Santaris Pharma A/S Obtains Exclusive License from Mass General Hospital for Intellectual Property Related to the Regulation of miR-33 for the Treatment of Cardiovascular Disorders

By Santaris Pharma As, PRNE
Sunday, February 27, 2011

miR-33, an important microRNA that regulates high density lipoprotein (HDL) levels or "good" cholesterol, is a promising therapeutic target for raising good cholesterol levels

HOERSHOLM, Denmark and SAN DIEGO, February 28, 2011 - Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused
on the research and development of mRNA and microRNA targeted therapies,
today announced that the Company has obtained an exclusive license from
Massachusetts General Hospital (MGH) for intellectual property related to the
regulation of miR-33 for the treatment of cardiovascular disorders. Santaris
Pharma A/S will utilize its Locked Nucleic Acid (LNA) Drug Platform to
develop a LNA-based drug targeting miR-33, an important microRNA that
regulates high density lipoprotein (HDL) levels or "good" cholesterol.

(Logo: photos.prnewswire.com/prnh/20100111/SPLOGO)

Cholesterol is an essential component of all cells and several important
hormones, but cholesterol levels that are out of balance or too high overall
lead to the formation of atherosclerotic plaques that cause heart attacks or
strokes. According to the World Health Organization, cardiovascular diseases
are the number one cause of death globally and by 2030, almost 23.6 million
people will die from cardiovascular diseases, mainly from heart disease and
stroke(1).

"We are excited to have licensed the intellectual property related to the
regulation of miR-33 from Mass General Hospital as it is a promising new
target to raise HDL or the 'good' cholesterol in patients who are suffering
from cardiovascular diseases," said Soeren Tulstrup, President and CEO of
Santaris Pharma A/S. "With our leadership position in microRNA drug
development having advanced miravirsen into Phase 2 clinical trials and a
keen focus on developing cardiovascular therapies, we are well positioned to
expand our cardiovascular disease portfolio for the benefit of patients who
are in need of new therapies to better control their cholesterol levels."

Last year, MGH researchers published data in Science identifying tiny
segments of RNA, microRNAs, which play an important role in the body's
regulation of cholesterol and lipids(2). Their study found that the miR-33
family of microRNAs suppresses a protein known to be important for generation
of HDL and for the removal of cholesterol from peripheral tissues, including
cells that form atherosclerotic plaques. Data show that turning off miR-33
raises HDL levels suggesting miR-33 as a novel target in the treatment of
cardiovascular and metabolic disorders.

"Current treatments for such cholesterol abnormalities as low circulating
HDL levels are only modestly effective, and there is an urgent need for new
therapeutic strategies," said Anders Naar, Ph.D., of the MGH Center for
Cancer Research, who led the Science study. "Our discovery of miR-33 as a key
regulator of HDL has provided a novel therapeutic target for antisense-based
technologies to ameliorate cardiometabolic disorders."

The Santaris Pharma A/S LNA Drug Platform is the only RNA technology with
both mRNA and microRNA targeted drugs in clinical trials, demonstrating the
broad utility of the proprietary platform. In September 2010, Santaris Pharma
A/S successfully advanced miravirsen, a lead microRNA drug candidate
targeting miR-122, into Phase 2 studies for the treatment of patients
infected with the Hepatitis C virus. In addition, Santaris Pharma A/S is
advancing two mRNA-targeted drugs, SPC5001 targeting PCSK9 and SPC4955
targeting apoB, for the treatment of high cholesterol into Phase 1 in the
first half of 2011.

RNA-targeted drugs are a promising new class of therapeutics that are
enabling scientists to develop drugs to work through targets thought to be
inaccessible to small molecules and monoclonal antibodies. The LNA Drug
Platform utilizing Santaris Pharma A/S proprietary single-stranded LNA
chemistry may provide the key to delivering on the promise of RNA-targeted
therapies today by overcoming the limitation of earlier antisense and siRNA
technologies. The unique combination of small size and high affinity
achievable with Santaris Pharma A/S LNA technology allows LNA-based drugs to
potently and specifically inhibit RNA targets in different tissues without
the need for complex delivery vehicles.

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
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. LNA-based drugs are a promising
new class of therapeutics that are enabling scientists to develop drug
candidates to work through previously inaccessible clinical pathways. 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 allows this new class of drugs candidates to potently
and specifically inhibit RNA targets in many different tissues without the
need for complex delivery vehicles. The most important features of LNA-based
drugs include excellent specificity providing optimal targeting; increased
affinity to targets providing improved potency; and favorable pharmacokinetic
and tissue-penetrating properties that allow systemic delivery of these drugs
without complex and potentially troublesome 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 www.santaris.com for more information.

    (1) World Health Organization -
        www.who.int/mediacentre/factsheets/fs317/en/

    (2) Najafi-Shoushtari et al. 2010. "MicroRNA-33 and the SREBP Host Genes
        Cooperate to Control Cholesterol Homeostasis", Science 328: 1566-9.

Navjot Rai, Director, Global Communications of Santaris Pharma A/S, Office, +1-858-764-7064 ext. 206, Cell, +1-619-723-5450, navjot.rai at santaris.com

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