June 2013
Volume 54, Issue 15
ARVO Annual Meeting Abstract  |   June 2013
A Phase I Open Label, Dose Escalation Trial Of QPI-1007 Delivered By A Single Intravitreal (IVT) Injection To Subjects With Low Visual Acuity And Acute Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION)
Author Affiliations & Notes
  • Andrew Antoszyk
    Retina, Charlotte EEN&T Associates, Charlotte, NC
    Surgery, Uniformed Services University of the Health Sciences, Bethesda, MD
  • Bradley Katz
    Ophthalmology and Visual Sciences, University of Utah Health Sciences Center, Salt Lake City, UT
  • Rishi Singh
    Ophthalmology, Cleveland Clinic Foundation, Cleveland, OH
  • Rabia Gurses-Ozden
    Quark Pharmaceuticals, Inc., Fremont, CA
  • Shai Erlich
    Quark Pharmaceuticals, Inc., Fremont, CA
  • Daniel Rothenstein
    Quark Pharmaceuticals, Inc., Fremont, CA
  • Nir Sharon
    Quark Pharmaceuticals, Inc., Fremont, CA
  • Jennifer Hodge
    Quark Pharmaceuticals, Inc., Fremont, CA
  • Leonard Levin
    Ophthalmology, McGill University, Montreal, QC, Canada
    Ophthalmology and Visual Sciences, University of Wisconsin, Madison, WI
  • Neil Miller
    Wilmer Eye Institute, Johns Hopkins University, Baltimore, MD
  • Footnotes
    Commercial Relationships Andrew Antoszyk, Genentech/Roche (C), Regeneron/Bayer (C), Thrombogenics (C), Quark Pharmaceuticals Inc (F); Bradley Katz, QUARK Pharmaceuticals (F); Rishi Singh, Genentech (C), Alcon (C), Bausch and Lomb (R), Zeiss (R), Quark Pharmaceuticals, Inc. (F); Rabia Gurses-Ozden, Quark Pharmaceuticals, Inc. (E); Shai Erlich, Quark Pharmaceuticals, Inc. (E); Daniel Rothenstein, Quark Pharmacuiticals Inc. (E); Nir Sharon, Quark Pharmaceuticals, Inc. (E); Jennifer Hodge, Quark Pharmaceuticals (E); Leonard Levin, Quark (C), Inotek (C), Merz (C), Wisconsin Alumni Research Foundation (P), Cytodefense (I), Teva (C), Allergan (C); Neil Miller, Quark Pharmaceutical Company (C)
  • Footnotes
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Investigative Ophthalmology & Visual Science June 2013, Vol.54, 4575. doi:
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      Andrew Antoszyk, Bradley Katz, Rishi Singh, Rabia Gurses-Ozden, Shai Erlich, Daniel Rothenstein, Nir Sharon, Jennifer Hodge, Leonard Levin, Neil Miller, QPI-1007 NAION Study Group Consisting of Various Institutions in Israel and the United States; A Phase I Open Label, Dose Escalation Trial Of QPI-1007 Delivered By A Single Intravitreal (IVT) Injection To Subjects With Low Visual Acuity And Acute Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION). Invest. Ophthalmol. Vis. Sci. 2013;54(15):4575.

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      © ARVO (1962-2015); The Authors (2016-present)

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Subjects with long-standing low vision due to retinal or optic nerve pathology and subjects with acute NAION were studied in a 1 year, 2-stratum, Phase I, multi-center, open-label, dose escalation study to determine safety, tolerability and the structural and functional changes after a single IVT injection of QPI-1007, a synthetic, chemically modified siRNA that inhibits expression of caspase 2.


Low vision subjects with visual acuity (VA) ≤20/200 (Stratum I) and NAION subjects with ≤20/40 and symptom onset within 28 days prior to the study drug injection (Stratum II-S2) were enrolled in 6 cohorts (0.2-6 mg) and 3 cohorts (1.2, 2.4 & 6 mg), respectively. After receiving a single IVT injection, subjects were evaluated for VA, visual field (VF) and retinal nerve fiber layer(RNFL) thickness at days 1, 7, 14, 28, and months 2, 3, 6, 12.


48 subjects (18 low vision, 30 NAION) were enrolled. All expected study visits are complete for all subjects, except the final follow-up visit (Month 12) for the last enrolled cohort (S2, 6 mg). Available data from both strata through Month 3 (n=48), Month 6 (n=48) and Month 12 (n=38) were analyzed. 261 of 273 adverse events (AEs) were of mild-to-moderate severity. There were no serious AEs. The most common AEs were conjunctival hemorrhage (n=29), conjunctival chemosis (n=11), and eye pain (n=11). Among 28 NAION subjects in S2 with on-chart VA, maximum VA gain was at Month 2 (mean± SD: 16.4 ±10.4 letters). The proportion of subjects in S2 (Figure 1) improving by ≥3 lines at Months 3 and 6 were 53.6% (n=15), and 50.0% (n=14) compared with 39.7% (n=48), and 42.6% (n=52) of IONDT historical controls (p = 0.2 and 0.5, respectively; Fisher exact test) [IONDT, 2000]. Of all the S2 subjects with available follow-up data, no subject lost ≥3 lines of VA compared with 9.1% (n=11), 14.8% (n=18), and 15.8% (n=18) at Months 3, 6 and 12, respectively, in the IONDT historical controls. VF mean defect was comparable to baseline. Decrease in RNFL thickness was similar to historical controls [Contreras et al., 2007].


A single IVT injection of QPI-1007 was well tolerated in subjects with long-standing low vision or acute NAION. Further studies are needed to determine QPI-1007’s effect on visual function and RNFL thickness.

Keywords: 613 neuro-ophthalmology: optic nerve • 615 neuroprotection • 466 clinical (human) or epidemiologic studies: treatment/prevention assessment/controlled clinical trials  

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