Results from CHAMPION-NMOSD trial demonstrated Ultomiris reduced the risk of relapse in AQP4 Ab+ NMOSD by 98.6% compared to placebo

Ultomiris also showed a lower proportion of patients experiencing clinically important worsening in Hauser Ambulatory Index score, a measure of patient mobility

Strong results across several subgroup analyses add to growing body of evidence supporting C5 inhibition in NMOSD

Detailed positive results from the Phase III CHAMPION-NMOSD trial showed that Ultomiris (ravulizumab) significantly reduced relapse risk in adults with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD), compared to the external placebo arm from the pivotal Soliris PREVENT clinical trial. Data were presented today at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress.

NMOSD is a rare and debilitating autoimmune disease that affects the central nervous system (CNS), including the spine and optic nerves.1-3 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.4-6

Sean J. Pittock, MD, 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, said: “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.”

Michael Yeaman, PhD, Professor of Medicine at the UCLA School of Medicine, Director of the Institute for Infection and Immunity, Lundquist Institute at Harbor–UCLA and Chair Medical Advisor to the Guthy-Jackson Charitable Foundation for NMOSD, said: “In recent years, we have seen meaningful progress in bringing safe and effective treatments to patients with AQP4 Ab+ NMOSD, a rare disease that can disrupt many aspects of daily life, including mobility, vision, strength and balance. Based on insights from working with patients and their families every day, continued innovative research and clinical trials help empower people living with NMOSD by advancing new treatment options that may be more compatible with their individual needs and lifestyles.”

Gianluca Pirozzi, MD, PhD, Senior Vice President, Head of Development and Safety, Alexion, said: “CHAMPION-NMOSD is a remarkable example of the innovation required to design and execute rare disease clinical trials that are both scientifically rigorous and clinically meaningful. By seeking input from patients and coordinating with health authorities, this Phase III trial put the needs of patients first and evaluated measures that mattered most to them. We are excited by the significance of the results and the potential of Ultomiris to advance care for the NMOSD community.”

CHAMPION-NMOSD is a global Phase III, open-label, multicentre trial evaluating the safety and efficacy of Ultomiris in adults (n=58). 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 active treatment, was compared to the external placebo arm from the pivotal Soliris PREVENT clinical trial.7

Data showed zero adjudicated relapses were observed among Ultomiris patients with a median treatment duration of 73 weeks (relapse risk reduction: 98.6%, hazard ratio (95% CI): 0.014 (0.000, 0.103), p<0.0001). Additionally, 100% of patients receiving Ultomiris remained relapse-free at 48 weeks, compared to 63% of patients in the external placebo arm.7

The CHAMPION-NMOSD 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 (a scale to assess mobility).7

Summary of efficacy results from primary treatment periodi,ii


Ultomiris reduced the risk of relapse by 98.6% compared with placebo
see & read more on
https://www.astrazeneca.com/media-centre/press-releases/2022/ultomiris-showed-zero-relapses-in-adults-with-neuromyelitis-optica-spectrum-disorder-nmosd-with-median-treatment-duration-of-73-weeks.html