Troubled Alexion takes another hit as star drug Soliris falls short in transplant trial

Alexion has pushed hard to expand the list of uses for its star orphan med Soliris to keep its blockbuster sales flowing, but now it's coming up short. The drug failed a trial in organ transplant patients, for an indication one analyst says it shouldn't have targeted in the first place.

Soliris missed its primary endpoint in a phase 2/3 trial testing the med in kidney transplant patients at high risk of delayed graft function (DGF), an organ transplant complication. At seven days post-transplant, the incidence of DGF, death, graft loss and discontinuation measured 35.9% among Soliris-treated patients and 41.7% for patients on placebo, a difference that didn’t prove statistically significant.

Some analysts, including Piper Jaffray’s Joshua Schimmer, dismissed the flop as “not surprising or important.” For one, Soliris had failed in the transplant setting before, coming up short in preventing antibody mediated rejection in living-donor kidney transplant recipients. And for two, DGF represented a sales opportunity of less than $50 million annually, according to calculations from Leerink Partners analyst Geoffrey Porges.

The way Porges sees it, though, while the misfire “does not materially impact our long-term Soliris forecasts or growth rate,” it “does represent another example of Alexion ploughing Soliris profits into high-risk trials for low-value indications”—and that’s a situation he’s not pleased with. He called the experiment a failure “to protect the profits” on Alexion's lead drug.

And these days, Alexion needs as much from Soliris as it can get. Amid sales fraud allegations and a recent management overhaul that brought in a new CEO and CFO, the Connecticut company has been leaning especially hard on its star med to keep its business stable and investors happy. Porges has gone as far as to call Alexion “one of the rare, once or twice per decade, activist investment situations in the biopharmaceutical industry,” with the “recent turmoil” presenting an “opportunity” for shareholders.

Luckily for the company, though, it has other, more significant Soliris expansion efforts in progress. Alexion is planning to file for a new generalized myasthenia gravis indication in both the U.S. and Europe this quarter, and Barclays analyst Geoff Meacham has estimated that a label expansion could kick in about $200 million in unadjusted incremental sales by 2020, conservatively speaking.