After Fraud Probe, Alexion Reports Failed Soliris Trial
December 22, 2016
By Alex Keown, BioSpace.com Breaking News Staff
NEW HAVEN, Conn. – The bad news keeps coming for Alexion Pharmaceuticals . The company continues to grapple with allegations of improper sales practices for its blood-disorder drug Soliris and the loss of two top executives and this morning it announced a failure of a Phase II/III trial for expanded use of Solaris.
The drug was being tested for the prevention of delayed graft function (DGF) after kidney transplantation in adult recipients of a deceased donor kidney. In an announcement this morning, the company said the Phase II/III trial that tested two doses of Soliris (eculizumab) against placebo did not reach statistical significance. Delayed graft function is a complication of organ transplantation in which the transplanted organ fails to function normally following transplantation. For the purpose of the study, DGF was defined as the requirement for dialysis for any reason in the first seven days post-transplant. The study assessed 286 patients who received a transplant. The incidence of DGF was 35.9 percent for patients receiving a two-dose regimen of eculizumab compared with 41.7 percent for patients receiving placebo, the company said.
“We are disappointed that this trial did not meet its primary endpoint, given the urgent need for preventive therapies for patients at risk of DGF and the potential role of the complement cascade in the development of this serious and life-threatening complication,” Martin Mackay, executive vice president and global head of R&D at Alexion, said in a statement. “We continue to analyze the data to better understand what the findings mean for patients undergoing a kidney transplant. Importantly, the safety of eculizumab in this trial appears consistent with the favorable safety profile observed in nearly a decade of real-world experience with this highly innovative therapy.”
With an annual price tag of about $440,000, Soliris is known as the world’s most expensive drug. It is used to treat paroxysmal nocturnal hemoglobinuria, a rare blood disorder that affects about one or two people out of every million. Eculizumab received orphan drug designation (ODD) in 2014 from the U.S. Food and Drug Administration for the prevention of DGF in renal transplant patients, and from the European Medicines Agency for the prevention of DGF after solid organ transplantation. Soliris (eculizumab) is not approved in any country for the treatment or prevention of DGF. Currently, there are no approved therapies to prevent or treat DGF after kidney transplantation.
December has been a rough month for the company and the failure to expand the use of Soliris adds to the woes Alexion faces. Earlier this week, there were reports that an unnamed, but large U.S.-based drug company, approached company leaders regarding a possible merger. However, Alexion neither confirmed nor denied the news.
Earlier this month, Chief Executive Officer David Hallal and Chief Financial Officer Vikas Sinha resigned from the company amid its internal investigation regarding sales practices. Although neither man has been accused of participating in the improper sales practices, their departure could hint at some of the investigating committee’s findings.