Jim.henderson / Wikimedia Commons / CC0 / September 2014 Eastview building archive, resized
Regeneron said a single dose of an experimental medicine reduced liver fat by 58% to 80% across dose groups after 36 weeks in a small early-stage trial, offering an initial signal for a treatment aimed at metabolic dysfunction-associated steatotic liver disease, or MASLD.
The company’s November 8 announcement described the first results from a randomized, double-blind, placebo-controlled Phase 1/2 study of CIDEB siRNA. Thirty participants received the medicine across four dose levels, while 10 received placebo. The study’s main purpose was to assess safety and tolerability; changes in liver fat and liver-injury markers were secondary measures.
At Week 36, the placebo group had a mean liver-fat reduction of 2.9%. Among participants receiving CIDEB siRNA, 62% reached liver fat below 5%, the trial’s definition of normalization, compared with none on placebo. The measurements were made using an MRI-based test.
Regeneron also reported improvements in markers of liver injury, including lower mean ALT levels across all doses, sustained through Week 36. It said the findings were presented at the American Association for the Study of Liver Diseases meeting and published simultaneously in The New England Journal of Medicine.
MASLD involves excess fat building up in the liver and can include more serious disease with inflammation and scarring. The new results measure liver fat and injury markers, not a demonstrated reversal of fibrosis. They do not establish that the experimental medicine is a cure or that it prevents liver failure or other long-term complications.
Overall adverse-event rates were generally similar between the active-treatment and placebo groups, according to Regeneron. Events were mild or moderate except for one serious episode of severe hyperglycemia in a treated participant, which was attributed to a later diagnosis of adult-onset Type 1 diabetes rather than the study medicine. There were no trial discontinuations.
CIDEB siRNA is designed to mimic the protective effect of rare genetic changes in the CIDEB gene. Regeneron developed the approach with Alnylam and holds exclusive development and commercialization rights, while Alnylam is entitled to milestone and royalty payments.
The medicine remains investigational, and Regeneron said its safety and efficacy have not been evaluated by a regulatory authority. Longer-term safety and tissue-based outcomes are being studied in ongoing trials. The company will discuss its liver-disease pipeline at a virtual investor event Tuesday, November 10, at 8:30 a.m. Eastern, available through its events website.
JBizNews Desk | Tarrytown, New York
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