Nefecon is a potential treatment for patients with IgAN at risk of developing ESRD.

It is a proprietary oral formulation of budesonide, designed to deliver budesonide to the ileum where the so-called Peyer’s patches, which harbor the majority of B-cells producing IgA antibodies, are found. By delivering budesonide locally instead of systemically, Nefecon greatly reduces the side-effect burden observed with high dose steroid treatment while optimizing the effective dose level of the drug where it is required.

Budesonide has been used to treat patients with asthma, inflammatory bowel disease and allergic rhinitis for over 35 years. It is rapidly degraded soon after entering the circulatory system, making it an ideal basis for drugs such as Nefecon because local delivery to disease tissue minimizes the systemic effects seen with other corticosteroids.

Nefecon has been granted orphan drug designation for IgAN by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).

Other potential indications

Calliditas has identified two potential follow-on indications for Nefecon, based on its localized delivery in the intestine and resulting first passage though the liver. These are autoimmune hepatitis (AIH) and primary biliary cholangitis (PBC).

Clinical development

Nefecon has successfully completed the Phase 2b study (NEFIGAN) in 149 IgA nephropathy (IgAN) patients at risk of developing ESRD and will initiate a global Phase 3 study in 2018.

In 2017, the Lancet published the results of the randomized, double-blinded controlled Phase 2b study of nine months’ treatment with either Nefecon or placebo demonstrated the effectiveness of Nefecon in reducing proteinuria and also demonstrated that the treatment was safe and tolerable for the patients. This study strongly supports that local treatment with budesonide is effective.