Galectin Therapeutics has reached FDA agreement on the design and endpoints of its pivotal Phase 3 trial for belapectin in patients with compensated MASH cirrhosis and portal hypertension, advancing toward a potential full approval pathway.
Written By: Pavan Kumar Chikkula, PharmD
Reviewed By: Pharmacally Editorial Team
Galectin Therapeutics has reached agreement with the U.S. Food and Drug Administration (FDA) on key elements of its planned Phase 3 program for belapectin, advancing the drug candidate toward a potential full approval pathway in patients with metabolic dysfunction-associated steatohepatitis (MASH) cirrhosis and portal hypertension.
The agreement follows a recent Type C meeting in which the FDA aligned with the company on the study design, primary endpoint, and overall regulatory framework for the pivotal trial. The outcome provides greater clarity for one of the few late-stage programs targeting patients with compensated MASH cirrhosis, a population with limited treatment options and a high risk of progression to liver-related complications.
Belapectin Targets Portal Hypertension
Belapectin is a galectin-3 inhibitor that targets pathways involved in inflammation, fibrosis, and portal hypertension. The therapy is being developed to slow disease progression in patients with advanced liver disease.
Portal hypertension is a major driver of complications in MASH cirrhosis, including the development of esophageal varices, hepatic decompensation, and liver failure. Despite significant investment across the broader MASH field, no approved therapies currently address disease progression in this advanced patient population.
Trial Design and Endpoint Agreement
The planned Phase 3 study will be a randomized, double-blind, placebo-controlled trial evaluating belapectin in patients with compensated MASH cirrhosis and portal hypertension.
A key outcome from the FDA discussions was agreement on a composite liver outcome as the primary endpoint. The endpoint will include prevention of large esophageal varices greater than 5 mm, a clinically meaningful marker of worsening portal hypertension and disease progression.
The agency also accepted Galectin’s blinded central review methodology for endoscopic assessment of esophageal varices. The process was previously used in the global Phase 2b/3 NAVIGATE trial, which evaluated belapectin in a similar patient population.
Based on efficacy findings from prior studies, the company plans to evaluate a single 2 mg dose of belapectin in the Phase 3 trial. Galectin expects the study size to be broadly comparable to NAVIGATE.
Full Approval Strategy
Unlike earlier-stage MASH programs that may pursue accelerated approval using histologic endpoints, belapectin will follow the traditional approval pathway required for therapies targeting patients with established cirrhosis.
According to the company, the FDA agreed that the proposed composite clinical outcomes and study framework could support a full approval application. Galectin also believes a successful Phase 3 trial could qualify under the agency’s evolving approach allowing a single adequate and well-controlled confirmatory study to support marketing approval in certain settings.
Chief Executive Officer Joel Lewis said the FDA agreement provides important clarity for advancing the program, while Chief Medical Officer Khurram Jamil highlighted the agency’s acceptance of the centralized endoscopy review process as validation of the methodology established through NAVIGATE.
Regulatory Path Forward
Galectin plans to submit its final Phase 3 protocol in the third quarter of 2026 and is actively exploring strategic and financial partnerships to support development and commercialization efforts.
If successful, the study could position belapectin among the first therapies approved to prevent disease progression in patients with MASH cirrhosis and portal hypertension, an area that remains a major unmet need in liver disease.
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About the Writer
Chikkula Pavan Kumar (LinkedIn), PharmD is a Doctor of Pharmacy with a keen interest in clinical pharmacy, pharmacovigilance, and evidence-based practice. In his words, he is passionate about patient safety and translating complex medical information into clear, research-driven communication.