Santaris Pharma Begins Human Clinical Testing of the Worlds First Medicine Targeted at a Human microRNA

SPC3649 (LNA-antimiR-122) being developed as a potential new therapy for Hepatitis C virus (HCV) infection

Ground-breaking Phase I volunteer study made possible through the success of Locked Nucleic Acid (LNA) chemistry

COPENHAGEN, Denmark--(BUSINESS WIRE)--Santaris Pharma, the Danish biopharmaceutical company, announced today that it has commenced a Phase I human volunteer trial of the worlds first microRNA medicine to be tested in man - SPC3649 (LNA-antimiRTM-122). The study is being conducted by PhaseOneTrials A/S, Copenhagen, and will include a maximum of 48 healthy male volunteers. The Company also announced that the first cohort of healthy volunteers in the study have completed treatment satisfactorily. SPC3649 is being developed by Santaris Pharma as a potential new approach to the treatment for Hepatitis C infection. Phase II studies in hepatitis patients will follow.

SPC3649 specifically targets microRNA-122, a small, liver-expressed, regulatory ribonucleic acid (RNA) that has recently been shown to facilitate human Hepatitis C virus replication in liver cells. Keith McCullagh, President & CEO, Santaris Pharma, said:

The mechanism of action of this drug represents a potential breakthrough in medical science. The ability to switch off the functions of particular microRNAs may enable clinicians to modulate entire networks of genes associated with disease or ill-health. Santaris Pharma scientists recently published the results of successful microRNA silencing with SPC3649 in non-human primates.1 We are excited now to be able to evaluate the drugs efficacy and safety in human subjects. If successful, such trials may lead to the development of a new approach to the treatment of Hepatitis C and more generally, contribute to the development of a major new class of therapeutic agents.

The Phase I First-In-Man clinical trial is a placebo-controlled, double-blind, randomised, single dose, dose-escalating safety study of SPC3649 in a total of 48 healthy male volunteers. The volunteers are divided into six groups with eight persons in each. In each group six persons will receive SPC3649 and two will receive placebo (non-effective substance). Each volunteer will receive a single two hour intravenously infusion with either SPC3649 or placebo. SPC3649 will be administered at six escalating dose levels and each volunteer will be followed for three months.

Santaris Pharma forward looking statements

This written announcement contains forward-looking statements, identified by the use of words such as "believes," "expects," "may," "will," "should", "potential," "anticipates," "plans" or "intends" and similar expressions. Such forward-looking statements involve risks, uncertainties and other factors that may cause actual results, events or developments to be materially different from the future results, events or developments indicated in this announcement. Such factors include, but are not limited to the timing, success and cost of clinical studies; the ability to obtain regulatory approval of products, market acceptance of and future demand for Santaris Pharma products and the impact of competitive products and pricing. These factors should be considered carefully and readers are cautioned not to place undue reliance on such forward-looking statements. No assurance can be given that the future results covered by the forward-looking statements will be achieved. All information in this press release is as of the date of this press release and Santaris Pharma does not intend to update this information.


About Santaris Pharma

Santaris Pharma is a Danish clinical stage biopharmaceutical company. The Company was formed in 2003 and has the exclusive rights to LNA technology, a 3rd generation antisense chemistry used to develop new classes of RNA medicines, called RNA antagonists. Santaris Pharmas messengerRNA antagonists and microRNA antagonists are being developed to silence mRNAs and microRNAs associated with various diseases, including cancer, metabolic disorders and viral infections. Santaris Pharma completed a Euro 40m second round of equity financing in May 2006 and a Euro 20m third round of equity financing in December 2007. Santaris Pharma has a global alliance with Enzon Pharmaceuticals of New Jersey to develop and co-commercialise a series of Santaris Pharma RNA antagonists for the treatment of cancer and a worldwide strategic alliance with GlaxoSmithKline for the discovery, development and commercialization of novel medicines against viral diseases.

About Santaris Pharmas Locked Nucleic Acid technology

LNA is the first true conformational analogue of RNA (ribonucleic acid). The ribose sugar in LNA is locked in the three-dimensional shape of RNA by virtue of its rigid bicyclic structure. The result is that when incorporated into oligonucleotides, LNA conveys dramatically enhanced binding affinity to complementary RNA sequences. Drug molecules with multiple LNA substitutions therefore have truly unprecedented potencies. The greater potency of LNA in binding complementary RNA sequences means that LNA oligonucleotide drugs can be made significantly shorter than previous antisense or siRNA drugs. These shorter RNA antagonist drugs are taken up efficiently by cells and tissues, thereby overcoming many of the delivery problems of RNAi to date. As LNA drugs are resistant to degradation when given systemically, have long tissue half lives, and are taken up readily by many tissues they have greater potency than other oligonucleotide chemistries.

About microRNAs

MicroRNAs are a newly discovered class of small regulatory molecules which control many biological processes in cells. In addition, microRNAs are implicated in many diseases, such as cancer, viral infections, cardiovascular disease and neurological disorders and, therefore, represent a new class of targets for therapeutic intervention. Santaris Pharmas unique LNA technology enables development of short, synthetic RNA-binding molecules that can effectively antagonize disease-causing microRNAs and, thus may yield patient benefits unobtainable by other therapeutic approaches.

About SPC3649:

SPC3649 is the first of the new class of microRNA antagonists based on Santaris Pharmas proprietary LNA technology. SPC3649 targets the liver specific microRNA122. SPC3649 works by blocking microRNA-122 in liver cells leading to a rapid loss of Hepatitis C virus replication. Another effect of the drug is to enhance the metabolism of cholesterol and fats.

About Hepatitis C (HCV):

There is a huge need to develop effective therapeutics for the treatment of HCV. 170 million people world-wide are chronically infected with the disease. 75-80% of newly infected patients develop chronically HCV infection which leads to severe morbidity and mortality caused by cirrhosis, cancer and liver failure. Six different types (genotypes) of HCV have so far been identified. The standard therapy, which is a combination therapy (pegylated interferon and ribavirin) is often poorly tolerated by the patients. This treatment is furthermore only efficient in 50 % of the cases against the most frequent HCV genotype (1).

About PhaseOneTrials

PhaseOneTrials A/S is a Danish Contract Research Organization (CRO) that conducts highly specialized clinical phase I trials and early phase II trials of new drug candidates. PhaseOneTrials has extensive experience planning and conducting First-in-Man trials for both classical pharmaceutical and new biological drugs having worked with recombinant peptide hormones, enzymes and growth factors. The location at a university hospital, proximity to their intensive care unit and collaboration with distinguished specialists all mean that the Company easily meet the increasing government safety requirements for such trials.

1 See reference: Elmen, J, Lindow, M. et al., (2008) LNA-mediated microRNA silencing in non-human primates. Nature, VOL 452, April 17, pages 896-899, 2008


Santaris Pharma
Randi Krogsgaard, +45 4517 9879
Cell +45 2048 8384
Director, Corporate Communications

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