Quark Announces First Patient Dosed in a Phase IIa Trial Evaluating QPI-1007 for Neuroprotection in Patients With Acute Primary

Quark Announces First Patient Dosed in a Phase IIa Trial Evaluating QPI-1007
for Neuroprotection in Patients With Acute Primary Angle Closure Glaucoma

FREMONT, Calif., June 25, 2014 (GLOBE NEWSWIRE) -- Quark Pharmaceuticals, Inc.
today announced that the first patient has been dosed in a double-masked,
randomized sham controlled Phase IIa safety, tolerability and pharmacokinetics
study of Quark's proprietary neuroprotective agent QPI-1007 when administered
by single intravitreal (IVT) injection in patients suffering an acute attack
of primary angle closure glaucoma (PACG). QPI-1007 is a siRNA designed to
temporarily inhibit the expression of Caspase 2, a protein that has been shown
to play a role in retinal ganglion cell (RGC) cell death. QPI-1007 is
currently under investigation for the treatment of non-arteritic ischemic
optic neuropathy (NAION). In a Phase I/IIa multicenter clinical study recently
completed in the USA and Israel a single intravitreal injection of QPI-1007
resulted in a neuroprotective effect in NAION patients.

The study is a double-masked, single dose, randomized sham controlled study of
QPI-1007 conducted in several centers in the United States, Vietnam, and
Singapore. Approximately 60 patients with unilateral acute PACG (APACG) will
be randomized at a 1:1 ratio to receive 1.5 mg IVT injection of QPI-1007
(active group) or sham injection (control group). Once the acute attack has
been treated by standard of care methods (medical therapy and laser iridotomy)
and the intraocular pressure in the study eye becomes less than 25 mmHg,
subjects will be randomized into the study within 120 hours of the attack.
Randomization will be stratified by time from symptom onset to the study drug
administration or sham procedure (≤ 72 hours and > 72 hours). The primary
objective of the study is to evaluate the safety, tolerability and
pharmacokinetics of QPI-1007 in subjects with acute primary angle-closure
glaucoma. In addition, the biological activity of the drug will be studied by
comparing the active group and control group study eyes at Month 4 with
respect to a series of visual function parameters.

"We are delighted to announce that patient dosing has begun in the Phase I/IIa
trial of QPI-1007 in patients with acute angle closure glaucoma," said Dr.
Daniel Zurr, CEO of Quark. "Acute attacks of angle closure glaucoma are
potentially associated with significant ocular morbidity and may lead to
permanent vision loss from ocular ischemia and consequent RGC death. The
ability to protect ganglion cells from cell death could represent a
breakthrough in the effort to save the vision of patients who suffer acute
angle closure glaucoma attacks and in the broader area ofneuroprotection.
With its unique properties, we believe that QPI-1007 is a potential game
changer in the future treatment of all glaucoma patients."

About QPI-1007

QPI-1007 is an investigational chemically modified siRNA drug designed to
temporarily inhibit the expression of Caspase 2 developed by Quark based on
entirely internally developed intellectual property of Quark. Intravitreal
administration of QPI-1007 in rat models of optic nerve crush and axotomy as
well as in acute retinal ischemia achieved by acute transient elevation of the
intraocular pressure had ocular neuroprotective effects.

In the Phase I/IIa study in patients with NAION, an intravitreal injection of
QPI-1007was well tolerated.There were no reported serious adverse events, no
dose limiting toxicities and no discontinuations due to adverse events. The
study was not designed for determining the efficacy of the drug, however,
best-corrected visual acuity (BCVA) was compared to historical control. The
proportion of NAION patients improving by ≥ 3 lines (≥ 15 letters) at month 3
was 51.7% (n=29) compared with 39.7% (n=121) of IONDT historical controls [The
IONDT Research Group (1995). JAMA, Vol.273, pp.625 – 632]. None of the NAION
patients lost three or two lines (and only 1 patient lost 1 line only) of VA
at months 3 and 6 compared with 9.1% and, 14.8% respectively, in the
historical controls. One patient (3.6% of patients) with QPI-1007 treatment
lost ≥ 3 lines of VA at 12 months.This was the first proof of concept in
human for this unmet medical need.

About Acute Angle Closure Glaucoma

Acute angle-closure glaucoma represents an ocular emergency, in which the
outflow of aqueous humor through the anterior chamber angle is blocked. As a
result, intra-ocular pressure (IOP) rises to levels high above the normal,
causing pain, hyperemia, corneal epithelium edema, iris ischemia and ocular
tissue damage including damage to the lens, and increased risk of ischemic
damage to the neurosensory retina and the optic nerve. The high IOP during the
acute attack was found to cause progressive long-term damage and structural
and functional changes to the retina. Contributing to this damage is likely to
be ischemic injury sustained RGCs during the attack. Some cells may not
survive the acute attack, while other cells initiate apoptotic cascade. The
visual morbidity from APACC is significant. The natural history in untreated
patients is progressive vision loss that may result in unilateral or bilateral
blindness. It is estimated that all forms of PACG account for about 10% of
glaucoma cases in the U.S., but in 35% up to 50% of glaucoma cases in Asian
populations. Acute attacks of ACG seem to occur in 20%-40% of PACG.

About Quark Pharmaceuticals, Inc.

Quark Pharmaceuticals, Inc., the world leader in novel therapeutic RNAi
discovery and development, has the largest clinical-stage siRNA pipeline in
the industry.The Company's fully integrated drug development platform spans
therapeutic target identification to drug development. Quark's approach to
delivery allows targeting of tissues and organs including the eye, kidney,
ear, lung, skin, spinal cord and brain.Quark has three siRNA product
candidates in clinical development in five different disease indications of
which four are in Phase II. Quark's Joint venture in China, Kunshan Ribo-Quark
Pharmaceutical Inc, and its strategic partner in India, Biocon Limited, are
part of Quark's world wide clinical studies network.

Quark is headquartered in Fremont, California and operates research and
development facilities in Boulder, Colorado and Ness-Ziona, Israel.For
additional information please visit: www.quarkpharma.com

CONTACT: Quark Pharmaceuticals, Inc.
         Juliana Friedmann
         +972 89 30 5111
         jfriedman@quarkpharma.com
 
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