Disease Indications

Age-related Macular Degeneration (AMD)
Age-related macular degeneration is a disease leading to the loss of central vision and is the leading cause of blindness in individuals 65 years and older. The clinical course of the disease is divided into two stages. The early stage is characterized by the development and accumulation of drusen (“dry” AMD). In the late stage, choroidal neovascularization (CNV) and leakage leads to disciform scarring (“wet” AMD), resulting in loss of vision. Current interventions include photodynamic therapy to eliminate abnormal blood vessels, and drug therapies in the form of VEGF blockade to inhibit abnormal angiogenesis that leads to choroidal neovascularization and eventual retinal neural apoptosis/death. Since they act exclusively on blood vessel formation, there is a need for additional therapies that directly protect the retinal neurons within existing lesions and enhance survival of damaged neurons, thus promoting the restoration of visual acuity. The incidence of the neovascular form of AMD is about 200,000 new cases per year in the US with an estimated prevalence of AMD resulting in visual impairment of approximately 1.7 million Americans over age 65.

Diabetic Macular Edema (DME)
Diabetic macular edema is swelling of the macula as a result of retinal microvascular changes that occur in patients with diabetes and is the major cause of visual impairment in diabetic patients. Diabetic macular edema is a common complication of diabetic retinopathy and is the cause of most of the functional visual loss in patients with this condition. Diabetes mellitus and its systemic and ophthalmic complications represent an enormous public health threat in the United States. The prevalence of diabetes in the United States is currently estimated at 9% or approximately 21 million, though only about 55% are estimated to be diagnosed. DME affects up to 10% of all patients with diabetes with 75,000 new cases occurring every year. Currently, laser photocoagulation is the standard of care for the treatment of DME associated with diabetic retinopathy. However, laser photocoagulation is a late and destructive procedure that does not specifically address the underlying etiology of diabetic retinopathy. To date, no pharmacological treatment has received regulatory approval for the treatment of diabetic retinopathy, including DME. Therefore an unmet medical need exists in the management of DME.

Delayed Graft Function (DGF) in Kidney Transplantation
DGF is one of the most common complications during the immediate postoperative period in renal transplantation and affects 25-40% of the deceased donor renal transplant patients. DGF results most often from ischemia-reperfusion injury that occurs when blood flow is re-established to the transplanted kidney, initiating a chain of events that can lead to severe renal damage. DGF is associated with longer hospital stays, higher rates of graft rejection, patient morbidity and shortened survival time of the transplanted kidney. DGF is an unmet medical need. The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) granted Orphan designation of QPI-1002 for prophylaxis of DGF in kidney transplantation.

Acute Kidney Injury (AKI)
AKI is a clinically devastating disease that complicates approximately 5% of hospital admissions and up to 30% of admissions to intensive care units. The incidence of AKI is rising. In patients undergoing major cardiovascular surgery, post-surgical AKI develops within hours to days as a result of ischemic conditions caused by reduced local blood flow to the kidneys during the time the patient is on cardiopulmonary bypass. Reperfusion of the kidneys upon removal of the patient from bypass initiates a chain of events that can lead to renal damage. The rate of AKI development in most patients undergoing cardiovascular surgery is low, but rate can be as high as 22% in high-risk patients. The 30-day mortality rate following onset of AKI after surgery is greater than 50%. AKI is an unmet medical need.

Non-arteritic Anterior Ischemic Optic Neuropathy (NAION)
Optic nerve atrophy is defined as the loss of the fibers of the optic nerve, and results from the death of retinal ganglion cells (RGCs). Since they are unable to divide, loss of RGCs results in irreversible loss of vision. Therefore, intervention with neuroprotective agents in diseases that lead to optic nerve atrophy could preserve RGCs and thereby preserve vision. Optic nerve atrophy is the hallmark of optic neuropathies, the most common of which is glaucoma. Ischemic optic neuropathy (ION) is another important subtype of optic nerve atrophy that includes a variety of disorders associated with ischemia to the optic nerve. NAION is the most common cause of sudden optic nerve-related vision loss. NAION typically presents as an abrupt, painless mono-ocular vision loss that varies widely, ranging from minor loss of visual acuity to complete blindness. Improvement or further deterioration of visual acuity and/or visual field typically occurs during the first 6 months after onset. The cumulative probability of developing NAION in the fellow eye ranges from 12-19% over 3-5 years. When NAION develops in the second eye, there is no correlation in the visual outcome in the two eyes. The estimated mean annual incidence of NAION is about 8,000/year in the US. Currently there are no approved therapies for NAION.