Indian biopharmaceutical firm Biocon and US-based Quark Pharmaceuticals have announced the initiation of the pivotal global Phase II/III trial (QRK207) of siRNA (small interfering RNA) drug candidate, QPI-1007, for ocular neuroprotection.
Approved by the Drug Controller General of India (DCGI), QRK207 is the first ever clinical trial of a siRNA therapy in India.
The randomised, double masked, sham-controlled trial is designed to evaluate the efficacy of QPI-1007 to treat acute non-arteritic ischemic optic neuropathy (NAION).
Around 465 patients will be enrolled in this trial, to be conducted across 95 hospitals in several nations such as India, US, Israel, Germany, Australia, Italy and China.
In the trial, QPI-1007 will be administered through multi-dose, intravitreal injections to subjects with NAION, which is a rarely occurring ocular disorder.
Quark chairman and CEO Dr Daniel Zurr said: "siRNA therapeutics is a new class of breakthrough medicines, with the potential to revolutionise drug development, as well as offer therapeutic precision and widespread applicability and we are delighted to initiate QRK207 within India in this multi-national study to seek a treatment for NAION, a debilitating condition.
"QPI-1007 represents a novel therapeutic strategy for treating NAION and future plans are to develop it for additional optic neuropathies, including glaucoma, which, similar to NAION, are characterised by the death of retinal ganglion cells."
Jointly developed by Biocon and Quark, the QPI-1007 is a double-stranded RNA molecule chemically modified by Quark's patented technology.
The drug temporarily inhibits the caspase 2 subsequently blocking the apoptotic death of retinal ganglion cells.
An initial Phase I/IIa trial has demonstrated the tolerability of QPI-1007, as well as its efficacy in treating long-standing low vision or acute NAION.