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Aquestive Therapeutics Announces Positive Topline Results from Oral Allergy Syndrome (OAS) Challenge Study for Anaphylm™ (epinephrine) Sublingual Film

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Aquestive Therapeutics (NASDAQ: AQST) announced positive topline results from its Oral Allergy Syndrome (OAS) challenge study for Anaphylm™ sublingual film. 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. The pharmacokinetic profile remained consistent with previous studies, showing a 12-minute time to maximum plasma concentration. The company plans a pre-NDA meeting with FDA in Q4 2024, aims to submit an NDA in Q1 2025, and targets product launch in Q1 2026.

Aquestive Therapeutics (NASDAQ: AQST) ha annunciato risultati positivi dalle fasi iniziali del suo studio di sfida sull'Anaphylm™ film sublinguale per la Sindrome Allergica Orale (OAS). Lo studio ha raggiunto entrambi gli obiettivi primari e secondari, dimostrando una rapida risoluzione dei sintomi che inizia due minuti dopo la somministrazione. Il tempo mediano per la completa risoluzione dei sintomi è stato di dodici minuti, significativamente più veloce rispetto ai settantaquattro minuti di riferimento. Il profilo farmacocinetico è rimasto coerente con studi precedenti, mostrando un tempo di dodici minuti per la concentrazione plasmatica massima. L'azienda prevede un incontro pre-NDA con la FDA nel quarto trimestre del 2024, punta a presentare un NDA nel primo trimestre del 2025 e mira a lanciare il prodotto nel primo trimestre del 2026.

Aquestive Therapeutics (NASDAQ: AQST) anunció resultados positivos de las pruebas iniciales de su estudio de desafío sobre la Anaphylm™ película sublingual para el Síndrome de Alergia Oral (OAS). El estudio cumplió con tanto los objetivos primarios como secundarios, demostrando una rápida resolución de síntomas que comienza dos minutos después de la administración. El tiempo mediano para la resolución completa de síntomas fue de doce minutos, significativamente más rápido que los setenta y cuatro minutos de referencia. El perfil farmacocinético se mantuvo consistente con estudios anteriores, mostrando un tiempo de doce minutos para alcanzar la concentración plasmática máxima. La empresa planea una reunión pre-NDA con la FDA en el cuarto trimestre de 2024, tiene como objetivo presentar un NDA en el primer trimestre de 2025 y busca lanzar el producto en el primer trimestre de 2026.

Aquestive Therapeutics (NASDAQ: AQST)Anaphylm™ 서브링구얼 필름의 구강 알레르기 증후군(OAS) 도전 연구에서 긍정적인 초기 결과를 발표했습니다. 이 연구는 주요 및 부차적 목표를 모두 충족하며, 투여 후 2분 후에 증상이 빠르게 완화되는 것을 보여주었습니다. 증상이 완전히 해결되는 데 소요된 중위 시간은 12분으로, 74분의 기준 시간보다 훨씬 빠릅니다. 약리학적 프로파일은 이전 연구와 일관되었으며, 최대 혈장 농도에 도달하는 데 12분이 걸립니다. 회사는 2024년 4분기에 FDA와의 사전 NDA 회의를 계획하고 있으며, 2025년 1분기에 NDA를 제출할 예정이며, 2026년 1분기에 제품 출시를 목표로 하고 있습니다.

Aquestive Therapeutics (NASDAQ: AQST) a annoncé des résultats préliminaires positifs de son étude de défi pour le film sublingual Anaphylm™ dans le cadre du syndrome d'allergie orale (OAS). L'étude a atteint à la fois les objectifs primaires et secondaires, démontrant une résolution rapide des symptômes commençant deux minutes après l'administration. Le temps médian pour la résolution complète des symptômes était de douze minutes, ce qui est significativement plus rapide que la ligne de base de soixante-quatorze minutes. Le profil pharmacocinétique est resté cohérent avec les études précédentes, montrant un temps de douze minutes pour atteindre la concentration plasmatique maximale. L'entreprise prévoit une réunion pré-NDA avec la FDA au quatrième trimestre 2024, vise à soumettre un NDA au premier trimestre 2025, et cible le lancement du produit au premier trimestre 2026.

Aquestive Therapeutics (NASDAQ: AQST) gab positive vorläufige Ergebnisse aus seiner Herausforderungstudie zur Anaphylm™ sublingualen Folie für das Orale Allergiesyndrom (OAS) bekannt. Die Studie erreichte sowohl die primären als auch die sekundären Endpunkte und zeigte eine schnelle Linderung der Symptome, die zwei Minuten nach der Verabreichung begann. Die mittlere Zeit zur vollständigen Symptomlinderung betrug zwölf Minuten, deutlich schneller als die Basislinie von 74 Minuten. Das pharmakokinetische Profil blieb mit früheren Studien konsistent, wobei ein Zeitpunkt von zwölf Minuten bis zur maximalen Plasmakonzentration festgestellt wurde. Das Unternehmen plant ein Pre-NDA-Meeting mit der FDA im vierten Quartal 2024, beabsichtigt, im ersten Quartal 2025 ein NDA einzureichen, und zielt darauf ab, das Produkt im ersten Quartal 2026 auf den Markt zu bringen.

Positive
  • Study met all primary and secondary endpoints
  • Rapid symptom resolution (12 minutes vs. 74-minute baseline)
  • Consistent pharmacokinetic profile maintained during allergic conditions
  • Clear regulatory pathway with planned NDA submission in Q1 2025
  • All adverse events were mild/moderate and resolved without intervention
Negative
  • None.

Insights

The OAS challenge study results for Anaphylm represent a significant milestone in developing an alternative to traditional epinephrine auto-injectors. The data shows remarkable efficacy with symptom resolution beginning at 2 minutes and 50% of symptoms resolving by 5 minutes, compared to 74 minutes without treatment. Critical pharmacokinetic parameters remained consistent with and without allergen exposure - Tmax at 12 minutes and comparable Cmax levels.

Particularly noteworthy is the successful resolution of oral swelling within 5 minutes, a important factor for severe allergic reactions. The study's design, including both single and repeat dosing arms with allergen exposure, provides robust evidence for real-world effectiveness. The consistency in PK profiles despite allergen-induced physiological changes strongly supports the reliability of sublingual delivery even during allergic reactions.

This successful study positions Aquestive strongly in the $2+ billion epinephrine market. The sublingual film format addresses key pain points in the current auto-injector dominated market - ease of use, portability and administration during oral symptoms. With planned NDA submission in Q1 2025 and potential launch in Q1 2026, Aquestive could capture significant market share, particularly among patients seeking needle-free alternatives.

The positive data significantly de-risks the regulatory pathway, potentially accelerating market adoption if approved. The pediatric study expansion to subjects over 30kg will further broaden the addressable market. Current market leaders like EpiPen could face substantial competition from this innovative delivery system, especially given the demonstrated rapid onset and consistent performance during allergic reactions.

  • Completed OAS challenge study meets both primary and secondary endpoints
  • Demonstrates rapid resolution of allergen-related symptoms beginning two minutes after administration
  • Pharmacokinetic (PK) profile after allergen exposure comparable to non-allergen PK profile
  • On track for a pre-NDA meeting on Anaphylm in Q4 2024

WARREN, N.J., Oct. 24, 2024 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ: AQST), a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies, today announced positive topline results from its Oral Allergy Syndrome (OAS) challenge study for Anaphylm™ (epinephrine) Sublingual Film. This marks the completion of the final supportive adult study in the Anaphylm development program prior to meeting with the U.S. Food and Drug Administration (FDA).

The OAS challenge study was designed as a two-part investigation to evaluate the PK and pharmacodynamics (PD) of Anaphylm in adults with allergen-induced oral physiological change. Part 1 of the study enrolled subjects with confirmed OAS into a three-period study with the following arms: (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. Six subjects who received single dose in Part 1 received repeat dose; and six subjects who received repeat dose in Part 1 received single dose. Anaphylm was administered with allergen exposure, while IM was administered without allergen exposure. 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. There were no reports of difficulty administering Anaphylm to subjects in the study.

Following allergen exposure, all subjects reported symptoms consistent with those experienced with their known allergies. Approximately twenty-five percent of subjects reported swelling of their tongues, lips, cheeks, and/or throat. Additional mucosal allergic symptoms included tingling, pain, and nasal congestion. Ninety-four percent of subjects were categorized as having moderate or severe symptoms according to the pre-defined oral severity score.

The median time for complete symptom resolution for subjects in the study following administration of Anaphylm was twelve minutes. This is faster than the median time to complete symptom resolution at screening, which was seventy-four minutes. After Anaphylm administration, symptoms began resolving as early as two minutes in some subjects and fifty percent of all symptoms across all subjects were resolved by five minutes. Importantly, all instances of symptomatic swelling were completely resolved by five minutes after administration of Anaphylm.

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. Anaphylm PK results in subjects with allergen exposure remained similar to previous profiles from the Company’s pivotal study in healthy subjects. The time to maximum plasma concentration, or Tmax, remained at twelve minutes in subjects with and without allergen exposure following a single dose of Anaphylm. The maximum plasma concentration, or Cmax, was comparable between Anaphylm administered with and without allergen exposure. In addition, Anaphylm was safe and well-tolerated with all adverse events categorized as mild or moderate and resolving without medical intervention.

“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, physician at LeBonheur Children’s Hospital, and Chair of the Annual Meeting Program for the American College of Allergy, Asthma, and Immunology (ACAAI). “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.”

"We are extremely pleased with the positive results from our OAS challenge study, which further validate Anaphylm's potential as a game-changing treatment option for severe allergic reactions, including anaphylaxis, if approved by the FDA" said Daniel Barber, President and Chief Executive Officer of Aquestive. "These results demonstrate that Anaphylm maintains its consistent PK and PD profile even when administered during oral allergic conditions, such as swelling. In addition, Anaphylm demonstrated its ability to resolve symptoms following the introduction of an oral allergen. This is a critical finding as we advance towards our NDA submission, as it confirms Anaphylm's potential effectiveness in real-world allergic scenarios."

Aquestive has requested a pre-NDA meeting with the FDA and expects to meet 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 by the FDA, Aquestive is poised to initiate a full product launch of Anaphylm in the first quarter of 2026.

A presentation containing additional information about this topline data is available on the Events and Presentations page within the Investor page of the Aquestive website.

About Anaphylm™ (epinephrine) Sublingual Film
Anaphylm™ (epinephrine) Sublingual Film is a polymer matrix-based epinephrine prodrug product candidate. Anaphylm is similar in size to a postage stamp, weighs less than an ounce, and begins to dissolve on contact. No water or swallowing is required for administration. The packaging for Anaphylm is thinner and smaller than an average credit card, can be carried in a pocket, and is designed to withstand weather excursions such as exposure to rain and/or sunlight. The Anaphylm trade name for AQST-109 has been conditionally approved by the FDA. Final approval of the Anaphylm proprietary name is conditioned on FDA approval of the product candidate.

About Aquestive Therapeutics
Aquestive is a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies. We are developing orally administered products to deliver complex molecules, providing novel alternatives to invasive and inconvenient standard of care therapies. Aquestive has five commercialized products marketed by the Company and its licensees in the U.S. and around the world, and is the exclusive manufacturer of these licensed products. The Company also collaborates with pharmaceutical companies to bring new molecules to market using proprietary, best-in-class technologies, like PharmFilm®, and has proven drug development and commercialization capabilities. Aquestive is advancing a late-stage proprietary product candidate for the treatment of severe allergic reactions, including anaphylaxis, and an earlier stage epinephrine prodrug topical gel for various dermatology conditions. For more information, visit Aquestive.com and follow us on LinkedIn.

Forward-Looking Statement
Certain statements in this press release include “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “believe,” “anticipate,” “plan,” “expect,” “estimate,” “intend,” “may,” “will,” or the negative of those terms, and similar expressions, are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements regarding the advancement and related timing of our product candidate Anaphylm™ (epinephrine) Sublingual Film through clinical development and approval by the FDA, including the timing of submission of supporting and pediatric clinical studies, holding a pre-NDA meeting with the FDA and filing the NDA for Anaphylm with the FDA, and the following launch of Anaphylm, if approved by the FDA; that the results of the Company’s clinical studies for Anaphylm are sufficient to support submission of the NDA for approval of Anaphylm by the FDA; the potential indications and potential benefits our product candidates could bring to patients; and business strategies, market opportunities, and other statements that are not historical facts.

These forward-looking statements are based on our current expectations and beliefs and are subject to a number of risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Such risks and uncertainties include, but are not limited to, risks associated with our development work, including any delays or changes to the timing, cost and success of our product development activities and clinical trials and plans, including those relating to Anaphylm (including for pediatric patients); risk of delays in advancement of the regulatory approval process through the FDA of our product candidates, including the filing of the respective NDAs, including for Anaphylm, or the failure to receive FDA approval at all of any of these product candidates; risk of the Company’s ability to generate sufficient clinical data for approval of our product candidates, including with respect to our pharmacokinetic and pharmacodynamic comparability submission for FDA approval of Anaphylm; risk of the Company’s ability to address the FDA’s comments on the Company’s clinical trials and other concerns identified in the FDA Type C meeting minutes for Anaphylm, including the risk that the FDA may require additional clinical studies for approval of Anaphylm; risks and uncertainties inherent in commercializing a new product (including technology risks, financial risks, market risks and implementation risks and regulatory limitations); risk of sufficient capital and cash resources, including sufficient access to available debt and equity financing, including under our ATM facility and the Lincoln Park Purchase Agreement, and revenues from operations, to satisfy all of our short-term and longer-term liquidity and cash requirements and other cash needs, at the times and in the amounts needed, including to fund commercialization activities relating to fund future clinical development and commercial activities for our product candidates, including Anaphylm, should these product candidates be approved by the FDA; risk of eroding market share for Suboxone® and risk as a sunsetting product, which accounts for the substantial part of our current operating revenue; risk of default of our debt instruments; risks related to the outsourcing of certain sales, marketing and other operational and staff functions to third parties; risk of the rate and degree of market acceptance in the U.S. of Anaphylm and our other product candidates, should these product candidates be approved by the FDA, and for our licensed products in the U.S. and abroad; risk of the success of any competing products including generics; risk of the size and growth of our product markets; risk of compliance with all FDA and other governmental and customer requirements for our manufacturing facilities; risks associated with intellectual property rights and infringement claims relating to our products; risk that our patent applications for our product candidates, including for Anaphylm, will not be timely issued, or issued at all, by the U.S. Patent and Trademark Office; risk of unexpected patent developments; risk of legislation and regulatory actions and changes in laws or regulations affecting our business including relating to our products and products candidates and product pricing, reimbursement or access therefor; risk of loss of significant customers; risks related to claims and legal proceedings against Aquestive including patent infringement, securities, business torts, investigative, product safety or efficacy and antitrust litigation matters; risk of product recalls and withdrawals; risks related to any disruptions in our information technology networks and systems, including the impact of cybersecurity attacks; risk of increased cybersecurity attacks and data accessibility disruptions due to remote working arrangements; risk of adverse developments affecting the financial services industry; risks related to inflation and rising interest rates; risks related to the impact of the COVID-19 global pandemic and other pandemic diseases on our business, including with respect to our clinical trials and the site initiation, patient enrollment and timing and adequacy of those clinical trials, regulatory submissions and regulatory reviews and approvals of our product candidates, availability of pharmaceutical ingredients and other raw materials used in our products and product candidates, supply chain, manufacture and distribution of our products and product candidates; risks and uncertainties related to general economic, political (including the Ukraine and Israel wars and other acts of war and terrorism), business, industry, regulatory, financial and market conditions and other unusual items; and other uncertainties affecting us including those described in the "Risk Factors" section and in other sections included in the Company’s Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K filed with the U.S. Securities and Exchange Commission. Given those uncertainties, you should not place undue reliance on these forward-looking statements, which speak only as of the date made. All subsequent forward-looking statements attributable to the Company or any person acting on its behalf are expressly qualified in their entirety by this cautionary statement. The Company assumes no obligation to update forward-looking statements after the date of this press release whether as a result of new information, future events or otherwise, except as may be required by applicable law.

PharmFilm® and the Aquestive logo are registered trademarks of Aquestive Therapeutics, Inc. All other registered trademarks referenced herein are the property of their respective owners.

Investor Inquiries
ICR Healthcare
Stephanie Carrington
stephanie.carrington@icrhealthcare.com
646-277-1282


FAQ

What were the key results of AQST's Anaphylm OAS challenge study?

The study met both primary and secondary endpoints, showing rapid symptom resolution beginning at 2 minutes, with a median complete resolution time of 12 minutes. The pharmacokinetic profile remained consistent with previous studies.

When does Aquestive (AQST) plan to submit the Anaphylm NDA?

Aquestive plans to submit the New Drug Application (NDA) for Anaphylm to the FDA in the first quarter of 2025.

What is the expected launch timeline for AQST's Anaphylm?

If approved by the FDA, Aquestive plans to launch Anaphylm in the first quarter of 2026.

How quickly did Anaphylm resolve symptoms in AQST's OAS study?

Symptoms began resolving as early as 2 minutes, with 50% of symptoms resolved by 5 minutes, and complete resolution achieved at a median time of 12 minutes.

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