April 28, 2020—(BRONX, NY)—A compound originally discovered by Albert Einstein College of Medicine’s Vern Schramm, Ph.D., is being evaluated by BioCryst Pharmaceuticals, Inc., in Brazil as a treatment for COVID-19 patients. The randomized, double-blind, placebo-controlled trial is funded by the National Institute of Allergy and Infectious Diseases.

The compound, BCX4430, resulted from Dr. Schramm’s long-term collaboration with scientists at the Ferrier Research Institute of Victoria University of Wellington, N.Z., to develop compounds called transition state inhibitors. BCX4430 was one of several of Dr. Schramm’s compounds licensed to BioCryst Pharmaceuticals, Inc.

BioCryst subsequently discovered that BCX4430 showed promise against RNA viruses (i.e., their genetic information is stored in the form of RNA rather than DNA). BioCryst has been developing the drug, now called galidesivir, for use against a number of viruses and announced the start of the COVID-19 trial in an April 9 press release.

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BioCryst’s animal studies have shown that galidesivir can prevent some of the world’s most dangerous pathogens from multiplying, including Ebola, Marburg, Zika, and yellow fever. In laboratory studies, the drug has demonstrated activity against more than 20 RNA viruses in nine different families, including the coronaviruses that caused the MERS and SARS outbreaks.

“Since COVID-19 is also caused by a coronavirus, there’s reason to hope that galidesivir can help in treating people sickened by this pandemic,” said Dr. Schramm, professor and Ruth Merns Chair in Biochemistry at Einstein. He noted that galidesivir has already been found safe when administered both intramuscularly and intravenously (IV) to healthy human volunteers.

Galidesivir acts as a lethal Trojan Horse after it’s absorbed by virus-infected cells. Viral enzymes unwittingly use galidesivir molecules as building blocks for constructing new viral RNA, cranking out defective RNA and bringing viral replication to a halt.

“We have begun to see COVID-19 cases in Brazil, and we have a good opportunity to enroll and treat patients earlier in their disease course to determine if galidesivir can benefit patients with COVID-19,” said Dr. Esper Kallas, professor of medicine at the University of São Paulo and the clinical trial’s principal investigator. Dr. Kallas was quoted in the BioCryst press release cited above.

The first part of the Brazilian trial, which is now enrolling 24 hospitalized adults with confirmed COVID-19, is aimed at finding an optimal dosing regimen for galidesivir. Three cohorts of eight patients in a 2:1 ratio will receive different IV doses of galidesivir or placebo every 12 hours for seven days.

Since COVID-19 is also caused by a coronavirus, there’s reason to hope that galidesivir can help in treating people sickened by this pandemic.

Vern Schramm, Ph.D

Part two of the trial will enroll up to 42 hospitalized COVID-19 patients who will be randomized 2:1 to receive either the optimal IV dose of galidesivir or placebo. Measures of effectiveness will include time until clinical improvement, time until discharge from hospital, time until the virus is no longer detectable, and mortality from all causes.