Demonstrating the upside benefits of being a thinly-traded stock, Dicerna Pharmaceuticals (DRNA +21.5%) jumps on turnover of 353K shares, a 2x surge in volume. The upside move is being driven by the news that DCR-PH1, its therapeutic candidate for primary hyperoxaluria type 1 (PH1), inhibits the gene implicated in the pathogenesis of the disease. Investors' enthusiasm might be a bit premature considering that the encouraging results were observed in a preclinical animal model.
In genetically-modified PH1 mice, researchers noted a 97% reduction in in the HAO1 transcript in the liver after a single dose of DCR-PH1 and a significant reduction in urinary oxalate levels, a key marker of the disease. Urinary oxalate levels returned to near baseline in DCR-PH1-treated mice.
PH1 is a rare inherited liver disorder that often results in progressive and severe kidney damage.