ALN-BCAT / Alnylam - LARVOL DELTA

RNAi mediated inhibition of beta-catenin demonstrates anti-tumor efficacy and immune microenvironment modulation in preclinical hepatocellular carcinoma models (AACR 2024) - "For the 40% to 50% of HCC patients who are candidates for systemic therapies, the landscape has evolved significantly in the last 10 years, with most recently, immunotherapies such as combination of atezolizumab and bevacizumab emerging as the current first-line systemic therapy for patients with intermediate or advanced disease. Finally, ALN-BCAT treatment results in changes in the tumor microenvironment in a mouse syngeneic HCC model, with increases in CD4+ and CD8+ T cells and reductions in M2 macrophages, consistent with a shift to a more immune stimulatory anti-tumor microenvironment. Here, we demonstrate that ALN-BCAT provides a novel approach to the inhibition of the WNT pathway as a therapeutic approach for treatment of HCC patients and WNT pathway activated tumors."

IO biomarker • Preclinical • Gastrointestinal Cancer • Hepatocellular Cancer • Oncology • Solid Tumor • CD4 • CD8 • CTNNB1

ALN-BCAT-001: A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma (clinicaltrials.gov) - P1 | N=158 | Recruiting | Sponsor: Alnylam Pharmaceuticals | Not yet recruiting ➔ Recruiting

Combination therapy • Enrollment open • Metastases • Monotherapy • Gastrointestinal Cancer • Hepatocellular Cancer • Oncology • Solid Tumor

A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma (clinicaltrials.gov) - P1 | N=158 | Not yet recruiting | Sponsor: Alnylam Pharmaceuticals

Combination therapy • Metastases • Monotherapy • New P1 trial • Gastrointestinal Cancer • Hepatocellular Cancer • Oncology • Solid Tumor

Alnylam Pharmaceuticals Reports Second Quarter 2024 Financial Results and Highlights Recent Period Activity (Businesswire) - "In mid- and late 2024, Alnylam intends to: Submit a supplemental New Drug Application (sNDA) for vutrisiran to the FDA using a Priority Review Voucher. Initiate a Phase 3 study of ALN-TTRsc04 in patients with ATTR-CM at or around year-end. Report interim results from Part B of the Phase 1 study of mivelsiran (ALN-APP) in patients with Alzheimer’s disease. Initiate a Phase 2 study of mivelsiran in patients with Alzheimer’s disease at or around year-end. Initiate a Phase 1 study of ALN-BCAT, in development for the treatment of hepatocellular carcinoma."

FDA filing • New P1 trial • New P2 trial • New P3 trial • P1 data • Alzheimer's Disease • Amyloidosis • Cardiomyopathy • CNS Disorders • Hepatocellular Cancer • Liver Cancer • Metabolic Disorders • Oncology • Solid Tumor