Key Takeaways:
Galectin Therapeutics secured FDA agreement on a Phase 3 trial design for belapectin in MASH cirrhosis, clearing a regulatory path toward full approval in a population with no approved therapies.
Galectin Therapeutics Inc. reached alignment with the US Food and Drug Administration on a Phase 3 program for belapectin in patients with MASH cirrhosis and portal hypertension, a population facing the highest risk of life-threatening decompensation events.
"Alignment on the study design, primary endpoint, and regulatory framework provides important clarity, as we advance the program with renewed confidence," Joel Lewis, chief executive officer of Galectin Therapeutics, said in a statement.
The randomized, double-blind, placebo-controlled trial will evaluate a single 2 mg dose of belapectin, a carbohydrate-based galectin-3 inhibitor, using a composite liver outcome as the primary endpoint. The endpoint includes prevention of large esophageal varices greater than 5 mm, a clinically meaningful marker of worsening portal hypertension. The FDA accepted Galectin's proposed blinded central review process for endoscopic assessment, a methodology the company developed and validated in the global Phase 2b/3 NAVIGATE trial. The company expects the Phase 3 study to be comparable in size to NAVIGATE.
Unlike earlier-stage MASH populations eligible for accelerated approval based on histologic endpoints, the cirrhosis population requires a traditional approval pathway. The FDA confirmed that a single adequate and well-controlled trial could support marketing approval, giving Galectin a defined route to a broad indication. The company plans to submit the final Phase 3 protocol in the third quarter of 2026 and is actively pursuing strategic partnership opportunities.
Belapectin targets galectin-3, a protein that drives inflammatory, fibrotic, and malignant disease processes. In MASH cirrhosis, the drug aims to prevent progression to decompensated liver disease — a condition that excludes patients from most other clinical trials. The FDA acknowledged the substantial unmet medical need in this population during the Type C meeting, according to the company.
Khurram Jamil, chief medical officer at Galectin, said the agency's acceptance of the centralized endoscopy review process confirms the scientific rigor of the methodology the company pioneered in NAVIGATE. Standardized, blinded assessment of esophageal varices is critical to ensuring reliable endpoint evaluation in global studies, he said.
The competitive landscape for MASH cirrhosis remains sparse. Madrigal Pharmaceuticals' Rezdiffra (resmetirom) won FDA approval in 2024 for non-cirrhotic MASH with moderate-to-advanced fibrosis, but no therapy is approved specifically for patients with cirrhosis and portal hypertension. Galectin's belapectin, if successful, would address that gap.
Galectin has incurred operating losses since inception and has not disclosed its current cash runway. The company's lead development program is in MASH cirrhosis, with additional programs in combination immunotherapy for advanced head and neck cancers. Advancement of those programs depends on finding a suitable partner.
The NAVIGATE trial, which enrolled patients with MASH cirrhosis and portal hypertension, provided the efficacy data supporting the single-dose approach. The 2 mg dose showed strong efficacy across prior clinical studies, the company said, forming the basis for the planned registration trial.
This article is for informational purposes only and does not constitute investment advice.
