The professor of neurology and director of translational research and epilepsy clinical trials at NYU Langone spoke about the results of a phase 2b open-label feasibility study of this novel delivery method for abortive seizure treatment in patients with epilepsy.
“Ten minutes doesn’t sound like a lot, but when you’re about to have a seizure, 10 minutes is an eternity. So what can we do to shorten that time?”
There are a number of available abortive seizure treatments for patients with epilepsy, however, these nasal preparations are accompanied by a number of challenges—the time to effect being chief among them. When patients take an abortive medication in preparation for a seizure event, a window up to 10 minutes for the medication to take effect can present a serious issue.
To help address this, Jacqueline French, MD, and colleagues have been working with an inhaled delivery platform of alprazolam (Staccato Alprazolam, Engage Therapeutics) with the hope of bypassing the challenges of the mucus-membrane based pathway of the nasal delivery. This option offers a route through the lungs, which French told NeurologyLive® offers “an enormous quantity of space for the drug to be absorbed” into the bloodstream. In just 2 minutes, serum concentrations of the drug in the blood and brain are much higher.
In conducting a phase 2b open-label feasibility study, the professor of neurology and director of translational research and epilepsy clinical trials, NYU Langone, and Chief Medical Innovation Officer, Epilepsy Foundation, and colleagues found that a 1-mg dose was both safe and efficacious in 8 patients with epilepsy and predictable seizure episodes. Overall, 5 patients were considered responders (62.5%), and preliminary safety analysis showed no serious or severe adverse events.
The next phase of the study will be a randomized, double-blind trial including 100 patients who will be assigned to 1 of 2 active treatment arms evaluating 1 mg or 2 mg of Staccato alprazolam compared with placebo. To find out more about the study, NeurologyLive® spoke with French at the 2019 American Academy of Neurology Annual Meeting in Philadelphia, Pennsylvania.
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French J, Liow K, Vazquez B, et al. A two-part, double-blind, placebo-controlled, inpatient, dose-ranging efficacy study of Staccato alprazolam (STAP-001) in patients with epilepsy with a predictable seizure pattern: results from the initial open-label feasibility part. Presented at: 2019 American Academy of Neurology Annual Meeting. May 4-10, 2019; Philadelphia, PA.