On November 13, 2017, Kunshan RiboQuark Pharmaceutical Technology Co. Ltd., a siRNA pharmaceutical company jointly established by Suzhou Ribo Life Science Co. Ltd. and Quark Pharmaceutical Co. Ltd., announced that in the global pivotal II/III study QRK207 of its innovative siRNA (small interfering RNA) drug QPI-1007 for optic nerve protection, the investigational product has been administered to the first subject by Professor Wang Ningli's team at Beijing Tongren Hospital, Capital Medical University, China. QPI-1007 is a synthetic siRNA designed to temporarily inhibit the expression of the pro-apoptotic protein asparaginase 2. The study is the first clinical trial of oligonucleotide therapeutics conducted in China, and the subject became the first patient in China to receive oligonucleotide therapeutics.

Dr. Liang Zicai, Chairman of Ribo and RiboQuark, said, "RiboQuark is committed to developing innovative drugs for unmet clinical needs, and the initiation of QPI-1007 phase II/III clinical studies for NAION in China is an important step towards this goal. We are very excited that Chinese NAION patients can benefit from QPI-1007 treatment and we can be the first company to provide siRNA therapy."

The QRK207 study is conducted to evaluate the effect of QPI-1007 on visual function in nonarteritic anterior ischemic optic neuropathy (NAION). NAION, often referred to as "eye stroke," is a very devastating disease that will eventually lead to blindness. Currently, there is no approved treatment for NAION. The administration in the first patient announced today marks a milestone in the development of oligonucleotide therapeutics in China. RiboQuark owns all the R&D and industrialization interests of QPI-1007 in China and most Asian countries.

Previously, Quark Pharmaceuticals had been approved by previous CFDA to conduct the QRK207 study in China. QRK207 was conducted by Quark in collaboration with the Neuro-Ophthalmology Research Disease Investigator Consortium (NORDIC) and is currently enrolling subjects in eight countries, including China.

"Oligonucleotide therapeutics are a groundbreaking new class of drugs that have the potential to revolutionize drug development by providing precision therapy while having broad applicability. QRK207 is an international multicenter study seeking treatment for NAION, and we are pleased that this study can be initiated in China", said Dr. Daniel Zurr, Chairman and CEO of Quark Pharmaceuticals and Vice Chairman of RiboQuark." QPI-1007 represents a brand-new treatment strategy for NAION. We plan to expand it for other optic neuropathies that cause retinal ganglion cell apoptosis, such as glaucoma, which closely resembles NAION."

"Translating cutting-edge scientific research into commercial treatments to help patients requires very strong cooperation. We are deeply pleased to have RiboQuark, an innovative company that leads siRNA R&D in China." Dr. Daniel Zurr further stated.

About QPI-1007

QPI-1007 is a synthetic siRNA designed to temporarily inhibit the expression of the pro-apoptotic protein asparaginase 2. QPI-1007 uses a Quark-patented siRNA construct that can maintain activity while reducing off-target and immunostimulatory effects.

The Phase I/IIa, open-label, single-dose, escalation, safety, tolerability, and pharmacokinetic study of QPI-1007 first in human has been completed in 21 clinical sites in the United States and Israel (16 in the United States and 5 in Israel). Studies have shown that this drug has good safety as well as optic nerve protection in patients with NAION. The US FDA has recognized the drug as an orphan drug for anterior ischemic optic neuropathy (AION), a subtype of AION.

About QRK207 study

This is a pivotal Phase II/III, randomized, double-blind, sham-injection-controlled study comparing the safety and efficacy of QPI-1007 between two dose groups and a sham injection group by administering multiple doses of QPI-1007 by vitreous injection to subjects with NAION. This international multicenter study is being conducted at approximately 90 clinical sites including China, US, Israel, Germany, Australia, Italy, Singapore, and India. Approximately 465 patients will be recruited worldwide. Each patient's participation lasts 12 months.

About RNA interference

RNA interference (RNAi) is a general mechanism that uses non-coding RNA in living cells to control whether a gene is active and how active it is. This natural mechanism was discovered in 1998 and the scholar was awarded the Nobel Prize in 2006, bringing great reforms to experimental biology research. Currently, it has bright prospects in the field of clinical pharmaceutical applications. Effector molecules of RNA interference mechanisms are various types of short double-stranded RNA, some of which target specific genes and inhibit their expression in a sequence-dependent manner and are called small interfering RNA (siRNA). siRNA can be designed, synthesized, produced and used as a drug based on the sequence information of any gene. Oligonucleotide therapeutics inhibit the expression of any target gene, regardless of the previous classification of this target gene as "druggable" or "non-druggable." This class of drugs has higher specificity and safety than other small molecule drugs. Quark's siRNA platform includes patented siRNA compound structures and chemical modifications that enhance pharmacological properties. The company's strong intellectual property assets provide a broad space for its development in the siRNA field.