rapirosiran (ALN-HSD) / Regeneron, Alnylam - LARVOL DELTA

NASHGEN-2: A Precision Medicine Approach Using Gene Silencing to Treat a Chronic Liver Disease Called Non-Alcoholic Steatohepatitis (NASH) in Adult Participants at Increased Genetic Risk for This Condition (clinicaltrials.gov) - P2 | N=120 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Active, not recruiting ➔ Recruiting

Enrollment open • Hepatology • Metabolic Disorders • Metabolic Dysfunction-Associated Steatohepatitis

A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH) (clinicaltrials.gov) - P1 | N=6 | Terminated | Sponsor: Alnylam Pharmaceuticals | Completed ➔ Terminated; Study ALN-HSD-001 was terminated due to business reasons and changes in the development strategy of the product. No safety issues were found.

Trial termination • Hepatology • Metabolic Dysfunction-Associated Steatohepatitis

A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH) (clinicaltrials.gov) - P1 | N=6 | Completed | Sponsor: Alnylam Pharmaceuticals | Active, not recruiting ➔ Completed

Trial completion • Hepatology • Metabolic Dysfunction-Associated Steatohepatitis

NASHGEN-2: A Precision Medicine Approach Using Gene Silencing to Treat a Chronic Liver Disease Called Non-Alcoholic Steatohepatitis (NASH) in Adult Participants at Increased Genetic Risk for This Condition (clinicaltrials.gov) - P2 | N=120 | Active, not recruiting | Sponsor: Regeneron Pharmaceuticals | N=300 ➔ 120 | Trial completion date: Dec 2026 ➔ Sep 2027 | Trial primary completion date: Dec 2026 ➔ Sep 2027

Enrollment change • Trial completion date • Trial primary completion date • Hepatology • Metabolic Disorders • Metabolic Dysfunction-Associated Steatohepatitis

NASHGEN-2: A Study to Evaluate the Efficacy and Safety of ALN-HSD in Adult Participants With Non-alcoholic Steatohepatitis (NASH) With Fibrosis With Genetic Risk Factors (clinicaltrials.gov) - P2 | N=300 | Active, not recruiting | Sponsor: Regeneron Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • Fibrosis • Hepatology • Immunology • Non-alcoholic Steatohepatitis

A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH) (clinicaltrials.gov) - P1 | N=6 | Active, not recruiting | Sponsor: Alnylam Pharmaceuticals | N=114 ➔ 6 | Trial completion date: May 2024 ➔ Dec 2023 | Trial primary completion date: May 2024 ➔ Dec 2023

Enrollment change • Trial completion date • Trial primary completion date • Hepatology • Non-alcoholic Steatohepatitis

A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH) (clinicaltrials.gov) - P1 | N=114 | Active, not recruiting | Sponsor: Alnylam Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • Hepatology • Non-alcoholic Steatohepatitis

Phase 1 study of the RNA interference therapeutic ALN-HSD in healthy adults and patients with nonalcoholic steatohepatitis (EASL-ILC 2023) - P1, P2 | "ALN-HSD has exhibited an encouraging safety and tolerability profile to date, with robust reduction in liver HSD17B13 mRNA expression. A Phase 2 study has been initiated to further investigate ALN-HSD in patients with NASH (NCT05519475). Co-funded by Alnylam Pharmaceuticals and Regeneron Pharmaceuticals, Inc."

Clinical • P1 data • Addiction (Opioid and Alcohol) • Fibrosis • Gastrointestinal Cancer • Gastrointestinal Disorder • Hepatocellular Cancer • Hepatology • Immunology • Infectious Disease • Liver Failure • Non-alcoholic Steatohepatitis • Oncology • Otorhinolaryngology • Respiratory Diseases • Solid Tumor

A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH) (clinicaltrials.gov) - P1 | N=114 | Recruiting | Sponsor: Alnylam Pharmaceuticals | Active, not recruiting ➔ Recruiting | Trial completion date: Feb 2024 ➔ May 2024 | Trial primary completion date: Feb 2024 ➔ May 2024

Enrollment open • Trial completion date • Trial primary completion date • Hepatology • Non-alcoholic Steatohepatitis

NASHGEN-2: A Study to Evaluate the Efficacy and Safety of ALN-HSD in Adult Participants With Non-alcoholic Steatohepatitis (NASH) With Fibrosis With Genetic Risk Factors (clinicaltrials.gov) - P2 | N=300 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • Fibrosis • Hepatology • Immunology • Non-alcoholic Steatohepatitis

A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH) (clinicaltrials.gov) - P1 | N=103 | Active, not recruiting | Sponsor: Alnylam Pharmaceuticals | Trial completion date: Jan 2023 ➔ Feb 2024 | Trial primary completion date: Jan 2023 ➔ Feb 2024

Trial completion date • Trial primary completion date • Hepatology • Non-alcoholic Steatohepatitis

NASHGEN-2: A Study to Evaluate the Efficacy and Safety of ALN-HSD in Adult Participants With Non-alcoholic Steatohepatitis (NASH) With Fibrosis With Genetic Risk Factors (clinicaltrials.gov) - P2 | N=300 | Not yet recruiting | Sponsor: Regeneron Pharmaceuticals | Trial completion date: Aug 2026 ➔ Dec 2026 | Initiation date: Oct 2022 ➔ Jan 2023 | Trial primary completion date: Aug 2026 ➔ Dec 2026

Trial completion date • Trial initiation date • Trial primary completion date • Fibrosis • Hepatology • Immunology • Non-alcoholic Steatohepatitis

NASHGEN-2: A Study to Evaluate the Efficacy and Safety of ALN-HSD in Adult Participants With Non-alcoholic Steatohepatitis (NASH) With Fibrosis With Genetic Risk Factors (clinicaltrials.gov) - P2 | N=300 | Not yet recruiting | Sponsor: Regeneron Pharmaceuticals

New P2 trial • Fibrosis • Hepatology • Immunology • Non-alcoholic Steatohepatitis

A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH) (clinicaltrials.gov) - P1 | N=103 | Active, not recruiting | Sponsor: Alnylam Pharmaceuticals | Trial completion date: Jun 2023 ➔ Jan 2023 | Trial primary completion date: May 2023 ➔ Jan 2023

Trial completion date • Trial primary completion date • Hepatology • Non-alcoholic Steatohepatitis

A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH) (clinicaltrials.gov) - P1 | N=103 | Active, not recruiting | Sponsor: Alnylam Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • Hepatology • Non-alcoholic Steatohepatitis

A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH) (clinicaltrials.gov) - P1; N=128; Recruiting; Sponsor: Alnylam Pharmaceuticals; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Hepatology • Non-alcoholic Steatohepatitis

A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH) (clinicaltrials.gov) - P1; N=128; Not yet recruiting; Sponsor: Alnylam Pharmaceuticals

Clinical • New P1 trial • Hepatology • Non-alcoholic Steatohepatitis