The data are in from another phase 3 trial of vTv Therapeutics’ Alzheimer’s drug azeliragon and they’re not pretty—it missed a pair of coprimary endpoints, failing to improve cognitive or functional outcomes over placebo.
If this story sounds familiar, that’s because it is. vTv revealed in April that azeliragon had flubbed an identical phase 3 study, but essentially said: hold on, let’s not pronounce death until we’ve evaluated data from both studies.
The two trials, called Part A and Part B of the STEADFAST study, each enrolled 400 patients and evaluated the use of the drug in patients with mild Alzheimer’s. After the Part A disappointment, vTv pulled the plug on all clinical trials involving azeliragon—including Part B—but said enough patients would have finished 12 months of treatment that it could analyze the data.
Specifically, azeliragon failed in Parts A and B to beat placebo in improving patients’ Alzheimer's Disease Assessment Scale-cognitive subscale (ADAS-cog) and Clinical Dementia Rating Scale Sum of Boxes (CDR-sb) scores after 12 months of treatment.
Despite the double flop, vTv is staying optimistic. It’s following up with “expedited discussions” with the FDA to “propose a pathway for future clinical development” that could support an approval for the candidate.
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Its ray of hope is a correlation between lower maximal plasma concentrations of azeliragon and improvements in efficacy.
“We are eager to obtain early feedback from the FDA based on the results we have achieved and the data that we have observed associating lower plasma levels of the drug and improved efficacy,” said vTv CEO Steve Holcombe in a statement. “As a result, we believe that azeliragon has the potential to address the pressing unmet need in Alzheimer’s disease.”
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It has been a hard road for azeliragon. Pfizer dumped the drug after a trial using a 20-mg dose was stopped after signs of cognitive worsening developed in patients. vTv picked it up, saying that a lower, 5-mg dose showed promise in ADAS-Cog scores. So much promise that vTv got off a $117 million IPO built around the Pfizer castoff.
Editor's note: This story has been updated to correct this history of azeliragon. It previously conflated the drug with Axovant's intepirdine.