8-K: Aquestive Therapeutics Announces Q1 2025 Financial Results and Anaphylm NDA Submission

Aquestive Therapeutics reports its Q1 2025 financial results, highlights the NDA submission for Anaphylm, and provides a business update.

Summary

• Aquestive Therapeutics announced its first quarter 2025 financial results and provided a business update.
• The company submitted the NDA for Anaphylm (epinephrine) sublingual film, a potential first-in-class treatment for severe allergic reactions.
• FDA acceptance of the NDA is expected in the second quarter of 2025, with a potential U.S. launch in the first quarter of 2026, pending FDA approval.
• The company is de-emphasizing other activities, such as the advancement of AQST-108, and will not appeal the court decision on Libervant to focus resources on the Anaphylm launch.
• Libervant scripts for patients aged two to five years old continued to grow during the first quarter of 2025.
• However, following a recent court decision, the FDA revised Libervants full approvaloriginally granted in April 2024to a tentative approval status.
• As a result of the change in regulatory status, sales and marketing activities for the product have paused.
• The company plans to provide patient access to Libervant in the U.S. in 2027, upon the expiration of the orphan drug market exclusivity granted by the FDA to an approved intranasal product of another company, or sooner if market access is granted by the FDA.
• Total revenues decreased to $8.7 million in the first quarter 2025 from $12.1 million in the first quarter 2024.
• The net loss for the first quarter 2025 was $22.9 million, or $0.24 for both basic and diluted loss per share, compared to the net loss for the first quarter 2024 of $12.8 million, or $0.17 for both basic and diluted loss per share.
• Cash and cash equivalents were $68.7 million as of March 31, 2025.
• The company has revised its full-year 2025 financial guidance, expecting total revenues of $44 to $50 million and a non-GAAP adjusted EBITDA loss of $47 to $51 million.

Sentiment

Score: 5

Explanation: The sentiment is neutral. While the NDA submission for Anaphylm is a positive milestone, the company faces challenges with Libervant and reported a larger net loss with decreased revenues. The revised financial guidance also tempers enthusiasm.

Positives

• The NDA submission for Anaphylm represents a significant milestone for Aquestive.
• Positive topline results from the pediatric clinical trial for Anaphylm support a label aligned with the 0.3 mg epinephrine autoinjector.
• Launch preparations for Anaphylm are underway in advance of a potential U.S. commercial introduction in the first half of 2026.
• The company has a strong balance sheet with $68.7 million in cash and cash equivalents as of March 31, 2025.
• Manufacturing operations continue to generate cash flow.

Negatives

• The FDA revised Libervant's full approval to a tentative approval status, pausing sales and marketing activities.
• Total revenues decreased to $8.7 million in the first quarter 2025 from $12.1 million in the first quarter 2024.
• The net loss for the first quarter 2025 was $22.9 million, compared to a net loss of $12.8 million for the first quarter 2024.
• The company revised its full-year 2025 financial guidance downward.

Risks

• The FDA may not accept the NDA for Anaphylm or may require additional clinical studies.
• Anaphylm may not receive FDA approval or may face delays in the approval process.
• The company may not be able to successfully commercialize Anaphylm.
• Libervant's market access is restricted due to orphan drug market exclusivity granted to another company.
• The company may face challenges in manufacturing and supplying its products.
• The company may face increased competition from other products in the market.
• The company's financial performance may be affected by economic and market conditions.

Future Outlook

Aquestive expects total revenues of approximately $44-$50 million and a non-GAAP adjusted EBITDA loss of approximately $47-$51 million for 2025.

Management Comments

• Daniel Barber, President and Chief Executive Officer of Aquestive, stated that the NDA submission for Anaphylm represents a critical step toward delivering the first oral, non-invasive epinephrine treatment.
• Management believes that they are the potential best-in-class epinephrine therapy in a multi-billion dollar growing market.

Industry Context

Aquestive is positioning Anaphylm as a potential alternative to existing epinephrine delivery methods, such as autoinjectors, in a growing market for allergy treatments. The company is also developing AQST-108 for alopecia areata, targeting a market currently dominated by JAK inhibitors.

Comparison to Industry Standards

• The existing therapies for alopecia areata are janus kinase (or JAK) inhibitors.
• These systemic treatments with known side effects come with a 'black box' warning and are expensive for patients.
• Even with these limitations, the current estimated market opportunity for JAK inhibitors is over one billion U.S. dollars.
• Aquestive is developing Libervant as an alternative to the device-based products currently available for patients with refractory epilepsy, including a rectal gel and nasal spray products.

Stakeholder Impact

• Shareholders: The NDA submission for Anaphylm could positively impact shareholder value if the product is approved and successfully commercialized.
• Patients: Anaphylm could provide a new treatment option for patients with severe allergic reactions.
• Employees: The company's focus on Anaphylm could impact employee roles and responsibilities.
• Customers: The company's manufacturing business supports the production of licensed products for other companies.

Next Steps

• Aquestive anticipates receipt of FDA's determination of acceptance of their NDA submission in the second quarter of 2025.
• The company is actively preparing for a potential U.S. launch of Anaphylm in the first quarter of 2026, if approved by the FDA.
• Following NDA acceptance, if granted by the FDA, Aquestive plans to initiate regulatory submissions in key international markets, including Europe, the United Kingdom, and Canada.
• Aquestive is currently preparing for a potential Advisory Committee meeting during the FDA review process.
• Aquestive plans to initiate its Phase 2a clinical trial of AQST-108 in the first half of 2026, after the launch of Anaphylm.
• The Company plans to provide patient access to Libervant in the U.S. in 2027, upon the expiration of the orphan drug market exclusivity granted by the FDA to an approved intranasal product of another company, or sooner if market access is granted by the FDA.

Key Dates

Keywords

Anaphylm, Aquestive Therapeutics, Epinephrine, Sublingual Film, NDA Submission, Financial Results, Libervant, AQST-108, Alopecia Areata, Pharmaceuticals