The U.S. government’s top infectious disease expert said that early results of a closely watched clinical trial offered “quite good news” regarding a potential COVID-19 therapy made by the biotechnology company Gilead Sciences Inc.
Anthony Fauci, the head of the National Institute of Allergy and Infectious Diseases, which is conducting the study, said at a White House meeting with President Donald Trump and Louisiana Gov. John Bel Edwards that the trial showed a significant positive effect in treating the virus.
Early Wednesday morning, Gilead issued a news release saying it had become aware of results from the NIAID trial showing its experimental drug remdesivir helped patients recover more quickly than standard care, suggesting it could become the first effective treatment for an illness that has turned modern life inside out.
NIAID’s trial enrolled more than 1,000 patients internationally and compared remdesivir treatment alongside supportive care with a placebo. Patients who got the drug recovered in an average of 11 days, while those who got a placebo recovered in 15 days, Fauci said.
The results were “highly significant if you look at the time to recovery,” Fauci said. The U.S. Food and Drug Administration indicated it is in talks with Gilead to make the medicine available quickly. The New York Times reported ”citing a senior administration official” that the agency could grant an emergency authorization as soon as Wednesday.
The FDA “has been engaged in sustained and ongoing discussions with Gilead Sciences regarding making remdesivir available to patients as quickly as possible, as appropriate,” said Michael Felberbaum, an agency spokesman, in an email.
UAB was one of the sites of the international trial and was also involved in the initial development of remdesivir.
Dr. Paul Goepfert and Dr. Nathan Erdmann with the UAB’s Division of Infectious Diseases were part of the trial.
Goepfert was the principal investigator of the study.
“This is the first step, the first drug that we’ve tested in humans, really, that have direct anti-virus activities, so it actually targets the virus directly,” Goepfert said. “And it worked. It’s 31% effective over placebo, which doesn’t sound fantastic, but it is likely going to be saving lives and it gives the researchers direction on how to target this virus and come up with even better therapy.”
Erdmann supported enrollment in the study and oversaw the trial.
“What is most relevant to this study is that we actually know the implications of this,” he said. “So, we now have a drug that, when compared against placebo, has been demonstrated to have significant therapeutic benefits. So, we know that this is something that will actually help patients.”
Having a treatment that has been tested and found to work is an important milestone, Erdmann said.
“We now have confidence that we have at least one drug that’s available that will improve overall outcomes,” he said, stopping short of calling it a game changer just yet. “It’s not a miracle drug in that people take it and then are immediately better. So there are still many hurdles to come, but to have actual positive progress this quickly in the global outbreak is certainly encouraging.”
UAB joined the study in late March with 16 patients. Some patients continue to participate in the study.
Goepfert said the quick emergence of remdesivir as a potential treatment is surprising.
“It’s pretty incredible because I’ve done a lot of trials for a long time and I don’t think I’ve ever gotten a positive result so quickly,” he said. “This will likely become standard of care. The challenge is going to be for Gilead, the drug maker, to come up with enough doses to treat the number of people who are going to need it. I suspect that soon everybody that gets hospitalized with severe infection, the doctors are going to want this drug to treat them right now.”
Remdesivir is also providing hope, Goepfert said.
“This is a very exciting time and I think it’s very hopeful,” he said. “It certainly isn’t a home run at this point, but it is extremely hopeful. At least now we have something that can make a change in somebody’s life who has severe coronavirus infection.”
Goepfert said the FDA can either approve remdesivir or allow it to continue to be used on a “compassionate use” basis.
Finding a treatment for COVID-19, the respiratory disease caused by the coronavirus, could move the world closer to easing lockdown measures put in place to help slow its spread. News of the positive results helped send the U.S. stock market soaring Wednesday despite data showing a deep contraction in the economy in the first quarter of the year.
Gilead shares jumped as much as 8.1% Wednesday after the company’s statement.
The broader stock market and Gilead itself have been churned in recent weeks by a series of early looks and leaks of trial data on remdesivir. Last week, the market swooned after apparently discouraging results from a Chinese trial that was halted early after researchers’ struggles to enroll patients were accidentally posted on a World Health Organization web page.
Data confirming those more downbeat results were published in the U.K. medical journal The Lancet on Wednesday. Fauci said at the White House Wednesday that that trial was “not an adequate study.”
Originally developed to treat other novel viruses, remdesivir has placed Gilead at the head of the race to develop a treatment for COVID-19. The drug, which has been tested on Ebola, isn’t approved for use anywhere in the world.
A separate study of 397 patients released by Gilead Wednesday found remdesivir appears equally effective when given for half as long as the 10 days used in the NIAID and other studies, and that some patients may fare even better when given less medication. The potential ability to treat patients for less time would help ease strain on stretched health systems that must infuse the medicine, and make supplies of the drug last longer.
Two weeks after treatment, 65% of those who got the five-day dose had recovered, compared with just 54% of those who received the infusion for 10 days. The death rate showed a similar trend, with 8% of patients getting less drug dying, compared with 11% in the 10-day group. The difference wasn’t statistically significant, which means it could have happened by chance.
The five-day regimen “could significantly expand the number of patients who could be treated with our current supply of remdesivir,” said Merdad Parsey, Gilead’s chief medical officer.
UAB’s Goepfert said the study grew from that original 400 to more than 1,000 because the coronavirus spread so quickly around the world. That created the opportunity for a study on this scale.
“This is probably unprecedented,” he said. “I think in some ways we’re a bit lucky to be able to do this.”
Taken together, Gilead’s study and the NIAID trial could signal a profound shift in the race to get the novel coronavirus under control. The availability of a treatment could allow the world to start reopening economies, as well as offer psychological relief to billions of people who have been self-isolating to hide from the virus.
In the most severe cases, COVID-19 can lead to respiratory failure and death. More than 1 million cases have been confirmed in the U.S., and over 200,000 people have died around the globe from the illness.
Multiple trials of the drug are still underway. The NIAID trial measured how quickly patients taking it were able to be removed from supplemental oxygen therapy or were discharged from the hospital. The agency plans to publish the findings in a medical journal in the near future, Fauci said.
Remdesivir will likely be approved for use in certain settings on the basis of the positive result, said Jefferies health-care strategist Jared Holz, at least until better alternatives are available.
“There is a massive bias to look at the data with half-glass-full approach,” he said, adding that it’s “totally fair given the current predicament.”
UAB’s Goepfert said, “This drug has the potential to prevent infection as well,” and he said patients who take remdesivir in early onset of infection may reduce the severity of COVID-19. Those are among the areas that future studies may test, he said.
The next phase of the trials will also see what drugs are effective working with remdesivir, UAB’s Erdmann said.
“What we’re already building in place for the next phase of study … we’re actively changing the arms to eliminate the placebo and then be able to transition to adding on additional agents along with remdesivir now that we have a new baseline of how to manage patients,” he said. “I anticipate various iterations of this over the next weeks to months as the pandemic sort of waxes and wanes.”
The results from the Chinese study in The Lancet have been long-anticipated. The trial was originally meant to enroll 761 patients in Wuhan, the early center of the country’s outbreak, but researchers couldn’t find enough people after its epidemic ebbed in early March.
Still, the results from 237 adults in the randomized trials, in which neither investigators nor patients knew who was taking the real drug and who was taking a placebo, were seen by some as a setback for the drug.
Erdmann said the small number of patients in the Chinese study didn’t provide the data that are becoming available through the much larger NIAID study.
Last week, the WHO prematurely and accidentally published results of the China trial but retracted it soon after. The WHO post showed that the drug didn’t show benefit for these patients in terms of preventing death and reducing virus load.
About 100 clinics around the world worked together on the NIAID study, with a central goal of seeing whether remdesivir could help more patients recover more quickly after 29 days of followup.
Unlike the Chinese trial, which was running even as strict containment measures were clamping down on the numbers of infected people, NIAID’s study had the benefit of casting a net for patients just as their numbers were exploding. After initially expecting to enroll 572 patients, researchers later said the total sample size could be more than 800 people.