8-K: Aquestive Therapeutics Advances Epinephrine Pipeline, Highlights Positive Clinical Data at Investor Day

Aquestive Therapeutics provided updates on its epinephrine product candidates, Anaphylm sublingual film and AQST-108 topical gel, at its virtual investor day.

Summary

• Aquestive Therapeutics hosted a virtual investor day to discuss its pipeline, focusing on Anaphylm (epinephrine) Sublingual Film and AQST-108 (epinephrine) Topical Gel.
• The company has completed enrollment in the oral allergy syndrome (OAS) challenge study for Anaphylm and expects topline data soon.
• A pre-NDA meeting request has been submitted to the FDA for Anaphylm, with an NDA submission targeted for Q1 2025 and a potential launch at the end of 2025 or early 2026.
• Aquestive is developing AQST-108 for Alopecia areata, having completed a first-in-human study showing no serious adverse events or topical adverse events.
• The company plans to hold a pre-IND meeting with the FDA in Q1 2025 and commence a Phase 2 study in the second half of 2025.
• The Adrenaverse platform is designed to control absorption and conversion rates of epinephrine, enabling local delivery across mucosal surfaces and skin.
• Anaphylm has shown consistent pharmacokinetics with a time to peak drug concentration (Tmax) of 12-15 minutes and onset of pharmacodynamics (PD) effects within 2-5 minutes.
• AQST-108 demonstrated significant local absorption in rat ears for over one hour without systemic exposure.
• Non-clinical studies of AQST-108 in minipigs showed high exposure in both the dermis and epidermis lasting over 6 hours with no systemic exposure.
• The company estimates a potential peak annual net sales of $500M for AQST-108.
• Aquestive expects 2024 total revenues of approximately $57 to $60 million and a non-GAAP adjusted EBITDA loss of approximately $20 to $23 million.

Sentiment

Score: 7

Explanation: The document presents a positive outlook with advancements in the pipeline, but also acknowledges the risks and challenges associated with drug development and commercialization. The company is making progress, but there are still uncertainties.

Positives

• Anaphylm has completed enrollment in its oral allergy syndrome (OAS) challenge study, moving closer to potential market launch.
• The pre-NDA meeting request for Anaphylm has been submitted to the FDA, indicating progress in the regulatory process.
• AQST-108 has shown promising results in its first-in-human study with no serious adverse events or topical adverse events.
• The Adrenaverse platform has demonstrated the ability to control absorption and conversion rates of epinephrine.
• Anaphylm has shown rapid absorption and onset of action, comparable to standard of care epinephrine auto-injectors.
• AQST-108 has demonstrated significant local absorption without systemic exposure, potentially reducing side effects.
• The company has a clear plan for the clinical development of both Anaphylm and AQST-108.
• Aquestive has a diversified pipeline with multiple product candidates in development.
• The company has a strong intellectual property portfolio with over 150 patents worldwide.
• Aquestive has a cash runway extended into 2026.

Negatives

• The company is still in the clinical development phase for both Anaphylm and AQST-108, with no guarantee of FDA approval.
• The company is currently operating at a loss, with an expected non-GAAP adjusted EBITDA loss of $20 to $23 million for 2024.
• The company faces competition from other pharmaceutical companies developing treatments for anaphylaxis and Alopecia areata.
• The company's revenue is heavily reliant on Suboxone, which is a sunsetting product.
• There is a risk of delays in the regulatory approval process for both Anaphylm and AQST-108.
• The company may need to raise additional capital to fund its clinical development and commercialization activities.
• There is a risk of unexpected patent developments that could impact the company's intellectual property.
• The company faces risks related to claims and legal proceedings.

Risks

• There are risks associated with the development of Anaphylm and AQST-108, including potential delays or changes to the timing, cost, and success of clinical trials.
• The company may not receive FDA approval for Anaphylm or AQST-108.
• There is a risk that the company may not be able to generate sufficient clinical data for approval of its product candidates.
• The company may not be able to address the FDA's comments on its clinical trials.
• There is a risk of the success of competing products.
• The company may not be able to overcome the orphan drug market exclusivity for Libervant.
• There are risks and uncertainties inherent in commercializing new products.
• The company may not have sufficient capital and cash resources to fund its operations.
• There is a risk of eroding market share for Suboxone.
• The company faces risks related to intellectual property rights and infringement claims.
• There are risks related to disruptions in information technology networks and systems, including cybersecurity attacks.
• The company faces risks related to inflation and rising interest rates.
• There are risks related to the impact of the COVID-19 global pandemic and other pandemic diseases on the business.

Future Outlook

Aquestive anticipates an NDA submission for Anaphylm in Q1 2025 and a potential launch at the end of 2025 or early 2026. The company also plans to commence a Phase 2 study for AQST-108 in the second half of 2025. The company expects to receive full FDA approval for Libervant for patients ages six and up in January 2027.

Management Comments

• Daniel Barber, President and CEO, stated that the pipeline is progressing and they are excited about the next chapter for growth.
• Daniel Barber noted that they recently submitted their pre-NDA meeting request to the FDA for Anaphylm and are on track to report topline data from their oral allergy challenge study.
• Carl Kraus, MD, Chief Medical Officer, mentioned that the Adrenaverse platform has demonstrated the ability to harness the therapeutic potential of epinephrine through highly differentiated prodrug formulations.
• Carl Kraus stated that AQST-108 for the treatment of Alopecia areata is a natural next step in the evolution of the Adrenaverse platform.

Industry Context

The announcement highlights Aquestive's efforts to innovate in the epinephrine delivery space, addressing unmet needs in anaphylaxis and Alopecia areata. The company is leveraging its proprietary PharmFilm and Adrenaverse technologies to develop novel products that could potentially disrupt the market. The focus on topical delivery for Alopecia areata is a unique approach compared to existing systemic treatments.

Comparison to Industry Standards

• Anaphylm's rapid absorption and onset of action are comparable to epinephrine auto-injectors like EpiPen and Auvi-Q, but with the advantage of being an oral film.
• The development of AQST-108 as a topical treatment for Alopecia areata is a novel approach compared to existing treatments like JAK inhibitors, which are systemic and have known side effects.
• The company's focus on local delivery of epinephrine through the Adrenaverse platform is a differentiated approach compared to traditional epinephrine delivery methods.
• The company's estimated peak annual net sales of $500M for AQST-108 is a significant potential market opportunity, especially considering the high cost of existing JAK inhibitors for Alopecia areata.
• The company's diversified pipeline and proprietary technologies position it as a potential leader in the oral thin film and epinephrine delivery space, with a focus on addressing unmet needs in various therapeutic areas.

Stakeholder Impact

• Shareholders may benefit from the potential success of Anaphylm and AQST-108.
• Patients with anaphylaxis and Alopecia areata may benefit from new treatment options.
• Employees may benefit from the company's growth and success.
• Customers may benefit from the company's innovative products.
• Suppliers may benefit from the company's increased demand for raw materials.
• Creditors may benefit from the company's improved financial performance.

Next Steps

• Complete the oral allergy syndrome (OAS) challenge study for Anaphylm.
• Hold a pre-NDA meeting with the FDA for Anaphylm.
• File an NDA with the FDA for Anaphylm.
• Hold a pre-IND meeting with the FDA for AQST-108.
• Commence a Phase 2 study for AQST-108.
• Continue to develop and commercialize Libervant.
• Continue to advance the Adrenaverse platform.

Key Dates

Keywords

Anaphylm, AQST-108, epinephrine, Alopecia areata, Adrenaverse, sublingual film, topical gel, clinical trials, FDA, prodrug, PharmFilm, regulatory approval