FDA approves IND for SPC2996 in Chronic Lymphocytic Leukaemia (CLL)

Copenhagen, 8th November 2005: Santaris Pharma, the Danish biopharmaceutical company developing innovative RNA-silencing drugs for the treatment of cancer, today announced that the US Food and Drug Administration (FDA) has approved an Investigational New Drug Application (IND) for the Company’s international Phase I/II study of SPC2996 in Chronic Lymphocytic Leukaemia (CLL).

SPC2996 is the first of a new class of LNA-based investigational drugs known as RNA Antagonists and is designed to reduce the level of Bcl-2 protein within tumor cells by binding and inactivating Bcl-2 messenger RNA, thereby inducing programmed cell death (apoptosis). Bcl-2 is highly over-expressed in CLL cells and appears to play a role in pathogenesis of the disease, preventing lymphocyte apoptosis and being strongly correlated with poor clinical outcome.

The Phase I/II clinical trial, the European arm of which is already ongoing in Denmark, UK and France, is an open-label, escalating, repeated-dose, multi-center study of SPC2996 in patients with relapsed or refractory CLL requiring therapy. The primary objective is to investigate the safety and potential efficacy of SPC2996 in CLL patients and the Company plans to enrol an overall total of 42 patients. The participation of US centers is intended to facilitate the further development of this novel investigational agent.

Lene Worsaae Dalby, Vice-President of Clinical Development at Santaris Pharma, commented:

“The FDA approved the SPC2996 dossier as submitted and without questions. To receive IND approval is always pleasing, but to obtain approval for the first IND for a new class of molecule without any regulatory issues or concerns is particularly gratifying and reflects the dedication and skill of the team at Santaris.”

The first US center to join the study will be the Holden Comprehensive Cancer Center at the University of Iowa where the trial will be under the direction of Dr. James Wooldridge.

ENDS

For further information, contact:

At Santaris
Keith McCullagh, President & CEO
Tel. +45 4517 9819

km@santaris.com

Lene W. Dalby, Vice President, Clinical Development.
Tel. +45 4517 9870
lwd@santaris.com


At Northbank Communications
Douglas Pretsell
Tel: +49 89 57 00 18 06

d.pretsell@northbankcommunications.com

Sue Charles
Tel: +44 20 7886 8152
s.charles@northbankcommunications.com

NOTES FOR EDITORS

About Santaris Pharma
Santaris Pharma A/S is a clinical-stage biopharmaceutical company focussed on developing innovative RNA-silencing drugs for the treatment of cancer. Created in May 2003, Santaris Pharma is backed by a broad group of Scandinavian life-science venture capital investors.

The Company’s drug pipeline is comprized of novel RNA antagonising drugs based on unique Locked Nucleic Acid (LNA) chemistry. LNA drugs, with their high potency and biostability, have the potential to transform the field of RNAi therapeutics, making specific and effective gene silencing a reality in human medicine. If this potential is realized, even in part, it should be possible to design new drugs to treat a wide variety of human diseases by switching off the expression of harmful genes. Santaris Pharma holds the world wide patent rights to the exploitation of LNA in pharmaceuticals.

With the initiation of clinical studies, Santaris Pharma is currently raising new equity funds to finance the continued clinical development of SPC2996 and its other RNA Antagonist drugs in development. The Company plans to raise approximately 40m Euro of new equity in 2005, approximately 50% of which is expected to come from existing shareholders and 50% from new investors.

For further company information see www.santaris.com

About Chronic Lymphocytic Leukaemia
Chronic Lymphocytic Leukaemia (CLL) is a cancer of the blood characterized by a progressive accumulation of long-lived, functionally incompetent lymphocytes. CLL is the second most common type of leukaemia and 8,000 new cases are diagnosed each year in the US alone. The condition mostly affects people over 50 years of age and there is currently no cure, although progression of the cancer can often be delayed by current therapeutic regimens.

About SPC2996
SPC2996 is an investigational medicinal product and the first of a new class of drug known as RNA Antagonists. The developmental drug is composed of a short single chain of nucleotides including LNA, a conformational analogue of RNA, which confers very high specific RNA binding activity on the drug. SPC2996 acts by inhibiting the synthesis of Bcl-2, a key intracellular protein that protects cells against apoptosis (programmed cell death). The protein is expressed in most cancers including CLL and high expression levels correlate with low response rates and resistance to chemotherapy, faster time to relapse and shorter survival time. Down-regulation of Bcl-2 expression thus provides an attractive means by which CLL and many other cancers can be re-sensitized to natural apoptotic stimuli and to chemotherapeutic agents that provoke apoptosis.

About RNA-silencing, Locked Nucleic Acid (LNA) and “RNA Antagonists”
One route to blocking or silencing genes that are involved in human illness is by interfering with the process by which genetic information controls the proteins active in the body. The information from a particular gene is transferred from a strand of DNA in the nucleus to a strand of RNA in the cytoplasm of the cell. The information contained in the RNA is then “translated” into proteins in the process of protein synthesis. Blocking or silencing particular RNAs, or RNA antagonism, offers a powerful means to prevent the harmful effects of these disease-related genes.

In recent years it has been discovered that the human body uses RNA silencing itself to control many cellular processes, through short double-stranded RNA molecules known as microRNAs (miRNAs). There has been much interest in mimicking nature’s process by making similar synthetic short interfering RNAs (siRNAs) as drugs but unfortunately, the poor stability of such agents in the body has made this difficult to accomplish. Now, Santaris has developed a means to make stable short single-stranded RNA mimics with unprecedented target affinity and bio-stability using a unique new chemistry known as Locked Nucleic Acid (LNA). Santaris Pharma has used the term "RNA Antagonists" to describe this exciting new class of synthetic oligonucleotide drugs.