Fremont, Ca – October 27, 2015– Quark Pharmaceuticals, Inc, a late clinical-stage pharmaceutical company, who is a leader in the discovery and development of novel RNA interference (RNAi)-based therapeutics, announced today that it has initiated the U.S. arm of a global Phase II/III pivotal study (NCT02341560) of QPI-1007 for treatment of acute non arteritic anterior ischemic optic neuropathy (NAION). Additionally, the Company plans to begin recruiting patients at sites outside the U.S. during the fourth quarter of 2015. . QPI-1007 has received FDA orphan drug designation in the U.S.

“We are delighted to initiate this multi-national trial to seek a treatment for NAION, a debilitating disease with a clear, unmet medical need,” stated Shai Erlich, Chief Medical Officer at Quark.  “This pivotal trial is a significant milestone for us in bringing our siRNA-based neuroprotective drug QPI-1007 closer to patients. QPI-1007 represents a novel therapeutic strategy for treating NAION and future plans are to develop it for additional optic neuropathies, including glaucoma, which, similar to NAION,  are characterized by the death of retinal ganglion cells.”

 About RNAi

RNA interference (RNAi) is a universal mechanism within living cells that employs non-coding RNA to control which genes are active and how active they are. This natural mechanism, which was discovered in 1998 and was awarded the Nobel Prize in 2006, has already revolutionized experimental biology and currently holds the highest promise for therapeutic purposes.  Various types of short double-stranded RNAs act as effector molecules of the RNAi mechanism. Some of them, targeting specific genes in a sequence-dependent manner and inhibiting their expression, are called short interfering RNAs (siRNAs). siRNAs can be designed based on the sequence information of virtually any gene, produced synthetically and used as drugs.  siRNA drugs can cause inhibition of expression of any gene, regardless of its traditional attribution to potentially “druggable” or “non-druggable” targets. These drugs are highly specific and potentially safer than other drugs such as promiscuous small molecule therapies. Quark’s siRNA platform includes proprietary siRNA compound structures and chemical modifications with improved pharmacological properties, while the Company’s strong IP portfolio provides freedom to operate in the siRNA space.

 About QPI-1007

QPI-1007 is an siRNA molecule chemically modified by Quark’s proprietary technology. The drug is designed to temporarily inhibit the expression of caspase 2. QPI 1007 is being developed as a neuroprotectant for the treatment of non-arteritic ischemic optic neuropathy (NAION) and in the future other optic neuropathies such as glaucoma that result in the death of retinal ganglion cells (RGCs). QPI-1007 has been evaluated in a human, dose escalation, Phase I/IIa Study (Protocol QRK007), delivered by single intravitreal injection to Optic Nerve Atrophy patients with low visual acuity and thereafter in acute NAION patients. This study was conducted at 22 sites in the US and 6 sites in Israel. This study showed that a single intravitreal injection of QPI-1007 was well tolerated in subjects with long-standing low vision or acute NAION. The drug has also demonstrated protective activity compared to historical data. QPI-1007 was also studied in a Phase IIa clinical trial in acute angle closure glaucoma patients in the United States, Vietnam and Singapore.

Quark licensed this drug to its partner in India, Biocon and to its joint venture in China, Kunshan Ribo-Quark Pharmaceutical Inc.

About the Study

This multi-national, pivotal study is a double masked, randomized, sham-controlled efficacy and safety study that will enroll approximately 352 subjects with recent-onset NAION. It is anticipated that ~60 sites will participate in the study, in the USA, Canada, China, India, Germany and Israel. Participants will receive multiple doses of the study drug (or sham) and will be followed through 6 months and 12 months. More information is available at

About Quark Pharmaceuticals

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 two are in pivotal Phase III studies. Quark’s Joint Venture in China, Kunshan Ribo-Quark Pharmaceutical Inc, and its strategic partner in India, Biocon Limited, are part of Quark’s worldwide 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:


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Quark Pharmaceuticals, Inc.Juliana Friedmann+972 89 30 5111



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