Clinical-stage biotech BioVie has seen its Phase III Alzheimer’s disease (AD) study impeded by Good Clinical Practice (GCP) violations and protocol deviations as it announces efficacy results.
The topline efficacy data from the Phase III study (NCT04669028) demonstrated that NE3107 showed an advantage over the placebo group in patients living with mild to moderate AD.
However, the trial’s results have been significantly impacted by what the sponsor has called a series of ‘significant deviations’ from established protocol and GCP guidelines, resulting in data from only 81 out of the initially enrolled 439 patients being taken as valid.
BioVie said that upon trial completion, it found violations at 15 out of 39 sites. The company has now referred all of these sites to the US Food and Drug Administration’s (FDA) Office of Scientific Investigations (OSI) for further investigation. BioVie said that the violations all occurred within the same unnamed geographic area.
BioVie president Cuong Do said: “I am very proud of the integrity our team displayed in taking immediate action to identify and report the potentially problematic sites to the FDA for independent investigation.”
The trial was launched during the Covid-19 pandemic in August 2021, which restricted patient enrollment. The last patient’s final visit concluded in September 2023.
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The biotech has employed Pentara, a specialised biostatistics consulting firm, to assist with data examination.
Pentara’s CEO Suzanne Hendrix said: “The unblinding of topline efficacy data from the trial confirmed an unusual pattern we saw with the blinded data – that patients in a particular demographic group within the trial seemed to have a data pattern different from historical evidence for this demographic group.
“Patients from this demographic group in this trial reportedly experienced cognitive improvements that were improbable scientifically, and inconsistent with the pathology of this disease.”
Despite these setbacks, NE3107-treated patients with mild or moderate AD showed improved performance compared to the placebo group on all cognitive and functional assessments.
While patients in the placebo group saw worsening cognitive test results as their condition progressed, patients treated with NE3107 experienced a treatment advantage after six months, which was similar to other currently approved treatments for AD.
GlobalData’s Pharmaceutical Intelligence Centre currently estimates that NE3107 could see sales of $35m by 2025, with that figure expected to rise to potential sales of $112m by the end of 2029.
GlobalData is the parent company of Clinical Trials Arena.
The relationship between sponsors and clinical research organisations (CROs) is often plagued by numerous difficulties. At the recent Outsourcing Clinical Trials conference in Copenhagen, Denmark, representatives from sponsors sat down with staff from CROs for a series of talks on how to avoid allowing strained relationships to lead to trials running into difficulties.