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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 | May 28, 2008 | Santaris Pharma, the Danish biopharmaceutical company, announced today that it has commenced a Phase I human volunteer trial of the world’s 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 C 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 sssociated 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 drug’s 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.
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 Pharma’s 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 Pharma’s 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 Pharma’s 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 Pharma’s 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.
SOURCE: Santaris Pharma |
