-- Staccato alprazolam achieved statistically significant response rate in both treatment arms compared to placebo --
-- Average time to seizure cessation was 30 seconds --
-- Staccato alprazolam generally well-tolerated --
-- Data to be presented at American Academy of Neurology on April 26 --

SUMMIT, N.J., March 12, 2020 (GLOBE NEWSWIRE) -- Engage Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing an orally inhaled therapy designed to terminate an active epileptic seizure, today announced that its Phase 2b StATES study of Staccato® alprazolam met its primary endpoint which was proportion of responders achieving cessation of seizure activity within two minutes of treatment administration and no recurrence within two hours.

“With statistically significant and clinically meaningful Phase 2 results in this randomized, placebo-controlled trial, Staccato alprazolam has demonstrated the ability to rapidly terminate seizures in patients with epilepsy in two minutes or less and prevent recurrence of seizure within two hours,” said Jaqueline French, MD, the study’s principal investigator and professor of neurology and co-director of epilepsy research and epilepsy clinical trials at NYU Langone Health’s Comprehensive Epilepsy Center, and founder/director of the Epilepsy Study Consortium. “We are now one step closer to bringing to patients an EpiPen®-like rescue treatment that works fast enough to terminate an active seizure episode. We look forward to initiating a Phase 3 study in the outpatient setting later this year.”

The multi-center StATES Study (Staccato Alprazolam Terminates Epileptic Seizures - NCT03478982) was designed to evaluate the safety, efficacy and usability of Staccato alprazolam in adults living with focal or generalized epilepsy who have a predictable seizure pattern in an in-patient environment. Data from the trial will be presented during the American Academy of Neurology (AAN) Annual Meeting in Toronto on Sunday, April 26, at noon.

There were 116 patients enrolled in the double-blind portion of the trial and randomized to one of two treatment arms (1mg of Staccato alprazolam, N=38 or 2mg N=38) or placebo (N=40). In the two treatment arms of the trial, 50 of 76 total patients achieved the primary endpoint of seizure cessation with no recurrence:

 Placebo1 mg Staccato alprazolam2 mg Staccato alprazolamCombined treatment arms
Patients40383876
Responders17 (42.5%)25 (65.8%)25 (65.8%)50 (65.8%)
P value 0.03920.03920.0158

In assessing the primary endpoint of seizure activity cessation within two minutes, Staccato alprazolam showed rapid onset of action by ceasing seizure activity in approximately 30 seconds, on average.

Staccato alprazolam was generally well-tolerated in both treatment arms, as the majority of adverse events were mild in nature and there were no treatment-related serious adverse events. The most common adverse event was somnolence which was reported by 11 (14.5 percent) of the 76 patients in the treatment arms. Other adverse events included cough (14.5 percent), dysgeusia or a distortion of taste (13.2 percent), dizziness (5.3 percent), sedation (2.6 percent) and throat irritation (2.6 percent).

“As a parent, I have personally experienced the need for a product that can work fast enough to terminate an active seizure when it occurs,” said Gregory T. Mayes, Founder and CEO of Engage Therapeutics. “We believe these data from the StATES study show what we hoped could be done – can be done. As we move the clinical development forward, we want to see Staccato alprazolam become a tool that patients with epilepsy can use to rapidly stop a seizure.”

About Staccato Alprazolam
Staccato alprazolam is an investigational drug designed to be used as a single-use, epileptic seizure rescue therapy that combines the Staccato delivery technology, which is currently used in a U.S. Food and Drug Administration (FDA) approved product, with alprazolam, an FDA-approved benzodiazepine. It is a small, hand-held inhaler device designed for easy delivery of alprazolam with a single normal breath potentially providing a way for people with epilepsy and their caregivers to stop an active seizure. The Staccato system rapidly vaporizes alprazolam to form an aerosol, with particle size designed for deep lung delivery to produce a rapid, systemic effect.

About Engage Therapeutics, Inc.
Engage Therapeutics is developing Staccato alprazolam for the immediate termination of an active epileptic seizure, or Rapid Epileptic Seizure Termination (REST). The Company anticipates advancing Staccato alprazolam to a Phase 3 registration trial in 2020. Engage Therapeutics is based in Summit, N.J. For additional information please see www.engagetherapeutics.com.

Contact:
Mark Theeuwes
mtheeuwes@engagetherapeutics.com