sweden, is a non-profit organization dedicated to finding a cure for corticobasal degeneration. CBD Solutions supports and works in partnership with world leading scientists active within the field of CBD research and who are actively seeking new partners interested in new therapeutic opportunities in CBD. More information can be found at www.cbdsolutions.se.    

About Cortice Biosciences

Cortice Biosciences is a clinical-stage drug development company pioneering novel therapies for the treatment of oncologic and neurologic disease indications with urgent unmet medical need. More information can be found at www.corticebiosciences.com.

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