Novartis announced today positive results of the first pivotal Phase III trial of ACZ885 in patients with systemic juvenile idiopathic arthritis (SJIA), a rare and serious childhood auto-inflammatory disease. The results, presented at the 2011 European Pediatric Rheumatology Congress in Bruges, Belgium, showed all primary and secondary endpoints of the study were met.
Most ACZ885 patients (83.7%) experienced at least a 30% improvement in symptoms vs. 9.8% for placebo (p<0.0001) and a third of ACZ885 patients (32.6%) achieved a 100% improvement vs. 0% for placebo.
"SJIA is the most severe form of juvenile arthritis. Many of my patients suffer terribly from this disease, resulting in a critical need for new treatment options," said Daniel Lovell, M.D., one of the study investigators and Professor of Pediatrics at the Cincinnati Children's Hospital Medical Center. "This study showed ACZ885 effectively relieved the systemic and arthritic disease components evaluated in the trial, demonstrating a much-needed benefit for this patient population."
SJIA affects less than one child per 100,000 worldwide. It is called 'systemic' because the inflammation affects the whole body, as well as most of the joints. The condition is characterized by potentially life-long and recurrent arthritis flares, which can involve skin rash, daily spiking fever, joint pain and swelling.
In this study, patients were evaluated according to the adapted American College of Rheumatology (ACR) Pediatric criteria, which includes absence of fever. The ACR criteria are regularly used to assess the success of treatments in SJIA.
"These results are a positive development for patients suffering from this very severe auto-inflammatory condition," said David Epstein, Head of the Pharmaceuticals Division of Novartis. "We are committed to investigate ACZ885 in a range of inflammatory diseases where interleukin-1 beta plays a key role and high unmet medical needs exist."
The results of a second pivotal Phase III trial, aimed at determining whether ACZ885 can extend the time to next flare and reduce or eliminate corticosteroid use, will be presented later this year. Worldwide regulatory submissions for ACZ885 in SJIA are planned for 2012.
Machine learning identifies key genes linking rheumatoid arthritis and osteoporosis