In the current fiscal environment, everything in the U.S. budget is open to scrutiny. Fortunately for the biodefense industry, protecting the nation from bioterrorism tends to have bipartisan support, especially when potential threats get extensive news coverage.
This month alone, there have been ricin-laced letters sent to President Barack Obama, Sen. Roger Wicker and Mississippi judge Sadie Holland, and claims that the chemical weapon sarin was used during the Syrian civil war.
Funding for biodefense has gone through changes over the years, starting with Project BioShield in 2004 to stockpile products that were close to FDA approval. Eventually the Biomedical Advanced Research and Development Authority (BARDA) was formed in 2006 to fund development of biodefense and pandemic flu products.
"The government has come a long ways since 9/11, but we have a long way to go," Francesca Cook, senior vice president of policy and government affairs at PharmAthene Inc., told BioWorld Insight.
PharmAthene's second-generation anthrax vaccine, SparVax, a recombinant antigen produced in E. coli rather than toxin purified from the anthrax bacteria, has received government funding commitments of up to $213 million.
Funding through BARDA wasn't directly affected by the government's sequester because projects were funded through advanced appropriations. Funding in fiscal year 2014, though, is still up in the air.
"Given the recent events I think we will see adequate funding," Cook said. "Funding is the most visible sign of the commitment to be as prepared as we can be for attacks."
Last week, BARDA issued a request for information from companies, the starting point for funding, for developing a countermeasure for ricin.
Soligenix Inc., of Princeton, N.J., is working on a vaccine, RiVax, to induce ricin-neutralizing antibodies with funding from the National Institutes of Health. The firm has completed two Phase I trials in healthy volunteers showing RiVax is safe and immunogenic.
For potential chemical attacks by nerve agents such as sarin and tabun, Annapolis, Md.-based PharmAthene is developing a bioscavenger that binds to a broad spectrum of chemical nerve agents.
The drug, funded through the Department of Defense (DoD), is a recombinant version of butyrylcholinesterase (BChE) that is present in low levels of human plasma, so low that one 500-mg dose would require 500 liters of plasma.
The complex enzyme proved difficult to make in cell culture, so PharmAthene developed transgenic goats that were capable of producing BChE in their milk. While the project was successful and completed a Phase I trial, the DoD decided it needed considerably more BChE than first requested.
"That wasn't economical using the transgenic goat platform," explained John Troyer, PharmAthene's vice president of chemical defense product development.
So PharmAthene went back to the drawing board, eventually working out a way to express the protein in a human cell line using a new technology that Troyer wasn't able to talk about because of the benefit to national security.
"The main takeaway is that the recombinant protein is virtually indistinguishable from the protein seen in plasma. In other systems, animal cells decorate it with different sugars so the immune system could recognize it as foreign," Troyer said.
With new DoD funding to develop the recombinant BChE, PharmAthene is running pharmacokinetic tests and expects to run nerve agent challenge studies in animals starting early this summer.