Engage Therapeutics Presents Data from Part 1, Open-label Run-in of StATES Phase 2b Study of Staccato® Alprazolam at 2019 American Academy of Neurology Annual Meeting

May 8, 2019 / Portfolio News
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  • 8 patients with predictable generalized or focal epileptic seizures treated with Staccato alprazolam
  • 62.5% of patients respond with the cessation of seizure activity within two minutes of therapy administration and no recurrence of seizure activity within two hours
  • Feasibility of both self-administration or caregiver-administration demonstrated
  • Enrollment of additional 115 patients in double-blind, placebo-controlled part 2 of the study underway

SUMMIT, N.J., May 6, 2019 /PRNewswire/Engage Therapeutics, Inc., a clinical stage biopharmaceutical company developing a Rapid Epileptic Seizure Termination (REST) therapy for people with epilepsy who experience a predictable pattern of seizures presented data from part 1 of its Phase 2b StATES Study (Staccato Alprazolam Terminates Epileptic Seizures) at the 2019 American Academy of Neurology (AAN) Annual Meeting on Sunday in Philadelphia.

Eight adult patients with either generalized or focal predictable seizures were enrolled and treated with a single dose of Staccato alprazolam, an investigational therapy that combines FDA-approved Staccato delivery technology with alprazolam, an FDA approved benzodiazepine, in the open-label, run-in portion of this two-part study. Outcome measures were safety, efficacy, pharmacokinetics, and feasibility of self- or staff-caregiver-administration. Highlights from the AAN 2019 poster, entitled, “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,” include:

  • Five of eight (5/8) or 62.5% of patients responded with the cessation of seizure activity within two minutes of treatment administration and no recurrence of seizure activity within two hours;
  • Responses were observed irrespective of seizure type, BMI or concurrent use of CYP450 inducers;
  • No serious or severe adverse events reported;
  • Study procedures for the administration of 1 mg Staccato alprazolam to patients with predictable seizures were deemed feasible;
  • The double-blind, part 2 portion of the study has initiated and will randomize 115 patients to one of two active arms (1 mg or 2 mg Staccato alprazolam) vs placebo.

“These encouraging data not only illustrate a potential efficacy signal for Staccato alprazolam in rapidly arresting seizures in patients with epilepsy but also support the safety and feasibility of administering this unique therapy to patients just as they begin to experience seizure activity,” said Jaqueline French, MD, the study’s principal investigator and professor in the Department of Neurology at NYU Langone Health’s Comprehensive Epilepsy Center, and founder/director of the Epilepsy Study Consortium. “Currently available seizure therapies are limited to preventing additional seizures, leaving patients with epilepsy in constant fear. A rescue therapy capable of rapidly aborting a seizure could alleviate that fear in many people.”

About the StATES Study
The multi-center StATES Study (Staccato Alprazolam Terminates Epileptic Seizures -NCT03478982) is a two-part phase 2b trial designed to evaluate the safety, efficacy, and usability of Staccato alprazolam in subjects with epilepsy who have a predictable seizure pattern. In addition to the eight patients enrolled in the open-label, part 1 portion of the study, up to 115 patients will be enrolled in part 2 of the study, double-blinded and randomized to one of two active arms (1 mg or 2 mg Staccato Alprazolam) vs placebo. The primary endpoint of the study is cessation of seizure activity within two minutes of treatment administration and no recurrence within two hours based on clinical observation. Additional endpoints include safety and tolerability, the severity of the seizures compared to a subject’s prior seizures, utilization of rescue medication and incidence of adverse events. The StATES study is being conducted at approximately 50 trial sites in the United States, Australia, Canada, and Jamaica. Please visit www.epilepsyhealthstudy.com for more information about the StATES study and www.engagetherapeutics.com/study-locations/ for a list of study locations.

About Staccato Alprazolam

Staccato alprazolam is a single-use, investigational 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, easy-to-use, hand-held inhaler that delivers alprazolam with a single breath potentially providing a way for people with epilepsy and their caregivers to stop an ongoing seizure. The Staccato system rapidly vaporizes alprazolam to form an aerosol, with particle size designed for deep lung delivery, producing
a rapid, systemic effect. In a phase 2a proof-of-concept study, Staccato alprazolam demonstrated rapid reduction of seizure-like activity in a photosensitivity model.

About Engage Therapeutics, Inc.

Engage Therapeutics is developing Staccato alprazolam for the immediate cessation of active and acute epileptic seizures. The investigational product is in the Rapid Epileptic Seizure Termination (REST) category of products. Engage Therapeutics is based in Summit, N.J.

Media Contact

Mark Theeuwes – mtheeuwes@engagetherapeutics.com

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