Aquestive Therapeutics Inc. (AQST) Provides Business Update

Aquestive Therapeutics, Inc. (NASDAQ: AQST), a pharmaceutical company advancing medicines to solve patients' problems with current standards of care and provide transformative products to improve their lives, today provided a business update on its lead pipeline programs and base business results.

The Company recently received comments from the U.S. Food and Drug Administration (FDA) on its pivotal Phase 3 pharmacokinetic (PK) clinical study protocol for Anaphylm™ (epinephrine) Sublingual Film. Anaphylm is a polymer matrix-based epinephrine prodrug administered as a sublingual film that is applied under the tongue for the rapid delivery of epinephrine. Anaphylm comes in a highly portable package with the durability to withstand many of the norms of daily life.

In its comments, the FDA indicated that the Company’s proposed endpoints, sample size, and statistical analysis are reasonable. As anticipated, the FDA also reminded the Company that PK sustainability post-dosing (30 – 60 minutes) is an important factor and recommended using repeat-dose data to support PK sustainability. The Company has incorporated the FDA’s feedback into its upcoming studies and remains on track to start the pivotal Phase 3 study in the fourth quarter 2023. Table 1 below provides a list of anticipated clinical and regulatory activities to be completed prior to the planned New Drug Application (NDA) submission for Anaphylm in the second half of 2024.¹

¹Timeline does not include chemistry, manufacturing and controls (CMC), preclinical and human factors activities.
²Determination of whether a sublingual angioedema study is needed will occur in the Type C Meeting.

As previously announced, the Company’s NDA for approval of Libervant for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) in patients between two and five years of age was recently accepted for review by the FDA. Diastat^® (diazepam) Rectal Gel is the only approved treatment currently available to this patient population for this indication. Based on the latest information available to the Company, the review for this NDA remains on track and there are currently no outstanding information requests from the FDA. The NDA for Libervant has a PDUFA assignment date of April 28, 2024.

In addition, the Company’s base business continues to generate strong results. Based on preliminary third quarter 2023 results and binding supply purchase orders, the Company anticipates increasing current full-year 2023 guidance for revenue and decreasing full-year guidance for non-GAAP-adjusted EBITDA loss. Updated guidance will be provided during the Company’s third quarter earnings call in early November.

“We are pleased with the progress we have made in multiple areas of our business,” stated Daniel Barber, Chief Executive Officer of Aquestive. “Our base business remains on track to have double-digit growth in 2023, we are on track to bring Libervant to pediatric patients under the age of 6, if approved by the FDA for U.S. market access, and we remain on track to launch Anaphylm in 2025, if approved by the FDA. We believe these advancements are important steps forward for the Company and for the patients who may benefit from our products.”

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