Veru has lost a split decision at its FDA advisory committee meeting, with experts voting eight to five against the known and potential benefit-risk profile of VERU-111 in hospitalized moderate to severe COVID-19 patients who are at high risk for acute respiratory distress syndrome.
The chances of Veru securing a clear win at the meeting took a hit when the FDA published its briefing document. While the agency acknowledged that Study 902 met its primary endpoint of all-cause mortality at Day 60, it “identified a number of uncertainties with the data” from the “small trial,” including high placebo arm mortality rate, potential for unblinding events and baseline imbalances in standard of care.
Those uncertainties shaped the discussion at the advisory committee. Ultimately, the vote went against Veru by eight experts to five, but the comments revealed uncertainty on both sides of the fence, as Brigham and Women’s Hospital’s Lindsey Baden, M.D., articulated in the explanation of his vote.
“I agree with all of the previous voters, both yes and no. We're all on the edge of how do we weigh the efficacy signal and the absence of a safety signal, but the absence of safety data?” Baden said.
While Baden voted yes, he agreed with the arguments of other experts who voted no. Daniel Gillen, Ph.D., from the department of statistics at the University of California, Irvine set out the case against VERU-111, also known as sabizabulin.
“Given the data that we have currently, I believe we have a limited both efficacy and safety data set with a new molecule where we don't have a full understanding of the mechanism of action,” Gillen said. “I don't know if we’re ever guaranteed to know that completely, but certainly we're far from it at this point.” Gillen voted no.
The National Institutes of Health’s Daniel Chertow, M.D., voted yes but expressed reservations about the data, concluding that there are neither clearly known benefits nor clearly known risks. In the context of the patient population, Chertow said the potential benefits outweigh the potential risks and, with the FDA asking the experts to consider the “known and potential” risks and benefits, that led to the yes vote.
In the context of the emergency use authorization sought by Veru, potential benefits could be enough to get VERU-111 over the line at the FDA given the unmet medical need in COVID-19 patients failed by standard of care.