Novartis has presented late-stage data which shows that its investigational biologic canakinumab achieved "rapid and long-lasting remission" in people suffering from a group of rare forms of arthritis after just one dose.

The Phase III data, which was presented at the American College of Rheumatology meeting in San Francisco, showed that ACZ885 (canakinumab) proved effective as a treatment for a group of rare but potentially life-threatening autoinflammatory diseases called cryopyrin-associated periodic syndromes. Due to the long duration of response, patients only needed further treatment every two months, Novartis noted.

The results “are exciting for patients and for the medical community," said Philip Hawkins of the National Amyloidosis Centre in London. He noted that current treatments “are not always effective, and are also short-acting and often poorly tolerated by patients” so ACZ885 provides “an important and much-needed development for both children and adults with CAPS”.

Novartis claimed that unlike other agents, canakinumab solely blocks interleukin-1 beta, the form of the interleukin-1 protein that sustains autoinflammatory diseases such as CAPS. The latter, including Muckle-Wells Syndrome, are characterised by a single gene mutation that activates excessive production of IL-1 beta and leads to symptoms such as fever, fatigue, skin rash, painful joints and muscles, plus severe headache.

The Swiss firm also presented preliminary results of a Phase I/II study in systemic juvenile idiopathic arthritis which showed that most patients treated with ACZ885 achieved substantial clinical improvement within 15 days. In addition to CAPS and SJIA, it is also being investigated in rheumatoid arthritis, “using an innovative tailored approach with biomarkers to predict response to treatment”, Novartis said. If successful, “these will give suitable patients a personalised approach to the treatment of their disease”.

Orphan drug status has been granted to ACZ885 in the European Union and the USA for treating CAPS and SJIA, and Novartis says that filing is on track in CAPS in the two territories.