Santaris Pharma has announced that it has commenced a Phase I human volunteer trial of SPC3649 (LNA-antimiRTM-122), the world’s first microRNA medicine to be tested in man. 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 although Phase II studies in hepatitis patients will not commence until 2009.
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.
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, randomized, 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.