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Abstract Details
The FALCON program: Two phase 2b randomized, double-blind, placebo-controlled studies to assess the efficacy and safety of pegbelfermin in the treatment of patients with nonalcoholic steatohepatitis and bridging fibrosis or compensated cirrhosis
Contemp Clin Trials. 2021 Feb 28;106335. doi: 10.1016/j.cct.2021.106335. Online ahead of print.
Manal F Abdelmalek1, Edgar D Charles2, Arun J Sanyal3, Stephen A Harrison4, Brent A Neuschwander-Tetri5, Zachary Goodman6, Richard A Ehman7, Morten Karsdal8, Atsushi Nakajima9, Shuyan Du10, Giridhar S Tirucherai10, George H Klinger10, Johanna Mora10, Masayuki Yamaguchi10, Diane E Shevell10, Rohit Loomba11
Author information
1Duke University School of Medicine, Durham, NC, United States of America.
2Bristol Myers Squibb, Princeton, NJ, United States of America. Electronic address: elizabeth.artwick@medicalexpressions.com.
3Virginia Commonwealth University, Richmond, VA, United States of America.
4Pinnacle Clinical Research, San Antonio, TX, United States of America.
5Saint Louis University, St. Louis, MO, United States of America.
6Inova Fairfax Hospital, Falls Church, VA, United States of America.
7Mayo Clinic College of Medicine, Rochester, MN, United States of America.
8Nordic Bioscience, Herlev, Denmark.
9Yokohama City University, Yokohama, Japan.
10Bristol Myers Squibb, Princeton, NJ, United States of America.
11University of California at San Diego, San Diego, CA, United States of America.
Abstract
Background: Nonalcoholic steatohepatitis (NASH) is the progressive form of nonalcoholic fatty liver disease (NAFLD); no approved therapies for NASH currently exist. Pegbelfermin (PGBF), a human fibroblast growth factor 21 analog, has metabolic effects that may provide benefit for patients with NASH.
Design: The FALCON 1 and 2 studies are Phase 2b, multicenter, double-blind, placebo-controlled, randomized trials to assess safety and efficacy of PGBF treatment in patients who have histologically-confirmed NASH with stage 3 liver fibrosis (FALCON 1; NCT03486899) or compensated cirrhosis (FALCON 2; NCT03486912). In both studies, randomized patients receive once weekly subcutaneous injections of PGBF (10, 20, or 40 mg) or placebo during a 48-week treatment period and are then followed for an additional 4 weeks.
Endpoints: The primary efficacy endpoint for FALCON 1 is the proportion of patients who achieve ≥1 stage improvement in fibrosis (by NASH CRN fibrosis score) without NASH worsening or NASH improvement (≥2 point decrease in NAFLD Activity Score) without fibrosis worsening at Week 24. For FALCON 2, the primary efficacy endpoint is ≥1 stage improvement in fibrosis without NASH worsening at Week 48. Key safety endpoints for both studies include incidence and frequency of adverse events, bone mineral density and immunogenicity.
Summary: Previous clinical trial data show that PGBF can reduce hepatic fat and improve metabolic factors and biomarkers of hepatic injury and fibrosis. The FALCON studies aim to evaluate PGBF treatment specifically in patients with NASH and advanced fibrosis, who are at greatest risk of poor clinical outcomes over time.