Cellceutix Initiates Dose Escalation in Phase 2 Trial of Brilacidin for Ulcerative Proctitis

Cellceutix Initiates Dose Escalation in Phase 2 Trial of Brilacidin for Ulcerative Proctitis

Cellceutix has initiated the dose escalation of brilacidin (PMX-30063) in the second cohort of patients enrolled in a Phase 2 clinical trial evaluating the drug candidate for the treatment of ulcerative proctitis and ulcerative proctosigmoiditis (UP/UPS), two types of ulcerative colitis (UC).

UP is a mucosal inflammatory bowel disease of unknown cause involving only the rectum. UP evolves to UPS when both the rectum and the distal colon are involved.

Brilacidin is the company’s lead drug candidate modeled after host defense proteins (HDPs), the front-line of defense in the immune system. It’s a small synthetic molecule that kills pathogens and functions in a robust immunomodulatory capacity, lessening inflammation and promoting healing.

After reviewing safety data from the first cohort (on 50 mg), researchers concluded that brilacidin administered for six weeks (42 consecutive days) as a retention enema appeared to be well-tolerated by patients with no measurable systemic absorption reported. This provided the green light researchers needed to initiate a dose escalation to 100 mg once daily.

The ongoing Phase 2 open-label, proof-of-concept clinical trial began enrolling its first patients in June. The trial is evaluating brilacidin for induction of remission, not merely maintenance, in patients suffering from mild to moderate UP/UPS. The trial includes three sequential, progressively escalating cohorts with six patients enrolled in each. The cohorts started at 50 mg, then 100 mg, and if that proves successful, will move up to 200 mg of Brilacidin.

The primary objective of this Phase 2 trial is to assess the frequency of clinical remission with brilacidin per rectum by enema in patients with active UP or UPS after six weeks of treatment. Additional objectives include the evaluation of the drug candidate’s safety and tolerability, assessment of systemic exposure and assessment of the effectiveness of the drug at the end of the treatment.

“We remain extremely encouraged by the results that continue to emerge from this trial,” Arthur P. Bertolino, MD, PhD, president and chief medical officer of Cellceutix, said in a press release. “In addition, the clinical sites at which the study is being performed are now even more enthusiastic about recruiting additional patients to the trial.”

“Such enthusiasm is a good indicator of brilacidin’s potential in treating ulcerative colitis and other inflammatory bowel diseases,” he said, adding the company looks forward to seeing data from the next cohorts.