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A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of ALN-CIDEB in Adults With Fibrotic Metabolic Dysfunction-Associated Steatohepatitis (MASH)

Study to Evaluate ALN-CIDEB in Adults With Fibrotic Metabolic Dysfunction-Associated Steatohepatitis (MASH)

Somersan-Karakaya Selin

Regeneron Pharmaceuticals, Inc.

777 Old Saw Mill River Road, Tarrytown, NY 10591 United States

clinicaltrials@regeneron.com

Rosario Chikako

Parexel International Inc.

Kayabacho Tower, 1-21-2, Shinkawa, Chuo-ku

+81-80-8929-3137

Clinicaltrial-registration@parexel.com

Pending

Jan. 19, 2027

Interventional

randomized controlled trial

double blind

placebo control

parallel assignment

treatment purpose

1. A diagnosis of MASH documented in the participant's medical history, or a clinical suspicion of MASH based on non-invasive biomarkers and clinical risk factors, including having a history of 1 or more elements of metabolic syndrome as described in the protocol
2. Screening percutaneous liver biopsy demonstrating a NAFLD Activity Score (NAS) >=4 and fibrosis stage F2 or F3 as described in the protocol
3. Has a FibroScan Aspartate aminotransferase (FAST) score >0.35 either at Screening Visit 1 or within approximately 3 months of Screening Visit 1 as described in the protocol
NOTE: Other Protocol-defined Inclusion Criteria Apply

1. Known chronic liver disease other than Metabolic dysfunction-Associated steatotic Liver Disease (MASLD), as determined by the investigator as described in the protocol
2. Prior or current suspected or known drug-induced liver injury within approximately 1 year prior to Screening Visit 1
3. History of liver transplantation, current placement on a liver transplant list, or MELD score >12
4. Known history of alcohol or other substance abuse within the last year or at any time during screening based on investigator's discretion and/or a score on the AUDIT questionnaire >=8
5. Prior current, or planned future use of a Glucagon-Like Peptide-1 (GLP-1) receptor agonist-based therapy or any medication approved for the treatment of MASH unless used at a generally stable dose and regimen since at least 3 months prior to Screening Visit 1 or the qualifying historical liver biopsy and throughout the screening period with no change to the dose or regimen anticipated during the treatment period as described in the protocol
NOTE: Other Protocol-defined Exclusion Criteria Apply

Both

Metabolic Dysfunction-Associated Steatohepatitis (MASH)

ALN-CIDEB Dose 1
- Drug: ALN-CIDEB
- Administered per the protocol

ALN-CIDEB Dose 2
- Drug: ALN-CIDEB
- Administered per the protocol

Placebo
- Drug: Placebo
- Administered per the protocol

Percent change from baseline in liver fat by Magnetic Resonance Imaging-derived Proton Density Fat Fraction (MRI-PDFF)

- Resolution of MASH with no worsening of Nonalcoholic Steatohepatitis-Clinical Research Network (NASH-CRN) fibrosis on liver biopsy
- Percent change from baseline in liver fat by MRI-PDFF
- Achievement of a >=30% reduction in liver fat by MRI-PDFF
- Achievement of <=5% liver fat by MRI-PDFF
- Percent change from baseline in liver fat by MRI-PDFF for each dose level of ALN-CIDEB
- Improvement of NASH-CRN Fibrosis Stage (F) by >=1 with no worsening of MASH
- Occurrence of Treatment-Emergent Adverse Events (TEAEs)
- Severity of TEAEs
- Change from baseline in FibroScan Controlled Attenuation Parameter (CAP)
- Change from baseline in FibroScan Liver Stiffness Measurement (LSM) by Vibration Controlled Transient Elastography (VCTE)
- Change from baseline in Aspartate Aminotransferase (AST)
- Change from baseline in Alanine Aminotransferase (ALT)
- Change from baseline in Enhanced Liver Fibrosis (ELF)
- Change from baseline in PRO-C3
- Change from baseline in ADAPT
- Change from baseline in NIS2+
- Percent change from baseline in liver fat by MRI-PDFF in genetic subpopulations

+81-3-3822-2131

Yes

All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing.

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