Fremont, California-based biotech Quark Pharmaceuticals has dosed patients in two pivotal Phase III studies of siRNA candidates that mark a first in China and India and build on a joint venture there with Suzhou Ribo Life Science.
That joint venture, Kunshan RiboQuark Pharmaceutical Technology, was launched two years ago after a Series A round that raised above $7 million to develop a siRNA ocular neuroprotectant led by BVCF--formerly BioVeda China.
Quark, in a release, said that in February it dosed the first patients in each of two pivotal Phase III studies.
"One for prevention and amelioration of the severity of Delayed Graft Function (DGF) in kidney transplant patients and the other for the preservation of visual acuity in Acute Nonarteritic Ischemic Optical Neuropathy (NAION)," according to the release.
The DGF study with QPI-1002, Quark said is based on a Phase II trial with 326 subjects and covers approximately 75 hospitals in North America, Latin America, Australia and Europe. "Quark has received Orphan Drug status by the (U.S.) FDA and the EMA for this indication," the release said.
Novartis ($NVS) holds a global license option for QPI-1002, according to the Quark website. In July 2014, Quark missed its primary endpoint in a Phase II trial for QPI-1002, but at the time said it would press on with further studies, citing patient subgroup data with potential milestones from Novartis worth up to $680 million at stake.
For the NAION study with QPI-1007, the Phase II/III study is being conducted at approximately 66 hospitals in the U.S., Europe, Israel, India and China, according to the release. Kunshan RiboQuark Pharma holds China rights for QPI-1007.
"The approval for this study by the Indian (DCGI) and Chinese (CFDA) health authorities marks a major milestone because it is the first time that an siRNA molecule was approved by these authorities to be used in humans in clinical studies," according to the release. "Quark received Orphan Drug status from the (U.S.) FDA on this indication. The siRNA drug molecule is based on Quark's unique new proprietary structure."
- here's the release