Santaris Pharma A/S Advances New Cholesterol-Lowering Drug, SPC5001 Inhibiting Exciting New Target PCSK9, Into Phase 1 Clinical Trials for the Treatment of High Cholesterol

HOERSHOLM, Denmark and SAN DIEGO, May 4, 2011 - — Phase 1 clinical study to assess safety and tolerability of SPC5001, a
RNA-targeted drug inhibiting PCSK9, which is a protein involved in removing
low-density lipoprotein cholesterol (LDL-C) or "bad" cholesterol from the
bloodstream

— In a study representing 147 million people, the World
Health Organization recently reported that most patients with high
cholesterol levels are not getting the treatment they need to reduce their
risk of cardiovascular diseases such as heart attack and stroke

— Data from preclinical studies showed that SPC5001 provided
fast-acting, potent and long-lasting inhibition of PCSK9 and reductions in
LDL-C by up to 74%

— Developed using Santaris Pharma A/S Locked Nucleic Acid
Drug Platform, SPC5001 is one of the drugs in the company's multi-faceted
cardiometabolic program aimed at helping patients gain better control of
their target cholesterol levels

Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused
on the research and development of mRNA and microRNA targeted therapies,
today announced that it has advanced SPC5001 into Phase 1 clinical trials for
the treatment of high cholesterol. SPC5001 is a mRNA-targeted drug that
inhibits the exciting new target, Proprotein Convertase Subtilisin/Kexin type
9 (PCSK9), a protein involved in removing low density lipoprotein cholesterol
(LDL-C) or "bad" cholesterol from the bloodstream.

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

In a study representing 147 million people, the World Health Organization
recently reported that most patients with high cholesterol levels are not
getting the treatment they need to reduce their risk of cardiovascular
diseases such as heart attack and stroke(1).

The randomized, dose-escalation, double-blind, placebo-controlled Phase 1
study will assess safety, tolerability, pharmacokinetics, and
pharmacodynamics of SPC5001. The study aims to enroll 32 healthy volunteers
and 8 patients with familial hypercholesterolemia who will be randomized to
receive weekly subcutaneous injections of SPC5001 or placebo. Santaris Pharma
A/S aims to first use SPC5001 in patients with familial hypercholesterolemia,
a genetic disorder characterized by high cholesterol levels, specifically
very high levels of LDL-C in the blood and often leads to early
cardiovascular disease.

SPC5001 is one of the drugs from the company's multi-faceted
cardiometabolic program aimed at helping patients achieve target cholesterol
levels. Santaris Pharma A/S also plans to advance SPC4955 inhibiting
apolipoprotein B (apoB) for the treatment of high cholesterol in the first
half of 2011. In yet a third approach, in February 2011 Santaris Pharma A/S
obtained an exclusive license from Massachusetts General Hospital for
intellectual property related to the regulation of miR-33, an important
microRNA that regulates high density lipoprotein (HDL) levels or "good"
cholesterol.

"Having been recently discovered, PCSK9 is a high-value target for the
treatment of high cholesterol and advancing SPC5001 into Phase 1 clinical
studies is a significant step in potentially helping patients achieve their
target cholesterol levels," said Arthur A. Levin, PhD, Vice President and
Chief Development Officer of Santaris Pharma A/S. "Santaris Pharma is
committed to helping patients better manage their cholesterol levels by
moving multiple therapeutics from our cardiometabolic research programs such
as SPC4955 and SPC5001 as a way to provide new treatment options for patients
to better manage their high cholesterol levels and reduce the risks of
cardiovascular disease."

In preclinical studies, SPC5001 provided fast-acting, potent and
long-lasting inhibition of PCSK9 and provided reductions of 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.

In addition to its cardiometabolic programs, 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.

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.

With its partners, Santaris Pharma A/S also has a robust product pipeline
consisting of mRNA and microRNA drug discovery and development
collaborations. These include partnerships with Pfizer, Inc. (delivery of
lead candidates against up to 20 targets), miRagen Therapeutics
(cardiovascular diseases), Shire plc (rare genetic disorders),
GlaxoSmithKline (four viral disease drug candidates) and Enzon
Pharmaceuticals (eight cancer targets successfully delivered - three are now
in Phase 1 clinical studies).

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 cardiometabolic 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 cardiometabolic
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) www.who.int/mediacentre/news/notes/2011/cholesterol_20110201/en/

(2) Oral and poster presentation PCSK9 Conference, Locked Nucleic Acid
antisense oligonucleotide inhibition of PCSK9, March 11, 2010

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