Alcobra took a body blow last year when the FDA placed a clinical hold on its lead drug metadoxine, but a failed phase 3 trial has now delivered a coup de grâce for the program.
The Israeli company released top-line results from its trial of metadoxine in attention-deficit hyperactivity disorder (ADHD) in adult patients today, revealing that the drug was no better than placebo at managing symptoms.
"We consider the negative findings of the study to be conclusive," said Alcobra's chief executive Yaron Daniely on a call with investors today, saying it is now unlikely that the company will invest further in metadoxine for ADHD.
The finding dashes Alcobra's hopes of shaking up the market for ADHD drugs with a drug specifically developed for adult patients with the condition, and shares in the company had already slid more than 40% this morning.
The FDA placed the MEASURE trial on a clinical hold last September, introducing a block on any additional dosing with the drug as well as patient recruitment after reports of potentially damaging effects on peripheral nerve function in animal studies.
At the time, Daniely played down the significance of the regulatory move, noting that metadoxine has been on the market outside the U.S. for nearly 40 years as an antidote for alcohol intoxication with no significant safety concerns. He also said the doses used in the animal study were much higher than would be encountered in clinical practice.
In December the FDA agreed to relax the clinical hold from full to partial and allow a phase 1 safety assessment, but fast forward to this month and those developments are of course redundant.
Where now for the company? Daniely had little to offer shareholders on the call today, simply noting that the company would take some time to reassess its priorities and will communicate a new plan "in the next few weeks."
Options could be to continue to develop metadoxine in other indications—notably Fragile X syndrome outside the U.S—or indeed other pipeline projects, but there were few details to be had on the latter other than assertions that Alcobra has been stepping up efforts to identify promising projects since last year.
"I don't believe the efficacy outcome of this study has any bearing on Fragile X or any other cognitive disorder," said Daniely. Alcobra may explore studying the drug in Fragile X in Europe, but would need a partner for that project, he suggested.
Meanwhile, Alcobra is sitting on around $50 million in cash, according to chief operating officer Tomer Berkovitz, who said the company will look for ways to reduce its costs in the coming months.