Connect Biopharma Holdings Limited announced positive data at Week 48 from Phase 2 trial (CN002) for icanbelimod (formerly known as CBP-307), a once-daily, orally administered, selective sphingosine 1-phosphate receptor 1 (S1P1) modulator in development for the treatment of ulcerative colitis (UC). Trial Design (Maintenance Period): The maintenance period of CN002 was a 36-week treatment period that followed the 12-week induction period (N=145) where icanbelimod 0.2 mg, administered orally once daily (PO QD), showed statistically significant improvements in clinical remission and clinical response compared to placebo, and a numerical improvement in change from baseline in adapted Mayo score. The maintenance period included patients who achieved clinical response at the end of the induction period of the trial and continued treatment on icanbelimod 0.2 mg (n=21), 0.1 mg (n=12) or placebo (n=13) PO QD, and an icanbelimod 0.2 mg PO QD open-label arm for all non-responders (n=40) from the induction period.

Icanbelimod 0.2 mg PO QD Efficacy Data Highlights from Maintenance Period: 86% (18/21) of patients who received icanbelimod completed the maintenance period. 67% (12/18) of patients who completed the study through Week 48 achieved clinical remission. 80% (8/10) of patients who achieved clinical remission at the end of the induction period sustained it through Week 48.

Overall, 57% (12/21) of patients with clinical response at the end of the induction period achieved clinical remission at the end of the maintenance period. Safety Data Highlights from Maintenance Period: Icanbelimod was well-tolerated and long-term safety data through Week 48 remained consistent with safety findings observed in the induction period. Frequencies of treatment emergent adverse events were similar between icanbelimod and placebo groups, and most were mild to moderate in severity with no new safety signals noted.

About CN002 Trial: CBP-307CN002 is a Phase 2 study evaluating the efficacy and safety of icanbelimod as an induction and maintenance therapy in adult patients with moderate-to-severe UC. The randomized, double-blind, placebo-controlled, multi-center study enrolled a total of 145 patients in two active dose arms (icanbelimod 0.1 mg (n=39); icanbelimod 0.2 mg (n=53)) and a placebo arm (n=53) from over 60 sites in 4 countries. About Ulcerative Colitis: Ulcerative Colitis (UC) is an idiopathic inflammatory condition of the mucosal and submucosal colon that has a globally increasing prevalence thought to be driven by societal changes.

There are approximately 600,000 to 900,000 people in the United States living with ulcerative colitis. When insufficiently controlled, UC leads to progressive organ damage that presents as functional impairment and anatomical changes such as dysplasia, which may ultimately progress to cancer. Despite the availability of new treatments that have advanced the standard of care, a “therapeutic ceiling” exists, meaning that treatment options remain limited and clinical remission is still not achieved in 70–80% patients.

About Icanbelimod (formerly known as CBP-307): Discovered internally using Connect Biopharma's proprietary Immune Modulation Technology, icanbelimod is an orally administered small molecule designed to modulate sphingosine 1-phosphate receptor 1 (S1P1), which is a validated target for the treatment of several inflammatory diseases, including UC. Icanbelimod was observed to be generally well tolerated and showed evidence of clinical activity in both the 12-week induction and 36-week maintenance periods of the 48-week Phase 2 clinical trial in adults with moderate-to-severe UC, suggesting a potential for a differentiated risk–benefit profile compared with data from clinical trials of current orally administered therapies.