Axovant races to PhIII dementia study as nelotanserin clears an early hurdle, but questions linger
Axovant says that its drug nelotanserin has cleared an early hurdle in a Phase II study for Lewy body dementia, triggering a decision to expand the mid-stage trial and hitting the green light for a Phase III to get started later this year. But investors are likely to question a stumble on key secondary goals.
A snapshot of results for the first tiny group of 11 patients concluded that they were demonstrating statistically significant responses to the drug on the primary endpoint. These patients included people suffering from Parkinson’s as well as Lewy body dementia. They were recruited after suffering from frequent visual hallucinations.
“The primary outcome measures of the study were extrapyramidal signs as assessed by Unified Parkinson’s Disease Rating Scale (UPDRS) Parts II + III and safety,” the company reported.
There was a big hitch, though. The trial did not hit secondary endpoints laid out by investigators to track the frequency of visual hallucinations, including both a standard SAPS assessment as well as an endpoint they created internally at Axovant. And that may have weighed in on investors’ response to the results, with Axovant’s stock up only 5% in pre-market trading.
Axovant, the first of Vivek Ramaswamy’s biotechs to get started, plans to hustle into the expanded Phase II for the drug — a 5HT2A antagonist — as it wastes no time in getting the Phase III up and running. Ramaswamy has made no secret of his plans to accelerate R&D at his companies. And Axovant is the most advanced with a big trial underway for Alzheimer’s.
Axovant already faces an established market player. Acadia’s rival drug Nuplazid won FDA approval for Parkinson’s dementia last spring, but only after regulators expressed their safety concerns, noting a distinct increase in the number of deaths as well as the rate of adverse events among the patients taking the drug compared to the control arm of the study — even if there was no obvious clue what was triggering those events.
Axovant’s hope is that it can come out of clinical trials with a better success rate and a cleaner safety profile as both companies target a broad patient population suffering from different forms of dementia.
“I am intrigued by the benefits observed on the UPDRS in this study,” stated Dr. James Leverenz, Chair, Scientific Advisory Council of the Lewy Body Dementia Association and Director of the Cleveland Lou Ruvo Center for Brain Health at the Cleveland Clinic. “If a single drug could simultaneously address the motor and neuropsychiatric symptoms of Lewy body dementia, which the published literature suggests may be the case for 5HT2A antagonists, it would represent a unique and important advance for the treatment of this condition. I look forward to reviewing the full dataset later this year.”