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 potentially 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.
Apoptosis (programmed cell death) is thought to be the main cause of the death of retinal ganglion cells in non-arteritic anterior ischemic optic neuropathy and glaucoma Animal studies of QPI-1007 demonstrated (i) uptake of siRNA in retinal ganglion cells following intravitreal administration, (ii) RNAi-mediated mechanism of action of QPI-1007 in ocular tissues harvested after intravitreal administration and (iii) efficacy of QPI-1007 in three animal models. QPI-1007 exhibited a favorable safety profile in toxicity studies. Based these preclinical studies, Quark believes that QPI-1007 may be an effective neuroprotective agent, with potential use in NAION and also in major diseases involving retinal ganglion cell loss, including 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.