Aquestive Therapeutics announced positive topline results from its Oral Allergy Syndrome (OAS) challenge study for Anaphylm™ (epinephrine) Sublingual Film, marking the completion of the final supportive adult study in the Anaphylm development program before a planned meeting with the U.S. Food and Drug Administration (FDA). The data indicate rapid symptom resolution and consistent pharmacokinetic (PK) profiles, reinforcing Anaphylm's potential as a reliable alternative for severe allergic reactions, including anaphylaxis.
OAS Challenge Study Details
The OAS challenge study was a two-part investigation evaluating the PK and pharmacodynamics (PD) of Anaphylm in adults experiencing allergen-induced oral physiological changes. In Part 1, subjects with confirmed OAS were enrolled into a three-period study. The arms included: (1) Anaphylm with allergen exposure (n=18 single dose; n=18 repeat dose); (2) Anaphylm without allergen exposure (n=15 single dose; n=13 repeat dose); and (3) Adrenalin intramuscular (IM) injection without allergen exposure (n=18 single dose; n=17 repeat dose). Part 2 was an optional follow-on study to Part 1. During allergen exposure arms in Parts 1 and 2, subjects were exposed to a fruit they were known to be allergic to, and the resulting symptoms were documented for location, severity, and duration.
Symptom Resolution and PK Profile
Following allergen exposure, subjects reported symptoms consistent with their known allergies, with approximately 25% reporting swelling of the tongue, lips, cheeks, and/or throat. Ninety-four percent of subjects experienced moderate to severe symptoms based on a pre-defined oral severity score. The median time for complete symptom resolution following Anaphylm administration was 12 minutes, significantly faster than the 74-minute median time observed at screening. Symptom resolution began as early as two minutes in some subjects, with 50% of all symptoms resolving within five minutes. Notably, all instances of symptomatic swelling were completely resolved within five minutes after Anaphylm administration.
Both primary and secondary endpoints of the OAS challenge study were successfully met, with no significant differences found between Anaphylm PK results in subjects with and without allergen exposure. The time to maximum plasma concentration (Tmax) remained at 12 minutes in both groups following a single dose. The maximum plasma concentration (Cmax) was also comparable. Anaphylm was safe and well-tolerated, with all adverse events categorized as mild or moderate and resolving without medical intervention.
Expert Commentary
"Symptom relief is the most real-world scenario whereby subjects know their rescue product is working," said Jay Lieberman, M.D., Professor at the University of Tennessee Health Science Center. "I am reassured by the speed of symptom relief seen in the OAS Study and by the continued and consistent rapid absorption profile of Anaphylm. These data provide strong evidence that Anaphylm could provide a reliable alternative to the approved epinephrine medical devices currently available to patients."
Regulatory and Commercialization Plans
Aquestive has requested a pre-NDA meeting with the FDA in the fourth quarter of 2024. The company remains on track to commence a pediatric study in subjects weighing 30 kgs and above in the fourth quarter 2024 and to submit a New Drug Application (NDA) to the FDA in the first quarter 2025. If approved, Aquestive plans to initiate a full product launch of Anaphylm in the first quarter of 2026.
