Alexion has already seen major success expanding flagship Soliris into multiple indications. And it may just have another nod on the horizon.
Monday, the Boston-based biotech said that in a phase 3 study of the drug in patients with Neuromyelitis Optica Spectrum Disorder (NMOSD), a rare CNS disease that can cause blindness, paralysis and death, Soliris nailed its primary endpoint. Treatment with the drug reduced patients’ risk of relapse by 94.2% compared with placebo, and at the 48-week mark, 97.9% of Soliris patients were relapse-free.
“These results far exceeded our expectations. The remarkable reduction in relapse risk ... suggests a promising new treatment for NMOSD,” John Orloff, Alexion’s R&D head, said in a statement. “Given that patients currently have no approved therapies, we are moving quickly to discuss these results with regulators and file for approval in the U.S., EU and Japan," he added.
The trial was far from a sure thing in the eyes of investors, Leerink Partners analyst Geoffrey Porges wrote in a Monday note to clients. Though the drug had posted successful results in a phase 1 study, “we believed a positive phase 3 outcome was not appreciated by consensus given significant trial design changes from phase 1 to phase 3 that added uncertainty about the phase 3 trial,” Porges wrote.
Now, though, “the results appear to show robust treatment effect and should result in rapid approval after filing later this year,” he predicted, adding that the development should pad Alexion’s top line by $500 million to $700 million.
A go-ahead in NMOSD would follow up on Soliris’ latest FDA OK, a 2017 green light in myasthenia gravis. That nod—which analysts said could bring in $1 billion—brought Soliris’ indication count to three.
But another indication for Soliris also might make the company more attractive to potential buyers. Bernstein’s Ronny Gal, for one, has suggested that Amgen—facing a biosimilar onslaught—could go after Alexion to help ease some competitive pressure.