Alexion’s – part of the AstraZeneca Rare Disease group – Ultomiris (ravulizumab) significantly reduced relapse risk in adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD) compared to an external placebo arm, positive results from a phase 3 trial CHAMPION-NMOSD have shown.
Regulatory submissions for Ultomiris for the treatment of NMOSD are currently under review with multiple health authorities, including in the US, EU and Japan, the company said in a statement.
NMOSD is a rare and debilitating autoimmune disease that affects the central nervous system, including the spine and optic nerves. Most people living with NMOSD experience unpredictable relapses, characterised by a new onset of neurologic symptoms or worsening of existing neurologic symptoms, which tend to be severe and recurrent and may result in permanent disability.
Due to the potential long-term functional impact of NMOSD relapses and available effective treatment options, a direct placebo comparator arm was precluded for ethical reasons. Instead Ultomiris was compared to the external placebo arm from the company’s pivotal Soliris PREVENT clinical trial.
Zero adjudicated relapses were observed in the CHAMPION-NMOSD trial among Ultomiris patients, with a median treatment duration of 73 weeks. Moreover, 100% of patients receiving Ultomiris remained relapse-free at 48 weeks, compared to 63% of patients in the external placebo arm.
The trial also met key secondary efficacy endpoints, including adjudicated on-trial annualised relapse rate – total number of relapses in the study divided by total number of patient years – and clinically important change from baseline in mobility – ability to walk – as measured by Hauser Ambulation Index.
In the subgroup analysis of the study, based on time to first adjudicated on-trial relapse, Ultomiris was superior to the external placebo arm in patients receiving monotherapy and in patients receiving concomitant therapy.
The robust treatment effect of Ultomiris was also observed across pre-specified efficacy subgroups, including age, sex, Asian and white races and geographic region.
Overall, the safety and tolerability of Ultomiris was consistent with previous clinical studies and real-world use and no new safety signals were observed, the company reported.
“The CHAMPION-NMOSD trial showed zero relapses with a median treatment duration of 73 weeks, providing evidence that ravulizumab may offer patients sustained reduction in the risk of relapse with dosing every eight weeks and underscoring the efficacy of C5 inhibition in managing NMOSD,” said Sean Pittock, director of Mayo Clinic’s Center for Multiple Sclerosis and Autoimmune Neurology and of Mayo’s Neuroimmunology Laboratory and lead primary investigator in the CHAMPION-NMOSD trial.