Hitherto, alicaforsen has been granted orphan drug designation and is prescribed as an unlicensed medicine in accordance with international regulation, for the treatment of pouchitis and left-sided ulcerative colitis.

The differential response highlights the confounding symptoms of a large subset of subjects whose needs are apparently not being met by the current clinical trial design.

The efficacy of alicaforsen was durable in these sub-groups, with significantly improved duration of clinical response with no maintenance therapy, suggesting a disease-modifying effect.

However the co-primary endpoints (an adaptation of the Mayo Score of improvement in endoscopic remission and bowel frequency) were not met, perhaps due to the high discontinuation rate of 35% that compromised the sample size available for statistical analysis.

Clinical outcomes published in case series have confirmed durable disease remission in patients with Ulcerative Colitis with no treatment related SAEs.

[13] •    UC case series publications confirming prolonged duration of action: Ø    Digestive Diseases Journal (Nov 2017); 10/12 patients with left-sided UC/proctitis responded to treatment, with a median durable response of 18 weeks Ø    Gastrodagarna Congress, Sweden (May 2016); all 7 distal UC patients that completed treatment responded, 57% remained in remission for 5-20 months Ø    Irish Society of Gastroenterology (Nov 2014); reported remission achieved in 57% of patients with UC with a durable response 1 No drug-related SAEs have been reported in any usage of alicaforsen enema