Accueil / Communiqués / Dicerna Announces Dosing of First Volunteer in Phase 1 Clinical Trial of DCR-HBVS Designed for the Treatment of Chronic Hepatitis B Virus

Dicerna Announces Dosing of First Volunteer in Phase 1 Clinical Trial of DCR-HBVS Designed for the Treatment of Chronic Hepatitis B Virus

Monday, January 28th 2019 at 12:30pm UTC

— Clinical Proof-of-Concept Data Expected in Second Half of 2019 —

CAMBRIDGE, Mass.–(BUSINESS WIRE)– Dicerna
Pharmaceuticals, Inc
. (NASDAQ: DRNA), (the “Company”) a leading
developer of RNA interference (RNAi) therapeutics, today announced the
dosing of the first human volunteer in its Phase 1 clinical trial of
DCR-HBVS, the Company’s investigational GalXCTM-based therapy
for the treatment of chronic hepatitis B virus (HBV) infection in
adults. The Company anticipates human proof-of-concept data from the
Phase 1 trial, which is known as DCR-HBVS-101, in the 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
effectively,” said Ralf Rosskamp, M.D., chief medical officer of
Dicerna. “We are hopeful that this three-part Phase 1 trial will
validate RNA interference as a viable clinical strategy against chronic
hepatitis B infection, based upon our encouraging preclinical data on
DCR-HBVS.”

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 Edward Gane, MBCHB, M.D., deputy
director and hepatologist of the New Zealand Liver Transplant Unit at
Auckland City Hospital and clinical professor of Medicine at the
University of Auckland School of Medicine. “Given the encouraging
inhibitory activity of DCR-HBVS in animal studies, as well as its
favorable preclinical safety profile, we eagerly anticipate the first
results from healthy volunteers in the DCR-HBVS-101 trial, and then in
the second part of the study, from patients with chronic hepatitis B.”

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 World Health Organization. Chronic HBV
infection, a condition characterized by the presence of the HBV surface
antigen (HBsAg) for six months or more, claims approximately 780,000
lives annually; an estimated 650,000 of these deaths are caused by
cirrhosis and liver cancer as a result of chronic hepatitis B, and
130,000 of these deaths result from complications associated with acute
disease.1

About DCR-HBVS

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.

About Dicerna Pharmaceuticals, Inc.

Dicerna Pharmaceuticals, Inc., is a biopharmaceutical company focused on
the discovery and development of innovative, subcutaneously delivered
RNAi-based therapeutics for the treatment of diseases involving the
liver, including rare diseases, chronic liver diseases, cardiovascular
diseases, and viral infectious diseases. Dicerna is leveraging its
proprietary GalXC™ RNAi technology platform to build a broad pipeline in
these core therapeutic areas, focusing on target genes where connections
between target gene and diseases are well understood and documented.
Dicerna intends to discover, develop and commercialize novel
therapeutics either on its own or in collaboration with pharmaceutical
partners. Dicerna has strategic collaborations with Boehringer
Ingelheim, Eli Lilly and Company, and Alexion Pharmaceuticals. For more
information, please visit www.dicerna.com.

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
Securities and Exchange Commission (SEC). These risks and uncertainties
include, among others, the cost, timing and results of preclinical
studies and clinical trials and other development activities; the
unpredictability of the duration and results of regulatory review of New
Drug Applications and Investigational NDAs; market acceptance for
approved products and innovative therapeutic treatments; competition;
the possible impairment of, inability to obtain and costs of obtaining
intellectual property rights; and possible safety or efficacy concerns,
general business, financial and accounting risks and litigation. The
forward-looking statements contained in this press release reflect
Dicerna’s current views with respect to future events, and Dicerna does
not undertake and specifically disclaims any obligation to update any
forward-looking statements.

References

1.  

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.

Contacts

Investors:
Rx Communications Group
Paula Schwartz,
917-322-2216
pschwartz@rxir.com

Media:
SmithSolve
Alex Van Rees, 973-442-1555 ext. 111
alex.vanrees@smithsolve.com

Source: Dicerna Pharmaceuticals, Inc.


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