Top-line Results from a Phase 2b Study of PTI-125 in Alzheimer’s Disease Does Not Meet Primary Endpoint

- Study Showed High Variability in Levels of CSF Biomarkers Over 28 Days -

- Drug Was Safe and Well-tolerated -

AUSTIN, TX, USA I May 15, 2020 I Cassava Sciences, Inc. (Nasdaq: SAVA) today reported top-line results from a Phase 2b study of PTI-125, its lead investigational drug, in patients with Alzheimer’s disease. This study did not meet its primary endpoint. The pre-specified primary endpoint was a statistically significant effect of PTI-125 versus placebo on cerebrospinal fluid (CSF) levels of tau protein and other biomarker assessments from baseline to Day 28. PTI-125 significantly reduced a secondary endpoint, CSF levels of IL1-beta (p<0.035), a core biomarker of neuroinflammation, from baseline to Day 28.  Drug was safe and well-tolerated.

A post-hoc analysis of biomarker data revealed high variability in levels of CSF biomarkers over 28 days. For example, placebo-treated patients recorded changes in levels of CSF tau and p-tau ranging from -54% to +34% and -49% to +253%, respectively, from baseline to Day 28. Biomarker analysis was conducted by outside labs.

The drug effects of PTI-125, if any, may have been masked in this study by high variability in levels of biomarkers of disease. In the months ahead the Company plans to re-analyze CSF biomarkers from all study participants.

“Today’s top-line results disappoint and are not consistent with previous clinical experience for reasons that are unclear at the moment,” said Remi Barbier, President & CEO. “We plan to thoroughly analyze these top-line data, and to re-analyze CSF biomarkers from study participants, to better understand the outcome of this study. Alzheimer’s is a disease in dire need of new treatments. It is worth reflecting on what we can learn from this study and how to move forward with drug development plans for PTI-125 in Alzheimer’s disease.”

Phase 2b Study Design
Phase 2b was a double-blind, randomized, placebo-controlled study of PTI-125 in 64 patients with mild-to-moderate Alzheimer’s disease, 50-85 years of age, with MMSE 16 to 26. Participants received PTI-125 100 mg, 50 mg or matching placebo, twice-daily, for 28 continuous days. 

About Alzheimer's Disease
Alzheimer’s disease is a progressive brain disorder that destroys memory and thinking skills. Currently, there are no drug therapies to halt Alzheimer’s disease, much less reverse its course. In the U.S. alone, approximately 5.8 million people are currently living with Alzheimer’s disease, and approximately 487,000 people age 65 or older developed Alzheimer’s in 2019.1 The number of people living with Alzheimer’s disease is expected to grow dramatically in the years ahead, resulting in a growing social and economic burden.2

About Cassava Sciences, Inc.
The mission of Cassava Sciences, Inc. is to detect and treat neurodegenerative diseases, such as Alzheimer’s disease. Over the past 10 years, Cassava Sciences has 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 technologies, without royalty obligations to any third-party.

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SOURCE: Cassava Sciences

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