PTI-125 has a novel mechanism of action that demonstrated improvement in biomarkers of Alzheimer’s disease in plasma and lymphocytes.

The new data demonstrated clinical evidence of PTI-125’s mechanism of action and drug-target engagement including:

• Improvements in biomarkers of Alzheimer’s disease in plasma and lymphocytes.
• Consistency across biomarker improvements in CSF, plasma, and lymphocytes.
• Significant reductions (p<0.01) in both nitrated and phosphorylated forms of tau protein.
• Evidence that each individual patient showed biomarker responses to PTI-125.
• Evidence that PTI-125 reversed the shape of altered filamin A in lymphocytes.

Cassava Sciences expects to publish a manuscript of these new clinical data in a peer-reviewed medical journal.

About PT-125 Targets

PTI-125 targets both neurodegeneration and inflammatory components of Alzheimer’s disease. As previously reported in a Phase 2a study funded by the National Institutes of Health (NIH), open-label treatment with PTI-125 for 28 days significantly improved key CSF biomarkers of Alzheimer’s pathology, neuroinflammation and neurodegeneration.

PTI-125 target is an altered form of filamin A (FLNA), a scaffolding protein. Published studies have shown that altered FLNA in the brain disrupts the normal function of neurons leading to Alzheimer’s pathology, neurodegeneration, and neuroinflammation. Cassava Sciences’ lead drug candidate, PTI-125, is a small molecule that restores the normal shape and function of FLNA in the brain. This action improves the function of certain receptors in the brain, which slows neurodegeneration and exerts powerful anti-neuroinflammatory effects.

Cassava Sciences

Cassava Sciences is now evaluating PTI-125 in a confirmatory Phase 2b study.  

Cassava Sciences is also developing an investigational diagnostic to detect Alzheimer’s disease with a simple blood test. This program, called PTI-125Dx, also receives significant scientific and financial support from NIH.

The underlying science for Cassava Sciences’ programs in neurodegeneration is published in prestigious peer-reviewed technical journals, including the Journal of Neuroscience, Neurobiology of Aging, and Journal of Biological Chemistry. As previously announced, NIH has awarded Cassava Sciences two research grants following an in-depth, confidential review of its science and technology. These two grant awards represent up to $6.7 million of non-dilutive financing. 

• Cassava Sciences’ mission is detecting and treating neurodegenerative diseases, such as Alzheimer’s disease.
• Cassava Sciences has spent ten years using combined state-of-the-art technology with new insights in neurobiology to develop novel solutions for Alzheimer’s disease. 
• Cassava Sciences owns worldwide development and commercial rights to its research programs in Alzheimer’s disease, and related technology without royalty obligations to any third-party. 

Prohost Observations

Cassava Sciences’ product PTI-125 is bringing good news toward treating Alzheimer’s Disease and other neurological diseases. The product has achieved positive results in what it has been investigated for. We are looking forward to seeing these positive results improving the symptoms of Alzheimer’s Disease in the recruited patients.

We tend to be optimistic about PTI-125 based on the successful clinical trial results to date.

Happy New Year 2020 

The End of the Year Issue of the Prohost Letter Will Be Posted Soon!

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