FREMONT, Calif. Oct. 26 — Quark Pharmaceuticals, Inc., a development-stage pharmaceutical company discovering and developing novel RNA interference (RNAi)-based therapeutics, today announced issuance of Japanese Patent Number: 4354633, titled, “Hypoxia regulated genes,” on August 7, 2009. Granted to Quark from a Japanese National Phase application of International (PCT) Patent Application No. PCT/US98/17296, the patent corresponds to U.S. Patent No.s 6,455,674, 6,555,667 and 6,740,738 issued by the United States Patent and Trademark Office.
The granted claims broadly cover various aspects of the RTP801 gene, including inhibitors targeting either the mRNA or peptide products of the gene. This patent is part of the intellectual property estate that is the basis of PF-4523655 (formerly RTP801i-14), a synthetic siRNA molecule licensed to Pfizer on an exclusive worldwide basis that is designed to inhibit the expression of RTP801 and is being evaluated in clinical studies for the treatment of Diabetic Macular Edema (DME), Age-related Macular Degeneration (AMD). PF-4523655 is currently in Phase II clinical studies for DME and for wet AMD.
By targeting the RTP801 gene, PF-4523655 has been shown to inhibit abnormal blood vessel growth and leakage, and to reduce inflammation and suppress apoptosis in animal models of retinal diseases. Importantly, RTP801 inhibition differentiates PF-4523655 from other therapeutics designed to specifically inhibit angiogenesis by targeting either VEGF or its receptors, because it acts via a VEGF-independent pathway. Hence, PF-4523655 has the potential to show efficacy when used either as a monotherapy or in combination with existing VEGF-based therapies.
Daniel Zurr, Ph.D., President and Chief Executive Officer of Quark, said, “Patents are essential in the drug development process as they protect the significant investments in R&D and regulatory activities necessary to obtain marketing approvals and to generate commercial sales. We are vigilant about upholding our industry leadership position in siRNA R&D by protecting our pipeline with an aggressive and comprehensive approach to intellectual property generation and management. . We are also focused on broadening our intellectual property estate to further protect and enhance the value of our product pipeline.”
About the PF-4523655 (RTP801i-14) Clinical Program
PF-4523655 (RTP801i-14) is a synthetic, siRNA molecule designed to inhibit the expression of Quark’s proprietary target, RTP801. The product candidate is licensed to Pfizer on an exclusive worldwide basis. Quark discovered RTP801 using its BiFAR(TM) target discovery platform, which identifies clinically relevant genes and proteins that are critical to reversing a given disease phenotype when inhibited. The Company owns a family of patents covering the RTP801 gene and protein sequences, specific antibodies, and gene inhibition across different disease indications.
The ongoing Phase II study is a prospective, randomized, multi-center, dose-ranging study is designed to evaluate the safety and efficacy of PF-4523655 versus laser therapy in 160 patients with DME. In this study, patients are randomized to receive intravitreal injections of one of three dose levels of PF-4523655, or laser therapy. Therapeutic effect will be evaluated by assessing visual acuity and retinal morphology.
About Quark Pharmaceuticals, Inc.
Quark Pharmaceuticals, Inc. is a development-stage pharmaceutical company engaged in discovering and developing novel RNAi-based therapeutics. Quark has a fully integrated drug development platform that spans therapeutic target identification to drug development. Quark’s RNAi technology includes novel siRNA structures and chemistry providing Quark with freedom to operate in the siRNA intellectual property arena, as well as the ability for non-invasive delivery of siRNA to other target tissues including the eye, ear, lung, spinal cord and brain.
In addition to PF-4523655, Quark’s current clinical pipeline includes QPI-1002, the first systemically administered siRNA drug in human clinical trials, developed by Quark for the prevention of acute kidney injury (AKI) following major cardiovascular surgery and the prophylaxis of delayed graft function after kidney transplantation. Quark has licenses from Silence Therapeutics and from Alnylam Pharmaceuticals for the structure of these products.
QPI-1007, a siRNA that utilizes a proprietary structure developed by Quark, is being evaluated in advanced IND-enabling preclinical studies as a neuroprotective agent in eye diseases. In addition, Quark has a broad pipeline of siRNA drug candidates based on novel structures developed internally. The Company expects to utilize the structures to develop additional RNAi drug candidates.
Quark is headquartered in Fremont, California and operates research and development facilities in Boulder, Colorado and Ness-Ziona, Israel. Additional information is available at www.quarkpharma.com.