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发表于 2008-3-1 12:46
Fri Jan 11, 2008 8:30am EST
First Patient Treated With Systemically Delivered Gene Silencing
Therapeutic Designed to Reduce Both Viral Titer and Destructive HBV
Antigen Load
HORSHAM, Pa.--(Business Wire)--Nucleonics, Inc., a privately held biotechnology company focused
on the development of novel RNA interference (RNAi)-based
therapeutics, announced today that the company has begun treating
patients in a Phase 1 human safety study of its experimental treatment
for chronic Hepatitis B virus (HBV) infection, NUC B1000.
NUC B1000 (http://www.nucleonicsinc.com/products/hepb.html) is an
expressed interfering RNA (eiRNA)-based product consisting of a
plasmid DNA construct designed to produce four short interfering RNA
(siRNA) molecules, formulated with a proprietary cationic-lipid
delivery system. Each of the four siRNAs targets a different sequence
of the HBV genome, collectively leading to the potential destruction
or elimination of all RNA species produced by HBV within an infected
cell. The result is a potent antiviral effect designed for efficacy
against all HBV genotypes, including drug resistant strains.
Additionally, unlike currently available HBV therapeutics, NUC B1000
is designed to specifically reduce viral antigen load in addition to
viral titer, thereby reducing the destructive effects of hepatitis and
increasing the potential for resolution of viral infection.
"Despite the widespread availability of multiple prophylactic
vaccines against HBV, chronic HBV infection remains a public health
problem around the world that can lead to such serious diseases as
cirrhosis, fibrosis and liver cancer in up to one-third of patients,"
said Principal Investigator, Robert G. Gish, M.D., Medical Director,
Liver Transplant Program and Chief, Division of Hepatology and Complex
GI, California Pacific Medical Center, San Francisco. "Current
anti-HBV drug therapies are of limited effectiveness and are not often
used in patients with mild to moderate disease due to risks of
developing drug resistance. Thus there is significant need for new
treatments that can improve patient response rates to therapy and
prevent the emerging drug resistance associated with the use of
conventional antiviral therapeutics, enabling more patients to benefit
from treatment."
Nucleonics plans to enroll a total of 15 patients infected with
HBV with mild to moderate disease and no evidence of cirrhosis at
three U.S. clinical sites and two sites in Eastern Europe. The
patients are being organized into five escalating dose groups of three
patients per group. Primary endpoints are safety-related, with
secondary endpoints tracking biological markers of efficacy, including
HBV viral levels in the blood and circulating HBV surface antigen
(HBsAg) levels. An independent safety monitor as well as a data safety
monitoring board (DSMB) will oversee the trial. There is a planned
stop and interim safety analysis after completion of the third dose
group before proceeding to the fourth and fifth cohorts.
"We are very pleased to initiate this clinical study, which
represents one of the first systemic administrations of any RNAi-based
therapeutic and is the first RNAi treatment directed against HBV,"
said Robert Towarnicki, President and Chief Executive Officer of
Nucleonics, Inc. "We believe Nucleonics' eiRNA approach offers several
major advantages over other RNAi approaches. Each eiRNA molecule
entering the nucleus of a targeted cell produces thousands of copies
of siRNA, and allows for a sustained therapeutic response versus the
repeat administration expected to be required for synthetically
created RNAi drugs. Moreover, one eiRNA drug molecule has the
potential to encode four or more different siRNAs, enabling the attack
of multiple targets using a single therapeutic, thereby decreasing the
chances of developing drug resistance. Moreover, plasmid DNA is
non-immunogenic and a stable drug format that benefits from
well-characterized manufacturing methods, storage techniques,
regulatory requirements and the likelihood of a favorable safety
profile based on earlier DNA vaccine clinical trials."
For additional information about clinical studies with NUC B1000,
please visit our web site here. If you have further
questions, please email [email protected].
About Nucleonics, Inc.
Nucleonics, founded in January 2001, is an emerging biotechnology
company focused on the development of novel RNA interference-based
therapeutics for viral and other diseases. Privately owned, Nucleonics
is headquartered in Horsham, Pennsylvania. For more information on the
company, please visit the Nucleonics website at
www.nucleonicsinc.com.
Nucleonics, Inc.
Robert Towarnicki, CEO, 267-518-0101
[email protected]
or
Kureczka/Martin Associates
Joan Kureczka, 415-821-2413 (Media)
[email protected]
Copyright Business Wire 2008 |
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