Watch out, AstraZeneca: UCB uncorks late-stage success for $2.1B Soliris rival
Just over two years after spending more than $2 billion to acquire the drug, UCB announced that zilucoplan proved effective at treating the rare autoimmune disease myasthenia gravis in a large Phase III trial.
Although UCB declined to share any specific numbers, the Belgian pharma said patients on the drug showed significant improvement over placebo on all primary and secondary endpoints, notably including three different scales used to track symptoms of the complex disease.
The study, which had 174 patients according to clinicaltrials.gov, also found the drug was “well-tolerated,” UCB said, with a similar number of serious treatment-emergent adverse events in both placebo and drug arms.
The company plans to file for approval later this year.
UCB acquired the drug in 2019, when it bought out Ra Pharma for $2.1 billion. The idea at the time was that the drug could challenge Alexion’s blockbuster Soliris, which is now owned by AstraZeneca. Zilucoplan goes after the same target as Soliris, blocking complement factor 5, a critical node in the immune system.
Without actual data, it’s impossible to tell where zilucoplan will actually be able to compete, should it be approved. But the market has already gotten more crowded in the short span since UCB’s buyout.
In addition to AstraZeneca’s follow-up drug Ultomiris, Apellis now has an approved complement-targeting rival called Empaveli, although it has yet to gain approval in myasthenia gravis. Both Empaveli and zilucoplan are given subcutaneously rather than by IV, making an easier dose regimen for patients.
Several companies have also pursued — and in one case got approved — molecules that block a receptor called FcRn, helping clear antibodies that contribute to diseases such as myasthenia gravis from the body.
UCB, though, has hedged its bets. The company has its own FcRn antibody called rozanolixizumab that’s already in Phase III trials. It’s also developing an extended release form of zilucoplan and oral complement inhibitor that, if successful, would be the first of its kind.