Novartis‘ ACZ885 (canakinumab) has demonstrated positive results in a Phase III trial involving patients with systemic juvenile idiopathic arthritis, a rare and serious childhood auto-inflammatory disease.

ACZ885 is a fully human monoclonal antibody that works by neutralising IL-1 beta for a sustained period of time, thereby inhibiting inflammation.

The four-week, randomised, double-blind and placebo-controlled study involved 84 patients aged between two and 19, who were given either a single subcutaneous dose of ACZ885 (4mg/kg, up to 300mg) or a placebo.

The primary endpoint was the proportion of patients achieving the adapted ACR Paediatric 30 criteria, demonstrating a 30% improvement from baseline in at least three of the six variables at day 15.

The secondary endpoints included the proportion of patients achieving the adapted ACR Pediatric 50, 70, 90 and 100 criteria, demonstrating a 50%, 70%, 90% and 100% improvement in at least three variables from baseline at days 15 and 29.

The six variables included physician’s assessment of disease activity, parent’s or patient’s assessment of overall well-being, functional ability, number of joints with active arthritis, number of joints with limitation of motion and C-reactive protein, a laboratory measure of inflammation.

The data demonstrated that 83.7% of patients who took the compound achieved at least a 30% improvement in symptoms versus 9.8% among those who took the placebo placebo. In addition, 32.6% of patients given the drug achieved a 100% improvement, versus none for the placebo.

Professor Pierre Quartier of the Necker-Enfants Malades Hospital in Paris said that the study data suggested that ACZ885 could become an important treatment option for children with systemic juvenile idiopathic arthritis.

"ACZ885 provided rapid and long-lasting symptom relief by targeting interleukin-1 beta, a key inflammatory mediator of the disease," Quartier said.

The company aims to present the results of a second pivotal Phase III trial, determining whether ACZ885 can extend the time to next flare and reduce or eliminate corticosteroid use, later this year.