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    Neuroprotective drug may be possible NAION therapy

    International trial under way with new siRNA; interim analysis may be available mid-year



    Trial cohorts

    The drug (QPI-1007, Quark Pharmaceuticals) is a synthetic small interfering RNA (siRNA) that targets the mRNA of the caspase 2 gene. The drug is under development as a neuroprotectant to treat NAION and glaucoma, as well as other optic neuropathies that kill retinal ganglion cells. The FDA has given the drug an orphan designation for NAION, according to Quark.

    The trial has five patient cohorts as follows:

    1. Subjects receiving a single low-dose intravitreal injection of QPI-1007
    2. Subjects receiving a single high-dose intravitreal injection of QPI-1007
    3. Subjects receiving three low-dose intravitreal injections of QPI-1007
    4. Subjects receiving three high-dose intravitreal injections of QPI-1007
    5. Subjects receiving a sham injection or injections

    The trial will enroll about 530 subjects over the next 2 years; subjects will be randomly assigned into one of the aforementioned groups. Researchers have recruited more than 100 patients so far, which is on target with the research plans, Dr. Miller said. The trial’s main outcome is loss of vision, followed by safety and tolerability.

    Dr. Miller is part of the Neuro-Ophthalmology Research Disease Investigator Consortium (NORDIC), a group organized by the North American Neuro-Ophthalmology Society and that receives funding from the National Eye Institute and other sources. The trial is co-sponsored by Quark and NORDIC and eventually will take place at about 90 sites worldwide, although 60 are participating so far. Most sites are in the United States, with participants also in India, Israel, Germany, Italy, Australia, and Singapore.

    Results for an interim analysis may be available by the middle of this year, Dr. Miller said. 


    Neil R. Miller, MD

    e: [email protected]

    Dr. Miller did not indicate any financial interest in the subject matter.

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