New Data Shows Breakthrough microRNA-Targeted Therapy Developed Using Santaris Pharma A/S Proprietary LNA Technology Holds Promise as New Treatment for Hepatitis C
- SPC3649 Successfully Inhibits miR-122, a microRNA Important
for Hepatitis C Viral Replication, Thereby Significantly Reducing
Hepatitis C Virus in the Bloodstream in Chimpanzees Chronically
Infected With the Hepatitis C Virus
- SPC3649 Demonstrates Efficacy With No Evidence of Viral
Resistance and No Serious Adverse Events in the Treated Animals -
Signaling a Potential Benefit for Patients Who are Not Responsive
to or Cannot Tolerate Current Standard of Care Therapies
- SPC3649 is the First microRNA-Targeted Drug to Enter Human
Clinical Trials; Phase 1 Clinical Trials Ongoing
- Santaris Pharma A/S Proprietary Locked Nucleic Acid (LNA) Drug
Platform Fundamental in Developing Effective RNA-Targeted Therapies
With High Affinity, Target Specificity and Remarkable Potency for a
Range of Diseases
HOERSHOLM, Denmark and SAN DIEGO, California, December
3/PRNewswire/ -- A study published online in this week's
Science shows that SPC3649, a breakthrough microRNA-targeted
therapy developed by Santaris Pharma A/S using its proprietary
Locked Nucleic Acid (LNA) technology, holds promise as a novel
treatment for patients infected with the Hepatitis C virus
(HCV).
(Logo: http://www.newscom.com/cgi-bin/prnh/20090921/360545 )
The World Health Organization estimates about 3% of the world's
population has been infected with HCV and that some 170 million are
chronic carriers at risk of developing liver cirrhosis and/or liver
cancer[1]. Approximately 3-4 million Americans are chronically
infected with an estimated 40,000 new infections per year[2]. In
Europe, there are about 4 million carriers[3].
Santaris Pharma A/S, the first company to have advanced both
mRNA and microRNA targeted drugs into clinical trials, is an
international biopharmaceutical company focused on the discovery
and development of RNA-targeted therapies for a range of diseases
including metabolic disorders, infectious and inflammatory
diseases, cancer and rare genetic disorders.
In this preclinical study, SPC3649 successfully inhibited
miR-122, a liver-expressed microRNA important for Hepatitis C viral
replication. By inhibiting miR-122, SPC3649 dramatically reduced
Hepatitis C virus in the liver and in the bloodstream in
chimpanzees chronically infected with the Hepatitis C virus[4].
Four HCV chronically infected chimpanzees were treated weekly with
5 or 1 mg/kg of SPC3649 for 12 weeks followed by a treatment free
period of 17 weeks. The two animals that received the 5 mg/kg dose
had a significant decline in viral levels in the blood and liver of
approximately 2.5 orders of magnitude or approximately 350
fold[5].
"In collaborating with Santaris Pharma, we proved that the drug
worked exceptionally well in treating HCV infections in
chimpanzees," said Robert Lanford, Ph.D., a scientist at the
Southwest Foundation for Biomedical Research and one of the lead
authors on the study. "The current standard anti-HCV treatment,
which combines pegylated interferon-alpha with ribavirin, is
effective in only about 50% of patients and is often associated
with severe side effects. Because of the unique mechanism of action
of SPC3649 and its tolerability profile, this new therapy could
have the potential to replace interferon to treat disease
progression or be combined with current treatments."
SPC3649 provided continued efficacy in the animals up to several
months after the treatment period with no adverse events and no
evidence of viral rebound or resistance, an important factor that
distinguishes SPC3649 from direct antiviral HCV therapeutics.
Current antiviral therapies that target the virus directly are
challenged as the HCV continually mutates to develop resistance to
treatment. Because SPC3649 inhibits miR-122, an important microRNA
involved in HCV replication, the HCV is blocked from replicating
without the apparent selection of resistant mutants. SPC3649 has
other important properties that make it attractive as a therapeutic
agent for HCV. The preclinical data show changes in the expression
of key genes that may help patients who do not respond to
interferon treatment to become responsive.
SPC3649 is the first microRNA-targeted drug to enter human
clinical trials and is currently undergoing Phase 1 clinical
studies in healthy volunteers. These preclinical data provide even
greater impetus to further examine the potential of SPC3649 for
treating patients infected with HCV.
"Advancing the first microRNA-targeted therapy, SPC3649, into
human clinical trials was certainly a breakthrough in science and
we are very encouraged by these preclinical findings demonstrating
that SPC3649 has the potential to be an effective treatment for
patients infected with the Hepatitis C virus," said Henrik 0rum,
PhD, Vice President and Chief Scientific Officer of Santaris Pharma
A/S. "In drug discovery and development programs internally and
with our partners, we continue to demonstrate that our proprietary
LNA Drug Platform is fundamental in developing effective
RNA-targeted therapies with high affinity, target specificity and
remarkable potency for a range of diseases."
Santaris Pharma A/S has a robust product pipeline based on its
proprietary LNA technology including mRNA and microRNA drug
discovery and development partnerships and collaborations with
Shire (rare genetic disorders), Wyeth, now part of Pfizer,
(delivery of lead candidates against up to ten targets),
GlaxoSmithKline (four viral disease drug candidates) and Enzon
Pharmaceuticals (eight cancer targets successfully
delivered).
About Locked Nucleic Acid (LNA) Drug Platform
The Locked Nucleic Acid (LNA) Drug Platform developed by
Santaris Pharma A/S creates synthetically modified chemical
versions of the normal nucleic acid building blocks of ribonucleic
acids (RNA). These modified chemical versions called LNAs improve
the drug-like qualities of resulting therapeutics
(oligonucleotides) by improving affinity to their target RNA,
boosting resistance to metabolism and improving tissue uptake. Upon
systemic administration of these "naked" molecules, LNA-based
therapeutics are delivered to many different tissues where they
show potencies many-fold better than other oligonucleotide
therapeutics.
About Santaris Pharma A/S
Santaris Pharma A/S is a clinical-stage biopharmaceutical
company focused on the discovery and development of RNA-targeted
therapies. The Company's proprietary Locked Nucleic Acid (LNA) Drug
Platform, in combination with its highly specialized and targeted
drug development capabilities, offers potential partners
pre-screened drug candidates for commercial consideration across a
multitude of disease states. Santaris Pharma A/S research and
development activities focus on infectious diseases and metabolic
disorders while partnerships with major pharmaceutical companies
include a range of therapeutic areas including cancer, rare genetic
disorders and inflammatory diseases. Santaris Pharma A/S, founded
in 2003, is privately-held and headquartered in Denmark with
operations in the US. As part of its broad patent estate, the
Company holds exclusive worldwide rights to all therapeutic uses of
LNAs. The Company has strategic partnerships with Enzon
Pharmaceuticals, GlaxoSmithKline, Wyeth Pharmaceuticals (now part
of Pfizer) and Shire plc. Please visit http://www.santaris.com for
more information.
---------------------------------
[1] World Health Organization -
http://www.who.int/csr/disease/hepatitis/Hepc.pdf
[2] American Association for the Study of Liver Diseases -
http://www.aasld.org/patients/Pages/LiverFastFactsHepC.aspx
[3] World Health Organization -
http://www.who.int/csr/disease/hepatitis/Hepc.pdf
[4] The chimpanzee is the only animal other than humans that is
susceptible to HCV
[5] Lanford et al, Therapeutic silencing of microRNA-122 in
primates with chronic hepatitis C virus infection, Science
online
Media Contact:
Navjot Rai
Santaris Pharma A/S
Office: +1-858-764-7064 ext. 206
Cell: +1-619-723-5450
e-mail: navjot.rai@santaris.com
Photo: http://www.newscom.com/cgi-bin/prnh/20090921/360545
Source: Santaris Pharma A/S
Media Contact: Navjot Rai, Santaris Pharma A/S, Office:
+1-858-764-7064 ext. 206, Cell: +1-619-723-5450, e-mail:
navjot.rai@santaris.com
Posted: December 2009
