Cassava Sciences (NASDAQ:SAVA) reported disappointing topline results from its Phase 2b study of PTI-125 for the treatment of Alzheimer’s disease (AD).
The study enrolled 64 patients with mild-to-moderate AD, 50-to-85 years of age, who received either 50 mg of PTI-125, 100 mg of PT-125, or placebo, twice-daily, for 28 continuous days.
The study failed to meet its primary endpoint of a statistically significant effect of PTI-125, compared with placebo, on cerebrospinal fluid (CSF) levels of tau protein from baseline to day 28.
A post-hoc analysis revealed a high variability in CSF biomarkers, and Cassava noted that the effects of PTI-125, if any, may have been masked by the high variability in biomarker levels.
“Today’s topline results disappoint and are not consistent with previous clinical experience for reasons that are unclear at the moment,” Remi Barbier, Cassava’s president and CEO, said in a statement.
“We plan to thoroughly analyze these topline data, and to reanalyze CSF biomarkers from study participants, to better understand the outcome of this study. AD 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 AD,” he added.
