CONCORD, Mass. — Bacainn Therapeutics, Inc., a Company developing novel therapies to address uncontrolled inflammation, today announced the successful completion of the company’s Phase 1, first-in-human clinical trial of BT051, a novel, orally administered, gut-selective inhibitor of migration and activation of neutrophils. During active flares of ulcerative colitis, neutrophils migrate into the submucosa and lumen of the colon where they contribute to tissue damage by releasing a cocktail of destructive enzymes, DNA, histones and pro-inflammatory mediators.
“There is significant evidence to support the role of overactive neutrophils in the large intestine as a driver of inflammation in inflammatory bowel disease, and BT051 has the potential to be a first-in-class medicine.”
“The safety and tolerability results from this first-in-human trial of BT051 are encouraging as they support our efforts to develop BT051 as a treatment for inflammatory bowel disease,” said Chris Stevens, MD, Chief Medical Officer at Bacainn Therapeutics. “There is significant evidence to support the role of overactive neutrophils in the large intestine as a driver of inflammation in inflammatory bowel disease, and BT051 has the potential to be a first-in-class medicine.”
The completed clinical trial was a phase 1, randomized, double-blind, placebo-controlled, single-center study. Healthy subjects (n=50) were randomized into 5 sequential, single-ascending-dose cohorts to receive BT051 (100 mg, 300 mg, 700 mg, 1500 mg, or 3500 mg) or placebo. Safety assessments included monitoring of adverse events, clinical laboratory testing, vital signs, physical examinations, and ECG assessments. Single doses of BT051 up to 3500 mg were generally safe and well tolerated in all healthy subjects exposed. The majority of adverse events were mild in severity, none lead to study discontinuation, and there were no serious adverse events reported.
Systemic exposure and stool concentrations following BT051 oral administration were also assessed. Across the doses evaluated there was either undetectable or minimal systemic exposure, while high stool concentrations were observed as was expected, given that the drug is designed to avoid absorption. Based on pharmacokinetic findings as well as the favorable safety profile of BT051 seen in this study, Bacainn Therapeutics plans to initiate a Phase 1b clinical trial of BT051 in patients with moderate to severe active ulcerative colitis in 2021.
Full results from the completed phase 1 clinical trial will be shared at a future scientific congress.
BT051 is a rationally designed new chemical entity which was discovered by Bacainn Therapeutics, Inc. to have activity limited to the GI tract. It takes advantage of Bacainn’s insights into the mechanisms responsible for controlling neutrophil migration into the lumen of the GI tract, as well as these immune cells’ subsequent activation via inhibition of multi-drug resistance protein 2 (MRP2) and formyl peptide receptor 1 (FPR1). It has been shown that the pathology of moderate to severe ulcerative colitis results in part from the highly damaging effects of dysregulated neutrophil accumulation and activation.
About Bacainn Therapeutics
Bacainn Therapeutics was founded based on the extensive insights into neutrophil biology generated by Dr. Beth McCormick’s laboratory at the University of Massachusetts Medical School. The Company takes its name from the Irish word for barrier, reflecting Dr. McCormick’s contributions into understanding neutrophil transepithelial migration, as well as her family’s historic roots. Bacainn was co-founded in 2017 by Morningside Ventures and recently closed a $25 million financing to advance the ongoing clinical development program in addition to supporting further pipeline build.