The Medical Need

From its headquarters in Summit, New Jersey, Engage Therapeutics is developing Staccato® alprazolam, an easy to use, hand-held inhaler that could be the first rescue treatment with the potential to stop epileptic seizure activity dead in its tracks.  Epilepsy is a condition that causes seizures and is the fourth most common neurological problem in the world according to the Epilepsy Foundation. Approximately three million patients in the United States are living with epilepsy, and one-third of those patients have uncontrolled seizures because no available treatment works for them. Twenty percent of those patients have a seizure pattern that they can predict and need a rescue treatment to stop on-going seizure activity before their seizure progresses.

Clinical Trials

In a phase 2a proof of concept trial, Staccato alprazolam aborted seizure-like activity in less than two minutes. The therapy, which has entered a phase 2b study, combines the FDA-approved Staccato delivery technology with alprazolam, a well-known benzodiazepine that has potent and effective anti-epileptic properties, to rapidly terminate seizure activity once it has begun. If successful, this would be the first product approved for such an indication.

Regulatory Pathway

Because the active ingredient and the inhaler are already approved for different indications, Engage Therapeutics is using the 505(b)(2) regulatory pathway to develop and seek approval for Staccato alprazolam. This should result in a less expensive and time-consuming development program.  In a phase 2a study, Staccato alprazolam demonstrated a reduction in seizure-like activity in a photosensitivity model.

How It Works

Alprazolam, which has decades of safety data supporting its continued commercial availability, has shown anti-epileptic activity, and the Staccato system, achieves an unprecedented speed of onset. When patients draw a normal breath through the Staccato system, the substrate surface instantaneously heats to create a condensation aerosol. The patient inhales particles ideally sized for systemic delivery of excipient free drug down into the narrower, more vascularized tissue of the deep lung, allowing fast and more complete absorption into the bloodstream.

The Staccato system is the only technology on the market that non-invasively delivers a drug as quickly as an IV route of administration and has a bioavailability greater than ninety percent (90%). This innovation in aerosol delivery has the potential to revolutionize the treatment of epileptic seizure activity by stopping the seizure almost as soon as it starts.

Financing

Engage Therapeutics closed on a $23 million Series A financing round in September, 2017. TPG Biotech led the Series A financing, and was joined by Adage Capital Management and Lumira Capital. These established cross-over and venture capital firms have track records of investing in life science companies that have produced truly innovative products based on transformative technologies.

1. Should Engage focus on one project (epilepsy) or create optionality with multiple shots on goal (other possible uses like acute panic disorder)?
2. How should you select outsourced providers like a CRO whose success you depend upon?
3. What are the keys to success when starting a new emerging life science venture?

6:00 – Dinner and Drinks
7:00 – Greg Mayes President, CEO and Founder of Engage, will deliver the company's "elevator" pitch to the group

7:20 – The three questions for discussion listed above will be addressed

8:00 – Q&A Session

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Questions or Coments?

Contact: David S. Blatteis

david@rxpcci.com | (201) 390-1782