Durham company to provide drug to sick boy

Mar. 11, 2014 @ 08:52 PM

A family’s call for Chimerix Inc. to give their ill 7-year-old boy an experimental drug got national attention this week.

And on Tuesday night, after voicing reluctance to do so, the company relented.

Wednesday, Chimerix will make the drug available to Josh Hardy through a new pilot study, company officials said.

Josh Hardy is hospitalized at St. Jude Children’s Research Hospital in Memphis with adenovirus infection, which he contracted after receiving a bone marrow transplant. According to multiple media accounts and information posted on Facebook, the transplant was part of a treatment for cancer.

His parents, Aimee and Todd Hardy of Fredericksburg, Va., did interviews with national media outlets such as CNN and Fox News in their quest to get an antiviral drug under development by Durham-based Chimerix Inc. for their son.

The drug, called brincidofovir, is not yet approved by the U.S. Food and Drug Administration. But the family and others had urged the company to make the drug available through the FDA’s expanded access or “compassionate care” program.

According to information from the FDA, “compassionate use” allows patients with serious or life-threatening diseases who have exhausted currently available treatment options to access experimental drugs outside of clinical trials. Drug companies must be willing to provide an experimental drug to the patient and to the doctor.

The company was not planning to release the drug through the compassionate use route. Duane Adams, a family friend, said he was part of an effort to gather several hundred people outside Chimerix’s headquarters in Durham on Thursday to urge the company to do so. In addition, thousands of people had supported their campaign on Facebook.

There was a time when Chimerix made the drug available through compassionate use, a company spokesman said in an email, and 430 people got it that way.

But in an emailed statement, Chimerix CEO Ken Moch said that making the drug available through compassionate use had the potential to slow the drug’s development. In the past several years, Chimerix has received hundreds of requests for the compassionate care use of brincidofovir, he said.

However, later in the evening Tuesday, the company announced that it had reached an agreement with the U.S. Food and Drug Administration for the immediate launch of a pilot trial of open-label brincidofovir for the treatment of adenovirus infections in patients with compromised immune systems. Josh Hardy was expected to be the first patient enrolled in the study on Wednesday.

“Josh Hardy's story brought to public attention the often-devastating impact of adenovirus infection, and helped accelerate a discussion between the FDA and Chimerix regarding the need for additional clinical development to assess brincidofovir's potential in adenovirus infection,” the release said.

St. Jude confirmed in its own release that the company was expected to send brincidofovir to the hospital within 48 hours so the drug could be used to treat Josh’s adenovirus. Because the drug is experimental and not approved by the FDA, the drug would be administered under a new treatment protocol.

“St. Jude will continue to pursue state-of-the-art treatment for Josh and all of our patients,” the statement said. “We are grateful for the efforts of Chimerix, the FDA and many others who worked to achieve this outcome. We ask that you continue to keep Josh and his family in your thoughts.”

Chimerix’s statement also said the FDA committed to “work expeditiously” with Chimerix to design a full Phase 3 study to continue the pilot.

The company already is testing brincidofovir in Phase 3 trials to see if it’s safe and effective against cytomegalovirus in patients who received bone marrow transplants. The company also already finished an exploratory Phase 2 trial for brincidofovir as a pre-emptive treatment for adenovirus in bone marrow transplant patients.

Moch said in the statement that 20 patients will participate in the open-label study announced Tuesday, and it will inform the drug’s use in adenovirus and other serious DNA viral infections.

“We are pleased to be providing access to brincidofovir in a manner consistent with our focus on progressing clinical development toward a potential regulatory approval that would make it widely available to patients who might benefit from its use,” said Dr. Herve Mommeja-Marin, vice president of clinical research for Chimerix, in a statement. “We are grateful to the FDA for their continuous guidance and assistance in expediting brincidofovir's development path forward.”