Genkyotex picks out silver linings from failed PhII, braces for pivotal study in primary biliary cholangitis
Genkyotex’s Phase II primary biliary cholangitis trial was mainly supposed to measure reduction in gamma glutamyl transpeptidase (CGT). But that may not be obvious if you just read the company’s release on the topline efficacy results.
That’s likely because their drug, a NOX1&4 inhibitor dubbed GKT831, failed the primary endpoint. They did report a 19% reduction in CGT, but it’s not statistically significant after 24 weeks. Yet what the French biotech would like to spotlight are the secondary endpoints — a statistically significant reduction in alkaline phosphatase (p=0.002) and, for a subgroup of patients who had more severe fibrosis to start with, a 22% reduction in liver stiffness compared to a 4% increase in placebo (p=0.038).
The study involved 111 patients who were divided into three groups to receive the standard of care drug plus either placebo, GSK831 400 mg once a day or GKT831 400 mg twice a day. No changes were observed in bilirubin, a marker of liver injury that the biotech earlier described as a measure of safety, across the three arms.
CEO Elias Papatheodorou says the results are promising enough to advance GSK831 into late-stage trials for both PBC and other fibrotic liver diseases like NASH. In parallel, the company is launching a Phase II in lung fibrosis funded by the NIH.
There’s just one more small hurdle to overcome: Get the money to fund a pivotal.
Papatheodorou is looking at various options — including partnering and doing an equity raise — to complement the $8 million (€7.3 million) in the bank at last count, he said in a conference call according to Evaluate Vantage.
Its shares (Euronext Paris & Brussels: $GKTX) has fallen 7.5% since it announced the results, which came six months after a more upbeat interim readout.