Aquestive Therapeutics Advances Anaphylm and AQST-108, Reports Q3 2024 Financial Results
核心洞察
Aquestive Therapeutics plans to submit Anaphylm™ (epinephrine) Sublingual Film for FDA approval after completing adult supportive studies, with a pre-NDA meeting scheduled for Q4 2024.
AQST-108 (epinephrine) Topical Gel, a novel prodrug for alopecia areata (搜索), is progressing towards a Phase 2a study in 2025, pending FDA alignment after a pre-IND meeting in Q4 2024.
Libervant (搜索)® (diazepam) Buccal Film is now available through retail channels for patients aged 2-5, supported by a dedicated sales force and nationwide reimbursement coverage.
Aquestive Therapeutics, Inc. (NASDAQ:AQST) has announced its third-quarter financial results for 2024, alongside key updates on its pharmaceutical pipeline. The company is focused on advancing medicines through innovative science and delivery technologies to improve patient lives.
Anaphylm™ (epinephrine) Sublingual Film Development
Aquestive is progressing with Anaphylm™, an orally delivered epinephrine product, as an alternative to existing epinephrine auto-injectors like EpiPen for severe allergic reactions, including anaphylaxis (搜索). The company reported positive topline data from the OASIS study in October 2024, meeting both primary and secondary endpoints. The study demonstrated that Anaphylm maintained its pharmacokinetic (PK) and pharmacodynamic (PD) profile during allergen-induced oral physiological changes. Rapid symptom resolution was observed as early as two minutes after administration, with a median time to complete symptom resolution of twelve minutes, compared to seventy-four minutes at screening baseline. The safety profile was favorable, with only mild to moderate adverse events that resolved without medical intervention.
At the American College of Allergy Asthma and Immunology (ACAAI) 2024 Annual Meeting, Aquestive presented data showing Anaphylm's consistent PK and PD profile regardless of variable placement or intraoral movement. The analysis indicated that initial placement or subsequent movement of the sublingual film had no impact on epinephrine PK or PD comparability to epinephrine auto-injectors.
A clinically focused pre-NDA meeting with the FDA is scheduled for the fourth quarter of 2024, and the company anticipates a full product launch in the first quarter of 2026, pending FDA approval. This timeline is based on commencing a pediatric study in subjects weighing 30 kgs and above in the fourth quarter of 2024 and completing an NDA submission with the FDA in the first quarter of 2025.
AQST-108 (epinephrine) Topical Gel for Alopecia Areata
Aquestive is also developing AQST-108, a topically delivered adrenergic agonist prodrug, as a potential treatment for alopecia areata (搜索). The company plans to commence a Phase 2a study in the second quarter of 2025, following a pre-IND meeting with the FDA in the fourth quarter of 2024.
Alopecia areata (搜索) affects an estimated 6.7 million people in the United States, with 43% considered severe. Current treatments, such as janus kinase (JAK) inhibitors, have limitations, including systemic side effects and high costs. AQST-108 demonstrated no serious adverse events or topical adverse events in a Phase 1 clinical study. If approved, AQST-108 has the potential to capture significant market share due to its topical application and potentially reduced systemic side effects.
Libervant® (diazepam) Buccal Film Expansion
Libervant (搜索)® (diazepam) Buccal Film, approved by the FDA in April 2024, is available for the acute treatment of seizure clusters (搜索) in patients aged two to five years. Aquestive has expanded the launch of Libervant with a dedicated sales team and nationwide retail distribution capabilities. The company expects to file for approval of Libervant for the treatment of epilepsy (搜索) patients between six to twelve years of age prior to the expiration of the orphan drug market exclusivity block in January 2027.
Financial Highlights
Total revenues for the third quarter of 2024 increased to $13.5 million from $13.0 million in the third quarter of 2023. Research and development expenses increased to $5.3 million, driven by clinical trial costs for the Anaphylm development program. The company's net loss for the third quarter of 2024 was $11.5 million, or $0.13 per share, compared to a net loss of $2.0 million, or $0.03 per share, for the third quarter of 2023. Cash and cash equivalents were $77.9 million as of September 30, 2024.
Aquestive's full-year 2024 financial guidance includes total revenue of $57 to $60 million and a non-GAAP adjusted EBITDA loss of $20 to $23 million.
"Our innovative epinephrine prodrug platform remains the cornerstone of our development strategy," said Daniel Barber, President and Chief Executive Officer of Aquestive. "We've reached a significant milestone with the clinical development program for Anaphylm... Moreover, AQST-108, our promising pipeline candidate, is progressing towards a pre-IND meeting this quarter, setting the stage for a potential Phase 2a study in alopecia areata (搜索) next year, subject to FDA alignment."