BioCryst Pharmaceuticals, a small drug developer in Durham, raised its profile in the global fight against Ebola on Wednesday by announcing a $4.1 million award in federal research funds to develop a potential treatment for the deadly virus that has claimed more than 1,000 lives in its latest outbreak.
The funding from the National Institute of Allergy and Infectious Diseases will be used to test BioCryst’s experimental antiviral drug, which is known by the code BCX4430.
The antiviral will be tested for safety in humans and for effectiveness in primates as a treatment for the lethal family of viruses called hemorrhagic fever viruses, of which Ebola is the most feared. The National Institute of Allergy and Infectious Diseases is part of the National Institutes of Health.
People infected in the current Ebola outbreak will not be used as test subjects for BioCryst’s drug, but the monkeys in the experiments will be infected, said Serge Belanger, a New York analyst with Needham & Co.
“More likely the next steps in development include evaluating the efficacy of BCX4430 against the Ebola virus in non-human primates,” Belanger said in a research note.
The BioCryst drug is already being developed as a treatment for a related virus, Marburg, under a 5-year federal research contract for up to $22 million. The $4.1 million release represents an installment payment on last year’s award, bringing the total paid out to $13.5 million in less than a year.
Now BioCryst’s Marburg research will include Ebola, which has no known treatment or cure. BioCryst did not comment on the grant Wednesday but highlighted the Ebola link in its announcement.
“The ongoing Ebola epidemic in West Africa emphasizes the urgent need for safe and effective antiviral agents for hemorrhagic fever virus diseases,” William Sheridan, BioCryst’s chief medical officer, said in a statement.
One of the challenges of fighting Ebola is that it comes in five different species, four of which are deadly to humans. But BCX4430 has demonstrated at least some effectiveness against a number of deadly viruses, including Marburg and Ebola.
“We look forward to the body of evidence supporting BCX4430 evolving into a highly compelling package, enabling us to attract U.S. Government advanced development funding,” Sheridan said in the statement. “This is critical so that a new drug application can be filed as quickly as feasible for this unique, broad spectrum antiviral.”
In March, the journal Nature published a favorable report about BioCryst’s drug, noting that BCX4430 was successful in treating mice infected with Marburg and Ebola virus as well as macaque monkeys infected with Marburg, both within the filovirus family.
“Most importantly, BCX4430 completely protects cynomolgus macaques from Marburg virus infection when administered as late as 48 hours after infection,” Nature’s report said. “This is the first report, to our knowledge, of non-human primate protection from filovirus disease by a synthetic drug-like small molecule.”
Cell Research, another journal, described the results in April.
“Clinically useful treatments for Ebola and Marburg viruses have been elusive and until now, potential post-exposure therapies have faced practical, regulatory, safety and/or cost obstacles,” according to Cell Research. “If BCX4430 has minimal side effects and is cost-effective, it could be widely administered to high-risk individuals in an outbreak setting.”
BioCryst, a 24-year-old company that employes 40 people, doesn’t have a drug on the U.S. market. Its stock closed Wednesday at $13.82, up 43 cents.
As part of the $22 million research project, BCX4430 is on schedule to be evaluated for safety on human subjects in 2015, but it is considered to be several years from being available on the market.
BioCryst is developing BCX4430 as a multi-virus treatment in collaboration with the U.S. Army Medical Research Institute of Infectious Diseases. U.S. defense authorities are developing a viral killer to stockpile to protect populations against a natural epidemic or a bioterror attack.
The experimental anti-viral is designed to stop viruses from replicating in their human hosts. The lead compound in BCX4430 has demonstrated “broad-spectrum activity” against more than 20 RNA viruses, BioCryst said.
“BCX4430 appears to be an ideal product candidate,” Belanger wrote, “as the agency is intent on moving away from its historical ‘one bug, one drug’ approach, towards more broad-based therapeutic agents that are effective against a broad range of diseases/viruses.”