Positive interim results were reported in the first two groups of ulcerative colitis patients being treated with Brilacidin (PMX-30063) in a dose-escalating Phase 2 clinical trial. Patients for a third and final group — testing the highest dose — are now being recruited.
The open-label and proof-of-concept study is testing the treatment’s safety and effectiveness at three dose levels — 50 mg, 100 mg, and 200 mg — in a total of 18 people with ulcerative proctitis/ulcerative proctosigmoiditis (UP/UPS), both limited forms of ulcerative colitis. Its primary goal is disease remission, and patients are being given Brilacidin by retention enema daily at bedtime for 42 days, the treatment’s developer, Cellceutix Corporation, announced in a press release.
All 12 patients in groups A and B have completed treatment with Brilacidin, and all showed a good response to the treatment as assessed by the Modified Mayo Disease Activity Index (MMDAI), the company reported. Those patients’ evaluations, reviewed by a trial safety committee, were a requirement for enrolling a third and final group.
Cellceutix also reported that the study met its primary efficacy endpoint in six patients (three in each group), meaning that at day 42, these people were in clinical remission, as observed by decreases in stool frequency and rectal bleeding, and through endoscopy subscores. Two others were in partial remission, and the remaining four did not meet remission criteria.
Data from the first two cohorts also showed the drug to be well-tolerated, with no measurable systemic absorption detected in the patients’ plasma.
Improvements were seen in quality of life, as measured by the Short Inflammatory Bowel Disease Questionnaire (SIBDQ), in all 12 patients post-treatment, with more than 40% of patients reporting significant improvements.
Once analyses across all three groups are done, the optimal dose of Brilacidin for future study will be determined.
Brilacidin is Cellceutix’s non-corticosteroid lead candidate, and is modeled after host defense proteins, a front-line immune system. It is a small synthetic molecule designed to kill pathogens quickly so as to prevent drug resistance, and functions in a immuno-modulatory capacity to reduce inflammation and promote healing, according to the press release.