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  • 17 March 2016
    TopiVert reports successful Phase I study with TOP1288 for the treatment of ulcerative colitis

    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 tract and eye, today announced positive Phase I study results for TOP1288, its lead compound for the treatment of ulcerative colitis (UC). TopiVert will present the Phase I study data at the 11th Congress of ECCO in Amsterdam on Friday 18 March 2016. 

    The Phase I study results demonstrate that a rectal formulation of TOP1288, a potent inhibitor of key kinases involved in inflammation, has an excellent safety and tolerability profile in healthy volunteers when administered once or twice daily for five days at a dose of up to 200mg. Furthermore, TOP1288 was detected in tissue biopsies taken from the colon 24 hours after dosing, positive signals of target engagement and biomarker response were observed and only minimal drug was found in the systemic circulation. These results support the concept that NSKIs have 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 current systemically available therapies.

    UC is a relatively common inflammatory disease affecting the colon and approximately 40% of patients fail to respond to current drug therapy, which is often poorly tolerated. The disease can be serious enough to require surgery to remove the colon in some patients.

    TopiVert plans to start a Phase II proof of concept study with a rectal formulation of TOP1288 in UC patients in mid-2016, with results anticipated by mid-2017. This study is designed to demonstrate whether TOP1288 provides a clinical benefit in UC patients with active disease while avoiding significant exposure to healthy tissues outside the gastrointestinal tract.

    TopiVert is also planning to start a Phase I study of an oral formulation of TOP1288 in the second half of this year which will similarly report in mid-2017. An oral product offers greater patient acceptability and convenience over a rectal formulation.

    Dr Steve Webber, Chief Scientific Officer of TopiVert, said: “We are delighted that our first clinical study with an NSKI product demonstrated minimal systemic exposure when delivered locally, resulting in an excellent safety and tolerability profile. We are now eager to see whether the potent antiinflammatory effects observed with TOP1288 in preclinical testing translate into clinical benefits for patients with ulcerative colitis in a proof of concept study.”