Core Insights - Chemomab Therapeutics has successfully aligned with the FDA on a single Phase 3 registration study for its lead product candidate, nebokitug, aimed at treating primary sclerosing cholangitis (PSC) [2][3] - The Phase 3 trial design provides regulatory clarity and eliminates the need for liver biopsies or additional confirmatory studies, focusing on clinically relevant endpoints associated with PSC disease progression [3][5] - Nebokitug is a first-in-class dual activity monoclonal antibody that neutralizes CCL24, showing promise in treating PSC and other fibro-inflammatory diseases [7][11] Group 1: Trial Design and Objectives - The Phase 3 trial is a randomized placebo-controlled study with a 2:1 active to placebo ratio, enrolling approximately 350 PSC patients [6] - The primary endpoint is the time-to-first clinical event, which includes various adverse events related to PSC progression, such as acute cholangitis and liver transplantation [6][4] - The trial aims to collect sufficient clinical events to demonstrate statistically significant changes between treatment and placebo arms, with an estimated two-year timeline for participants to experience a clinically meaningful event without intervention [6][4] Group 2: Regulatory and Strategic Developments - The FDA's agreement on the trial design marks a significant milestone, providing a streamlined path to potential full regulatory approval based on a single pivotal trial [3][5] - The company is actively engaging with potential strategic partners while preparing for the Phase 3 program, which could launch before the end of the year [3][5] - The trial's design is similar to oncology registration trials, focusing on disease progression-related events, which may allow for broader labeling compared to symptom-related endpoints [5][6] Group 3: Clinical Background and Need - PSC is a rare and debilitating liver disease with no FDA-approved therapies, affecting an estimated 30,000 patients in the U.S. and 80,000 worldwide [8] - The disease is characterized by inflammation and fibrosis of the bile ducts, leading to severe complications such as liver failure and increased cancer risk [8] - There is a high unmet medical need for effective treatments to address PSC symptoms and slow disease progression, highlighting the importance of nebokitug's development [8][11]
Chemomab Completes Successful End-of-Phase 2 Meeting and Aligns with FDA on Clear and Efficient Path to Potential Regulatory Approval for Nebokitug (CM-101) in Primary Sclerosing Cholangitis