Novartis has unveiled promising late-stage data which show that its investigational biological therapy Ilaris can treat a rare but potentially life-threatening auto-inflammatory disease.

The data, from a one-year Phase III study, involved 35 patients aged nine to 74, who suffered from cryopyrin-associated periodic syndrome. The findings revealed that more than 90% of CAPS patients treated with Ilaris (canakinumab) remained in remission at the end of the final four-month phase of the study.

Novartis quoted the author of the study, Helen Lachmann of the UK National Amyloidosis Centre, as saying that patients experienced a benefit “within hours after receiving a single dose of canakinumab and only needed further treatment every two months to control their symptoms". Trevor Mundel, head of development at Novartis Pharma, said the firm is "extremely excited about the efficacy shown by Ilaris in patients with CAPS, and we hope to be able to extend these benefits to many more patients with other inflammatory diseases which are more widespread”.

Ilaris, a monoclonal antibody which selectively blocks interleukin 1-beta, is to be investigated also in other rare diseases such as systemic juvenile idiopathic arthritis. It will also be tested in more common ones such as some forms of gout, chronic obstructive pulmonary disorder, rheumatoid arthritis and type 2 diabetes.

Novartis has filed Ilaris for regulatory approval as a CAPS treatment in the USA, the European Union, Switzerland and Australia. It has orphan drug status in those territories.

Typhoid vaccine grant
The Basel-based major also noted that it has been awarded a grant from the UK’s Wellcome Trust to develop a bivalent vaccine for typhoid fever.

The 5.15 million euro grant will go to the Siena, Italy-based Novartis Vaccines Institute for Global Health which will be used for a vaccine that protects against both S Typhi and S Paratyphi A. Currently available vaccines for the former do not protect infants and young children, while S Paratyphi A causes 25%-50% of all typhoid cases.

Novartis chief executive Daniel Vasella, noting that there are more than 21 million cases of typhoid and more than 600,000 deaths each year, said that the bivalent vaccine being developed by NVGH “will use a novel approach to increase efficacy and address the needs of patients that other vaccines have not”.

He added that the treatment “will ultimately have the potential to eradicate this disease”.