- Quark Pharmaceuticals' RNA-targeting treatment for kidney damage has hit its goal in a Phase 2 study, an outcome that puts further pressure on rival drugmakers looking to be the first to market with an RNA interference (RNAi) therapy.
- QPI-1002 significantly reduced the risk of acute kidney injury (AKI) versus placebo in patients who had undergone cardiac surgery — the primary endpoint of the QRK-209 trial. The small interfering RNA (siRNA) drug also met several secondary endpoints, which evaluated factors such as mortality rates and the number of patients who required renal replacement therapy.
- Adverse events were similar between the experimental and control arms, according to a Thursday statement from Quark. In addition to AKI, QPI-1002 is in late-stage testing as a prophylaxis for delayed graft function (DGF) in patients receiving kidney transplants.
The positive results should help Quark usher its drug into Phase 3 testing for another indication, an important move given the robust late-stage pipeline of other RNAi drugmakers.
Alnylam Pharmaceuticals, considered a leader in the RNAi space, already has three candidates in those advanced trials: patisiran, for hereditary ATTR amyloidosis; inclisiran, for hypercholesterolemia; and fitusiran, for hemophilia and rare bleeding disorders. The biotech also plans to start the late-stage program for its acute hepatic porphyrias therapy, dubbed givosiran, later in 2017.
Whether or not Quark is first to market with an RNAi treatment, it could fetch strong returns with an AKI therapy. Research has shown the condition is becoming increasingly prevalent, and has even been described as reaching epidemic levels. A study published last year in the journal Annals of Cardiac Anaesthesia cited that 20% of cardiac surgery patients will develop AKI.
QRK-209 enrolled more than 340 participants ages 45 and older who were undergoing such surgeries and who were and at risk of AKI. Patients who received Quark's drug showed consistent treatment effects across a variety of subgroups, such as those with chronic kidney disease or diabetes.
"These trial findings support continued development of QPI-1002 for the prevention and amelioration of AKI associated with cardiac surgery in patients at risk for whom no approved therapy exists to date," Quark said in the July 27 statement.
Interestingly, Novartis holds an exclusive option to commercialize and develop QPI-1002. It's unclear whether these new clinical results will sway the Swiss drugmaker to act on the option.
Novartis hasn't been keen on RNAi therapies in the past, selling its business in that space for a paltry $7 million upfront to Arrowhead back in 2015.
Quark will present further data from the trial at a future medical meeting.