Hoersholm, Denmark/San Diego, California, December 3, 2009 — 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).  

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 Ørum, 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.  The Company’s own 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. The Company has strategic partnerships with Enzon Pharmaceuticals, GlaxoSmithKline, Wyeth Pharmaceuticals (now part of Pfizer) and Shire plc. Please visit www.santaris.com for more information.

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   
 
[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 

Hoersholm, Denmark/San Diego, California, October 28, 2009 — Santaris Pharma A/S, a privately-held biopharmaceutical company focused on developing RNA drugs targeted to disease-related mRNAs and microRNAs, has expanded its executive team with the appointment of Mark Wedel, MD, FACP as Vice President and Chief Medical Officer and Stuart Mackey, JD as Vice President Strategy and Chief Business Officer. Internally and through its partners, Santaris Pharma A/S has a robust drug development pipeline including programs to treat metabolic disorders, infectious and inflammatory diseases, cancer and rare genetic disorders.  

The newly appointed executives will hold global responsibility for advancing Santaris Pharma A/S proprietary RNA-targeted Locked Nucleic Acid (LNA) Drug Platform programs and Drug Discovery Engine capabilities. Dr. Wedel will be responsible for global clinical development programs and therapeutic portfolio management.  Mr. Mackey will lead Santaris Pharma A/S corporate strategy development, corporate communications and global business development programs.  

“I am pleased to have Dr. Wedel and Mr. Mackey join our management team,” said Søren Tulstrup, President and Chief Executive Officer of Santaris Pharma A/S. “As Santaris Pharma A/S continues to advance the development of its RNA-targeted drugs internally and with its partners, Dr. Wedel’s extensive experience in managing RNA-based clinical development programs coupled with Mr. Mackey’s experience in corporate strategy development will be valuable.”  

The newly appointed executives will be based out of the Company’s recently established US subsidiary. Increasing interest, new collaborations and breakthrough advancements led Santaris Pharma A/S, headquartered in Denmark, to establish US-based operations in San Diego, California.  

“The strong medical and business development expertise that Dr. Wedel and Mr. Mackey bring to the team further solidify the presence of Santaris Pharma A/S in the US as we continue to explore new licensing opportunities, scientific collaborations and increase visibility with capital markets,” said Dr. Arthur A. Levin, President of US operations and Vice President and Chief Development Officer of Santaris Pharma A/S.  

Dr. Wedel joins Santaris Pharma A/S from Isis Pharmaceuticals where he was Senior Vice President of Drug Development and Chief Medical Officer responsible for clinical operations, strategic therapeutic portfolio management, and overseeing Isis development programs including, cardiovascular, inflammatory, metabolic, infectious diseases, as well as oncology. Dr. Wedel received his medical degree from The Johns Hopkins School of Medicine in Baltimore, Maryland and holds board certifications in Internal Medicine,              Critical Care and Chest Medicine. He received a law degree from Thomas Jefferson School of Law, San Diego, California, and a B.A. from Valparaiso University, Valparaiso, Indiana.    

Mr. Mackey joins Santaris Pharma A/S after spending the last ten years at leading biotech company Amgen Inc. where most recently he was Managing Director of Amgen Ventures, a $100 M venture capital affiliate of Amgen Inc. with approximately 15 portfolio companies.  He had also been responsible for Amgen’s in- and outlicensing transactions group and Amgen’s Corporate Alliance Management function.  Mr. Mackey received his undergraduate degree from Harvard University and his law degree from the University of California, Berkeley’s Boalt Hall School of Law.

Recent scientific and business accomplishments at Santaris Pharma A/S include a breakthrough in medical science and new collaborations. Santaris Pharma A/S is the first company to advance a microRNA-targeted therapy into human clinical trials.  The drug SPC3649 specifically targets microRNA-122 (miR-122), a host factor for Hepatitis C virus replication. A single-dose Phase 1 trial in healthy volunteers has been completed and the drug continues to move forward in clinical trials.  

Santaris Pharma A/S recently announced a multi-year worldwide strategic alliance with Shire plc to discover and develop new RNA-targeted medicines to treat rare genetic disorders. This collaboration adds to the growing list of mRNA and microRNA drug discovery and development partnerships, which includes Wyeth (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 boosting resistance to metabolism, increasing half-life and improving tissue uptake.  LNA-based therapeutics demonstrate improved binding affinity to their target RNA, which increases potency many-fold over other nucleotide therapeutics. 

The greater potency of LNA in binding complementary RNA sequences also allows for the use of significantly shorter LNA oligonucleotide drugs which can be more effective than previous antisense or siRNA drugs.  

About Santaris Pharma A/S
Santaris Pharma A/S is a privately-held biopharmaceutical company focused on developing RNA medicines targeted to disease-related mRNAs and microRNAs. Utilizing the Company’s proprietary Locked Nucleic Acid (LNA) Drug Platform and Drug Discovery Engine, Santaris Pharma A/S provides fast and efficient generation of lead LNA drug candidates. 

The Company’s own 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 headquartered in Denmark with operations in the US. The Company has strategic partnerships with Enzon Pharmaceuticals, GlaxoSmithKline, Wyeth Pharmaceuticals and Shire plc. Please visit www.santaris.com for more information.  

Media Contacts:

Navjot Rai Santaris Pharma A/S USA Office + 1 858 764 7066 ext. 206 Cell: +1 619 723 5450 e-mail: nra@santaris.com

Randi Krogsgaard Santaris Pharma A/S, Denmark Office: +45 45179879  Cell: +45 20488384  e-mail: rmk@santaris.com

Hoersholm, Denmark/San Diego, California, September 16, 2009 — Santaris Pharma A/S, a privately-held biopharmaceutical company focused on developing RNA-based drugs targeted to  disease-related mRNAs and microRNAs, today announced it has established operations in the United States. Internally and through its partners, the Company has a robust drug development pipeline including programs to treat metabolic disorders, infectious and inflammatory diseases, cancer and rare genetic disorders.  

Increasing interest, new collaborations and breakthrough advancements utilizing the Company’s proprietary RNA-based Locked Nucleic Acid (LNA) Drug Platform and Drug Discovery Engine led Santaris Pharma A/S, headquartered in Denmark, to establish US-based operations in San Diego, California.  

To spearhead the US expansion, Santaris Pharma A/S has appointed leading RNA industry expert Arthur A. Levin, PhD, as President of its US operations.  In addition, Dr. Levin will hold global responsibility as Vice President and Chief Development Officer of Santaris Pharma A/S.   Dr. Levin has 15 years of experience in RNA-based research and more than 25 years of experience in the pharmaceutical industry. Prior to joining Santaris Pharma A/S, Dr. Levin was Senior Vice President of Drug Development at Isis Pharmaceuticals where he was instrumental in advancing more than a dozen RNA-based antisense drugs from basic research to clinical development.    

“We are pleased that Dr. Levin has joined our management team to establish operations in the US and oversee global development programs for Santaris Pharma A/S,” said Søren Tulstrup, President and Chief Executive Officer, Santaris Pharma A/S. “His wealth of RNA-based research and development experience  and industry knowledge will be instrumental in leveraging the Company’s proprietary Locked Nucleic Acid chemistry and Drug Discovery Engine to explore new licensing opportunities and scientific collaborations, increase visibility with capital markets and attract leading scientists.”  

Santaris Pharma A/S recently achieved a breakthrough in medical science as the first company to advance a microRNA-targeted therapy into human clinical trials.  A single-dose Phase 1 trial in healthy volunteers has now been completed and an additional Phase 1 multiple ascending-dose study is planned. The drug SPC3649 specifically targets microRNA-122 (miR-122), a host factor for Hepatitis C virus replication. In 2008, Santaris Pharma A/S published the results of successful microRNA silencing with SPC3649 in non-human primates in Nature[1].  

“Advancing the first microRNA therapy into human clinical trials is a testament to Santaris Pharma A/S leadership in the field,” said Henrik Ørum, Vice President and Chief Scientific Officer at Santaris Pharma A/S. “Our proprietary LNA Drug Platform incorporating short single-stranded LNAs offers higher affinity, greater specificity and remarkable potency compared to other RNA-based oligonucleotides. With a growing list of pharmaceutical partners and LNA rapidly becoming the chemistry-of-choice in RNA-targeted therapeutics, we are pleased to have Dr. Levin join our team to progress important new therapies into drug development.”

Last month, Santaris Pharma A/S announced a multi-year worldwide strategic alliance with Shire plc to discover and develop new RNA-based medicines to treat rare genetic disorders utilizing Santaris Pharma’s proprietary LNA Drug Platform. This collaboration adds to the growing list of mRNA and microRNA drug discovery and development partnerships, which includes Wyeth (delivery of lead candidates against up to ten targets), GlaxoSmithKline (four viral disease drug candidates) and Enzon Pharmaceuticals (eight cancer targets successfully delivered). Operations in the US aim to support existing relationships and generate new licensing and partnership opportunities.  

About Locked Nucleic Acid (LNA) Drug Platform
The Locked Nucleic Acid (LNA) Drug Platform developed by Santaris Pharma A/S creates synthetic chemical versions of the normal nucleic acid building blocks of ribonucleic acids (RNA). These synthetic chemical versions called LNAs improve the drug-like qualities of resulting therapeutics (oligonucleotides) by boosting resistance to metabolism, increasing half-life and improving tissue uptake.  LNA-based therapeutics demonstrate improved binding affinity to their target RNA, which increases potency many-fold over other nucleotide therapeutics.  The greater potency of LNA in binding complementary RNA sequences also allows for the use of significantly shorter LNA oligonucleotide drugs which can be more effective than previous antisense or siRNA drugs.  

About Santaris Pharma A/S
Santaris Pharma A/S is a privately-held biopharmaceutical company focused on developing RNA-based drugs targeted to disease-related mRNAs and microRNAs. Utilizing the Company’s proprietary Locked Nucleic Acid (LNA) Drug Platform and Drug Discovery Engine, Santaris Pharma A/S provides fast and efficient generation of lead LNA drug candidates.  The Company’s own 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 headquartered in Denmark with operations in the US. The Company has strategic partnerships with Enzon Pharmaceuticals, GlaxoSmithKline, Wyeth Pharmaceuticals and Shire plc. Please visit www.santaris.com for more information.  

Media Contacts:

Navjot Rai	Randi Krogsgaard
Santaris Pharma A/S, USA	Santaris Pharma A/S, Denmark
Office: + 1 858 764 7066 ext. 206	Office: +45 45179879
Cell: +1 619 723 5450	Cell: +45 20488384
e-mail: nra@santaris.com	e-mail: rmk@santaris.com

    
[1] Elmén et al. LNA-mediated microRNA silencing in non-human primates. Nature 2008, 452, 896-899