Santaris Pharma A/S Advances RNA-Targeted Drug Development Candidate Against PCSK9, an Important New Target for the Treatment of High Cholesterol
By Santaris Pharma As, PRNEMonday, April 26, 2010
HOERSHOLM, Denmark and SAN DIEGO, April 27, 2010 - — Santaris Pharma A/S selects lead research compound SPC5001 targeting
PCSK9 to advance into drug development for the treatment of high cholesterol
— PCSK9, an important new target, is a protein involved in regulating
LDL or "bad" cholesterol in the blood- a major risk factor for coronary heart
disease, heart attack and stroke
— Preclinical data presented at PCSK9 Conference show SPC5001 provided
potent, specific and long-lasting inhibition of PCSK9 and lowered LDL
cholesterol in primates by up to 74%
— Versatility and broad utility of Santaris Pharma A/S LNA Drug Platform
and Drug Discovery Engine critical in developing effective RNA-targeted
therapies for a range of diseases
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 into drug development a discovery research candidate
directed against PCSK9 (proprotein convertase subtilisin/kexin type 9), an
important new target for the treatment of high cholesterol. Using its
proprietary Locked Nucleic Acid (LNA) Drug Platform and Drug Discovery
Engine, Santaris Pharma A/S identified and advanced the new drug, SPC5001, in
just 18 months.
High cholesterol is a major risk factor for coronary heart disease, heart
attack and stroke. According to the World Health Organization, high
cholesterol is estimated to cause 18% of strokes and 56% of heart disease,
globally and amounts to approximately 4.4 million deaths and 40.4 million
disability-adjusted life years(1).
Santaris Pharma A/S presented preclinical data on SPC5001 last month at
the PCSK9 Conference "From gene to therapeutics" in Nantes, France where many
top pharmaceutical companies gathered to discuss efforts to develop therapies
aimed at inhibiting the important new target PCSK9, a protein involved in
regulating LDL (low-density lipoprotein) or "bad" cholesterol in the blood.
Data presented by Santaris Pharma A/S scientists showed that SPC5001
provided potent, specific and long-lasting inhibition of PCSK9 and lowered
mean LDL cholesterol by 50% in non-human primates with a sustained reduction
of 74% in the highest responder. SPC5001 did not change HDL (high-density
lipoprotein) levels or the "good" cholesterol in the blood(2). The
preclinical data suggest that SPC5001 has the potential to provide patients
with a new treatment option in managing their cholesterol levels.
"There is a lot of interest in PCSK9 as an exciting new target for the
treatment of high cholesterol and having demonstrated that SPC5001
effectively lowers LDL or the 'bad' cholesterol in preclinical studies, we
are quickly moving forward with plans to advance SPC5001 into clinical
studies as a potential new treatment to help patients better manage the risks
of cardiovascular diseases," said Henrik Orum, PhD, Vice President and Chief
Scientific Officer of Santaris Pharma A/S. "SPC5001 is a testament to the
efficiency of our LNA Drug Platform and Drug Discovery Engine as we
discovered, optimized and advanced SPC5001 into preclinical development in
just 18 months."
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 for a range of diseases
including metabolic disorders, infectious and inflammatory diseases, cancer
and rare genetic disorders.
"In addition to advancing SPC5001 into drug development for the treatment
of high cholesterol, we remain on track to begin Phase 2 clinical trials of
miravirsen (SPC3649) in patients with Hepatitis C in the second half of this
year," said Soren Tulstrup, President and Chief Executive Officer, Santaris
Pharma A/S. "In our internal programs and partnered programs, the versatility
and broad utility of Santaris Pharma A/S LNA Drug Platform and Drug Discovery
Engine continues to be critical in developing effective RNA-targeted
therapies with high affinity, target specificity and remarkable potency for a
range of diseases."
SPC5001 adds to the portfolio of Santaris Pharma A/S drugs in
development, including its most advanced compound miravirsen, the
International Nonproprietary Name (INN) or generic name for the compound
formerly known as SPC3649.
Miravirsen, the first microRNA-targeted drug to enter human clinical
trials, is a specific inhibitor of miR-122, a liver-specific microRNA that
the Hepatitis C virus requires for replication. Unlike Hepatitis C therapies
that directly target the virus, miravirsen works by removing a "helper"
molecule (miR-122) that the virus needs in order to make new copies.
Miravirsen is being studied in a Phase 1 trial multiple-ascending dose study
in healthy volunteers and a Phase 2 study in patients infected with Hepatitis
C is expected to begin in the second half of 2010.
Santaris Pharma A/S also has a broad range of drug discovery and
development programs with its partners: Shire plc (lead candidates against up
to five targets for rare genetic disorders), Pfizer (lead candidates against
up to ten targets), GlaxoSmithKline (options to drug candidates from up to
four viral disease programs) and Enzon Pharmaceuticals (lead candidates
against eight cancer targets successfully delivered).
About Locked Nucleic Acid (LNA) Drug Platform
The LNA Drug Platform utilizing Santaris Pharma A/S proprietary LNA
chemistry provides the key to delivering on the promise of RNA-targeted
therapies today by addressing these concerns and overcoming 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, infectious and inflammatory diseases, and
rare genetic disorders. The Company has strategic partnerships with Enzon
Pharmaceuticals, GlaxoSmithKline, Pfizer and Shire plc. As part of its broad
patent estate, the Company holds exclusive worldwide rights to all
therapeutic uses of LNAs. Santaris Pharma A/S, founded in 2003, is
headquartered in Denmark with operations in the United States. Please visit
www.santaris.com for more information.
(1) World Health Organization -
www.who.int/healthinfo/statistics/bod_cerebrovasculardiseasestroke.pdf
(2) Oral and poster presentation PCSK9 Conference, Locked Nucleic Acid
antisense oligonucleotide inhibition of PCSK9, March 11, 2010
Navjot Rai of Santaris Pharma A/S, +1-858-764-7064, ext. 206, or Cell, +1-619-723-5450, navjot.rai at santaris.com
Tags: April 27, denmark, Denmark And San Diego, Hoersholm, Norway, Santaris Pharma A/S, sweden, United Kingdom