AD04 Advancing to Phase III Amid Promising Results in Alcohol Use Disorder

PE: Were there any specific patient demographics or subgroups that demonstrated a notably different response to AD04 in terms of safety or efficacy?

Claiborne: The drug showed strong safety across all of the groups we tested. We knew this going in based on prior studies, but AD04 has also shown higher efficacy among certain patients with certain genetic mutations or genotypes in the 5-HT3 receptor area of the brain.

PE: How do these results impact AD04’s development timeline, and what are the next critical milestones toward FDA approval?

Claiborne: This study was a critical milestone. This is a key part of what the FDA was looking for. Anytime you have a repurposed drug, you have to do a PK study and show viral availability, how it compares to the reference drug you're going to be using a lot of the data from. This will pave the way for initiating our next planned Phase III study with the FDA, which we hope to get into early to mid-next year.

Full Interview Summary: The AD04 study aimed to assess the relative bioavailability, pharmacokinetics (PK), and safety of AD04, an ultra-low-dose ondansetron formulation, compared to a generic 4 mg ondansetron product. Key design elements included evaluating PK variability and dose proportionality between two AD04 doses (0.33 mg and 0.99 mg), assessing food effects on bioavailability, and confirming safety across patient demographics.

The results confirmed AD04's predictable pharmacokinetic profile, demonstrating that its bioavailability was unaffected by food intake or meal timing. These findings align with the drug's intended use for alcohol use disorder (AUD), particularly as a convenient treatment option. Notably, the study revealed enhanced efficacy in patients with specific genetic mutations in the 5-HT3 receptor region, supporting the drug’s targeted therapeutic approach.

The study addressed critical feedback from the FDA regarding bioavailability and PK data requirements for repurposed drugs. This milestone clears the path for initiating a Phase III study, expected to begin early to mid-next year, focusing on the genetically defined subgroup showing higher efficacy.

Safety results indicated strong tolerability across all tested groups, consistent with prior studies. The company is also exploring external collaborations for both development and commercialization, leveraging these findings to advance AD04’s clinical and market readiness. These efforts position the drug for meaningful progress toward FDA approval and broader adoption for AUD treatment.