Dicerna Announces Roche’s Initiation of GalXC™ RNAi Candidate RG6346 in Phase 2 Combination Trial for Treatment of Chronic Hepatitis B Virus Infection
Mar 04, 2021
– Phase 2 Initiation Triggers $25
“Currently available therapies fall short of the ultimate goal of providing patients with a functional cure for chronic HBV, which claims the lives of more than 880,000 people worldwide each year,” said
“We are very encouraged by the results from the Phase 1 trial of RG6346, which have shown a strong and sustained reduction in the levels of hepatitis B surface antigen,” commented
The Roche Phase 2 trial (NCT04225715) is a randomized, adaptive, open-label platform trial designed to evaluate the safety, tolerability and efficacy of multiple combinations of novel agents in patients infected with chronic HBV against a common control, allowing rapid inclusion of additional treatment arms as needed. A combination of Roche’s novel investigational TLR7 agonist and core protein allosteric modulator (CpAM) inhibitor is currently already running within this study. In
About Chronic Hepatitis B Virus (HBV) Infection
Hepatitis B virus (HBV) is the world’s most common serious liver infection and affects an estimated 292 million people worldwide.1 According to the
RG6346 is an investigational GalXC™ RNAi therapeutic candidate in development in collaboration with Roche for the treatment of chronic hepatitis B virus (HBV) infection. Current therapies for HBV, such as nucleos(t)ide analogs, can provide long-term viral suppression if taken continuously, but they rarely lead to long-term functional cures, as measured by the clearance of HBV surface antigen (HBsAg) and sustained HBV deoxyribonucleic acid (DNA) suppression in patient plasma or blood. By contrast, RG6346 is designed to employ RNAi to knock down selectively HBsAg messenger RNA (mRNA) and protein expression in hepatocytes, which is required for the HBV virus lifecycle. Preclinical data have demonstrated greater than 99.9% reduction in circulating HBsAg, as observed in mouse models of HBV infection. Results from a Phase 1 trial of RG6346 demonstrated that four monthly doses of RG6346 treatment resulted in substantial and durable reductions in HBsAg levels lasting up to one year following the last dose. We believe RG6346 has the potential to deliver a functional cure as part of a combination regimen for patients living with chronic HBV.
About Dicerna's GalXC™ RNAi Technology Platform
Dicerna’s proprietary GalXC™ RNA interference (RNAi) platform aims to advance the development of next-generation RNAi-based therapies designed to silence genes implicated in disease. Investigational therapeutics developed using our flagship GalXC technology utilize a proprietary N-acetyl-D-galactosamine (GalNAc)-mediated structure of double-stranded RNA molecules that are designed to bind specifically to receptors on liver cells, leading to selective hepatocyte internalization and access to the RNAi machinery within the cells. Dicerna is continuously innovating and exploring new applications for RNAi technology beyond GalNAc-mediated delivery to the liver, including alternative RNA structures and fully synthetic ligands that target other tissues and enable new therapeutic applications, referred to as GalXC-Plus™.
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Examples of forward-looking statements include, among others, statements we make regarding the collaboration agreement with Roche and the potential of RG6346 to result in a functional cure for chronic hepatitis B virus infection in combination with other agents with different mechanisms. The process by which investigational therapies could potentially lead to an approved product is long and subject to highly significant risks. Applicable risks and uncertainties include those relating to Dicerna’s clinical research and other risks identified under the heading "Risk Factors" included in the Company’s most recent filings on Forms 10-K and 10-Q and in other future filings with the
1 Polaris Observatory Collaborators. Global prevalence, treatment, and prevention of hepatitis B virus infection in 2016: a modelling study. The Lancet Gastroenterology and Hepatology. 2018;3(6):383-403.
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