Alexion Pharmaceuticals Inc (NASDAQ:ALXN) was one of the top risers in pre-market trading on Thursday after the multi-billion dollar biotech’s new blood disease drug sailed through a pivotal late-stage study.
In a Phase III trial, the Connecticut-based group’s ALXN1210 treatment for paroxysmal nocturnal hemoglobinuria – a rare and deadly blood disease – demonstrated “non-inferiority” compared to Soliris, the current standard of care which is also made by Alexion.
ALXN1210 as the new standard of care
Essentially, the company wanted to prove that its new drug is just as effective and safe as its existing one, which it did.
PNH patients currently receiving Soliris need 26 infusions a year, but one of the key benefits of ALXN1210 is that it only needs to be administered six times a year.
“We are very pleased with these positive data for ALXN1210 in the first and only head-to-head study versus Soliris, and the results reinforce our ambition to establish ALXN1210 as the new standard of care for patients with PNH,” said head of research and development John Orloff.
“Soliris has established a high bar for efficacy. Achieving non-inferiority on both co-primary and all key secondary endpoints, as well as seeing numeric results in favor of ALXN1210, in such a rigorous study met a very high hurdle.”
Alexion said it plans to make regulatory submissions in the US, EU and Japan in the second half of 2018.
Shares were up 9.8% to US$134.65 in pre-market trading on Thursday.