Below is the text of a press release from Santaris describing
the therapy and the new trials.
Hoersholm,
Denmark and San Diego, CA -- September 22, 2010 -- Santaris
Pharma A/S, a clinical-stage biopharmaceutical company focused
on the discovery and development of RNA-targeted therapies,
today announced that it has advanced miravirsen (SPC3649), the
first microRNA-targeted drug to enter clinical trials, into
Phase 2 studies to assess the safety and tolerability of the
drug in treatment-naive patients infected with the hepatitis
C virus (HCV).
Paving
the way to conduct the first clinical trials of a microRNA-targeted
drug in the United States, Santaris Pharma A/S also received
acceptance of its Investigational New Drug (IND) application
from the U.S. Food and Drug Administration (FDA). In addition
to the United States, the Phase 2a clinical trials will be conducted
in the Netherlands, Germany, Poland, Romania, and Slovakia.
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[2]. Approximately 3-4 million Americans are chronically
infected with an estimated 40,000 new infections per year(1).
In Europe, there are about 4 million carriers[2]. The current
standard of care, pegylated interferon in combination with ribavirin,
is effective in only about 50% of those treated[1].
Developed
using Santaris Pharma A/S proprietary Locked Nucleic Acid (LNA)
Drug Platform, miravirsen is a specific inhibitor of miR-122,
a liver specific microRNA that the hepatitis C virus requires
for replication. Miravirsen is designed to recognize and sequester
miR-122, making it unavailable to the Hepatitis C virus. As
a result, the replication of the virus is effectively inhibited
and the level of hepatitis C virus is reduced.
"Advancing
miravirsen, the first microRNA-targeted drug to enter clinical
trials, into Phase 2 studies in patients with Hepatitis C demonstrates
Santaris Pharma A/S leadership in developing RNA-targeted medicines,"
said Arthur A. Levin, PhD, Vice President, Chief Development
Officer and President, U.S. Operations. "Receiving IND
acceptance from the FDA to conduct the first clinical trials
with a microRNA-targeted drug in the United States brings Santaris
Pharma A/S one step closer to potentially providing a growing
number of patients chronically infected with HCV with a more
effective and better tolerated treatment option."
The
LNA Drug Platform is the only technology with both mRNA and
microRNA targeted drugs in clinical trials, reinforcing the
broad utility of the platform. The unique combination of small
size and very high affinity, which is only achievable with LNA-based
drugs, allows this new class of drugs to potently and specifically
inhibit RNA targets in many different tissues without the need
for complex delivery vehicles. LNA-based drugs are a promising
new type of therapy that enables scientists to develop drugs
to attack previously inaccessible pathways.
"Using
our LNA Drug Platform to advance the first microRNA-targeted
therapy into human clinical trials was certainly a scientific
breakthrough," said Henrik Oerum, PhD, Vice President and
Chief Scientific Officer of Santaris Pharma A/S. "We are
extremely pleased with the results of the Phase I trials and
excited to progress miravirsen into Phase 2 clinical trials.
Because of its unique mechanism of action and tolerability profile,
miravirsen has the potential to be an effective treatment option
for patients with HCV."
The
randomized, double-blind, placebo-controlled, ascending multiple-dose
Phase 2a study will assess the safety and tolerability of miravirsen
and is designed to enroll up to 55 treatment-naive patients
with chronic hepatitis C virus genotype 1 infection. Secondary
endpoints include pharmacokinetics of miravirsen and its effect
on viral load. Miravirsen will be given as subcutaneous injections
weekly or every other week for four weeks.
Data
from Phase 1 clinical studies with miravirsen in healthy volunteers
show that the drug is well tolerated. A recent study published
in Science demonstrated that miravirsen successfully inhibited
miR-122 and dramatically reduced hepatitis C virus in the liver
and in the bloodstream in chimpanzees chronically infected with
the Hepatitis C virus[3]. Miravirsen 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.
In
addition to miravirsen, Santaris Pharma A/S has a robust product
pipeline targeting mRNAs and microRNAs both internally as well
as in partnerships and collaborations with miRagen Therapeutics
(cardiovascular diseases), Shire PLC (rare genetic disorders),
Pfizer (undisclosed therapeutic areas), GlaxoSmithKline (viral
disease) and Enzon Pharmaceuticals (oncology).
About
microRNAs
MicroRNAs
have emerged as an important class of small RNAs encoded in
the genome. They act to control the expression of sets of genes
and entire pathways and are thus thought of as master regulators
of gene expression. Recent studies have demonstrated that microRNAs
are associated with many disease processes. Because they are
single molecular entities that dictate the expression of fundamental
regulatory pathways, microRNAs represent potential drug targets
for controlling many biologic and disease processes.
About
Locked Nucleic Acid (LNA) Drug Platform
The
LNA Drug Platform and Drug Discovery Engine developed by Santaris
Pharma A/S combines the company's proprietary LNA chemistry
with its highly specialized and targeted drug development capabilities
to rapidly deliver potent single-stranded LNA-based drug candidates
against RNA targets, both mRNA and microRNA, for a range of
diseases including metabolic disorders, infectious and inflammatory
diseases, cancer and rare genetic disorders. The LNA Drug Platform
overcomes the limitations of earlier antisense and siRNA technologies
to deliver potent single-stranded LNA-based drug candidates
across a multitude of disease states. The unique combination
of small size and very high affinity, which is only achievable
with LNA-based drugs, allows this new class of drugs to potently
and specifically inhibit RNA targets in many different tissues
without the need for complex delivery vehicles. LNA-based drugs
are a promising new type of therapy that enables scientists
to develop drugs to attack previously inaccessible clinical
pathways. The most important features of LNA-based drugs include
excellent specificity, providing optimal targeting; increased
affinity to targets providing improved potency; and strong pharmacology
upon systemic delivery without complicated delivery vehicles.
About
Santaris Pharma A/S
Santaris
Pharma A/S is a privately held clinical-stage biopharmaceutical
company focused on the discovery and development of RNA-targeted
therapies. The Locked Nucleic Acid (LNA) Drug Platform and Drug
Discovery Engine developed by Santaris Pharma A/S combine the
company's proprietary LNA chemistry with its highly specialized
and targeted drug development capabilities to rapidly deliver
potent single-stranded LNA-based drug candidates across a multitude
of disease states. The company'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, cardiovascular disease,
infectious and inflammatory diseases, and rare genetic disorders.
The company has strategic partnerships with miRagen Therapeutics,
Shire plc, Pfizer, GlaxoSmithKline, and Enzon Pharmaceuticals.
As part of its broad patent estate, the company holds exclusive
worldwide rights to all therapeutic uses of LNA. Santaris Pharma
A/S, founded in 2003, is headquartered in Denmark with operations
in the United States. Please visit http://www.santaris.com
for more information.
9/24/10
References
1.
American
Association for the Study of Liver Diseases.
2.
World
Health Organization.
3.
Science. 2010 Jan 8; 327(5962):198-201. Epub 2009 Dec
3.
Source
Santaris
Pharma. Santaris Pharma A/S Advances Miravirsen, the First MicroRNA-Targeted
Drug to Enter Clinical Trials, into Phase 2 to Treat Patients
Infected with Hepatitis C Virus. Press release. September 20,
2010.
