KUNSHAN, China--(BUSINESS WIRE)--Kunshan RiboQuark Pharmaceutical Technology Co., Ltd. (“RiboQuark”), a Chinese-foreign equity joint venture and clinical stage pharmaceutical company established by Suzhou Ribo Life Science Co., Ltd. (“Ribo”) and Quark Pharmaceuticals Inc., announced today enrollment of the first patient in China in QRK207, a global pivotal Phase II/III study of the ocular neuroprotectant QPI-1007. QPI-1007 is a synthetic siRNA designed to temporarily inhibit expression of the pro-apoptotic protein, Caspase 2.
“RiboQuark is committed to develop innovative therapies that address unmet medical needs, and the initiation of the Phase II/III study investigating QPI-1007 in NAION in China is an important step towards this goal. China has a significant NAION patient population, and we are pleased to be the first in the country to provide a siRNA-based therapy that is likely to benefit many patients in need, and to have the first patient ever dosed on a siRNA trial in China,” commented Prof. Liang Zicai, Chairman of Ribo and RiboQuark.
The QRK207 study will determine the effect of QPI-1007 on visual function in subjects with acute non-arteritic ischemic optic neuropathy (NAION). NAION is often referred to as “stroke in the eye” and is a devastating condition that results in the loss of vision. There is no approved treatment for NAION. Today’s announcement marks a milestone for introducing potential siRNA therapies to China. RiboQuark holds the whole rights of QPI-1007 development and commercialization within China as well as most Asia countries.
Quark Pharmaceuticals received approval from the China Food and Drug Administration (CFDA) to proceed with the QRK207 study, one of the first clinical trials of a siRNA therapy in China. QRK207 is being conducted by Quark in collaboration with “Neuro-Ophthalmology Research Disease Investigator Consortium” (NORDIC) and enrolling patients in eight countries, including China.
“siRNA therapeutics is a new class of breakthrough medicines with the potential to revolutionize drug development and offer therapeutic precision and widespread applicability. We are delighted to initiate QRK207 in China in this multi-national study to seek a treatment for NAION, a debilitating condition,” stated Dr. Daniel Zurr, Chairman and CEO of Quark, Vice-Chairman of RiboQuark. “QPI-1007 represents a novel therapeutic strategy for treating NAION, and we have plans to develop it for additional optic neuropathies, including glaucoma, which, similar to NAION, are characterized by the death of retinal ganglion cells.”
“It takes strong partnerships to translate novel science into commercial therapies that can help people, and we are extremely pleased to have RiboQuark lead the siRNA based drug class development in China,” added Dr. Zurr.
QPI-1007 is a synthetic siRNA designed to temporarily inhibit expression of the pro-apoptotic protein, Caspase 2. QPI-1007 utilizes Quark’s proprietary siRNA structure that preserves activity while attenuating off-target and immunostimulatory effects.
Quark completed a Phase I first in human, open label, single-dose, dose escalation, safety, tolerability and pharmacokinetic study of QPI-1007, conducted at 16 centers in the USA and five centers in Israel. The study showed QPI-1007 was safe and exhibited neuroprotective activity in NAION patients. Quark received Orphan Drug status from the U.S. Food and Drug Administration (FDA) on the acute non-arteritic ischemic optic neuropathy (NAION) indication.
About the QRK207 Study
QRK207 is a pivotal Phase II/III, randomized, double masked, sham-controlled trial of QPI-1007 delivered by multi-dose intravitreal injections to subjects with acute non-arteritic anterior ischemic optic neuropathy (NAION) to compare the safety and efficacy of two doses of QPI-1007 along with the sham group. This multi-national, multi-centric trial is being conducted in approximately 90 hospitals in several countries including China, US, Israel, Germany, Australia, Italy, Singapore and India. Approximately 465 patients will be enrolled globally. Each patient will be treated for 12 months.
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 small molecule therapies which are known to exhibit some degree of promiscuity. 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.
Ribo Life Science Co., Ltd. is an innovative R&D company devoted to the development of nucleic acid drugs and related products based on the RNA interference (RNAi) technology. Ribo has built a strong strength in nucleic acid liquid-phase synthesis technology and production of nucleic acid crude drugs, and has developed some great drug R&D projects with its innovative R&D capabilities, aiming to make contribution to the treatment of serious diseases troubling the Chinese population. Ribo’s founders include some of China’s most renowned scientists in the field of RNAi technology. Ribo received the prize of The First Innovative Team in Jiangsu China. Ribo is headquartered in Kunshan, Jiangsu Province and has a subsidiary in Beijing. For more information, please visit www.ribolia.com.
About Quark Pharmaceuticals, Inc.
Quark Pharmaceuticals, Inc. is a world leader in discovery and development of novel small interfering RNA, or siRNA, therapeutics for unmet medical needs. RNA interference is a biological process in which RNA molecules regulate expression of targeted genes. Quark’s fully integrated drug discovery and development platform spans the process from therapeutic target identification to drug development. Two products, QPI-1002 for delayed graft function (DGF) following kidney transplantation and QPI -1007 for non-arteritic ischemic optic neuropathy (NAION), have been granted orphan designation and are in global Phase 2/3 pivotal clinical studies. Quark’s broad pipeline of clinical and preclinical product candidates is generated from the company’s internally-developed siRNAi platform technology and focuses on extrahepatic indications. In July this year, Quark has successfully completed a randomized double-blinded Phase 2 trial exploring efficacy of QPI-1002 in prevention of acute kidney injury (AKI) following cardiac surgery that met the primary and multiple secondary endpoints. Novartis has an option to license this product. Another Quark’s siRNA pipeline drug, PF-655 is licensed to Pfizer. Quark is headquartered in Fremont, California and operates research facilities in Ness-Ziona, Israel. For additional information please visit: www.quarkpharma.com.