AD04-13 Study Highlights Consistent Bioavailability and Safety for Alcohol Use Disorder Treatment

PE: Can you elaborate on the key design elements of the AD04-13 study and how they contributed to achieving positive topline results?

Claiborne: The purpose of the study was to evaluate the relative bioavailability of AD04. We compared low dose ondansetron, which is what AD04-13 is, to a reference standard marketed product, which is generic ondansetron at 4 mg. We also looked at the PK variability and the dose proportionality between two doses of ADL4 through .33 mg and .99 mg. We also evaluated the food effect on the bioavailability of ADL4 and of course, we looked at safety.

PE: What do these results indicate about the pharmacokinetic profile of AD04, and how does this align with the drug’s intended therapeutic use for Alcohol Use Disorder?

Claiborne: We’re really happy to say that AD04 behaved as expected. It followed the normal dosing properties that we were expecting to see when we started the study, and it's not impacted at all by food intake or the timing of those meals.

Full Interview Summary: The AD04-13 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.