First Ulcerative Colitis Patients Dosed in Phase 2a Trial of TopiVert’s New Therapy

First Ulcerative Colitis Patients Dosed in Phase 2a Trial of TopiVert’s New Therapy

The first patients have been dosed in TopiVert’s Phase 2a trial to assess the effectiveness, safety, and tolerability of TOP1288 (rectal form) for moderate to severe symptomatic ulcerative colitis.

TopiVert focuses on developing narrow spectrum kinase inhibitors (NSKIs) into new locally-acting drugs to treat chronic inflammatory diseases of the gastrointestinal (GI) tract and eye. TOP1288, the company’s lead candidate for ulcerative colitis (UC), was designed to inhibit key kinases (enzymes that modify other proteins) involved in the signaling pathways of inflammatory diseases. Administered orally or rectally, the drug acts in the GI tract.

The company recently reported positive data from its Phase 1 clinical trial (NCT02463045) that evaluated the safety and tolerability of TOP1288 in single doses of 1, 10, 100, and 200 mg, or a 200 mg pill twice a day, and multiple doses (four days) of 10, 50, and 200 mg, or one 200 mg pill twice a day of the drug or the placebo in 61 healthy volunteers.

The results, including positive biomarker responses and minor systemic absorption, support TOP1288’s potential to produce continuous effects in mucosal tissues after local administration without causing the adverse side effects seen with currently available UC treatments. These data and the preclinical pharmacology of TOP1288 were presented at this year’s Digestive Disease Week in San Diego.

The placebo-controlled Phase 2a clinical trial (NCT02888379) will evaluate the safety, tolerability, and effectiveness of TOP1288 200 mg rectal solution once daily for four weeks versus a placebo in moderate to severe symptomatic patients with UC.

The trial’s primary endpoint is the drug’s effectiveness as measured by the Mayo Clinic modified endoscopic subscore. Secondary endpoints include multiple safety assessments. Up to 60 patients will be recruited in eight European countries. Results from the trial are expected by mid-2017.

TopiVert is also planning to begin a Phase 1 clinical trial of an oral formulation of TOP1288 early next year, with results also expected by mid-year.