AstraZeneca Ultomiris cuts risk of relapse by 98.6% in CNS disorder in trial
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AstraZeneca (NASDAQ:AZN) said its medicine Ultomiris reduced the risk of relapse by 98.6%, compared to placebo, in certain patients with a type of central nervous system (CNS) disorder in a phase 3 trial.
The company reported detailed data from the study, dubbed CHAMPION-NMOSD, which evaluated Ultomiris (ravulizumab) in adults with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD).
The British pharma giant said that 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. Ultomiris, the main treatment, was compared to the external placebo arm from the Soliris PREVENT clinical trial.
NMOSD is an autoimmune disorder which affects the CNS, mainly the eye nerves (optic neuritis) and spinal cord (myelitis).
The company said data showed 0 adjudicated relapses were seen among Ultomiris patients with a median treatment duration of 73 weeks (relapse risk reduction: 98.6%), p<0.0001). In addition, 100% of patients on Ultomiris remained relapse-free at 48 weeks, compared to 63% of patients in the external placebo arm.
The study also met key secondary efficacy goals, including adjudicated on-trial annualized relapse rate (total number of relapses in the trial divided by total number of patient years) and clinically important change from baseline in mobility (ability to walk), according to the company.
"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, lead primary investigator in the trial.