vTv Therapeutics reported the second part of its Phase III Alzheimer’s disease study failed to meet co-primary efficacy endpoints.
More disappointing news for North Carolina-based vTv Therapeutics has sent shareholders running. The company reported the second part of its Phase III Alzheimer’s disease study failed to meet co-primary efficacy endpoints.
This was the second failure for the company’s late-stage Alzheimer’s drug azeliragon. Although the company was disappointed in April when he first part of its Phase III azeliragon study failed to meet primary endpoints in the first part of the trial, the company had hopes for Part B. However, the second part of the trial for people with mild Alzheimer’s disease failed, as have so many other potential treatments for the mind-robbing disease. As could be expected investors have been dumping shares of vTv. The stock is down more than 27 percent in premarket trading to $1.37.
Despite the failure to achieve primary endpoints, Steve Holcombe, chief executive officer of vTv Therapeutics, remains optimistic that azeliragon could yet prove to be beneficial to Alzheimer’s patients. Holcombe pointed to some small gains seen in patients. Much like was seen in a post-hoc analysis of data from Part A of the Phase III trial, vTv pointed to a subpopulation of Alzheimer’s patients that showed a statistically significant benefit in comparison to placebo. Those patients consisted of trial participants with peak azeliragon blood plasma concentration of less than 7.5 ng/mL and Mini Mental State Examination (MMSE) scores between 19 and 27. vTv said the endpoints were statistically significant improvement in cognitive or functional outcomes as measured by the Alzheimer’s disease Assessment Scale-cognitive subscale (ADAS-cog) and Clinical Dementia Rating Scale Sum of Boxes (CDR-sb) at 12-months compared to placebo. Consistent with findings in Part A and the Phase 2b trial, lower maximal plasma concentrations of azeliragon in Part B were associated with improvements in efficacy relative to placebo, the company said.
When the results of the Part A and Part B trial are pooled and change from baseline at 12 months is compared, vTv said the azeliragon subgroup had a 1.8 point improvement in ADAS-cog, a 0.4 improvement in CDR-sb and a 2.3 improvement in Alzheimer’s disease Cooperative Study-Activities of Daily Living relative to placebo.
vTv said it will rely on the Fast Track designation and study results to “pursue expedited discussions” with the U.S. Food and Drug Administration “to propose a pathway for further clinical development in support of regulatory approval of azeliragon.”
“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,” Holcombe said in a statement. “As a result, we believe that azeliragon has the potential to address the pressing unmet need in Alzheimer’s disease.”
Azeliragon is a novel orally active small-molecule antagonist of RAGE, that vTv discovered through its proprietary drug discovery platform TTP Translational Technology. In its research, vTv said research has shown that receptor for advanced glycation endproducts (RAGE) activation contributes to the pathogenesis of Alzheimer’s disease.
Alzheimer’s and other forms of dementia are a growing healthcare concern. In the United States, Alzheimer’s disease is the sixth-leading cause of death across all ages and the fifth-leading cause of death in people over 65 years of age. By 2050 the number of global patients is expected to hit 131.5 million.
