Dive Brief:

• Alexion Pharmaceuticals Inc.'s follow-on complement inhibitor ALXN1210 demonstrated non-inferiority in a Phase 3 study comparing it to the company's aging blockbuster Soliris in patients with paroxysmal nocturnal hemoglobinuria (PNH).
• ALXN1210 showed similar effectiveness to Soliris on the co-primary endpoints of transfusion avoidance and normalization of lactate dehydrogenase (LDH) levels, as well as on four key secondary endpoints, including percentage change from baseline in LDH levels and change from baseline in quality of life as assessed by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scale.
• According to the company, study results showed no noticeable difference in safety between the two compounds. ALXN1210, however, narrowly missed proving superiority over Soliris in its effect on breakthrough hemolysis.

Dive Insight:

With positive data from the trial in hand, Alexion intends to file for approval of ALXN1210 in the U.S., EU and Japan in the second half of 2018.

This is a major milestone for a company that has been struggling over the last two years. Soliris (eculizumab) — once dubbed the most expensive drug in the world — has become a blockbuster seller despite the rare patient populations that it treats. However, the drug loses market exclusivity in 2021.

Soliris is expected to bring in $3.325 to $3.4 billion in 2018 and makes up the bulk of the Alexion's revenues. The biotech has been searching for a drug that could help further its franchise.

Beyond the stress dependence on Soliris has put on the company, Alexion has faced numerous other troubles, including a scandal related to its salesforce, a change in management and layoffs. The controversy involved accusations that prior management pressured the salesforce to act inappropriately.

Now that some of those issues have been addressed by new leadership, the success of the company depends on Alexion's ability to bring its pipeline forward. ALXN1210 is the linchpin of those efforts.

"This data is consistent with our thesis that ALXN1210 will allow Alexion to extend the IP of their complement franchise from the mid- to late-2020s to 2035 and this should also mitigate many of the competitive concerns related to other complement inhibitors in development," wrote Leerink analyst Geoffrey Porges in a March 15 note to clients.

"This consistent picture of positive trends in favor of ALXN1210 should reassure investors that Alexion's 2nd generation product is indeed better than Soliris; we expect the stock to react positively to this disclosure, although controversy will still exist about whether 1210 could have shown superiority in a larger trial, and whether the upcoming switch study could still meet the higher hurdle," he added.

Jefferies analyst Eun Yang agreed the data is enough to get ALXN1210 approved, but cautioned in a note to investors that non-inferiority (as opposed to superiority) might not be enough for ALXN1210 to command a premium price to Soliris once approved.

Alexion's shares initially jumped 10% in Thursday morning trading before paring back some of those gains to trade up between 8% and 9% over yesterday's close.