The new issue of Human Molecular Genetics brings the results of the study that contributed to dose selection and exposure estimates for the first studies with drug candidate RG3039 in human subjects.
RG3039 is an inducer of 'the backup SMN gene' - SMN2. It is orally bioavailable, brain-penetrant and has been shown to be an inhibitor of the mRNA decapping enzyme, DcpS. RG 3039 is currently in a phase 1b trial.
RG3039 is an inducer of 'the backup SMN gene' - SMN2. It is orally bioavailable, brain-penetrant and has been shown to be an inhibitor of the mRNA decapping enzyme, DcpS. RG 3039 is currently in a phase 1b trial.
The study demonstrated that RG3039 can extend survival and improve function in two SMA mouse models of varying disease severity and positively impacts neuromuscular pathologies. In one type of mice, RG3039 provided more than 600% survival benefit when dosing began at P4, highlighting the importance of early intervention.
The study determined the minimum effective dose and the associated pharmacokinetic (PK) and exposure relationship of RG3039 and DcpS inhibition ex-vivo. These data support the long PK half-life with extended pharmacodynamic outcome of RG3039 in 2B/- SMA mice. In motor neurons, RG3039 significantly increased the number of gems and cells with gems, which is used as an indirect measure of SMN levels.
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