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news.Repligen has begun Phase 1b clinical study of RG3039, drug candidate for the potential treatment of spinal muscular atrophy (SMA).
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The study will evaluate the safety and plasma pharmacokinetics of multiple doses of RG3039, an orally bioavailable small molecule inhibitor of an RNA processing enzyme called DcpS, in healthy volunteers.
Repligen president and chief executive officer Walter Herlihy said, "Completion of this stage of the RG3039 clinical development program in healthy volunteers, if successful, will provide the necessary foundation for more advanced trials involving SMA patients who are in critical need of a treatment for this devastating disease."
This double-blind, placebo-controlled, ascending multiple dose study being conducted at the Jasper Clinic in Kalamazoo, Michigan, will randomise each of 32 adult healthy volunteers with ascending oral doses of either RG3039 or placebo.
According to the study design, within each of the four dose cohorts, six subjects will be exposed to oral doses of RG3039 and two subjects to placebo for 28 days.
The correlation of RG3039 PK with a panel of molecular biomarkers, and determination of the inhibitory plasma concentrations of RG3039 for the target enzyme DcpS are the secondary objectives.
FSMA invested $13m in funding and directing the preclinical development of RG3039.