End of Phase 2 Meeting With the FDA: Adial Prepares to Advance AD04 for Alcohol Use Disorder

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Adial Pharmaceuticals today announced the completion of a successful end of phase 2 meeting with the US Food and Drug Administration (FDA), in which the FDA provided input into the AD04 phase 3 adaptive design clinical trial and the overall clinical program.¹ AD04 is Adial’s lead investigational treatment, a genetically targeted selective serotonin-3 receptor (5-HT3) antagonist and therapeutic agent for the treatment of alcohol use disorder (AUD) in patients who engage in heavy drinking (defined as < 8 drinks/drinking day).

The objective for the end of phase 2 meeting was to align with the FDA on the design of the phase 3 clinical development program for AD04. This included key elements of the planned adaptive study design elements, such as population, endpoints, inclusion/exclusion criteria, dose regime, and affirmation of the biomarker positive and biomarker negative groups.

Adial anticipates that the new clarity and validation from the FDA on the clinical development path is expected to strengthen their position in discussions with potential partners.

“We are highly encouraged by the FDA’s feedback and progress made over the past several months, which included a thorough analysis of our historical data through advanced statistical analytics and simulation modeling. These efforts have been instrumental in refining the trial design to focus on the AUD population most likely to benefit from AD04 treatment,” said Cary Claiborne, president and chief executive officer of Adial. “We look forward to advancing the clinical program for AD04 and are optimistic about achieving a successful outcome in the phase 3 program. Importantly, we believe the productive FDA meeting will also enhance our ability to advance ongoing partnership discussions, as regulatory clarity is a key consideration for potential collaborators.”¹

Following the meeting, Adial will advance AD04 into its phase 3 clinical development program for the treatment of AUD in individuals with heavy drinking and select genotype. In February 2025, the United States Patent and Trademark Office issued a patent for Adial’s identification of patients with specific genetic markers linked to substance use disorders and treatment with AD04. This new patent expands the covered methods of identifying patients with genetic markers associated with substance use disorders and treating them with AD04, an investigational selective serotonin-3 receptor (5-HT3) antagonist. The AD04 treatment involves first detecting the TT genotype of rs1042173 in the serotonin transporter gene (SLC6A4) and then administering AD04 for individuals with opioid use disorder, AUD, and other related conditions.²

In a recent interview with Psychiatric Times, Brigitte Robertson, MD, the acting chief medical officer at Adial Pharmaceuticals, shared that Adial is working hard to help the 29 million individuals who are estimated to be living with AUD. “Our work focuses on a nonabstinence-based approach that meets patients where they are. For some, reducing alcohol use may be a more realistic and acceptable goal than immediate sobriety. We are investigating the use of AD04, a genetically targeted therapeutic agent, to help reduce cravings and heavy drinking to support behavior change. The promise of personalized care through genetic biomarker targeting, where abstinence is not required, is Adial’s focus. Our vision is that this approach will help to remove barriers and make treatment accessible to a genetically predisposed population. Our approach is focused on respecting patient autonomy, reducing harm, and helping patients build healthier, more fulfilling lives on their own terms.”³

References

1. Adial Pharmaceuticals completes successful EOP2 FDA meeting. News release. August 6, 2025. Accessed August 6, 2025. https://www.adial.com/adial-pharmaceuticals-completes-successful-eop2-fda-meeting/

2. Kuntz L. Patent issued for methods of identifying patients with substance use-associated genetic markers, treatment with AD04. Psychiatric Times. February 12, 2025. https://www.psychiatrictimes.com/view/patent-issued-for-methods-of-identifying-patients-with-substance-use-associated-genetic-markers-treatment-with-ad04

3. Kuntz L, Robertson B. Combating misconceptions about alcohol use disorder: in conversation with Brigitte Robertson, MD. Psychiatric Times. May 13, 2025. https://www.psychiatrictimes.com/view/combatting-misconceptions-about-alcohol-use-disorder-in-conversation-with-brigitte-robertson-md