Aquestive Therapeutics (AQST) Investor Day 2024 summary

Strategic vision and pipeline outlook

• The Adrenoverse platform is expanding with over 20 epinephrine prodrugs, targeting anaphylaxis, immunomodulatory, and dermatological indications, with Anaphylm and AQST-108 as lead assets.

• 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 from 2028 onward, pending FDA approvals.

• Combined peak annual sales potential for Anaphylm, AQST-108, and Libervant is estimated at over $1.5 billion, with AQST-108 alone projected at $250M–$750M+ depending on market scenario.

• Commercial infrastructure is being built to support these launches, leveraging existing manufacturing capabilities with minimal additional investment.

• Dermatologists are a key prescriber group for AQST-108, with an estimated 11,000 specialists in the US targeted by the commercial strategy.

Scientific and clinical rationale

• Adrenoverse enables controlled, local delivery of epinephrine, overcoming barriers of poor absorption and systemic exposure.

• Preclinical studies confirm AQST-108's sustained local exposure, immunomodulation, and minimal systemic absorption in animal and human skin models.

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

• The platform's mechanism is distinct from JAK inhibitors, aiming to restore immune privilege via IL-10 upregulation and local immune suppression.

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

Development plans and next steps

• AQST-108 topical gel is advancing toward a Phase 2 clinical study for mild to moderate Alopecia areata, with a pre-IND meeting planned for Q4 2024 and endpoints including hair regrowth and digital imagery.

• Initial clinical studies confirm safety, lack of systemic exposure, and no serious or topical adverse events at selected doses.

• AQST-108 will be tested as monotherapy before considering combination with JAK inhibitors, aiming to avoid systemic side effects.

• Manufacturing and formulation are aligned with existing capabilities, supporting scalability and cost efficiency.

• Future expansion into other dermatologic and mucosal autoimmune conditions is planned, with potential follow-on indications including vitiligo, atopic dermatitis, and acne, while chronic spontaneous urticaria is deprioritized.