FDA Has Granted Request for End of Phase 2 Meeting to Discuss Alcohol Use Disorder Treatment, AD04

Pormezz/AdobeStock

Adial Pharmaceuticals today announced that the US Food and Drug Administration (FDA) has granted Adial’s request for an end of phase 2 meeting to discuss a proposed clinical development plan and FDA guidance on the phase 3 adaptive with enrichment design of the upcoming clinical trial for AD04. The meeting will take place on July 25, 2025. 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).¹

“We recently announced the successful results of our Type D Meeting with FDA confirming Adial’s 505(b)(2) regulatory bridging strategy. Concurrently, we have made strong progress in developing the design of our phase 3 trial and look forward to aligning with the FDA on key requirements to move AD04 forward. Our recent analyses have reinforced the selection of our target patient population and continue to support AD04’s potential to effectively treat AUD and related conditions. We remain confident in the path ahead and look forward to sharing a comprehensive update following our FDA meeting in July,” said Cary Claiborne, President and Chief Executive Officer of Adial.

In honor of this news, let’s take a look at the other recent devolpments on AD04.

• In February 2025, the FDA confirmed Adial’s proposed in vitro bridging strategy for the phase 3 formulation of AD04.²
• Additionally, on February 11, 2025, the United States Patent and Trademark Office issued patent number 12,221,654 for Adial Pharmaceuticals’ identification of patients with specific genetic markers linked to substance use disorders and treatment with AD04.³
• Post-hoc analyses of Adial’s prior clinical studies have indicated that patients with mutations in the 5HT3 receptor experience substantial and clinically meaningful reductions in alcohol consumption. The specific mutations that appear to respond to AD04 are single nucleotide polymorphisms (SNPs) on rs1150226-AG or rs1176713-GG genotypes in the gene that encodes the 5-HT3A receptor subunit. These genes are thought to affect the binding of AD04 to the 5HT3 receptor and its function. 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, alcohol use disorder, and other related conditions.
• AD04 was recently investigated in the pivotal phase 3 ONWARD clinical trial for the potential treatment of AUD in subjects with certain target genotypes identified using Adial’s companion diagnostic genetic test. The test will be used in future clinical studies and is expected to be commercially available at the time of AD04’s launch. AD04 had similar adverse events to placebo, which further supports that it is likely to be extremely safe and tolerable. AD04 is also believed to have the potential to treat other addictive disorders such as opioid use disorder, gambling, and obesity.

"Substance use severely harms individual health, increasing the risk of chronic diseases, mental health conditions, and tragically resulting in millions of preventable deaths every year. It places a heavy burden on families and communities, increasing exposure to accidents, injuries, and violence," said Tedros Adhanom Ghebreyesus, PhD, WHO Director-General. "To build a healthier, more equitable society, we must urgently commit to bold actions that reduce the negative health and social consequences of alcohol consumption and make treatment for substance use disorders accessible and affordable."⁴

References

1. Adial Pharmaceuticals granted end of phase 2 meeting from the U.S. Food and Drug Administration. News release. May 8, 2025. https://www.adial.com/adial-pharmaceuticals-granted-end-of-phase-2-meeting-from-the-u-s-food-and-drug-administration/

2. Kuntz L. Positive FDA feedback for proposed in vitro bridging strategy for alcohol use disorder treatment, AD04. Psychiatric Times. February 25, 2025. https://www.psychiatrictimes.com/view/positive-fda-feedback-for-proposed-in-vitro-bridging-strategy-for-alcohol-use-disorder-treatment-ad04

3. 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

4. Kuntz L. AD04 for alcohol use disorder: new positive results from pharmacokinetics study. Psychiatric Times. January 29, 2025. https://www.psychiatrictimes.com/view/ad04-for-alcohol-use-disorder-new-positive-results-from-pharmacokinetics-study