US-based Regulus Therapeutics has started dosing of RG-101, a sGalNAc-conjugated anti-miR targeting microRNA-122 (miR-122), in healthy subjects in a Phase I clinical study.
Regulus’ Phase I clinical study, which is being conducted in The Netherlands, will have four parts.
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The four parts include a single ascending-dose study in healthy volunteer subjects, a multiple-ascending dose study in healthy volunteer subjects, a single-dose drug-drug interaction study of RG-101 in combination with an approved oral direct-acting antiviral in healthy volunteer subjects, and a single-dose study in hepatitis C virus (HCV) patients to evaluate the safety and viral load reduction.
Primary objective of the Phase I study is to assess the safety and tolerability of RG-101, while the secondary objectives are to assess pharmacokinetics, viral load reduction and any impact an oral direct-acting antiviral may have on the pharmacokinetics of RG-101.
The study is expected to enrol approximately 100 healthy volunteer subjects and HCV patients.
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RG-101 is a key programme in Regulus Therapeutics’ ‘Clinical Map Initiative’, which outlines certain corporate goals to advance its microRNA therapeutics pipeline over the next several years.
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By GlobalDataUnder the Clinical Map Initiative, the company anticipates demonstrating human proof of concept results in the Phase I clinical study by the end of 2014.
Through conjugation to GalNAc, RG-101 is effectively targeted to hepatocytes for the treatment of HCV. GalNAc is a carbohydrate-based chemistry approach for asialoglycoprotein receptor-mediated delivery of oligonucleotides to hepatocyte cells of the liver.
Using the GalNAc conjugate chemistry has significantly improved the potency of the active oligonucleotide of RG-101 by achieving targeted delivery of the oligonucleotide to the infected hepatocytes.
Data evaluating RG-101 for in vitro and in vivo potency, pharmacokinetic/pharmacodynamics, toxicology and safety pharmacology and inhibition of HCV replication were earlier presented by the company.
Efficacy of RG-101 has also been tested by the company in a human chimeric liver mouse model infected with HCV genotypes 1a and 3a.
After a single dose of RG-101, up to a 2-log reduction in HCV viral load titre was observed in both genotypes in this model.
The duration of action observed for RG-101 supports the potential for a once-a-month dosing regimen.
According to Regulus Therapeutics, RG-101 has demonstrated an excellent preclinical safety profile and has been well-tolerated to date.
Regulus chief scientific officer Dr Neil W Gibson said: "We continue to be encouraged by the preclinical data seen to date and believe that RG-101 has the potential to be a best-in-class host factor agent, specifically due to its pan-genotypic properties, including demonstrated efficacy in the hard to treat HCV genotype 3, and the potential for a once monthly dosing regimen.
"We look forward to reporting data from the Phase I clinical study of RG-101 by the end of this year."
Image: RG-101 is a novel anti-miR-122 oligonucleotide therapeutic. Photo: courtesy of Freedigitalphotos.net.
