Letter from the CEO

Welcome to the website of Quark Pharmaceuticals, Inc. I thank you for taking the time to visit and learn more about our Company. It is a great pleasure for me to introduce to you our exciting accomplishments in the drive to discover new therapeutics to improve human health.

Founded in December 1994, Quark devoted its early years to developing its proprietary technology platform, “BiFAR™”, to identify novel drug targets. BiFAR™ combines high throughput gene inhibition technology with microarray expression profiling to identify genes responsible for disease processes. In collaboration with large pharmaceutical partners, we applied our BiFAR™ platform to identify new therapeutic targets in serious diseases with unmet medical needs. Today we are harvesting the fruits of the innovative targets we identified and the corresponding therapeutic concepts.

In early 2002, Quark determined that the most rapid means of achieving therapeutic intervention with the disease targets identified by BiFAR™ was to exploit the newly-discovered RNA interference, or RNAi, pathway. Since then, Quark has focused its drug discovery and development efforts on the exciting new class of drugs called small interfering RNAs, or siRNAs. We have developed a robust RNAi technology platform and established an independent intellectual property position in the siRNA field, and are now a world-leader in the development of RNAi-based therapeutics. Our siRNA technology capabilities include the identification of the optimal target sequences, design of optimal siRNA candidates using our expertise in chemical modifications to enhance pharmacologic properties, and our know-how in delivery of these compounds to target tissues and organs of interest.

I am now pleased to highlight some of the Company’s key milestones.

Clinical Programs

We recently initiated dosing in a Phase I study of the investigational neuroprotective agent, QPI-1007. This drug candidate has a proprietary siRNA structure that preserves RNAi activity while reducing potential off-target and immunostimulatory effects. The structure, we believe, provides Quark freedom to operate in the siRNA intellectual property space. QPI-1007 is being developed as a neuroprotectant for the treatment of optic neuropathies that result in the death of retinal ganglion cells (RGCs). We are currently evaluating QPI-1007 to treat sudden vision loss associated with non-arteritic anterior ischemic optic neuropathy (NAION), an unmet medical need. Demonstration of activity of QPI-1007 in NAION patients may provide the basis for examining activity in slower-progressing optic neuropathies such as glaucoma. The current Phase I study is an open-label, dose escalation, safety, tolerability and pharmacokinetic study in which QPI-1007 will be administered to all participating patients.

Quark’s development pipeline also features QPI-1002 (I5NP), the first systemically administered synthetic siRNA in human clinical trials. QPI-1002 is designed to temporarily inhibit expression of the stress-response gene, p53. QPI-1002 is being developed for the prevention of acute kidney injury (AKI) in patients undergoing major cardiovascular surgery, and for the prophylaxis of delayed graft function (DGF) in patients receiving deceased donor kidney transplants. We completed Phase I studies in these patient populations and an independent Data Safety Monitoring Board recommended continuation of QPI-1002 clinical development in both disease populations. QPI-1002 was granted Orphan designation by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the prophylaxis of delayed graft function in kidney transplant patients. This designation provides regulatory support, financial incentives and marketing exclusivity once the drug is approved for marketing.

Quark’s partner, Pfizer, Inc., is currently evaluating the first siRNA we developed, PF-655, in two Phase II clinical studies, one in diabetic macular edema (DME) patients and the other in patients with wet, age-related macular degeneration (AMD). We actively collaborate in conducting these clinical studies. PF-655 is a synthetic, chemically modified siRNA designed to inhibit the expression of the RTP801 gene that was discovered by Quark through the BiFAR™ platform. RTP801 is a stress response gene that, among other pathways, is involved in abnormal blood vessel development and leakage in the eye. Under the licensing agreement, Pfizer has exclusive development rights to siRNA-mediated therapies that inhibit RTP801 for ophthalmic and non-ophthalmic indications, while Quark is eligible for development and sales-based milestone payments and royalties.

Preclinical Pipeline Utilizing Proprietary siRNA Structures and Compositions

Quark has a broad pipeline of siRNA drug candidates based on its novel structures and chemical compositions in a variety of disease areas where we have confirmed effective delivery of our synthetic siRNAs. We expect to develop additional RNAi drug candidates without the need to in-license third party intellectual property.

Financial

In April 2008 we announced the closing of a private financing totaling an aggregate of $27 million. The major investors were investment vehicles of SBI Asset Management Co., Ltd. and SBI Investment Co., Ltd., subsidiaries of the prestigious SBI Holdings, Inc. SBI extended this financing in June 2010 with an additional financing of $10 million as continued support to Quark for advancement of its RNAi drug pipeline.

Our strengths include the dedication and skills of our employees, the quality of our science and the depth of our development pipeline. Working to the best of our abilities, it is our intention to develop novel medicines to treat serious diseases, to build a profitable business and to maintain our unique work environment that values innovation, integrity and achievement.


Daniel Zurr

President and CEO



 
8/18/2010
Quark Pharmaceuticals and Major Pharmaceutical Company Enter into Licensing Option Agreement for the p53 Suppressor Drug QPI-1002, the First siRNA Administered Systemically in Human
 
7/15/2010
Nitto Denko and Quark Pharmaceuticals to Enter into Collaboration to Develop a Novel siRNA Anti-fibrotic Drug
 
6/28/2010
Quark Pharmaceutical’s QPI-1007 Exhibits Neuroprotective Effects in Glaucoma Model
 
6/24/2010
Quark Pharmaceuticals Announces Presentations of its Two siRNA Drug Candidates at Glaucoma & Retinopathies 2010
 
6/15/2010
Quark Granted European Orphan Drug Status for QPI-1002 for the Prophylaxis of Delayed Graft Function in Kidney Transplant Patients
 
6/10/2010
Quark Pharmaceuticals Closes $10 Million Financing
SBI Holdings, Inc. CEO Yoshitaka Kitao Appointed to Board. Funds to Support Advancing Clinical Pipeline.
 
5/17/2010
Quark Pharmaceuticals Announces Nature Medicine Publication for Role of RTP801 in Pulmonary Injury and Emphysema
 
5/6/2010
Quark Pharmaceuticals Development Partner Pfizer Presents In Vivo Activity of PF-04523655 at ARVO
 
5/4/2010
Quark to Present Data on Predicting Risk of Delayed Graft Function in Kidney Transplant Patients at American Transplant Congress
 
5/4/2010
Quark to Present Phase I Data for QPI-1002 in DGF
 
3/1/2010
Quark Announces Dosing of the First Patient in Phase I Clinical Trial of its Ocular Neuroprotective Agent, QPI-1007
 
2/16/2010
FDA Grants Orphan Drug Status for Quark’s QPI-1002 for the Prophylaxis of Delayed Graft Function in Kidney Transplant Patients
 
12/6/2009
Quark Pharmaceuticals Poised to Advance Clinical Studies of QPI-1002
First Synthetic siRNA Administered Systemically to Humans for the Prophylaxis of Acute Kidney Injury and Delayed Graft Function
 
11/19/2009
Quark Reports Progress of siRNA Clinical Programs; 300 Patients Dosed With Quark Pharmaceuticals siRNA Drugs In Multiple Fully Enrolled Clinical Trials
 
11/2/2009
Quark Pharmaceuticals to Present Data on its siRNA Therapeutic Programs in Kidney and Lung Diseases
 
10/26/2009
Quark Pharmaceuticals Expands International Patent Protection for Compounds Targeting the RTP801 Gene
 
8/4/2009
Quark Pharmaceuticals to Participate in IBC’s Oligonucleotide Therapeutics: From Concept to Implementation Conference
 
7/28/2009
Quark Pharmaceuticals Announces Data Indicating Potential Utility of QPI-1007 for Treatment of Glaucoma
 
6/9/2009
Quark Announces the Presentation of Data Indicating Potential Utility of QPI-1002 in Chronic Kidney Disease at the RNA Interference Summit
 
5/14/2009
Quark Pharmaceuticals Presents Data on Synthetic siRNAs for Prevention of Ocular and Inner Ear Sensory Cell Damage
 
5/11/2009
Quark Pharmaceuticals Presented At ARVO Data Showing That PF-04523655 Enters Retinal Cells And Elicits Its Pharmacologic Effect Via Target Gene Knock-Down Without Activating TLR3
 
4/20/2009
Quark Pharmaceuticals Presents at American Society for Pharmacology and Experimental Therapeutics Annual Meeting
 
3/9/2009
Quark Pharmaceuticals Presents at Asia TIDES Conference
Highlights First Systemic siRNA Administered in Humans and Quark’s Proprietary siRNA Structures
 
1/8/2009
Quark Announces Dosing of the First Patient in Phase I/II Clinical Trial for Systemically Delivered siRNA Drug Candidate for Delayed Graft Function
Kidney transplantation to be Quark’s second indication with clinical-stage, systemically administered siRNA product candidate for Delayed Graft Function
 
12/1/2008
Quark Pharmaceuticals to Present at the 20th Annual Piper Jaffray Health Care Conference
Presentation to Highlight Results of Ocular Neuroprotection Candidate
 
10/13/2008
Quark Pharmaceuticals Announces Publication of Study on Use of siRNA against Proprietary Target for Inhibiting Tumor Growth and Sensitization of Cancer Cells to Chemotherapy
Quark siRNAs Examined Alone and in Combination with Chemotherapy
 
9/25/2008
Quark Announces First RNAi Drug Candidate Having a Novel, Proprietary Structure Covered by Quark Intellectual Property
Data Validates Neuroprotective Effect of Quark’s New RNAi product on dying Retinal Ganglion Cells, a Cause of Blindness in Glaucoma
 
9/17/2008
Quark Pharmaceuticals Announces Poster Presentations at RNAi Europe 2008
 
7/30/2008
Quark Announces First Patient Dosing by Pfizer in Phase II Trial of RNAi Therapy in DME
Clinical Program Leverages Quark’s RNAi Technology
 
4/7/2008
Quark Receives FDA Approval of IND For Kidney Transplant
Quark’s second siRNA drug delivered systemically
 
4/7/2008
Quark Closes $27 Million Financing
Funds Will Support Clinical Development of Pipeline Products
 
12/23/2007
Quark to Present at JPMorgan 26th Annual Healthcare Conference
Quark Pharmaceuticals to Present New Preclinical Proof of Concept Results at JPMorgan 26th Annual Healthcare Conference
 
11/19/2007
Quark Announces First Systemic siRNA Dosing
Quark Pharmaceuticals, Inc. Announces First Systemic siRNA Dosing in Humans. Commences Phase I Clinical Trial of Proprietary siRNA Molecule in Acute Renal Failure.
 
10/23/2007
Quark to Present at CIBC and Acumen BioFin Rodman & Renshaw
Quark Pharmaceuticals to Present at CIBC World Markets 18th Annual Healthcare Conference and Acumen BioFin Rodman & Renshaw 9th Annual Healthcare Conference
 
10/10/2007
Development Milestone Payment from Mitsubishi
Quark Pharmaceuticals, Inc. to Receive Development Milestone Payment from Mitsubishi Pharma Corporation
 
9/25/2007
Development of Proprietary siRNA Compounds for Noise-Induced Hearing Loss
Quark Pharmaceuticals Signs Collaboration Agreement with University of Michigan for Development of Proprietary siRNA Compounds for Noise-Induced Hearing Loss
 
8/28/2007
Patent Application on RNAi molecules
 Quark Pharmaceuticals Files Patent Application Covering Novel Chemical Modifications To Its RNAi Molecules
 
8/21/2007
Agreement with University of NY
Quark Pharmaceuticals Extends Research Agreement with State University of New York for Proprietary siRNA Compounds for Acute Hearing Loss
 
8/6/2007
Positive Preclinical Results
Quark Pharmaceuticals, Inc. Presented Positive Preclinical Results of Systemic RNAi Compound for Acute Renal Failure (ARF)
Quark to initiate Phase 1 trial of AKIi-5 as the first human clinical study involving the systemic delivery of siRNA
 
7/31/2007
Silence Therapeutics and Quark
Silence Therapeutics plc and Quark Pharmaceuticals, Inc. (“Quark”), announced today an expansion of their current strategic technology licensing agreement
 
9/26/2006
Pfizer and Quark
Pfizer expands research commitment to ophthalmology through licensing agreement with quark biotech...
 
6/19/2005
COPD
Quark Biotech Discovers Role of RTP801 as Signaling Molecule in Cigarette Smoking-Induced Pulmonary Injury...
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