8-K: Aquestive Therapeutics Announces Positive Phase 3 Results for Anaphylm and FDA Meeting Outcomes

Aquestive Therapeutics reports positive topline data from its Phase 3 study of Anaphylm, an epinephrine sublingual film, and successful completion of an FDA Type C meeting.

Summary

• Aquestive Therapeutics has released positive results from its Phase 3 clinical trial for Anaphylm, a sublingual epinephrine film designed to treat severe allergic reactions.
• The study compared Anaphylm to epinephrine injections and autoinjectors like EpiPen and Auvi-Q in healthy adults.
• In single-dose studies, Anaphylm achieved a maximum concentration (Cmax) of 470 pg/mL, comparable to EpiPen (469 pg/mL) and Auvi-Q (521 pg/mL).
• Anaphylm showed a median time to maximum concentration (Tmax) of 12 minutes, faster than EpiPen (20 minutes), Auvi-Q (30 minutes), and Adrenalin (50 minutes).
• The repeat-dose study showed that Anaphylm maintained epinephrine plasma concentrations equal to or greater than existing injection products for up to two hours.
• The median Tmax after the second dose was 10 minutes, and the 100 pg/mL threshold was reached within 5 minutes.
• A Type C meeting with the FDA addressed previous concerns, including product hold time, emesis, and mouth conditions.
• The FDA recommended an allergen study to assess the impact of oral conditions on Anaphylm's performance, which will be conducted in the second quarter of 2024.
• The company plans to file an NDA with the FDA before the end of 2024, with a pre-NDA meeting targeted for the second half of 2024.

Sentiment

Score: 8

Explanation: The document presents very positive clinical trial results and a successful FDA meeting, indicating a strong likelihood of regulatory approval and market success for Anaphylm. The sentiment is very positive, with some caution due to the inherent risks in drug development.

Positives

• Anaphylm demonstrated comparable pharmacokinetic performance to existing epinephrine autoinjectors.
• The sublingual film showed a faster absorption rate compared to autoinjectors and manual injections.
• The repeat-dose study showed sustained epinephrine levels, which is crucial for managing anaphylaxis.
• The FDA Type C meeting provided clarity on the path to NDA filing.
• The FDA did not outline any new clinical development requirements.
• Anaphylm was consistently well-tolerated with no serious adverse events reported in the studies.

Negatives

• The FDA has reserved judgement on the sufficiency of the Anaphylm clinical development program until completion of ongoing and planned studies.
• The FDA reiterated that concentrations of epinephrine above known EpiPen levels must be justified from a safety perspective.
• The FDA recommended that Aquestive begin its pediatric study after completion of the remaining adult studies, which may delay the product's availability for children.

Risks

• There are risks associated with the company's development work, including delays or changes to the timing, cost, and success of product development activities and clinical trials.
• There is a risk that the company may not generate sufficient data for FDA approval of Anaphylm.
• The FDA may require additional clinical studies for approval of Anaphylm.
• There is a risk of delays in or failure to receive FDA approval of Anaphylm.
• The company faces risks inherent in commercializing a new product, including technology, financial, market, and implementation risks.
• There is a risk of insufficient capital and cash resources to fund future clinical development activities.
• The company faces risks related to intellectual property rights and infringement claims.

Future Outlook

Aquestive aims to file an NDA for Anaphylm with the FDA before the end of 2024, following a pre-NDA meeting in the second half of 2024. The company plans to conduct an allergen study in the second quarter of 2024 and initiate pediatric studies after completing the remaining adult studies.

Management Comments

• The company is aligned with the FDA's recommendation to initiate pediatric studies after completing adult studies.
• Aquestive believes the FDA's proposed allergen study is significantly easier to perform and have agreed to move forward with this model.

Industry Context

This announcement is significant as it provides an update on a potential alternative to epinephrine autoinjectors, which are the current standard of care for anaphylaxis. The successful Phase 3 results and positive FDA feedback position Aquestive as a potential competitor in the epinephrine market.

Comparison to Industry Standards

• The study compared Anaphylm's performance to established products like EpiPen and Auvi-Q, which are widely used epinephrine autoinjectors.
• Anaphylm's Cmax values were bracketed by those of EpiPen and Auvi-Q, indicating comparable drug exposure.
• Anaphylm's Tmax was faster than both EpiPen and Auvi-Q, suggesting a quicker onset of action.
• The repeat-dose study showed that Anaphylm maintained epinephrine levels equal to or greater than existing injection products, which is a key factor for effective treatment.
• The results suggest that Anaphylm could be a viable alternative to existing epinephrine delivery methods, potentially offering a more convenient and faster-acting option.

Stakeholder Impact

• Shareholders will likely view the positive clinical results and FDA feedback favorably.
• Patients with severe allergies may benefit from a new, potentially faster-acting treatment option.
• Employees of Aquestive will be involved in the upcoming clinical studies and NDA filing process.
• The company's suppliers and partners may see increased demand for their services and products.

Next Steps

• The company will conduct an allergen PK study in the second quarter of 2024.
• A pre-NDA meeting with the FDA is targeted for the second half of 2024.
• Aquestive plans to file an NDA for Anaphylm with the FDA before the end of 2024.
• The company will initiate pediatric studies after completing the remaining adult studies.

Key Dates

Keywords

Anaphylm, epinephrine, sublingual film, anaphylaxis, Phase 3 clinical trial, FDA, pharmacokinetics, autoinjector, NDA, allergy