Aquestive Therapeutics (NASDAQ:AQST) executives used a presentation at the Leerink Global Healthcare Conference to outline the company’s strategic priorities, provide an update on its lead program Anaphylm following a recent FDA complete response letter (CRL), and discuss commercial planning, financing, and broader pipeline ambitions.
Management reiterated a broader platform story beyond Anaphylm
CEO Dan Barber emphasized that while Anaphylm is “the center of our attention and our resource work right now,” the company views itself as larger than a single product. Barber said the company has two in-house technologies and has achieved six FDA approvals over the years. He also highlighted “Adrenoverse,” a newer approach described as turning epinephrine into a prodrug, which management believes could create opportunities beyond Anaphylm and into additional indications.
CRL described as clear roadmap; company targets Q3 resubmission
Barber spent time clarifying what a complete response letter represents, calling it the FDA’s “complete response” after reviewing an application and noting that the key is identifying what deficiencies are cited—and what is not. He said the company viewed the CRL’s “silver lining” as the absence of clinical deficiencies. According to Barber, the clinical portion of the submission had “zero clinical deficiencies” identified by the FDA.
Management’s focus is now on executing the remediation steps in the CRL. Barber said the company believes it can resubmit by the third quarter of this year, which he said would imply a potential approval “early in 2027.” He also characterized the CRL as unusually straightforward, saying it “couldn’t be clearer” what the FDA wants to see.
The company plans two studies in response:
- A repeat human factors validation study
- A pharmacokinetics (PK) study
Barber said protocols are complete, contract research organizations have been selected, and study dates are set. He added that the company has already submitted a Type A meeting request to the FDA and expects that meeting within the next few weeks.
Human factors changes focus on packaging, instructions, and tolerability
Discussing the human factors work, Barber said the changes required appear “straightforward.” He outlined several updates the company is making:
- Package opening: The prior packaging required users to fold the top and then tear. Barber said the company has removed the fold so users “just tear,” addressing the FDA’s concern that the fold-and-tear step could be difficult for some people.
- Placement instructions: Barber said 19 out of 166 participants in the prior study placed the film on top of the tongue instead of under it. He noted that the prior foil lacked instructions or a picture, and the updated packaging now includes an image showing where to place the film.
- Tolerability and use education: Barber said tolerability issues were limited in the prior study (he cited four people) and that the company has added to the instruction base and plans additional education and demonstration materials in physician offices.
On the potential label outcome, Barber said he does not anticipate problematic label additions, describing the requested labeling instructions as “straightforward” and saying the company is “happy to agree to them.” He also suggested there could be an upside from the additional work by allowing more characterization that may ultimately be useful in the label.
PK study may include multiple arms; company expects core arms to be consistent
On the PK study, Barber said the design could include up to five arms. He described three arms that the company has conducted multiple times—an intramuscular (IM) arm, administration by a clinician, and self-administration—and said management expects those to serve as “approvability gates” and to look consistent with prior results.
He also discussed two additional arms intended to inform the FDA about outcomes when the product is not used as intended, such as placing it on top of the tongue or chewing it. Barber said the company does not view those as approvability arms, but rather as informational. He noted that the firm already has top-of-tongue data and would expect a lower Cmax and longer Tmax, while still producing what he called an efficacious dose. He also said pharmacodynamic measures are believed to be “robust” even with top-of-tongue placement.
Barber reiterated “high conviction” in the company’s timing, citing study slots and an experienced development team, while acknowledging that unexpected developments could always arise. On whether approval could come faster than the standard review clock after resubmission, he said review timing is ultimately up to the FDA, but pointed to precedent where a competitor was approved in four months after a CRL rather than the full six months. Barber said he believes there is an opportunity to “beat the clock” with clear objectives and engagement with the agency.
Commercial positioning, launch planning, and financing update
On market dynamics, Barber said he believes epinephrine treatment is shifting toward alternate delivery options and that he has not found experts who expect auto-injectors to remain dominant over the next three to five years. He described Anaphylm’s positioning as “easy to carry, easy to use, and works fast,” and cited survey work where patients chose the film when shown an auto-injector, a nasal spray, and the film.
CMO Matt Greenhawt discussed what he sees as a key barrier in anaphylaxis care: whether patients carry epinephrine and use it when needed. He argued that a thin film that can be carried behind a phone could increase the probability of having treatment available and reduce hesitation associated with needle-based administration. Greenhawt also said physicians are increasingly familiar with needle-free epinephrine options and that the allergy community is largely past the question of whether alternate delivery “will work.”
On initial adopters, Barber added that company research suggests teenagers through young adults—roughly ages 15 to 30—could be faster “prime movers,” citing compliance concerns for college-age patients.
Regarding launch plans, Barber said the company is evaluating an increase in salesforce size from 50 to 75 representatives to improve reach and frequency, which he said is important in a competitive landscape. He also said the company does not expect to focus on direct-to-consumer advertising, but views existing DTC activity in the category as beneficial for overall market growth. He cited category growth figures he attributed to the fourth quarter of 2025, saying the overall market grew 9% year-over-year and auto-injectors grew 5%.
Chief Financial Officer Ernie Toth said the company ended 2025 with $120 million in cash, which he said provides runway to complete remediation efforts, resubmit to the FDA, and keep the core commercial team in place to prepare for launch. Toth also referenced RTW’s role as a lead investor in an August capital raise and said the company announced that RTW extended a timeline related to the revenue interest financing to June 30, 2027. He described the extension as providing time to move Anaphylm through the process and supporting part of the launch financing if approved.
Toth said the company’s projected cash runway—ending 2026 with $70 million—does not include additional proceeds tied to approval. He also noted the company still owns all rights to Anaphylm, and said that an ex-U.S. partnership for Europe could bring in additional cash. He also referenced Libervant, saying it comes off ODE early next year and the company still holds U.S. rights, which he said could provide additional capital.
In closing remarks on what may be underappreciated, Barber pointed to skepticism in biotech about launch execution and said he believes the company could “surprise to the upside” commercially if it executes well, adding that Anaphylm has generated an unusually strong reaction when shown to people outside the company.
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.
