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QPI-1007 is the first drug candidate to utilize a novel siRNA structure developed by Quark, free of third party intellectual property. It relies on novel compositions and proprietary siRNA structures developed internally with the collaboration of BioSpring GmbH. QPI-1007 is currently being evaluated in advanced IND enabling preclinical studies as a neuroprotective agent for eye diseases.
Quark has completed preclinical studies with QPI-1007 in different animal models of acute and severe optic nerve injury. In collaboration with Prof. Ann Logan of the Division of Medical Sciences, Department of Medicine, University of Birmingham, UK, we examined effectiveness of QPI-1007 in a model of retinal ganglion cells (RGC) death following optic nerve crush (ONC). QPI-1007 was also examined in the optic nerve axotomy model in collaboration with Prof. Adriana Di Polo of the Department of Pathology and Cell Biology, Université de Montréal. In both models, QPI-1007 displayed neuroprotective activity towards retinal ganglion cells the death of which is the hallmark of glaucoma and of acute injuries such as ischemic optic neuropathy.
QPI-1007 is a chemically modified siRNA discovered by Quark and covered by Quark’s intellectual property related to novel structures for chemically modified siRNAs
Glaucoma, worldwide the leading cause of irreversible blindness, is a slowly progressive optic neuropathy characterized by the progressive loss of RGCs. Elevated intra-ocular pressure (IOP) is a risk factor, but even people with normal pressure can lose vision to glaucoma. IOP is normal in about 25% to 30% of US glaucoma cases, a condition known as normal tension glaucoma. There is no cure for glaucoma and nerve damage and visual loss from glaucoma cannot usually be reversed but medication or surgery can often slow further vision loss. Several classes of drugs approved to treat glaucoma are used to lower IOP. However, many patients do not respond to IOP reducers and some patients with glaucoma still go blind despite significant IOP reductions. Neuroprotection of the optic nerve has been proposed as a therapeutic approach that aims to promote survival of RGCs. In patients who experience progressive nerve damage and visual field loss, despite relatively normal intraocular pressures or who do not respond to IOP reduction, protection of the optic nerve from glaucomatous damage would be a major advance in preventing blindness.
Triggered by blockage of blood flow to the eye, NA AION is sudden but painless in onset and frequently leads to permanent blindness. This acute disease develops over a relatively short period of time and usually shows little progress of visual loss beyond the first month or so. In the United States this disease that, currently, has no treatment affects about 8000 patients annually.
Quark’s development plan is to proceed to human proof of concept for its ocular neuroprotection agent QPI-1007 in patients suffering of NA AION. In a later stage, clinical trials are planned to test QPI-1007 effectiveness in glaucoma patients.
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