Alexion fails to PROTECT Soliris franchise
- Alexion Pharmaceuticals' blockbuster drug Soliris (eculizumab) failed to reach statistical significance on the primary endpoint for a Phase 2/3 trial, the company said Wednesday night.
- The rare disease drug, thought to be the most expensive drug in the world, failed to prevent delayed graft function in kidney transplantation patients.
- The results of the PROTECT study are the latest bad news for Alexion after the company revealed it was conducting an internal investigation for fraud related to Soliris sales.
It has been a rough holiday season for the rare disease company and Santa delivered another lump of coal with the latest trial results.
The PROTECT trial assessed 286 patients who had received a kidney transplant. The incidence of delayed graft function, death, graft loss or loss to follow-up at 7 days post-transplant was 35.9% in patients receiving a two-dose regimen of Soliris, compared with 41.7% in those given a placebo. The result was not statistically significant.
Delayed graft function is a serious complication of kidney transplant that occurs immediately following the operation and affects nearly 25% to 50% of deceased-donor kidney transplant cases.
The trial isn't a major hit to Alexion — most investors and analysts had low expectations. Yet, it is another piece of bad news for a company struggling to right its image.
In November, a Securities and Exchange Commission filing revealed that the company was looking into whistleblower allegations that Alexion was conducting unethical sales practices for Soliris.
The investigation pushed the company to delay its quarterly filing with the SEC. It has yet to file the report and the company said it wouldn't do so until the investigation is complete.
Adding to the bad news, the company unceremoniously dismissed its CEO and CFO, saying the men were leaving to pursue other options.
"Given the low expectations for the DGF trial, we don't expect much of an impact on Alexion shares following the announcement that the trial failed. What investors really care about is how this new management team handles the ongoing crisis. More specifically, with how much transparency and how expeditiously the new management team will deal with its first major credibility test in front of investors," wrote RBC Capital Markets analyst Simos Simeonidis.
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