Adial Pharmaceuticals has successfully completed analytical validation of a cheek swab genetic test designed to identify patients most likely to benefit from its investigational alcohol use disorder treatment AD04. The partnership with Genomind represents a key milestone in advancing the precision medicine approach toward Phase 3 clinical trials and potential FDA registration.
The collaboration developed an analytically validated gene test in a CLIA-certified laboratory that identifies important single nucleotide polymorphisms (SNPs) in the serotonergic system. These genetic markers are crucial for patient selection in the upcoming Phase 3 program for AD04, a genetically targeted serotonin-3 receptor antagonist therapeutic agent.
Genetic Testing Capabilities and Validation
Through the partnership, Adial can now test SNPs in the HTR3A, HTR3B, and SLC6A4 genes, as well as long and short forms of the SLC6A4 promoter. Genomind validated the assay for accuracy, reproducibility, and robustness using commercially sourced human samples.
The validation process encompassed multiple parameters including inter- and intra-assay precision, diverse sample origins and ethnic backgrounds, different operators, instruments, and assay reagent lots. The assay demonstrated 100% concordance across all SNP testing, establishing its reliability for both clinical trial use and future commercial application.
Target Population and Regulatory Alignment
The AD04 program targets patients who are biomarker positive for AG+, representing a subset of the Alcohol Use Disorder population identified through Adial's proprietary genetic test. The AG+ biomarker is present in roughly 14% of the general population, as demonstrated in large-scale epidemiology studies and prior clinical study populations from previous AD04 trials.
During a recent End of Phase 2 meeting, the FDA confirmed that use of this genetic test in Phase 3 studies constitutes Non-Significant Risk, eliminating the requirement for filing an Investigational Device Exemption application. This regulatory alignment positions Adial to advance toward registrational Phase 3 development with streamlined protocols.
Clinical and Commercial Applications
The cheek swab collection method enables seamless patient identification in physician offices or at-home settings, with results reported within days. This accessibility supports both clinical trial enrollment and future commercial deployment strategies.
"Successful completion of the first phase of our partnership with Genomind, and the creation of this patient and physician-friendly test in the form of a cheek swab collection method, will play an important role in both the upcoming clinical program for AD04 as well as future commercial deployment," said Cary Claiborne, CEO of Adial Pharmaceuticals.
Precision Medicine Strategy
The validated genetic test represents Adial's commitment to precision medicine in neuropsychiatry. By integrating pharmacogenetic insights and validated biomarkers into the development strategy, the company aims to enhance clinical predictability for targeted therapeutics.
AD04 was recently investigated in the ONWARD pivotal Phase 3 clinical trial, which showed promising results in reducing drinking in heavy drinking patients with no overt safety or tolerability concerns. The drug is also believed to have potential for treating other addictive disorders including Opioid Use Disorder, gambling, and obesity.
Claiborne noted that the precision medicine approach, combined with recent patent filings expected to extend market exclusivity to 2045, positions AD04 as "an extremely attractive future commercial opportunity with meaningful patient impact." The company believes AD04 could become the first genetically targeted therapy for Alcohol Use Disorder, addressing a large, underserved patient population.
