Aquestive Therapeutics (NASDAQ:AQST) executives outlined the company’s near-term regulatory plans and longer-term platform strategy during a presentation at Oppenheimer’s 36th Annual Healthcare Life Sciences Conference, with most of the discussion centered on Anaphylm, its sublingual epinephrine film for severe allergic reactions.
Company focus: oral thin film drug delivery and “Adrenoverse” platform
CEO Dan Barber said Aquestive’s mission is to take “existing molecules” that are “sub-optimized” and use the company’s drug delivery technologies to improve patient experience and outcomes. He highlighted the firm’s oral thin film technology, which it uses to develop orally administered products intended to offer alternatives to “invasive and inconvenient” standard-of-care therapies.
In addition to its thin film platform, Barber described “Adrenoverse,” which he characterized as the company’s ability to deliver epinephrine with different release profiles. He said epinephrine has a short half-life (five minutes or less) and is difficult to control and absorb, and that the company’s prodrug approach is designed to create different absorption and release profiles using different functional groups. Barber said the company has an intellectual property portfolio around the platform and sees longer-term potential beyond allergy, including dermatology.
Pipeline overview: Anaphylm, Libervant, and AQST108
Barber said the company’s pipeline includes three programs:
- Anaphylm, an epinephrine sublingual film aimed at severe allergic reactions including anaphylaxis.
- Libervant, a diazepam buccal film for acute repetitive seizures, which Barber said has tentative FDA approval and is waiting for a competitor’s orphan drug exclusivity to expire in January of next year. He added that outside the U.S. the product is partnered with Pharmanovia, which has filed in “almost 20 countries.”
- AQST108, an epinephrine topical gel in Phase 1 for alopecia areata, which Barber said is intended to demonstrate Adrenoverse’s versatility.
Anaphylm: product concept, speed, and market opportunity
Barber demonstrated a “demo film” and emphasized ease of use and portability. He said the film is intended to be carried easily (including on the back of a phone), and described it as needle-free and not a medical device. He said patients would tear open a pouch, place the film under the tongue, and the film would turn into a gel “in a matter of seconds” as it hydrates and begins absorption.
Barber also highlighted speed, saying Anaphylm has shown a 12-minute Tmax in the company’s studies, compared with 20 minutes for an autoinjector comparator in those studies. He argued that earlier treatment can reduce the likelihood of needing greater medical intervention as a reaction progresses.
On market dynamics, Barber said the U.S. market continues to grow and cited “scripts” of just under $5 million last year, with expectations for 2025 to be over $5 million. He also said the market is still largely dominated by autoinjectors, representing more than 90% of the market based on the data the company has, with the remaining share in nasal spray.
Barber discussed patient behavior and the challenges of carrying autoinjectors, citing a survey he said asked EpiPen carriers whether they would go home to retrieve their device if they forgot it, in which “0%” said they would. He argued that Anaphylm’s portability could address behavioral barriers such as inconvenience, forgetfulness, fear of use, and complacency.
Barber also described a company survey in which participants were sent physical mock-ups of an autoinjector, a nasal spray, and the film, and then asked which option they would prefer to carry. He said that among 52 or 53 people, only one preferred nasal spray and one was indifferent between film and nasal spray, while the rest preferred the film. Barber said the results support the idea that “seeing is believing” and that product visibility with physicians and patients could drive adoption.
FDA complete response letter and timeline to resubmission
Barber said the company received a complete response letter (CRL) around its January 31 PDUFA date for Anaphylm. He said the FDA is requiring additional human factors work and an additional pharmacokinetic (PK) study, which he placed largely in the first and second quarters of the year for human factors and in the second quarter for the PK study.
Barber said the company has publicly guided to a resubmission in the third quarter and expects a six-month review clock, describing the resubmission as a “Type 2” review because it will include clinical data. Based on that timing, he said the company would expect approval in the first quarter of 2027, depending on when it files in Q3. He added the company would advocate for a faster review, arguing that the submission would not include a large amount of data.
Asked about the nature of the requested studies, Barber said they were “very straightforward” relative to prior clinical questions, adding that the company has done these types of studies before and intends to execute and resubmit with clean data.
Financial runway and funding options
CFO Ernie Toth said the company is close to an earnings call and did not provide formal guidance, but reiterated prior comments about liquidity. Toth said the company ended 2025 with “a little over $121 million,” which he said provides a “comfortable” cash balance to address the CRL remediation work and support commercial staff being maintained in preparation for a launch.
Toth also said the company previously stated it would end 2026 with a “healthy cash balance” that would extend into 2027 to prepare for launch. He noted this did not include potential additional funding from RTW upon approval and accessing that line, or other funds that could come from out-licensing. Toth said the company still owns all rights to Anaphylm and could monetize those rights, and added that Libervant in the U.S., once off orphan drug exclusivity next year, could also be available for licensing.
About Aquestive Therapeutics (NASDAQ:AQST)
Aquestive Therapeutics, Inc is a specialty pharmaceutical company focused on the development and commercialization of novel drug delivery systems. Leveraging its proprietary PharmFilm® technology, Aquestive designs thin-film formulations that facilitate sublingual, buccal and oral delivery of small molecules, offering rapid onset of action and improved patient compliance compared with traditional dosage forms.
The company’s lead product, Libervant® (diazepam) Buccal Film, is approved by the U.S.
