Adial Pharmaceuticals Achieves Key Milestone With Analytical Validation Of Genetic Test For AD04 Phase 3 Trials

Adial Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company focused on developing innovative treatments for addiction and related disorders, has announced the successful completion of the analytical validation for a cheek swab-based genetic testing method. This advancement represents a major step toward FDA registration of its lead candidate, AD04, which targets Alcohol Use Disorder (AUD) in genetically defined patient populations.

“We continue to make progress in preparation for the next phase of Adial’s journey,” commented Cary Claiborne, CEO of Adial. “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. The ease of the test collection method allows for seamless identification of patients best suited to AD04 therapy, through a precision health platform designed with the patient in mind. A simple cheek swab test kit will assist in future commercial strategies for both physician led promotion as well as at home sample collection. The simplicity allows for testing and results to be reported within a few days. With regulatory alignment on key protocol elements, including patient population, efficacy endpoints, biomarker stratification, and adaptive enrichment strategies-we are now positioned to advance a streamlined and scientifically rigorous program designed to maximize the probability of success.”

The validated test, developed in collaboration with Genomind, enables the identification of key single nucleotide polymorphisms (SNPs) within the serotonergic system—specifically the HTR3A, HTR3B, and SLC6A4 genes. This test is intended for both clinical trial use and future commercial deployment, allowing for easy cheek swab collection in clinical settings or at home. The validation confirmed the test’s accuracy, reproducibility, and robustness across diverse samples, instruments, and reagent lots, with 100% concordance observed in SNP testing.

He also said, “This milestone also demonstrates our commitment to precision medicine. By integrating pharmacogenetic insights and validated biomarkers into our development strategy, we are enhancing clinical predictability and believe we have set a new benchmark for targeted therapeutics in neuropsychiatry. This progress, coupled with the recent announcement of our new patent filing for AD04, expected to extend market exclusivity to 2045, frames the AD04 program as an extremely attractive future commercial opportunity with a meaningful patient impact. We remain focused on delivering a transformative solution for patients living with AUD.”

The development aligns with FDA guidance provided during Adial’s recent End-of-Phase 2 (EOP2) meeting. The FDA also confirmed that use of this genetic test in the upcoming Phase 3 study is a Non-Significant Risk (NSR) activity, eliminating the need for an Investigational Device Exemption (IDE) filing. The Phase 3 program will focus on biomarker-positive (AG+) patients—representing approximately 14% of the population —who are most likely to respond to AD04 therapy. The company’s progress positions it to advance rapidly toward registration and commercial readiness for a precision-based treatment in Alcohol Use Disorder.

“As we advance toward Phase 3, we believe we are executing from a position of strength across regulatory, clinical, and manufacturing fronts. This is also a key element in our strategy to secure strategic partners who have indicated the importance of a simple solution to testing with an easy-to-execute SNP biomarker test. Through disciplined execution, we believe AD04 is positioned to become the first genetically targeted therapy for AUD-addressing a large, underserved patient population and unlocking a highly attractive commercial opportunity,” concluded Mr. Claiborne.