Aquestive Therapeutics, Inc. (NASDAQ: AQST) is making strides in its pharmaceutical development pipeline, particularly with Anaphylm, a sublingual film for anaphylaxis, and AQST-108, a topical gel for alopecia areata. Recent updates from the company's virtual investor day and clinical study results have fueled optimism, reflected in an increased price target from H.C. Wainwright.
Anaphylm: Advancing Towards Potential FDA Approval
Anaphylm, Aquestive's epinephrine sublingual film, is designed for the emergency treatment of severe allergic reactions, offering a needle-free alternative to traditional auto-injectors. The company recently announced positive topline results from its Oral Allergy Syndrome (OAS) challenge study, a crucial step before meeting with the U.S. Food and Drug Administration (FDA).
The OAS challenge study evaluated the pharmacokinetics (PK) and pharmacodynamics (PD) of Anaphylm in adults experiencing allergen-induced oral physiological changes. The study met both primary and secondary endpoints, demonstrating rapid symptom resolution beginning two minutes after administration. The median time for complete symptom resolution was twelve minutes, significantly faster than the 74-minute baseline observed without treatment. Notably, all instances of symptomatic swelling were completely resolved within five minutes after Anaphylm administration.
"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."
The pharmacokinetic profile of Anaphylm remained consistent with previous studies, showing a 12-minute time to maximum plasma concentration (Tmax). The maximum plasma concentration (Cmax) was comparable between Anaphylm administered with and without allergen exposure. The drug was also found to be safe and well-tolerated, with all adverse events categorized as mild or moderate and resolving without medical intervention.
Aquestive plans to hold a pre-New Drug Application (NDA) meeting with the FDA in the fourth quarter of 2024 and submit the NDA in the first quarter of 2025. If approved, the company anticipates initiating a full product launch of Anaphylm in the first quarter of 2026.
AQST-108: Targeting Alopecia Areata
Aquestive is also developing AQST-108, an epinephrine topical gel, for the treatment of alopecia areata, a condition affecting as many as 6.7 million people in the U.S. The company completed its first human clinical study for AQST-108 and expects to hold a pre-Investigational New Drug (IND) meeting with the FDA in the first quarter of 2025, with a Phase 2 study expected to commence in the second half of 2025.
The alopecia areata treatment landscape includes oral medications like Eli Lilly's Olumiant (baricitinib) and Pfizer's Litfulo (ritlecitinib), approved by the FDA in June 2022 and June 2023, respectively. Given the high cost of these treatments, Aquestive anticipates that a topical alopecia areata treatment without a black box warning could capture significant market share if priced competitively. Analysts project that AQST-108 could reach over $1 billion in annual U.S. sales with sufficient market penetration.
Analyst Optimism and Financial Outlook
H.C. Wainwright raised its price target for Aquestive Therapeutics to $10 from $9 per share, reflecting the addition of AQST-108 to the valuation assessment. The analyst attributes an 85% probability of approval to Anaphylm for anaphylaxis and a 30% probability of approval to AQST-108 for alopecia areata.
This positive outlook is further supported by analysts' belief that Anaphylm has the potential to significantly expand the epinephrine market, with estimates suggesting peak sales potential between $300 million and $400 million for Aquestive. The company's commercial model is expected to be relatively lean, suggesting high operating margins over time.
As Aquestive moves forward with its NDA submission for Anaphylm and the development of AQST-108, the company is poised to potentially transform the treatment landscape for severe allergies and alopecia areata.
