Quark Pharmaceuticals' gene-silencing treatment for kidney transplant complications missed its primary endpoint in a Phase II trial, possibly jeopardizing a deal with Novartis ($NVS) worth up to $680 million.
The treatment, QPI-1002, is a small interfering RNA (siRNA) designed to shut off the p53 gene in kidney transplant patients, thereby protecting tissues from damage caused by a disorder called delayed graft function (DGF). Quark mounted a 331-patient Phase II trial in hopes the treatment could meet its primary endpoint of a 30% relative risk reduction compared to placebo, but QPI-1002 came through at just 15.1%, missing its main goal.
Quark was quick to point out that its drug crossed the 30% threshold in a few patient subgroups, and the biotech noted that safety signals were similar between the trial's treatment and placebo arms. QPI-1002's benefits were particularly pronounced in patients whose kidney donors were older than 45, Quark said, and the company believes the results merit further study.
Now Quark is planning to take its results to the FDA to map out a Phase III study in DGF, and the company hopes to start dosing patients in the first quarter of 2015, CEO Daniel Zurr told FierceBiotech. At the same time, Quark plans to launch a Phase II effort in acute kidney injury, a larger indication Zurr believes is integral to QPI-1002's potential.
But whether the company goes it alone on those efforts remains up in the air.
Back in 2010, Novartis stepped in and paid $10 million up front for an option to license QPI-1002, promising to hand over up to $670 million more in fees and milestones if the gene-silencing treatment met its standards for success. Novartis is currently reviewing the Phase II results, Zurr said, and the Swiss giant has 90 days to make a decision. Missing a primary endpoint in Phase II would seem like a deal-breaker, but Novartis is yet to provide an update on the outstanding agreement and declined to comment for this story.
Novartis has already curtailed much of its internal RNAi research efforts, telling FierceBiotech in April that that decision stemmed from "ongoing challenges with formulation and delivery and the reality that the current range of medically relevant targets where siRNA may be used is quite narrow."
Beyond QPI-1002, Quark's clinical siRNA pipeline includes the Pfizer ($PFE)-partnered PF-655, in midstage development for diabetic macular edema and wet age-related macular degeneration, and QPI-1007, a wholly owned treatment for optic neuropathy and glaucoma.
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