Cellceutix announced additional interim positive findings from its ongoing Phase 2a trial evaluating brilacidin (PMX-30063) as a treatment for two types of inflammatory bowel disease (IBD) – ulcerative proctitis and ulcerative proctosigmoiditis (UP/UPS).
The proof-of-concept, open-label, dose-escalation trial is designed to evaluate the effectiveness, safety and drug properties of three dosing regimens of brilacidin – 50 mg (cohort A), 100 mg (cohort B), and 200 mg (cohort C) – in patients with active mild to moderate UP/UPS.
Three of six patients in the third and final group (cohort C) of the study were enrolled earlier this week and the company anticipates the full study will be completed by the second quarter of 2017, just in time to present it at the 2017 Drug Discovery and Therapy World Congress July 10-13 in Boston.
Confirming the results in a previous announcement, all 12 patients in two groups completed treatment with brilacidin and all demonstrated a good response to treatment. The study also met its primary effectiveness endpoint in six patients (three in each group), meaning that at day 42, these patients were in clinical remission. Two others were in partial remission and the remaining four did not meet remission criteria.
Notable improvements were observed in 10 of 11 patients (one patient declined endoscopy at the end of treatment): 100% reduction in two patients in cohort A and two patients in cohort B; 50% to 75% reduction in two patients in cohort A and four patients in cohort B; and 20% reduction in one patient in cohort A.
Data from the first two cohorts had also shown that the drug was well-tolerated, with no measurable systemic absorption detected.
Brilacidin is a small synthetic molecule that is designed to kill pathogens, promote healing and decrease inflammation.
“A changing IBD treatment paradigm, as well as a regulatory one, is placing an increased emphasis on establishing Clinical Remission based on objective criteria, such as validated endoscopic response, and not just relying on subjective scoring systems,” Arthur P. Bertolino, MD, PhD, president and chief medical officer at Cellceutix, said in a press release.
“That a majority of patients treated with brilacidin at the end of six weeks showed mild disease or no disease at all based on MMDAI (Modified Mayo Disease Activity Index) Endoscopy subscores reinforces brilacidin’s therapeutic potential in treating IBD,” he said.
UP is a limited type of ulcerative colitis (UC) that manifests as a mucosal inflammatory disease of unknown cause involving the rectum. Once it involves both the rectum and the distal colon, it is called UPS. It is characterized by inflammation, redness, and ulcerations of the mucosa. The course of UP/UPS is variable and ranges from complete resolution to easily maintained remission to chronic relapses or refractory disease.
“To see such consistently strong results through the first two cohorts of UP/UPS patients treated with brilacidin is highly promising,” said Cellceutix CEO Leo Ehrlich. “With planned newer formulations, foam and oral, we believe brilacidin could one day be extended into treating a number of chronic IBD indications.”
The U.S. Food and Drug Administration (FDA) granted brilacidin a Qualified Infectious Disease Product (QIDP) designation as an antibacterial product for skin infections, qualifying it for Fast Track and possible Priority Review designations.