NORDIC and Quark Announcement

Safety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent-Onset NAION Patients

Quark Pharmaceuticals, Inc is sponsoring a multi-center, open-label, single-dose, dose escalation study in the US and Israel to determine the safety, tolerability and pharmacokinetics of QPI-1007, a neuroprotectant being developed for the treatment of non-arteritic anterior ischemic optic neuropathy (NAION) and other optic neuropathies in which loss of retinal ganglion cells (RGCs) results in irreversible loss of vision. QPI-1007 is a new synthetic siRNA designed to temporarily inhibit the expression of caspase 2 via the RNA interference pathway. The caspases play a major role in apoptosis, and caspase 2 has been shown to be activated specifically in rat models of retinal ischemic insult. QPI-1007 has exhibited significant RGC neuroprotective activity with preservation of RGCs in three different animal models and, thus, may have the potential to preserve vision in humans.

This first human study is enrolling patients in two strata: 1) patients who are legally blind in the study eye due to irreversible long-standing optic nerve atrophy or retinal degeneration, and 2) patients with recent-onset NAION in one eye only. In stratum I, enrollment in which is now complete, patients were enrolled into 6 sequential dose cohorts of 3 patients per cohort. In Stratum II, which is ongoing, patients are being enrolled into a maximum of 3 dose cohorts of up to 10 patients per cohort. Dosing of the first cohort in Stratum II commenced after review of the available safety data from the 3rd dose level in Stratum I. Dosings in additional cohorts are subject to per protocol safety review.  After receiving a single intravitreal (IVT) injection of QPI-1007, patients will undergo regularly scheduled comprehensive evaluations for 12 months. Serial ophthalmologic evaluations include; best-corrected visual acuity, visual field testing, slit-lamp exams, IOP measurements, and ophthalmoscopy, as well as fundus photographs and OCT of the macula and peripapillary retinal nerve fiber layer.

http://www.clinicaltrials.gov/ct2/show/NCT01064505?term=QPI-1007&rank=1