Adial Pharmaceuticals Provides Business Update Following Favorable Comments from FDA End-of-Phase 2 (EOP2) Meeting for AD04
Adial Pharmaceuticals (NASDAQ: ADIL) has received positive feedback from the FDA following their End-of-Phase 2 (EOP2) meeting regarding AD04, their lead drug candidate for Alcohol Use Disorder (AUD). The FDA has aligned with Adial's proposed Phase 3 adaptive trial design for AD04, a serotonin-3 receptor antagonist targeting AUD patients with specific genotypes.
Key elements approved include the biomarker-positive patient strategy, targeting the AG+ biomarker present in approximately 14% of the population. The FDA confirmed primary efficacy endpoints focusing on zero heavy drinking days during months 5 and 6 of the observation period. The agency also supported Adial's plans for interim analyses and provided guidance on statistical analysis plans.
The company views this regulatory milestone as pivotal for advancing toward Phase 3 trials and potentially becoming the first genetically targeted therapy for AUD. This development, combined with their recent patent filing potentially extending market exclusivity to 2045, positions AD04 as a significant commercial opportunity.
Adial Pharmaceuticals (NASDAQ: ADIL) ha ricevuto feedback positivo dalla FDA dopo l'incontro End-of-Phase 2 (EOP2) riguardante l'AD04, il loro candidato principale per disturbo da uso di alcol (AUD). La FDA ha allineato il design proposto di trial di Fase 3 adattivo per l'AD04, un antagonista del recettore serotoninergico-3 mirato ai pazienti con AUD con particolari genotipi.
Gli elementi chiave approvati includono la strategia di pazienti biomarker-positivi, che punta al biomarcatore AG+ presente in circa il 14% della popolazione. La FDA ha confermato gli endpoint primari di efficacia focalizzati su zero giorni di consumo pesante durante i mesi 5 e 6 del periodo di osservazione. L'agenzia ha anche supportato i piani di analisi intermedie e fornito indicazioni sui piani di analisi statistica.
L'azienda considera questa pietra miliare normativa fondamentale per avanzare verso studi di Fase 3 e potenzialmente diventare la prima terapia mirata geneticamente per AUD. Questo sviluppo, insieme all'HDR recente di brevetto che potrebbe estendere l'esclusività di mercato fino al 2045, posiziona AD04 come una significativa opportunità commerciale.
Adial Pharmaceuticals (NASDAQ: ADIL) ha recibido comentarios positivos de la FDA tras su reunión de End-of-Phase 2 (EOP2) sobre AD04, su principal candidato a fármaco para el Trastorno por uso de alcohol (AUD). La FDA se ha alineado con el diseño propuesto de ensayo adaptativo de Fase 3 para AD04, un antagonista del receptor de serotonina-3 dirigido a pacientes con AUD con genotipos específicos.
Los elementos clave aprobados incluyen la estrategia de pacientes biomarcador-positivos, orientada al biomarcador AG+ presente en aproximadamente el 14% de la población. La FDA confirmó los criterios de eficacia primarios centrados en cero días de consumo excesivo durante los meses 5 y 6 del periodo de observación. La agencia también apoyó los planes de análisis interinos y dio orientación sobre los planes de análisis estadísticos.
La compañía ve este hito regulatorio como crucial para avanzar hacia ensayos de Fase 3 y potencialmente convertirse en la primera terapia dirigida genéticamente para AUD. Este desarrollo, junto con su reciente solicitud de patente que podría ampliar la exclusividad del mercado hasta 2045, posiciona a AD04 como una oportunidad comercial significativa.
Adial Pharmaceuticals (NASDAQ: ADIL)는 알코올 사용 장애(AUD)에 대한 선도 후보 약물 AD04에 대해 FDA의 End-of-Phase 2(EOP2) 회의 이후 긍정적인 피드백을 받았습니다. FDA는 AD04에 대한 Adial의 제3상 적응형 시험 설계와 일치했으며, 특정 유전형을 가진 AUD 환자를 대상으로 하는 세로토닌-3 수용체 길항제를 지목합니다.
승인된 핵심 요소로는 바이오마커 양성 환자 전략이 포함되며, 이는 인구의 약 14%에서 발견되는 AG+ 바이오마커를 겨냥합니다. FDA는 관찰 기간의 5~6개월 동안 무중증 음주일(zero heavy drinking days)을 주요 효능 지표로 확인했습니다. 또한 중간 분석 계획에 대한 지원과 통계 분석 계획에 대한 지침도 제공했습니다.
회사는 이 규제 이정표를 3상 임상으로의 진행과 잠재적으로 AUD에 대한 최초의 유전적으로 표적화된 치료제가 될 수 있는 중요한 계기로 보고 있습니다. 최근 특허 출원이 2045년까지 시장 독점권을 연장할 수 있다는 점과 함께, AD04는 중요한 상업적 기회를 제공합니다.
Adial Pharmaceuticals (NASDAQ: ADIL) a reçu des retours positifs de la FDA après la réunion End-of-Phase 2 (EOP2) concernant l'AD04, leur candidat principal pour le trouble lié à l’usage d’alcool (AUD). La FDA a aligné le design proposé d’essai adaptatif de phase 3 pour l’AD04, un antagoniste du récepteur de la sérotonine-3 ciblant les patients AUD avec des génotypes spécifiques.
Les éléments clés approuvés incluent la stratégie de patients biomarqueur-positifs, visant le biomarqueur AG+ présent chez environ 14% de la population. La FDA a confirmé des critères d’efficacité primaires axés sur zéro jour de consommation lourde pendant les mois 5 et 6 de la période d’observation. L’agence a également soutenu les plans d’analyses intermédiaires et fourni des orientations sur les plans d’analyses statistiques.
L’entreprise voit cette étape réglementaire comme déterminante pour progresser vers les essais de Fase 3 et potentiellement devenir la première thérapie génétiquement ciblée pour l’AUD. Cette évolution, associée à leur récent dépôt de brevet pouvant étendre l’exclusivité du marché jusqu’en 2045, positionne l’AD04 comme une opportunité commerciale majeure.
Adial Pharmaceuticals (NASDAQ: ADIL) hat positives Feedback von der FDA nach dem End-of-Phase-2-Treffen (EOP2) zu AD04 erhalten, ihrem führenden Medikamentenkandidaten für Alcohol Use Disorder (AUD). Die FDA hat sich mit dem von Adial vorgeschlagenen adaptiven Phase-3-Studienentwurf für AD04 abgestimmt, einem Serotonin-3-Rezeptor-Antagonisten, der auf AUD-Patienten mit bestimmten Genotypen abzielt.
Zu den genehmigten Schlüsselelementen gehört die Biomarker-positiven-Patienten-Strategie, die auf den AG+ Biomarker abzielt, der in etwa 14% der Bevölkerung vorhanden ist. Die FDA bestätigte primäre Wirksamkeitsendpunkte, die sich auf null Tage mit starkem Trinken in den Monaten 5 und 6 der Beobachtungsperiode konzentrieren. Die Behörde unterstützte auch Zwischenanalysenpläne und gab Hinweise zu den statistischen Analyseplänen.
Das Unternehmen betrachtet diesen regulatorischen Meilenstein als entscheidend für den Fortschritt zu Phase-3-Studien und potenziell als erste genetisch zielgerichtete Therapie für AUD. Diese Entwicklung, zusammen mit der jüngsten Patentanmeldung, die die Marktexklusivität bis 2045 verlängern könnte, positioniert AD04 als eine bedeutende kommerzielle Chance.
Adial Pharmaceuticals (NASDAQ: ADIL) تلقّت تعليقات إيجابية من إدارة الغذاء والدواء الأمريكية (FDA) عقب اجتماع End-of-Phase 2 (EOP2) حول AD04، مرشحها الدوائي الرائد لـ اضطراب تعاطي الكحول (AUD). وافقت FDA على التصميم المقترح لتجربة المرحلة 3 التكيفية لـ AD04، وهو مضاد مستقبلات السيروتونين-3 يستهدف مرضى AUD الذين يمتلكون جينات معينة.
تشمل العناصر الرئيسية المعتمدة استراتيجية المرضى الإيجابيين للبيوماركر، المستهدفة للبيوماركر AG+ الموجود تقريباً في 14% من السكان. أكدت FDA متغيرات الفاعلية الأساسية التي تتركز على أيام عدم تَهْدُر كبيرة خلال شهري 5 و6 من فترة المراقبة. كما دعمت الوكالة خطط التحليلات الوسيطة وقدمَت إرشادات حول خطط التحليلات الإحصائية.
ترى الشركة أن هذا الإنجاز التنظيمي خطوة محورية نحو التجارب في المرحلة 3 وربما يصبح أول علاج موجه جينيًا لـ AUD. هذا التطور، إلى جانب تقديم طلب براءة قد يمدّ الحصرية السوقية حتى 2045، يجعل AD04 فرصة تجارية هامة.
Adial Pharmaceuticals (NASDAQ: ADIL) 在关于 AD04 的 EOP2（End-of-Phase 2）会议后，收到了美国食品药品监督管理局（FDA）的积极反馈。AD04 是用于 酒精使用障碍（AUD） 的领先药物候选。FDA 与 Adial 提出的对 AD04 的第三阶段自适应试验设计保持一致，这是一种针对具有特定基因型的 AUD 患者的血清素-3 受体拮抗剂。
批准的关键要素包括 生物标志物阳性患者策略，针对约 14% 人群存在的 AG+ 生物标志物。FDA 确认了主要疗效终点，聚焦观察期第 5 和第 6 个月的零大量饮酒日。监管机构还支持 Adial 的中期分析计划并就统计分析计划提供了指南。
公司将这一监管里程碑视为推进进入 III 期试验的关键节点，并有可能成为 首个基因靶向的 AUD 治疗。这一发展，加上其最近的专利申请可能将市场独占性延长至 2045 年，使 AD04 成为一个重要的商业机会。
- FDA recognized AUD as an unmet medical need
- FDA approved core elements of Phase 3 trial design including biomarker strategy
- Recent patent filing could extend market exclusivity to 2045
- Potential to become first genetically targeted AUD therapy
- Clear regulatory pathway established for Phase 3 development
- Limited target population with AG+ biomarker present in only 14% of population
- Complex trial design with multiple endpoints and analyses required
- Extended timeline needed for Phase 3 completion with 5-6 month observation period
Insights
FDA support for Adial's Phase 3 trial design for AD04 significantly de-risks their clinical pathway for this genetically-targeted AUD treatment.
The FDA's positive feedback from Adial's End-of-Phase 2 meeting represents a significant regulatory milestone that substantially de-risks their development program. The agency's support for Adial's adaptive trial design with genetically-targeted patient selection via the AG+ biomarker demonstrates regulatory receptiveness to precision medicine approaches in addiction treatment, where innovation has historically lagged.
The FDA's recognition of AUD as an unmet medical need is particularly noteworthy as it could potentially expedite the review process. Current AUD treatments show limited efficacy, with many patients experiencing inadequate response. A genetically-targeted approach could dramatically improve outcomes for the estimated 14% of AUD patients with the AG+ biomarker.
Importantly, the agency's endorsement of Adial's primary endpoint—zero heavy drinking days during months 5-6—aligns with contemporary regulatory thinking. This endpoint balances clinical relevance with statistical feasibility better than total abstinence metrics that have hindered previous development programs.
The FDA's guidance on handling homozygous populations and references to targeted therapy development for low-frequency molecular subsets suggests potential pathways for expedited approval in genetically-defined subgroups. This could enable a focused commercial strategy targeting the ~14% AG+ population, significantly reducing commercial risk compared to traditional broad-market approaches.
While this regulatory alignment substantially strengthens Adial's position, investors should note that Phase 3 execution remains the critical determinant of ultimate success, with capital requirements for completion representing the primary near-term challenge.
FDA Minutes Reflect Constructive Dialogue and Successful Meeting Outcome with Alignment on Upcoming Phase 3 Clinical Trial Design for AD04 In Alcohol Use Disorder
GLEN ALLEN, Va., Sept. 16, 2025 (GLOBE NEWSWIRE) -- Adial Pharmaceuticals, Inc. (NASDAQ: ADIL) (“Adial” or the “Company”), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment and prevention of addiction and related disorders, today announced receipt of the final meeting minutes from its End of Phase 2 (EOP2) Meeting with the U.S. Food and Drug Administration (FDA) held on July 29, 2025. The minutes provide the FDA’s formal input into the AD04 Phase 3 adaptive clinical trial design and broader clinical development strategy. This positive feedback marks a key milestone in advancing AD04 toward registration.
The objective for the EOP2 Meeting was to align with the FDA on the design of the Phase 3 clinical development program for AD04, the Company’s lead investigational drug, a serotonin-3 receptor antagonist, being developed for the treatment of Alcohol Use Disorder (AUD) in individuals with heavy drinking and select genotypes. The discussion included key elements of the planned adaptive study design elements, such as target population, clinical endpoints, inclusion and exclusion criteria, dosing regimen, and affirmation of the biomarker-positive and biomarker-negative groups.
Key Highlights from the FDA EOP2 Meeting:
- FDA recognized AUD as an unmet need.
- FDA supported Adial’s protocol and proposed adaptive trial design core elements, including the defined biomarker-positive and biomarker-negative patients, key inclusion criteria targeting moderate to severe AUD, trial duration, primary endpoints, interim analysis sample size, and safety monitoring framework.
- FDA confirmed the proposed primary efficacy endpoints for AD04, specifically, zero heavy drinking days during months 5 and 6 of the efficacy observation period.
- FDA advised that key secondary endpoints intended for future product labeling should be pre-specified in the protocol for consideration.
- The FDA supported Adial’s plan to account for homozygous populations and referenced guidance on developing targeted therapies for low-frequency molecular subsets, with implications for study design and potential labeling of rare subgroups.
- The FDA provided feedback on the planned interim analyses, Statistical Analysis Plan (SAP), and Data Monitoring Committee (DMC) structure, and emphasized the importance of alignment between the study protocol, simulation report, and SAP to ensure appropriate alpha control and minimize post-trial data analysis risk.
Adial is implementing FDA recommendations consistent with the meeting outcomes, ensuring its readiness to advance toward registrational Phase 3 development. The AD04 program is designed to bring focused and targeted commercial success after registration and approval, for patients who are biomarker positive for AG+, a subset of the AUD population, identified through Adial’s proprietary genetic test. The AG+ biomarker is present in roughly
“We believe we are at an inflection point in Adial’s journey,” commented Cary Claiborne, CEO of Adial. “The successful completion of our End of Phase 2 meeting with the FDA has provided critical guidance that strengthens the clinical and statistical framework for our upcoming Phase 3 trial of AD04. With regulatory alignment on key protocol elements, including patient population, efficacy endpoints, biomarker stratification, and adaptive enrichment strategies—we are now positioned to advance a streamlined and scientifically rigorous program designed to maximize the probability of success.”
“This milestone reflects more than regulatory progress-it demonstrates our commitment to precision medicine. By integrating pharmacogenetic insights and validated biomarkers into our development strategy, we are enhancing clinical predictability and setting a new benchmark for targeted therapeutics in neuropsychiatry. This progress, coupled with the recent announcement of our new patent filing for AD04 possibly extending market exclusivity to 2045, frame the AD04 program as an extremely attractive future commercial opportunity with a meaningful patient impact. We remain focused on delivering a transformative solution for patients living with AUD.”
“As we advance toward Phase 3, we believe we are executing from a position of strength across regulatory, clinical, and manufacturing fronts. This milestone is also a catalyst for strategic partnership discussions, which we expect to accelerate with FDA alignment now in place. With disciplined execution, we believe AD04 is positioned to become the first genetically targeted therapy for AUD—addressing a large, underserved patient population and unlocking a highly attractive commercial opportunity,” concluded Mr. Claiborne.
About Adial Pharmaceuticals, Inc.
Adial Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the development of treatments for addictions and related disorders. The Company’s lead investigational new drug product, AD04, is a genetically targeted, serotonin-3 receptor antagonist, therapeutic agent for the treatment of Alcohol Use Disorder (AUD) in heavy drinking patients and was recently investigated in the Company’s ONWARD™ pivotal Phase 3 clinical trial for the potential treatment of AUD in subjects with certain target genotypes identified using the Company’s companion diagnostic genetic test. ONWARD showed promising results in reducing drinking in heavy drinking patients, and no overt safety or tolerability concerns. AD04 is also believed to have the potential to treat other addictive disorders such as Opioid Use Disorder, gambling, and obesity. Additional information is available at www.adial.com.
Forward-Looking Statements
This communication contains certain “forward-looking statements” within the meaning of the U.S. federal securities laws. Such statements are based upon various facts and derived utilizing numerous important assumptions and are subject to known and unknown risks, uncertainties and other factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Statements preceded by, followed by or that otherwise include the words “believes,” “expects,” “anticipates,” “intends,” “projects,” “estimates,” “plans” and similar expressions or future or conditional verbs such as “will,” “should,” “would,” “may” and “could” are generally forward-looking in nature and not historical facts, although not all forward-looking statements include the foregoing. The forward-looking statements include statements regarding advancing AD04 toward registration, implementing FDA recommendations consistent with the meeting outcomes, ensuring the Company’s readiness to advance toward registrational Phase 3 development, designing the AD04 program to bring focused and targeted commercial success after registration and approval for patients who are biomarker positive for AG+, being at an inflection point in Adial’s journey, being positioned to advance a streamlined and scientifically rigorous program designed to maximize the probability of success, integrating pharmacogenetic insights and validated biomarkers into the Company’s development strategy, enhancing clinical predictability and setting a new benchmark for targeted therapeutics in neuropsychiatry, extending market exclusivity to 2045 with the new patent filing for AD04, delivering a transformative solution for patients living with AUD, executing from a position of strength across regulatory, clinical, and manufacturing fronts, accelerating strategic partnership discussions with FDA alignment now in place, AD04 becoming the first genetically targeted therapy for AUD, addressing a large, underserved patient population and unlocking a highly attractive commercial opportunity and the potential of AD04 to treat other addictive disorders such as Opioid Use Disorder, gambling, and obesity. Any forward-looking statements included herein reflect our current views, and they involve certain risks and uncertainties, including, among others, our ability to obtain regulatory approvals for commercialization of product candidates or to comply with ongoing regulatory requirements, our ability to develop strategic partnership opportunities and maintain collaborations, our ability to obtain or maintain the capital or grants necessary to fund our research and development activities, our ability to complete clinical trials on time and achieve desired results and benefits as expected, regulatory limitations relating to our ability to promote or commercialize our product candidates for specific indications, acceptance of our product candidates in the marketplace and the successful development, marketing or sale of our products, our ability to maintain our license agreements, the continued maintenance and growth of our patent estate and our ability to retain our key employees or maintain our Nasdaq listing. These risks should not be construed as exhaustive and should be read together with the other cautionary statement included in our Annual Report on Form 10-K for the year ended December 31, 2024, subsequent Quarterly Reports on Form 10-Q and current reports on Form 8-K filed with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was initially made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise, unless required by law.
Contact:
Crescendo Communications, LLC
David Waldman / Alexandra Schilt
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Email: adil@crescendo-ir.com
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