In May 2021, BioCryst Pharmaceuticals terminated a phase Ib trial due to their decision to no longer pursue indications studied in this trial (NCT03891420; BCX4430-108; DMID18-0022). The randomised, double-blind, placebo-controlled trial was initiated in April 2020 to evaluate the safety, clinical impact and antiviral effects of galidesivir in patients with COVID-19 infections. The trial enrolled 24 patients in Brazil. The trial protocol is approved by the Agência Nacional de Vigilância Sanitária (ANVISA) and the Brazilian National Ethics Committee (CONEP). Part 1 of the trial will enroll 24 hospitalized adults diagnosed with moderate to severe COVID-19. In part 2 of the trial, up to 42 hospitalized patients with COVID-19 will be randomized 2:1 to receive intravenous galidesivir or placebo   . In November 2020, the patients enrollments were completed in the trial  . In December 2020, BioCryst Pharmaceuticals released results form the part 1 of the trial. The company reported that, trial was not designed or sized to demonstrate clinical efficacy, and no clinical efficacy benefit with galidesivir treatment compared to placebo treatment was observed in the trial    .
As of March 2020, phase II development of galidesivir was underway for the treatment of yellow fever under contract with the National Institute of Allergy and Infectious Diseases (NIAID) and the US Department of Health and Human Services (HHS)  .
In May 2019, BioCryst Pharmaceuticals completed a phase I trial that evaluated the safety, tolerability and pharmacokinetics of escalating doses of galidesivir, following administration of single doses (5mg/kg, 10 mg/kg, 15 mg/kg and 20 mg/kg) by intravenous infusion, in healthy volunteers (NCT03800173; BCX4430-106; DMID18-0013; 272201300017C-18-0-1). The randomised, placebo-controlled, double-blind trial was initiated in December 2018, and enrolled 24 participants in the US. Earlier, in January 2019, the first patient was dosed. In May 2019, the company reported that the trial outcome was successful. During the trial, galidesivir was generally safe and well tolerated     .
In May 2016, BioCryst reported that it has completed a phase I trial which investigated the safety, tolerability and pharmacokinetics of escalating doses of galidesivir in healthy volunteers (BCX4430-101, DMID 14-0030; NCT02319772). The trial was initiated in December 2014, and enrolled 94 participants in the UK. In part 1, volunteers received a single dose of galidesivir (up to six cohorts) and in part 2, volunteers received galidesivir for 7 days (four cohorts). The trial was funded by the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health in the US. Galidesivir was generally safe and well tolerated through doses up to 10 mg/kg daily for seven days    .
BioCryst, in October 2017, reported positive data from a preclinical study of intramuscular galidesivir in rhesus macaques models of zika virus infection and ebola virus infection. The data was also presented at the IDWeek 2016 (IDW - 2017)  .
In August 2017, BioCryst announced that it will conduct an additional non-clinical efficacy study in a delayed treatment setting in Ebola virus infections before finalising the protocol for a phase I trial, based on discussions with the FDA, BARDA and NIAID  .
BioCryst Pharmaceuticals, in September 2016, released favourable data from a 28-day proof of concept study that evaluated galidesivir for the delayed treatment of Ebola virus infections in NHP models  .
In November 2014, BioCryst initiated non-human primate studies (NHP) to evaluate the efficacy of galidesivir against Ebola virus in NHP disease models. The company also reported that a dose-ranging study was conducted in cynomolgus macaques infected with Ebola. In December 2014, the company reported results from a proof-of-concept study in rhesus macaques infected with Ebola. The study was funded by the National Institute of Allergy and Infectious Diseases (NIAID) and was conducted at the United States Army Medical Research Institute of Infectious Diseases  .
BioCryst, in October 2016, reported data from a preclinical study that demonstrated favourable efficacy and safety of intramuscular galidesivir in rhesus monkeys infected with Zika virus  . The data was also presented at the IDWeek 2016 (IDW - 2016)  .
In May 2016, BioCryst reported that in collaboration with Utah State University and NIAID, preclinical development of galidesivir in a model of Zika virus infection in interferon-receptor-deficient mice is underway in the US. Studies showed improved survival with dosing delayed up to 7 days after virus challenge, reduction in viral titer in the blood and development of protective immunity in surviving animals  .
BioCryst has identified galidesivir as a lead candidate for the treatment of yellow fever, under the NIAID Animal Models of Infectious Disease Program. In November 2012, the company announced positive proof-of-principle pharmacodynamics data for galidesivir   . It has been shown to be active against more than 20 RNA viruses in nine different families, including filoviruses, togaviruses, bunyaviruses, arenaviruses, paramyxoviruses, coronaviruses and flaviviruses  .
BioCryst reported in September 2013 that galidesivir is also being developed for the treatment of Marburg virus disease in addition to Ebola, under a contract with the NIAID. In December 2013, the NIAID exercised an option to conduct the IND enabling programme and ultimately conduct a phase I trial of intravenous and intramuscular formulations of galidesivir. BioCryst received a total of $US7.5 million; the award could total up to $US22 million over 5 years   .
In February 2014, BioCryst announced the publication of preclinical efficacy results for galidesivir, in animal models of Marburg virus and Ebola virus infection, in the journal Nature. The publication represents the first report of protection from filovirus disease in non-human primates by a small molecule drug. In cynomolgus macaques, the administration of galidesivir by intramuscular injection resulted in complete protection from Marburg virus, 48-hours post-infection. Protection against Marburg virus and Ebola virus infections was also demonstrated in rodents. In addition, galidesivir has shown potential in vitro against a broad range of other RNA viruses, including the emerging Middle East Respiratory Syndrome Coronavirus (MERS-CoV)  .
In December 2012, BioCryst announced the company's strategic restructuring with focus on development of antiviral programmes, including the broad spectrum antiviral, galidesivir, and BCX 5191 for HCV  .
In November 2019, BioCryst Pharmaceuticals announced the completion of the underwritten public offering of 43,620,690 shares of its common stock. The net proceeds from the offering will be use for general corporate purposes, that may include, but are not limited to, worldwide development, manufacturing, regulatory and commercial activities for the prophylactic berotralstat program, primarily focusing on the U.S., EU and Japan; development of the BCX 9930 program; development of the BCX 9250 program; post-approval commitments for RAPIVAB®/ALPIVABTM; [see ADIS insight drug profiles800042435, 800040653 , 800056474, 800010752, respectively] funding clinical development of pipeline assets; and capital expenditures and other general corporate expenses  .
In August 2020, the National Institute of Allergy and Infectious Diseases (NIAID), awarded BioCryst a new contract 75N93020C00055 totalling of $US44 million and added an award of approximately $US3 million to its existing contract HHSN272201300017C, to support the development of galidesivir  . In December 2020, BioCryst Pharmaceuticals reported that, they expect NIAID/HHS to continue its support for the development of galidesivir with a focus on biodefense threats, such as Marburg virus disease, and to discontinue the pursuit of a COVID-19 indication for galidesivir  . In September 2018, National Institute of Allergy and Infectious Diseases (NIAID) granted BioCryst an additional funding of $US3.5 million to support clinical trials of galidesivir in patients with yellow fever. The additional funding brings the NIAID contract amount to $US43.0 million. The NIAID, in July 2016, awarded an additional funding worth $US5.5 million, to support the development of galidesivir for the treatment of haemorrhagic fever viruses. In September 2013, the NIAID had awarded a contract to BioCryst Pharmaceuticals, for the development of galidesivir for the treatment of Marburg virus disease and other filoviruses, including Ebola virus disease. The funding of up to $US26.3 million over five years, if all options are exercised, will support early stage development of galidesivir, including non-human primate proof-of-concept studies, as well as a phase I study in healthy subjects    .
In June 2014, BioCryst was awarded an option funding to conduct development of an IV formulation of galidesivir including, pre-formulation, stability studies and manufacture of the drug candidate, under this contract. NIAID exercised two additional options valued at $US4.1 million, under this contract in August 2014, during the time of the Ebola epidemic in West Africa. These funds will enable BioCryst to conduct efficacy studies of galidesivir in non-human primates, file an IND and conduct phase I safety trials of an intramuscular formulation of galidesivir. Later, in the same month, under the contract modification, the company was awarded with an additional $US2.4 million funds from NIAID for carrying out dose ranging efficacy study of galidesivir (IM) in non-human primates as a treatment for Ebola virus infection. BioCryst received $US4 million in September 2014, when NIAID exercised two more options, which allows for the GMP manufacturing of drug product of galidesivir. Exercising of these options increased the existing contract value by $US2 million; the grant amount revised to $US26.3 million. In November 2014, BioCryst announced that the NIAID has increased the contract value to $US28.7 million, the company also reported that it was awarded $US2.4 million in October 2014, to conduct second NHP study. An additional option was exercised by NIAID in February 2015 that provided the company with an additional $US2.7 million for submission of an IND application for intravenous galidesivir. The total funds awarded to date under the NIAID contract amounts to $US25.0 million, as at February 2015, with a potential total amount of $US29.1 million. Since March 2015, BARDA supported the continued development of galidesivir, under contract HHSO100201500007C, for the treatment for filoviruses. The total contract value to advance the program through toxicology studies and manufacturing work to support a new drug application is $US39.1 million, if all contract options are exercised       .