After finding itself in the glaring spotlight of 24-hour cable news reports--and the target of a furious social media outburst they helped inspire--little Durham, NC-based Chimerix ($CMRX) will evidently have no trouble finding the 20 patients it is recruiting for the pilot study of its antiviral that was used to defuse the situation. And it still isn't willing to provide its therapy under the FDA's "compassionate use" regulations.
Chimerix CEO Ken Moch tells FierceBiotech that as of Friday night, physicians had proposed 6 patients for the study, which is intended to develop into a Phase III trial. Brincidofovir is already in a Phase III study for patients at high risk of cytomegalovirus (CMV) infections. This new trial will add patients at risk of adenovirus infections. But aside from one more prospective pilot trial, that's going to max out its capacity.
"If historical patterns hold, as I presume they will, we have continued to receive many requests for the compassionate use of brincidofovir, for many different viruses and for many different indications," Moch wrote in an e-mail over the weekend, after I asked him if the company was still fielding appeals. "As in the past, we will sadly need to remain focused on our (now two) ongoing Phase III clinical trials. We are evaluating other potential indications for brincidofovir, and we do hope to launch at least one additional pilot trial in another indication during 2014."
The cable news shows arrived at Chimerix's doorsteps last week as word spread that the biotech had rejected a compassionate use request for its powerful, and experimental, antiviral brincidofovir for a dying 7-year-old named Josh Hardy, whose fight against cancer had left his immune system compromised and his life threatened by a common cold virus. Hardy's case became an overnight sensation. In the media circus that followed, company execs faced death threats and demands as a national audience was outraged by the company's defense: It couldn't afford to satisfy all the demands that it receives from desperate patients who believe that brincidofovir is their only hope of survival. But the pilot study announced Tuesday night allowed the company to declare that young Hardy would be the first patient treated on Wednesday morning.
Pilot studies may not offer any kind of practical alternative to compassionate use programs--unless some other company finds itself facing another patient as winsome as Josh Hardy and media coverage spurs the same kind of visceral response from a mass audience. But there's no question that Chimerix ultimately emerged from the battleground last week with a clear win. The national media attention and the quick resolution pushed Chimerix's shares up 45% for the week as investors considered the potential of a therapy that could stir up this kind of demand. Addressing an unmet medical need is a mantra in the R&D world, and there's no question now that that is exactly where Chimerix is placing its bets.
Brincidofovir is a lipid-conjugated version of cidofovir--one of Gilead's ($GILD) antivirals--that's specifically designed to amp up the antiviral impact with a pill while sparing kidneys from a toxic threat. Its ability to get into cells more effectively than cidofovir has vastly improved its punch against all 5 DNA viral infections but has also raised questions whether it could prove a threat to all rapidly growing cells, including cells in the bone marrow. That's one of the issues that has clouded its rep as a potential "miracle" therapy--as explained in detail in a piece last week from Forbes.com--which is being closely watched in the clinical studies.
Chimerix launched a Phase III study of the drug in late 2013 among immunocompromised patients at high risk of developing CMV infections following a bone marrow transplant and is looking for a readout in mid-2015. As of the end of 2013 Chimerix had burned through close to $163 million to get its pipeline to this point, with a Merck ($MRK) collaboration underway for CMX157. BARDA has been helping to fund work on brincidofovir as a counterterrorism agent that could be used against a smallpox outbreak.
The controversy helped Chimerix seal its reputation as a closely watched biotech. But now every data point, as well as the experience of every patient, including young Hardy, will be put under a microscope. That may not always be an advantage. -- John Carroll, editor-in-chief (email | Twitter)