For the second time this year, Chimerix, a small drug development company in Durham, has attracted national attention by releasing its promising experimental drug to a gravely ill patient at risk of dying.
This time, the high-profile recipient is Thomas Eric Duncan, the Liberian man who fell ill with Ebola last month and is now fighting for his life in Texas. Duncan began taking the Chimerix medication, which is not yet approved for general public use, on Saturday at 2:14 p.m., the Texas Health Presbyterian Hospital in Dallas confirmed Monday.
The emergency request from Duncan’s doctors was granted by the U.S. Food and Drug Administration’s Emergency Investigational New Drug Applications branch. The program supplies experimental treatments to patients with life-threatening conditions for which there are no medical options.
Chimerix also said Monday that it is working with the FDA to develop a drug trial that will administer, for the first time, its antiviral drug to people stricken with Ebola, almost all of whom currently live in Africa, where more than 3,400 people have died from the viral illness this year.
The emergency release of the drug to Duncan looks like a do-over of Chimerix’s ill-fated move this spring to initially withhold the treatment from then-7-year-old cancer survivor Josh Hardy. Chimerix quickly relented and released the drug to the ailing boy, but not until the company was vilified on social media and its CEO faced death threats.
The parallels between the dual scenarios break down, however, at the sheer magnitude of the Ebola epidemic that is roiling West Africa and could infect as many as 1.4 million people by January, according to a forecast by the U.S. Centers for Disease Control and Prevention.
Now the question turns to how the 75-employee company would make the drug available on a wider scale in dire emergencies.
Tablet form easy to ship
The company’s treatment, called brincidofovir, is at least two years from approval for public use. Yet it has proven so promising in fighting viruses that some suggest it may emerge as a leading candidate in the global fight against the deadly Ebola virus, for which there is no approved treatment or cure.
“Once efficacy is demonstrated in either infected monkeys or humans, brincidofovir could be used in Ebola patients broadly and Chimerix could receive stockpiling orders from various governments,” analyst Y. Katherine Xu of the William Blair & Co. firm wrote in a research note Monday.
“Furthermore, brincidofovir comes in a tablet form and is ready to ship for use and storage around the world immediately,” Xu wrote. “These attributes confer significant advantage to brincidofovir compared to some other approaches being developed for Ebola.”
Chimerix’s director of investor relations, Joseph Schepers, said potential dissemination of the drug into Ebola hot zones is under review, and noted that Chimerix was invited to two World Health Organization summits in the past two months to discuss potential strategies to thwart the spread of Ebola.
“We’re working with global organizations around the world on this Ebola outbreak,” Schepers said. “These things are in discussion now.”
Schepers declined to provide other details as to the available quantities of the drug, or how quickly it can be produced, except to say “we have enough for clinical trials.” The company is in the midst of testing the drug on another dangerous virus with a pool of 450 people, 300 of whom would take the brincidofovir, and the rest would take a placebo.
If brincidofovir proves effective for Duncan, whose condition has been deteriorating, Chimerix stock could spike again on the promise of a lucrative payoff down the road.
At the same time, Chimerix could be subject to another round of ethical scrutiny as it deliberates how and when to make the antiviral medication available, and who would pay to have it distributed in Africa, where more than 7,000 have been infected to date.
Given the potential scale of the health crisis, James Hodge, a professor of public health law at Arizona State University, predicted that international humanitarian organizations would likely get involved, or the U.S. government could purchase a stockpile for international distribution to stem a global health emergency.
But Philip Rosoff, director of clinical ethics at Duke University Hospital in Durham, warned that every course of action, or inaction, will have ethical perils for an American pharmaceutical company.
“The history of drug development is littered with examples of drugs that looked good on paper, or in computer simulations, or in test tubes, and then failed miserably either for too much toxicity or for lack of efficacy when put into people,” Rosoff said. “We have to be very careful that we’re not doing experiments on Africans who are dying without the precautions that we normally use in human investigations.”
Shareholders have not been hampered by moral indecision, pushing Chimerix stock to $31.47 Monday, up $1.41 a share. The stock is up 108 percent this year on a string of good news about brincidofovir.
Other antidotes possible
In the race to stop the Ebola outbreak, potential antidotes have been hindered by liabilities. A drug from Japan’s Tekmira Pharmaceuticals requires more safety testing and is administered by injection, Xu wrote. A treatment from Mapp Pharmaceuticals is produced from tobacco plants, and supplies have been depleted.
BioCryst Pharmaceuticals, also in Durham, is testing BCX 4430 on Ebola-infected monkeys and plans to apply to the FDA for human drug trials this year. The BioCryst antiviral has to be administered intravenously.
Brincidofovir, a simple pill, is being tested for a variety of potent viruses, including adenovirus, a common cold virus that is potentially lethal for patients who have compromised immune systems.
Josh Hardy, the four-time cancer survivor from Virginia, was infected with adenovirus after a bone marrow transplant and was slipping in and out of consciousness in March when his parents made a public plea for their son. After a regimen of brinicidofovir, Josh recovered and has since left the hospital.
Since 2011, the federal Biomedical Advanced Research and Development Authority has funded $22.3 million in research to develop brincidofovir as a measure against smallpox spreading as a result of accidental release or from a bioterror attack.
The drug has proven effective against Ebola in laboratory petri dishes and is now being tested on monkeys infected with the virus.
Last week, a Wall Street analyst said brincidofovir is an excellent candidate to be stockpiled by the U.S. government for a range of viral epidemics. The analyst, Joseph Schimmer of the Piper Jaffray firm, touted the drug’s safety record, based on more than 1,000 people having taken brincidofovir safely in drug trials and compassionate care program distributions in past years.
“We’re saying that it’s shown activity against multiple viruses,” Schepers said. “Cytomegalovirus, adenonvirus, smallpox and now Ebola.”