Aquestive touts PhII data — and speed — in potential EpiPen replacement candidate

A New Jersey biotech specializing in drug delivery via oral film is reporting new Phase II data, setting a countdown to have it before the FDA by end of 2023.

Aquestive Therapeutics announced new data Tuesday for its candidate AQST-109, an oral film using a prodrug of epinephrine to treat anaphylactic shock from an allergic reaction.

Per the biotech, a readout of the EPIPHAST II trial was to compare one dose of AQST-109 — basically a patch — to a dose of an EpiPen at 0.3 mg, and an intramuscular injection of 0.3mg of epinephrine that wasn’t EpiPen.

In a single dose test, the median Tmax (the time it takes for a drug to reach maximum concentration in the blood after administration) was 12 minutes for AQST-109, compared to 22.5 minutes for EpiPen and 45 minutes for the generic injection.

Ken Truitt

Aquestive CMO Ken Truitt tells Endpoints News the “standard” way of delivering epinephrine — through the muscle — takes time to actually have a therapeutic effect after entering the bloodstream and reaching organs.

“If you compare us to the tried-and-true standard of just regular old IM (intramuscular) epinephrine, you can see some room for improvement in the old standard profile. It can get into the body, but then there’s a delay time before they really start getting into the bloodstream and start having pharmacodynamic effects,” Truitt added.

Aquestive said the results for AQST-109 were “in line with expectations,” reporting no major adverse events. As for why a patch under the tongue, the CMO said that area is highly vascular and allows for faster absorption of the pro drug.

Shares of $AQST went up about 5% for the near penny stock player, currently floating around $1.18 each.

CEO Daniel Barber added that now with the data in hand, the biotech has already submitted the info to the FDA, and he told Endpoints the company will be meeting with the federal agency before the end of the year. The end goal so far, according to Barber, is to get three required studies up and running and completed over the next year, ready to file by the end of 2023.

Those three studies, according to the CEO, are a pivotal study, a small pediatric study, and a human factor study.

This is one of the more recent developments for the biotech under the new CEO, who replaced former chief executive Keith Kendall several months ago after seven and a half years at the helm. While Aquestive said that Kendall would remain a consultant through the end of 2022, the company didn’t give an exact reason for the departure at the time, only noting that it was “unrelated” to strategy, operations nor finances.

AQST-109 is not the only film in the biotech’s repertoire: Other films include opioid addiction medicine combo buprenorphine/naloxone or Parkinson’s symptom drug apomorphine, used to treat “off episodes” in PD patients.

Aquestive has also been down a long road for its oral film to treat seizures. Originally submitted in late 2019, the drug candidate was first rejected in September 2020 due to what the FDA said was lower-than-desired exposure levels in certain weight groups, per Aquestive.

After the company resubmitted its application in 2021, receiving a PDUFA date for that December which was punted again, it finally received tentative approval in the US just last month.

“Libervant has met all required quality, safety, and efficacy standards for approval but, due to an existing FDA regulatory grant of orphan drug market exclusivity for Valtoco, a diazepam nasal spray product, Libervant is not yet eligible for marketing in the United States,” Aquestive said in a statement.

The FDA cannot give final approval to Libervant until the orphan drug market exclusivity period is over, either by naturally expiring or through other options like a court order. The company expects to launch the product in the US market by 2027, according to Aquestive’s website.

In the meantime, Aquestive is looking for a marketing partner to take Libervant outside the US.

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