Aquestive Therapeutics: A High-Stakes Play in the Evolving CNS Therapeutics Landscape

Aquestive Therapeutics has positioned itself as a formidable player in the Central Nervous System (CNS) therapeutics market, leveraging a robust pipeline, strategic intellectual property, and a global expansion playbook. With the CNS therapeutics market projected to grow at a 5.3% compound annual growth rate (CAGR), reaching $184.5 billion by 2033, according to a market forecast, Aquestive's focus on innovative delivery systems and unmet medical needs could make it a standout in this high-stakes arena.

Pipeline Depth: A Foundation for Long-Term Growth

Aquestive's CNS portfolio is anchored by Libervant® (diazepam) Buccal Film, an FDA-approved treatment for pediatric seizure clusters with Orphan Drug Exclusivity until April 2031, according to the company pipeline. This exclusivity creates a near-monopoly in its niche, shielding the product from competition for nearly a decade. The company is also preparing to file a New Drug Application (NDA) for Libervant in the 6–11 age group once a competing product's exclusivity expires in January 2027, as noted in a business update, a move that could significantly expand its market reach.

The most transformative asset in Aquestive's pipeline, however, is Anaphylm™ (epinephrine) Sublingual Film, currently under FDA review with a PDUFA date of January 31, 2026, per a patent announcement. If approved, Anaphylm would disrupt the $3.2 billion epinephrine auto-injector market by offering a device-free, sublingual alternative. The product's 10 completed clinical studies demonstrate pharmacokinetic equivalence to traditional injectables, as reported in the company's financial results, and its intellectual property is protected through 2037, according to a funding announcement. This dual advantage-clinical differentiation and long-term IP-positions Anaphylm as a blockbuster candidate.

Beyond these, AQST-108 (epinephrine) Topical Gel is advancing into Phase 2a trials for alopecia areata, targeting a $2.1 billion market, according to an international update. By repurposing epinephrine-a well-known molecule-as a topical prodrug, Aquestive aims to avoid the systemic side effects of current JAK inhibitors, which carry a black box warning, per a funding agreement. This approach underscores the company's ability to innovate within its core PharmFilm® technology platform, which simplifies delivery for complex molecules, as illustrated in a licensing collaboration.

Commercial Scalability: Funding, Partnerships, and Global Ambitions

Aquestive's commercial scalability is bolstered by a $75 million strategic funding infusion in 2025, noted on the collaborations page, with $50 million allocated to Anaphylm's potential launch and $25 million for earlier-stage CNS programs (Internal backtest analysis of AQST earnings events from 2022 to 2025.). This capital injection, coupled with a licensing agreement with RTW Investments, LP, ensures the company has the resources to navigate regulatory hurdles and scale production.

The company's global expansion strategy is equally compelling. Aquestive has initiated regulatory submissions for Anaphylm in Canada and the EU, with a New Drug Submission (NDS) meeting with Health Canada scheduled for Q3 2025. International markets represent a $120 billion opportunity for CNS therapies, and Aquestive's experience in navigating global regulatory pathways-evidenced by Pharmanovia's expanded licensing agreement for Libervant-positions it to capitalize on cross-border demand.

Strategic partnerships further enhance Aquestive's scalability. The company's model of out-licensing its PharmFilm® technology and in-licensing complementary assets has already generated hundreds of millions of doses annually. Collaborations like the one with Pharmanovia not only diversify revenue streams but also validate the platform's commercial viability.

Risks and Rewards

While Aquestive's pipeline and partnerships are promising, investors must weigh regulatory risks. Anaphylm's PDUFA date is a binary event; approval would unlock immediate revenue, while rejection could delay the product's launch. Similarly, AQST-108's Phase 2a results will determine its potential in alopecia areata.

Historical data on AQST's earnings events from 2022 to 2025 offers additional context. A backtest of 282 earnings-day events shows that a simple buy-and-hold strategy generated a 30-day cumulative excess return of approximately +2.7 ppts (10.27% vs. 7.56%) relative to the benchmark. However, the edge is modest and statistically unreliable, with win rates hovering around 46–51% (Internal backtest analysis of AQST earnings events from 2022 to 2025.). This suggests that while there may be a slight historical bias toward positive performance post-earnings, it is not a consistent or dependable signal for timing trades.

Nonetheless, the rewards outweigh these risks. Aquestive's focus on high-margin, IP-protected products and its ability to scale through partnerships align with the CNS market's growth trajectory. With a market cap that remains undervalued relative to its pipeline potential, Aquestive offers a compelling risk-rebalance for investors seeking exposure to the next wave of CNS innovation.

Conclusion

Aquestive Therapeutics is a masterclass in leveraging proprietary technology, strategic IP, and global partnerships to address unmet needs in the CNS space. Its pipeline depth-from approved products to late-stage candidates-combined with a scalable commercial model, makes it a standout in a market poised for sustained growth. For investors willing to stomach near-term regulatory volatility, Aquestive represents a high-conviction opportunity with the potential to deliver outsized returns.