GlobeNewswire·Nov 8, 2026, 4:00 PM ET

CIDEB siRNA Normalizes Liver Fat and Improves Liver Injury Markers in MASLD Patients, as Highlighted at AASLD

REGN

A single subcutaneous dose of investigational CIDEB siRNA produced rapid, deep, & durable liver fat reductions of 58-80% sustained through 36 weeks in patients with metabolic dysfunction-associated steatotic liver disease (MASLD).
Liver fat normalized in 62% of recipients by week 36, with 93% achieving a ≥30% reduction linked to histological MASH resolution, compared to a 2.9% mean decrease in the placebo group.
Improvements in liver injury markers, including alanine aminotransferase (ALT) & aspartate aminotransferase (AST), were observed at early timepoints & maintained through week 36.
The first-in-human Phase 1/2 trial demonstrated a favorable safety profile with adverse events generally similar to placebo, no nausea or vomiting reported, & no trial discontinuations.
CIDEB siRNA is a first-in-class, liver-targeted small interfering RNA therapeutic that mimics protective effects of rare CIDEB gene mutations discovered by the Regeneron Genetics Center.
Results were published in The New England Journal of Medicine & presented orally at AASLD The Liver Meeting 2026, supporting the potential for twice-yearly dosing.
Ongoing Phase 2a, Phase 2b, & Phase 3 trials are recruiting to evaluate longer-term safety & histologic outcomes.
Regeneron will host a virtual investor event titled "Regeneron Roundtable" on Tuesday, November 10 at 8:30 a.m. ET to discuss its MASH pipeline programs.
The therapy targets a liver-expressed receptor to reduce fat accumulation in the spectrum of conditions ranging from simple fatty liver to metabolic dysfunction-associated steatohepatitis (MASH).
Regeneron holds exclusive rights for the development & commercialization of CIDEB siRNA following a collaboration with Alnylam, which receives milestone & royalty payments.

Company: Regeneron Pharmaceuticals, Inc.
Keywords: Nasdaq:REGN, Regeneron Pharmaceuticals, Inc.