Veru's COVID-19 drug hopeful scrapes efficacy hurdle, but questions remain

Little-known Miami biotech Veru saw its shares jump this week after reporting seemingly positive data from a small phase 2 in COVID-19 patients.

Better known for its oncology work, Veru decided to use its cancer asset VERU-111 in a phase 2 test across 40 hospitalized patients at high risk of acute respiratory distress syndrome (ARDS) from SARS-CoV-2, a common issue that heightens the risk of death from the virus.

The idea is that the oral drug has an impact on the so-called microtubules of the cytoskeleton of cells, blocking off the roads that the virus can travel.

The data showed Veru's treatment, when pitted against placebo, had a “statistically significant and clinically meaningful reduction” in the proportion of patients who are treatment failures (dead or alive with respiratory failure) with a 30% treatment failure rate in the placebo group (20 patients) compared to 5.6% in the VERU-111 treated group (18 patients) after 29 days.  

This, the primary endpoint, “represents an 81% relative reduction in treatment failures,” the biotech said, although it only just showed statistical significance, with p=0.05.

It was similar news across the subgroups in patients at high risk for ARDS; in an analysis of those aged 60 years of age and up diagnosed with COVID-19 who are at higher risk for death and respiratory failure, older patients saw treatment failures at 9% for VERU-111 versus 50% for placebo, with the p-value once again razor thin at p=0.046.

And, looking at the severity of COVID-19, an analysis of patients hospitalized and on oxygen at baseline: Treatment failures were 11% for VERU-111 versus 54% for placebo, again statistically significant, but not by much, at p=0.04.

Secondaries showed that in the intent to treat population, VERU-111 reduced the proportion of patients who died on study from 30% (6/20) in the placebo group to 5.3% (1/19) in the VERU-111-treated group (with p=0.044), an 82% relative reduction in mortality in the VERU-111-treated group.

VERU-111 also reduced the days on mechanical ventilation from an average of 5.4 days in the placebo group to 1.6 days in the VERU-111-treated group.

Based on the data, the biotech said it will talk with the FDA to run a similar but larger test in around 200 patients in a phase 3. Its stock jumped from around $11 a share to nearly $15 on the news and were up again premarket by 8%, with a market cap now of $1.1 billion.

But that phase 3 confirmation trial will need to yield stronger results than the very close to failure p-values seen in this small test. With so many drugs, whether experimental of repurposed, failing to help COVID-19 patients in hospital—with an generic steroid currently coming up best in tests—there is a continual need while vaccine rollouts drag for better medicines.  

“We are very pleased with the results of our Phase 2 trial, which demonstrated clinically meaningful reductions in relevant endpoints, including respiratory failure, days in the ICU and on mechanical ventilation and patient mortality,” said Mitchell Steiner, M.D., chairman, president and CEO of Veru.

“Due to the urgency of the global pandemic and need for more effective treatment options for patients, we are duty-bound to pursue this indication, even though it has not been the primary focus of Veru. We have the resources to conduct a phase 3 trial without impacting our cancer drugs’ clinical development. We look forward to our upcoming discussion with FDA concerning the regulatory and clinical development steps to move VERU-111 for COVID-19 forward.”

The company said the Biomedical Advanced Research and Development Authority has also already granted Veru a meeting to “discuss possible grant funding for the phase 3 study and manufacturing scale up,” should it come through its bigger test.

“We view [the] announcement as another positive for VERU stock,” analysts at Cantor said in a note to clients on the data. “VERU-111 in COVID-19 is not one of our top value drivers for VERU near to midterm, and thus if successful, could provide meaningful upside to our estimates and outlook.”