Dicerna Announces Dosing of First Volunteer in Phase 1 Clinical Trial of DCR-HBVS Designed for the Treatment of Chronic Hepatitis B Virus
Jan 28, 2019
— Clinical Proof-of-Concept Data Expected in Second Half of 2019 —
“The dosing of the first human in the DCR-HBVS-101 trial brings us a
step closer to the potential availability of an innovative therapy for
patients with chronic hepatitis B, a serious liver infection that can
lead to advanced hepatic disease or liver cancer if not treated
DCR-HBVS is comprised of a single GalXC molecule that targets HBV messenger RNAs (mRNAs) within the hepatitis B surface antigen (HBsAg) gene sequence region. Preclinical studies with a standard mouse model of HBV infection showed DCR-HBVS led to greater than 99% reduction in circulating HBsAg, suggesting superior HBsAg suppression (both in magnitude and duration of suppression), compared to targeting within the X gene sequence region.
“RNAi-based therapy has the potential to change the treatment paradigm
for patients with chronic HBV infection. By silencing not only the S
antigen but also other viral genes, through a powerful and long-acting
mechanism, RNAi-based therapy could tip the balance toward allowing the
patient’s own immune system to mount an effective immune response. This
approach could help eradicate HBV and remove the need for life-long
therapy,” said principal investigator
About the DCR-HBVS-101 Trial
The DCR-HBVS-101 clinical trial is a randomized, placebo-controlled study designed to evaluate the safety and tolerability of DCR-HBVS in normal healthy volunteers (NHVs) and in patients with non-cirrhotic chronic HBV. Secondary objectives are to characterize the pharmacokinetic (PK) profile of DCR-HBVS and to evaluate preliminary pharmacodynamics (PD) and antiviral efficacy on plasma levels of HBsAg and HBV in blood. The study is divided into three phases or groups:
- In Group A, 30 NHVs are to receive a single ascending-dose of DCR-HBVS (0.1, 1, 1.5, 3, 6, or 12 mg/kg), with a four-week follow-up period.
- Group B is a single-dose study of DCR-HBVS (3 mg/kg) in eight patients with HBV who are naïve to nucleoside analog therapy; these patients will be followed for at least 12 weeks. The Company expects to initiate Group B dosing in the third quarter of 2019.
- Group C is a multiple ascending-dose study of DCR-HBVS (1.5, 3, or 6 mg/kg) in 18 patients with HBV previously treated with nucleoside analogs with a follow-up period of 24 weeks or more. The Company expects to initiate Group C dosing in the second quarter of 2019.
For more information about the DCR-HBVS clinical study, please visit clinicaltrials.gov and use the identifier NCT03772249.
About Chronic Hepatitis B Virus (HBV) Infection
Hepatitis B virus (HBV) is the world’s most common serious liver
infection, with more than 292 million patients chronically infected,
according to an estimate by the
DCR-HBVS is an investigational drug in development for the treatment of chronic hepatitis B virus (HBV) infection. Current therapies for HBV, such as nucleoside analogs and pegylated interferon, aim to suppress the virus; however, although these treatments can provide long-term viral suppression if taken continuously, they rarely lead to a long-term immunological cure, as measured by the clearance of HBV surface antigen (HBsAg) and sustained HBV deoxyribonucleic acid (DNA) suppression in patient plasma or blood. By contrast, DCR-HBVS targets HBV messenger RNA (mRNA) and leads to greater than 99% reduction in circulating HBsAg, as observed in mouse models of HBV infection. Those data suggest that DCR-HBVS may induce long-term clearance of intrahepatic and serum HBsAg, as well as sustained HBV DNA suppression.
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: (i) the therapeutic
and commercial potential of the GalXC™ platform, including DCR-HBVS;
(ii) research and development plans and timelines related to GalXC,
including DCR-HBVS; and (iii) the potential of our technology and drug
candidates in our research and development pipeline. The process by
which an early stage investigational therapy such as DCR-HBVS and an
early stage platform such as GalXC could potentially lead to an approved
product is long and subject to highly significant risks. Applicable
risks and uncertainties include those relating to our clinical research
and other risks identified under the heading "Risk Factors" included in
our most recent Form 10-Q filing and in other future filings with the
Hepatitis B Foundation. Facts and Figures. 2019. Available at: http://www.hepb.org/what-is-hepatitis-b/what-is-hepb/facts-and-figures/. Accessed on January 17, 2019.
Rx Communications Group
Paula Schwartz, 917-322-2216
Alex Van Rees, 973-442-1555 ext. 111