First Patients Dosed in Phase 2a Proof-of-Concept Study With Rectal Formulation of TopiVert's TOP1288 in Moderate to Severe Ulcerative Colitis

LONDON, UNITED KINGDOM--(Marketwired - Oct 6, 2016) -  TopiVert Pharma Ltd ("TopiVert" or the "Company"), a clinical-stage biotechnology company developing Narrow Spectrum Kinase Inhibitors (NSKIs) as novel, locally-acting medicines for the treatment of chronic inflammatory diseases of the gastrointestinal (GI) tract and eye, announced today that the first patients have been dosed in its Phase 2a proof-of-concept study to evaluate the safety, tolerability and efficacy of a rectal formulation of TOP1288 in symptomatic ulcerative colitis (UC) patients with moderate to severe disease activity.

TOP1288, the Company's lead NSKI programme, is a potent inhibitor of key kinases involved in inflammation and it is being developed by TopiVert as a topical therapy for UC, a form of inflammatory bowel disease (IBD) which affects the colon. As many as 40% of patients fail to respond to current drug therapy, which is often poorly tolerated, and some patients require surgical removal of the colon in order to manage the disease.

TopiVert reported encouraging results from a Phase 1 study of a rectal formulation of TOP1288 in healthy volunteers earlier this year¹, and this has subsequently been supported by data from an additional patient cohort. These clinical data, which include encouraging biomarker responses and negligible systemic absorption, support the concept that TOP1288 has the potential to produce sustained effects in mucosal tissues after local administration, but without the undesirable side effects often seen in UC patients treated with systemically available therapies.

The ongoing Phase 2a study with TOP1288 is a randomised, double-blind, placebo-controlled study to evaluate the efficacy, safety and tolerability of TOP1288 administered once daily for four weeks in symptomatic UC patients with moderate to severe disease activity. The study, which aims to recruit up to 60 patients at sites across eight European countries, is expected to report in the second half of 2017. The current study utilises a rectal formulation of TOP1288, to help demonstrate POC, and a Phase 1 study with an oral formulation of TOP1288, as the intended commercial presentation, is on track to commence in early 2017, with results also anticipated in the second half of 2017.

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http://www.rns-pdf.londonstockexchange.com/rns/7634L_1-2016-10-5.pdf