Regeneron's Gene-Silencing Therapy for Liver Disease Shows Promise in Ongoing Clinical Study

Regeneron Pharmaceuticals is conducting a Phase 2 clinical study to evaluate the effectiveness of ALN-HSD, a gene-silencing drug, in reducing liver scarring in patients with MASH. The study is ongoing and recruiting participants. Positive results could enhance investor confidence and position Regeneron favorably in the gene-silencing space.

Title: Regeneron's Phase 2 Clinical Study on ALN-HSD for MASH: A Promising Development in Liver Disease Treatment

Regeneron Pharmaceuticals ((REGN)) has recently announced an update on their ongoing Phase 2 clinical study, titled ‘A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study of siRNA Gene Silencing for the Treatment of Metabolic Dysfunction-Associated Steatohepatitis (MASH) in Participants With Genetic Risk Factors’ [1]. The study aims to evaluate the effectiveness of ALN-HSD, a gene-silencing drug, in reducing liver scarring in patients with MASH, a serious liver condition that can lead to cirrhosis and liver failure.

The intervention being tested, ALN-HSD, is administered via subcutaneous injection. It is designed to improve liver function and reduce inflammation associated with MASH by silencing specific genes involved in the disease process. The study employs a randomized, double-blind, placebo-controlled design with a parallel intervention model, ensuring unbiased results. The primary purpose of the study is treatment-focused.

The study began on February 9, 2023, and is currently recruiting participants. The last update was submitted on July 29, 2025, indicating ongoing progress. These dates are crucial for tracking the study’s progress and anticipated milestones. This clinical update could influence Regeneron’s stock performance by showcasing their commitment to innovative treatments in the liver disease market. Positive results could enhance investor confidence and position Regeneron favorably against competitors in the gene-silencing space.

Meanwhile, Madrigal Pharmaceuticals (MDGL) has struck a licensing deal with CSPC Pharmaceutical Group (1093.HK) that could be worth more than $2 billion for the Chinese company if it results in a new drug for a serious form of liver disease [2]. The agreement gives Madrigal exclusive global rights to SYH2086, an oral GLP-1 receptor agonist designed to mimic the effects of popular weight-loss medications such as Ozempic and Wegovy. The drug is currently being tested in lab studies. Madrigal plans to combine SYH2086 with its flagship therapy Rezdiffra, the only U.S. Food and Drug Administration-approved therapy for metabolic dysfunction-associated steatohepatitis (MASH) with moderate-to-advanced liver scarring.

MASH, formerly known as nonalcoholic steatohepatitis (NASH), is a progressive liver disease linked to obesity, diabetes, and high cholesterol. It can lead to liver failure and is a growing cause of the organ's transplants globally. The deal between Madrigal and CSPC is part of a broader trend of Western drugmakers tapping Chinese biotech firms for innovative therapies, particularly in fast-growing areas such as GLP-1 drugs.

Madrigal Pharmaceuticals has announced a global license agreement with CSPC Pharmaceutical Group for SYH2086, a preclinical oral GLP-1 receptor agonist aimed at developing combination treatments for metabolic dysfunction-associated steatohepatitis (MASH), utilizing Madrigal's existing therapy, Rezdiffra™ [3]. The collaboration aims to enhance treatment efficacy for MASH by combining Rezdiffra’s benefits with SYH2086’s weight loss effects in a single daily pill. Madrigal intends to initiate clinical development of SYH2086 in early 2026, following an upfront payment of $120 million to CSPC, with additional milestone payments potentially reaching $2 billion.

References:
[1] https://www.tipranks.com/news/company-announcements/regenerons-gene-silencing-approach-to-combat-liver-disease-a-clinical-study-update
[2] https://www.reuters.com/business/healthcare-pharmaceuticals/madrigal-signs-potential-2-billion-plus-deal-with-chinas-cspc-develop-liver-drug-2025-07-30/
[3] https://www.quiverquant.com/news/Madrigal+Pharmaceuticals+Enters+Exclusive+License+Agreement+with+CSPC+for+SYH2086+to+Enhance+MASH+Treatment+Pipeline