QURK Quark Pharma (MM)

Quark Pharmaceuticals, Inc., a late clinical-stage pharmaceutical company and leader in the discovery and development of novel RNAi-based therapeutics for unmet medical needs, today announced the acceptance of several abstracts on two major products, teprasiran (previously known as QPI-1002) and QPI-1007.

Teprasiran, an siRNA targeting the p53 gene, is under development in two separate pivotal trials in patients with delayed graft function (DGF) following renal transplantation (NCT# 02610296) and in patients at risk of acute kidney injury (AKI) (NCT#03510897) and its consequences following major cardiac surgery. QPI-1007, an siRNA targeting the caspase-2 gene, is under development in a pivotal clinical trial in non-arteritic anterior ischemic optic neuropathy (NAION) (NCT#02341560).

Two of the abstracts have been accepted for presentation at the American Society of Nephrology (ASN) 2018 Kidney Week Meeting to be held October 23-28, 2018 in San Diego, CA. The data to be presented at ASN are from the Phase 2 Study (NCT#02610283) that met its primary endpoint and include:

• Oral presentation of the one-year mortality follow-up of a Phase 2 Study in subjects treated with teprasiran for the prevention of AKI following major cardiac surgery, and
• Poster on the use of serum cystatin C (SCysC) levels as part of a new, innovative primary endpoint to assess Day 90 Major Adverse Kidney Events (MAKE90) and AKI in the Phase 3 study using teprasiran in subjects following cardiac surgery                   

The initial efficacy results from this study were presented last year at the High-Impact Clinical Trials session of the ASN.

An abstract for QPI-1007 has been accepted for presentation at the American Academy of Ophthalmology (AAO) 2018 Annual Meeting to be held October 27–30, 2018 in Chicago, IL. The presentation at AAO will focus on an interim analysis of the baseline characteristics in patients with NAION from the QRK207 Study, a Phase 2/3, Randomized, Double-Masked, Sham-Controlled Trial of QPI-1007.

Details of presentations and abstracts:

Meeting	Type of presentation	Presentation details	Drug candidate	Abstract title
ASN	Oral presentation	Session Title: AKI: Can We Improve Outcomes? [OR0102-2] October 26, 4:30 PM to 6:30 PMPresentation Time: 5:18PM	teprasiran	One-year mortality follow-up in subjects at risk of Acute Kidney Injury (AKI) following cardiac surgery (CS), treated or non-treated with QPI-1002. Abstract ID: 3016225
ASN	Poster	Session Title: AKI: Clinical, Outcomes, Trials - I [PO0102-1]October 26, 10:00AM - 12:00 PM Poster Board #: FR-PO010	teprasiran	Utility of serum cystatin C (SCysC) levels to assess Acute Kidney Injury (AKI) and Day 90 Major Adverse Kidney Events (MAKE90) following cardiac surgery (CS) in patients treated with QPI-1002. Abstract ID: 3012699
AAO	Oral presentation	Session Title: OP11 Neuro-Ophthalmology Original Paper Session October 30Presentation Time: 8:30AM	QPI-1007	Screening data of 200 subjects with NAION. Results from an Interim Analysis of a Phase 2/3, Randomized, Double-Masked, Sham-Controlled Trial of QPI-1007 (QRK207 Study). Abstract ID: 30057057

About teprasiran (QPI-1002)Teprasiran is the first synthetic chemically-modified siRNA drug to enter human clinical trials with systemic administration and to complete several well-controlled clinical studies with efficacy endpoints that were conducted in hundreds of patients. It is an investigational drug designed to temporarily inhibit the expression of the cell death inducing gene, p53, to protect normal kidney cells from death arising from acute ischemia-reperfusion injury. Teprasiran completed dosing of 594 patients in a pivotal Phase 3 study (QRK306, NCT02610296) for delayed graft function (DGF) following kidney transplantation in January 2018. Topline results are expected in Q4 2018. Teprasiran also reported positive results in a 341 patient, multicenter double-blind and placebo-controlled Phase 2 trial for prevention of AKI in high-risk patients undergoing cardiovascular surgery in 2017 (QRK209, NCT#02610283).  A 1,088 patient Phase 3 trial in AKI (QRK309, NCT03510897) is ongoing. Both Phase 2 studies provided support for a protective effect of teprasiran under conditions of acute ischemia-reperfusion kidney injury. Teprasiran has been granted Orphan Drug designation in the USA and Europe for prophylaxis of delayed graft function following kidney transplantation. Under an August 2010 agreement, Novartis has an exclusive worldwide license option for the development and commercialization of teprasiran.

About QPI-1007QPI-1007 is a synthetic chemically modified 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. It has shown a significant and substantial neuroprotective effect in numerous animal models of acute and chronic ocular neurodegeneration. In the completed Phase 1/2 trial in patients with NAION, single intravitreal injection of QPI-1007 performed within 2-4 weeks of disease onset halted further visual deterioration otherwise observed in this disease.

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 pivotal clinical studies. Quark’s broad pipeline of clinical and preclinical product candidates is generated from the company’s internally-developed siRNA platform technology and focuses on extrahepatic indications. Quark is headquartered in Fremont, California and operates research facilities in Ness-Ziona, Israel. For additional information please visit: www.quarkpharma.com.

ContactQuark Pharmaceuticals, Inc.Investor Relations:Gavin Samuels+1 510 402 4020      gsamuels@quarkpharma.com