BioCryst Pharmaceuticals, Inc. has opened enrollment into a randomized, double-blind, placebo-controlled clinical trial to assess the safety, clinical impact and antiviral effects of galidesivir in patients with COVID-19. The trial is being funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health.

Galidesivir is an investigational broad-spectrum antiviral drug that was safe and well tolerated in previously reported Phase 1 trials in healthy subjects. Galidesivir has demonstrated broad-spectrum activity in vitro against more than 20 RNA viruses in nine different families, including the coronaviruses that cause MERS and SARS.

In the COVID-19 trial, efficacy measures include time to clinical improvement, time to hospital discharge, time to undetectable levels (as measured by polymerase chain reaction (PCR) in respiratory specimens) of SARS-CoV-2, the virus that causes COVID-19, and all-cause mortality.

The trial will be conducted in Brazil under a U.S. investigational new drug application, and the protocol also has been approved by the Agência Nacional de Vigilância Sanitária (ANVISA) and the Brazilian National Ethics Committee (CONEP).

“Galidesivir has been safe and well-tolerated in Phase 1 studies, and, as a potent broad-spectrum antiviral medicine, we are hopeful that we will see a benefit in patients with COVID-19. This trial is part of the scientific community’s effort to urgently find effective treatments for patients in this global health emergency,” said Dr. William Sheridan, chief medical officer of BioCryst.

“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, infectious diseases specialist and professor of medicine at the School of Medicine, University of São Paulo, and principal investigator of the COVID-19 clinical trial with galidesivir.

Part 1 of the trial will enroll 24 hospitalized adults diagnosed with moderate to severe COVID-19 confirmed by PCR. Three cohorts of eight patients will be randomized to receive intravenous (IV) galidesivir (n=6) or placebo (n=2) every 12 hours for 7 days. Upon completion of part 1 of the trial, an optimized dosing regimen of galidesivir will be selected for part 2 of the trial, based on part 1 results including safety, viral load reduction in respiratory tract secretions, improvement in COVID-19 signs and symptoms and clinical manifestations, and mortality. In part 2 of the trial, up to 42 hospitalized patients with COVID-19 will be randomized 2:1 to receive IV galidesivir or placebo. After treatment, the patients will remain hospitalized until resolution of COVID-19 symptoms allows release. All patients will be followed for mortality through Day 56.

The galidesivir development program is substantially funded with federal funds from NIAID and by the Biomedical Advanced Research and Development Authority (BARDA). Since September 2013, NIAID has supported BioCryst in developing galidesivir as a therapeutic for Ebola and Marburg viruses. Since March 2015, BARDA has supported the galidesivir development program for the continued development of galidesivir as a potential treatment for filoviruses.

Galidesivir, a broad-spectrum antiviral drug, is an adenosine nucleoside analog that acts to block viral RNA polymerase. It is in advanced development for the treatment of COVID-19, Marburg virus disease and Yellow Fever. Phase 1 clinical safety and pharmacokinetics trials of galidesivir by both intravenous and intramuscular routes of administration in healthy subjects have been completed. In animal studies, galidesivir has demonstrated activity against a variety of serious pathogens, including Ebola, Marburg, Yellow Fever and Zika viruses. Galidesivir has also demonstrated broad-spectrum activity in vitro against more than 20 RNA viruses in nine different families, including coronaviruses, filoviruses, togaviruses, bunyaviruses, arenaviruses, paramyxoviruses, and flaviviruses. BioCryst is developing galidesivir in collaboration with U.S. government agencies and other institutions.
(Source: BioCryst)