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news.Novartis has announced that new Phase III data demonstrate that ACZ885, a biological drug in development, achieved rapid and long-lasting clinical remission after just one dose in children and adults with a group of rare but potentially life-threatening autoinflammatory diseases called cryopyrin-associated periodic syndromes.
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Due to the long duration of response, patients only needed further treatment every two months, the company said.
In addition, preliminary results of a Phase I/II study in systemic juvenile idiopathic arthritis (SJIA) showed that most patients treated with ACZ885 achieved substantial clinical improvement within 15 days.
The six-month cryopyrin-associated periodic syndromes (CAPS) clinical trial involved patients aged nine to 74 years old and was divided into three parts. In the first part lasting two months, 35 patients received a single dose of ACZ885 by subcutaneous injection. All but one patient (97.1%) showed a rapid and long-lasting clinical and biochemical response.
After this, 31 patients who maintained their response proceeded to part two, a randomized six-month, double-blind, placebo-controlled withdrawal design study. Patients were treated every two months and if a relapse occurred, they discontinued and entered part three.
Part two of the study included the primary endpoint, a comparison between the number of patients treated every two months with ACZ885 who experienced disease outbreaks or ‘flares’ versus those on placebo. Results showed that no patients in the ACZ885 group experienced a disease flare compared to 81% (13 out 15 patients) in the placebo group (p<0.001). Markers of inflammation were normalized in patients treated with ACZ885, but increased significantly for those on placebo. The study is being concluded with a four-month open-label, active-treatment period to provide further efficacy and safety data. All patients are subsequently being offered the chance to take part in an additional Phase III study to provide long-term information about the efficacy and safety of ACZ885, said Novartis. Preliminary data from the Phase I/II study in SJIA demonstrated that patients aged four to 19 years treated with ACZ885 achieved substantial clinical improvement within 15 days. Importantly, four patients achieved complete remission of the disease, that is, no arthritis inflammation in joints, no fever and no disease activity according to the physicians' assessment.