AstraZeneca's Ultomiris hits primary endpoint for PhIII NMOSD study
AstraZeneca caught a trio of recent wins — scoring an expanded indication for its breast cancer drug Enhertu, nailing Farxiga’s Phase III win earlier this week, and now, another trial win.
The UK pharma said Thursday that its long-acting C5 complement inhibitor drug Ultomiris (ravulizumab-cwvz) hit on its primary endpoint in the Phase III CHAMPION-NMOSD study. That endpoint was to show a “statistically significant and clinically meaningful reduction in the risk of relapse in adults” with a specific type of a rare autoimmune disease that targets the central nervous system.
Neuromyelitis optica spectrum disorder, or NMOSD, is currently an incurable disease that targets the spine and optic nerves. Most people living with NMOSD tend to experience “relapses” (which happen when there is inflammation in the nervous system), or an onset of neurologic symptoms or worsening of existing neurological symptoms such as blindness, partial paralysis or spasms.
According to AstraZeneca, the drug met the primary endpoint of time to first on-trial relapse. No relapse was observed in 58 patients over a median treatment duration of 73 weeks.
The lead primary investigator for the trial, Sean J. Pittock out of Mayo Clinic’s Center for Multiple Sclerosis and Autoimmune Neurology, said in a statement, “Every NMOSD relapse can have debilitating and irreversible consequences, so reducing relapses is critical.”
The drug was first approved by the FDA back in 2018 to treat patients at least one-month-old to treat blood disease paroxysmal nocturnal hemoglobinuria — the same disease being targeted at BioCryst with its lead candidate. BioCryst announced this morning that the FDA imposed a partial clinical hold on its candidate as part of its Q1 earnings announcement.
The drug is also a follow-up to Alexion’s blockbuster Soliris, also a complement factor 5 inhibitor, which AstraZeneca now owns after paying a behemoth $39 billion last year to acquire the company. And so far, there is not a ton of competition in the space. To date, there are only three drugs approved for NMOSD, more specifically AQP4-Ab positive NMOSD: AstraZeneca’s Soliris, Viela Bio’s Uplinza and Enspryng from Genentech.