QPI-1007 is a synthetic siRNA designed to temporarily inhibit expression of the pro-apoptotic protein, Caspase 2. QPI-1007 utilizes a novel siRNA structure developed by Quark that preserves activity while attenuating off-target and immunostimulatory effects. QPI-1007 is being developed as a neuroprotectant for the treatment of non-arteritic ischemic optic neuropathy (NAION) and other optic neuropathies such as glaucoma that result in the death of retinal ganglion cells (RGCs).
Quark is conducting a
Phase I dose-escalation safety study using QPI-1007 in patients suffering from Optic Nerve Atrophy and NAION. NAION is triggered by blockage of blood flow to the optic nerve, resulting in sudden and painless vision loss that frequently leads to permanent blindness. This disease affects about 8,000 persons annually in the United States and currently there are no effective treatments.
Quark evaluated the activity of QPI-1007 in a variety of animal models of acute and severe optic nerve injury, including a rat model of RGC retrograde death following optic nerve crush and the rat optic nerve axotomy model. In both models, QPI-1007 displayed neuroprotective activity towards RGCs, the death of which is the hallmark of glaucoma and of acute injuries such as ischemic optic neuropathy. In addition, efficacy of QPI-1007 was evaluated in the rat intraocular hypertension model of glaucoma. In these studies, QPI-1007, administered 2 weeks after induction of increased intraocular pressure, exhibited sustained RGC neuroprotective activity for up to five weeks after disease induction, providing proof of concept for the potential use of QPI-1007 to treat glaucoma.
Quark is developing non-invasive delivery strategies for the potential use of QPI-1007 to treat chronic diseases like glaucoma. Glaucoma is a slowly progressing optic neuropathy characterized by the progressive loss of RGCs and is the leading cause of blindness worldwide. There is no cure for glaucoma, and nerve damage and visual loss from glaucoma is usually irreversible. Several classes of drugs approved to treat glaucoma are used to lower intraocular pressure. However, elevated intraocular pressure (IOP) is only a risk factor, and people with normal IOP can also develop glaucoma. Neuroprotection has been proposed as a therapeutic strategy in glaucoma to promote survival of retinal ganglion cells.
The chemical structure of QPI-1007 was developed by Quark in collaboration with BioSpring GmbH, and is believed to be free of third Party IP.