CEO, Engage Therapeutics, Summit, NJ, United States
Staccato Alprazolam (STAP-001) is a single-use, hand-held, drug-device combination product that provides rapid systemic delivery by inhalation of a thermally generated aerosol of alprazolam. STAP-001 represents a new dosage form for alprazolam and is based on the proprietary Staccato delivery system developed by Alexza Pharmaceuticals. STAP-001 is delivered orally to the deep lung for systemic delivery, and the pharmacokinetics are similar to what is achieved with drug delivery by IV injection.
The Staccato delivery system is user-friendly and easy to use. Device actuation, aerosol formation, and delivery of the aerosolized drug to the deep lung are all accomplished with a single, normal breath by the subject. Achievement of peak plasma levels within 2 minutes via a simple, user-friendly delivery system makes STAP-001 ideal for the acute treatment of seizures.
STAP-001 was assessed in a phase 2a study using the Intermittent Photic Stimulation (IPS) model in subjects with photosensitive epilepsy. This study was conducted to establish proof of concept for STAP-001 as an acute treatment for seizures and to establish the dose range for subsequent clinical trials in epilepsy. Five patients were enrolled and completed all treatment arms (0.5mg, 1 mg, 2 mg of STAP-001, and two doses of placebo). All doses of STAP-001 decreased the mean standardized photosensitivity range (SPR), with maximal or near-maximal effect occurring by 2 minutes post dose, and the reduction in SPR was observed out to the 6 hours of observation. Sedation was the most common adverse event. It was dose related and most common with the 2mg dose of STAP-001. Overall the STAP-001 treatment was well tolerated and no serious adverse events were reported.
The next study in the STAP-001 clinical program for epilepsy is a phase 2b study (StATES = Staccato Alprazolam Terminates Epileptic Seizures) that will be conducted in an in-patient setting. The StATES study will enroll adult subjects with predictable patterns of seizure episodes. The patients will be randomized to 0.5 or 1 mg of Staccato Alprazolam or to placebo. One predictable seizure episode per patient will be treated. The primary endpoint is proportion of responders defined as seizure episode cessation within 2 minutes and no seizure recurrence in 2 hours. The study has just been initiated and will be conducted in 40 centers across the US.