Aquestive Therapeutics (AQST) Investor Day 2024 summary

Strategic Vision and Pipeline Outlook

• Focus on expanding the Adrenoverse epinephrine prodrug platform, with Anaphylm as the lead candidate and AQST-108 topical gel advancing for alopecia areata and other dermatological indications.

• Anticipated sequential launches: Libervant for ages 2–5 in 2024, Anaphylm in 2026, Libervant for ages 6–11 and 12+ in 2027, and AQST-108 after 2028, pending FDA approvals.

• The Adrenoverse platform features over 20 epinephrine prodrugs, enabling tailored drug delivery for allergy, topical, and other indications.

• Commercial infrastructure is being built to support these launches, leveraging existing manufacturing capabilities for scalability.

• Urticaria is deprioritized due to limited market opportunity, with focus shifting to areas with higher unmet need.

Scientific Rationale and Preclinical Validation

• Adrenoverse enables controlled, localized delivery of epinephrine, overcoming previous absorption and vasoconstriction challenges.

• Preclinical studies in rats, mini pigs, and human skin confirm AQST-108 achieves sustained local exposure and immunomodulation without systemic absorption.

• AQST-108 demonstrated suppression of pro-inflammatory cytokines and inactivation of NK cells, supporting its potential in autoimmune skin diseases.

• The mechanism leverages epinephrine’s ability to induce IL-10 and suppress inflammatory cytokines, distinct from JAK inhibitors.

• Patent portfolio and applications could protect the platform through 2046 across multiple indications.

Clinical Development and Future Plans

• AQST-108’s initial clinical focus is on alopecia areata, aiming to restore immune privilege in hair follicles via local immunomodulation.

• AQST-108 is advancing toward a Phase 2 clinical study for mild to moderate alopecia areata, with a pre-IND meeting planned for Q4 2024.

• Early clinical studies confirm safety, tolerability, and lack of systemic exposure at selected doses.

• Phase IIa trials will prioritize monotherapy in mild to moderate alopecia areata, with plans to expand to severe cases based on data.

• Planned Phase 2 study for AQST-108 in alopecia areata will assess safety and efficacy over 12–24 weeks, with endpoints including hair regrowth and digital imagery.