BioVie Inc (NASDAQ:BIVI), the clinical-stage company specializing in drug therapies for neurological disorders, revealed new results from the Phase 3 trial of NE3107 in patients with mild to moderate Alzheimer's Disease (AD).
The trial, initiated during the COVID-19 pandemic, enrolled 439 patients across 39 sites.
Initial analysis of unblinded data from 57 per-protocol patients suggests that NE3107 demonstrates biological activity and may impact cognitive, functional, and biomarker endpoints.
Patients treated with NE3107 showed improvement compared to placebo on cognitive and functional assessments, outperforming expectations.
“These data show NE3107’s treatment advantage over placebo to potentially be equal to or greater than data reported from clinical trials for the approved medications for AD without the associated safety concerns,” said Cuong Do, BioVie’s CEO.
“The adaptive trial design gives us the flexibility to continue patient enrollment in the advancement of this potentially important treatment for AD, and we look forward to discussing our findings of NE3107’s magnitude of therapeutic impact with our potential partners.”
Despite positive signals, issues related to significant Good Clinical Practice (GCP) violations and protocol deviations emerged at 15 sites, mainly from one geographic area. As a response, the company excluded data from these sites and referred them to the Food and Drug Administration (FDA) for further investigation.
These exclusions led to the trial missing statistical significance for the primary efficacy endpoint, according to a statement from the company. The adaptive trial feature may allow continued enrollment to reach statistical significance for registrational purposes.
BioVie said it took swift action upon identifying protocol deviations, modifying the Statistical Analysis Plan (SAP) and amending the study protocol to address the issues responsibly.
Do told shareholders that he is “very proud” of the team for taking immediate action to identify and report the potentially problematic sites to the FDA for independent investigation.
“Importantly, we recognize that along with the development of new and innovative therapeutics, our foremost responsibility in clinical testing is to protect the rights and well-being of study patients and the integrity of the clinical research process.”
The company has also retained a new Contract Research Organization (CRO) for future trials.