Engage Therapeutics announced that data from a phase 2a study of Staccato alprazolam to suppress seizures in patients with epilepsy were published online ahead of print on July 7 in the peer reviewed journal Epilepsia. This proof-of-concept study demonstrated that Staccato alprazolam delivered the drug deep into to the lung and rapidly suppressed seizure or epileptiform activity in five photosensitive patients with epilepsy.
This proof-of-concept study demonstrated that three doses of Staccato alprazolam (0.5 mg, 1.0 mg, and 2.0 mg) rapidly suppressed epileptic activity in photosensitive patients with epilepsy. The primary endpoint of the study was the change in standardized photosensitivity range (SPR) in patients receiving each dose of Staccato alprazolam. Staccato alprazolam reduced epileptiform activity as measured by SPR at two minutes; the effect was sustained through four hours for the 0.5 mg dose and six hours for the 1.0 and 2.0 mg doses. The magnitude and duration of sedation and sleepiness were dose related and more prominent with higher doses. Peak plasma concentrations following administration were achieved following inhalation within minutes, and rapid onset of pharmacodynamic effects of somnolence and sedation were observed. Staccato alprazolam was generally well tolerated with a safety profile similar to what has been reported for oral alprazolam or Staccato alprazolam for other indications. There were no severe or serious adverse events. Four of five subjects experienced at least one adverse event during the study, but all were mild or moderate in intensity.
Enrollment is currently underway in the phase 2b StATES Study (Staccato Alprazolam Terminates Epileptic Seizures). The double-blind, randomized, parallel group, dose-ranging study is designed to evaluate the safety, efficacy and usability of Staccato alprazolam in 123 subjects with epilepsy who have a predictable seizure pattern. The StATES study is being conducted at approximately 50 trial sites in the United States, Australia and Jamaica.
About Staccato alprazolam
Staccato alprazolam is a single-use, investigational epileptic seizure rescue therapy that combines Staccato delivery technology with alprazolam, both of which have been approved separately for unrelated indications by the U.S. Food and Drug Administration (FDA). Alprazolam is a well-known and highly characterized benzodiazepine for the treatment of anxiety disorders. The Staccato® system aerosolizes a drug and, via inhalation, delivers it deep into the lung for rapid systemic exposure.