ProQR Therapeutics B.V. Announces Results For The Second Quarter Of 2015

LEIDEN, the Netherlands, Aug. 19, 2015 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe diseases such as cystic fibrosis (CF) and Leber's congenital amaurosis (LCA), today announced results for the second quarter of 2015.

"The second quarter of 2015 was an important quarter for our Company. Since inception of the Company, three years ago, employees and external partners have worked very hard to achieve the start of our first clinical trial, so we were pleased to announce that we have started enrollment of patients in our first clinical trial to test our drug candidate QR-010 in patients with cystic fibrosis," said Daniel de Boer, Chief Executive Officer of ProQR. "I am happy and grateful that we have accomplished this important milestone and want to thank everyone who was involved."

Financial Highlights

As of June 30, 2015, ProQR held cash and cash equivalents of €106.4 million, compared to €113.8 million at March 31, 2015. Net cash used in operating activities during the three month period ended June 30, 2015 was €5.9 million, compared to €3.2 million for the same period last year.

Research and development costs increased to €5.4 million for the quarter ended June 30, 2015 from €3.3 million for the same period in 2014 and comprised of allocated employee costs including share-based payments, the costs of materials and laboratory consumables, license and intellectual property costs and other allocated costs. The increase in expenses was primarily due to advancement of QR-010 into clinical development and QR-110 from research into development as well as increased investment into our RNA based therapeutics pipeline.

General and administrative costs increased to €1.8 million for the quarter ended June 30, 2015 from €1.5 million for the same period in 2014. The increase is primarily due to the growth of the G&A functions to support the strong overall growth of the company, as well as costs directly and indirectly associated with our NASDAQ listing.

Net result for the three month period ended June 30, 2015 was a €8.6 million loss or €0.37 per share, compared to a €4.8 million loss or €0.74 per share for the same period in 2014. For further financial information for the period ending June 30, 2015, please refer to the financial statements appearing at the end of this release.

Corporate Highlights

• Enrollment has started in study PQ-010-001, a global Phase 1b clinical study of QR-010, a novel investigational RNA therapeutic designed to repair the genetic mutation in the mRNA of cystic fibrosis (CF) patients due to the ∆F508 mutation. PQ-010-001 is a Phase 1b randomized, double-blind, placebo-controlled, 28-day study to be conducted in approximately 20 centers worldwide. This first-in-human study will evaluate the safety, tolerability and pharmacokinetics of single and multiple ascending doses of inhaled QR-010 in 64 CF patients homozygous (carrying two copies) of the DF508 mutation. As exploratory efficacy endpoints this study will also assess sweat chloride, weight gain, CFQ-R Respiratory Symptom Score and lung function, measured by FEV1.
• The Phase 1b trial will be conducted in parallel with a proof-of-concept Nasal Potential Difference (NPD) study of QR-010 that will enroll 16 CF patients that are either DF508 homozygous or compound heterozygous (one copy of the ∆F508 plus one other CF disease causing mutation) for the mutation and is expected to begin in 3Q2015.
• The Company selected the product candidate in our second program, QR-110 for the treatment of Leber's congenital amaurosis (LCA), the most common genetic blindness in childhood. QR-110 is an RNA oligonucleotide designed to repair the pCys998X mutation that results in abnormal CEP290 protein in LCA patients. QR-110 is the first program to be added to the pipeline behind the CF program from ProQR's internal innovation (discovery) unit and the Company intends to start the first clinical trial of QR-110 in patients in 2016.
• The European Committee (EC) through its Horizon 2020 program awarded ProQR and its academic partners a grant of €6 million to support the clinical development of QR-010. Horizon 2020 is one of the largest research and innovation programs in the European Union with nearly €80 billion in available funding for qualified projects from 2014 to 2020. Under the Horizon 2020, the EC works closely with the European Research Council, the European Institute of Innovation and Technology and EURATOM and evaluates projects on three key pillars of Excellent Science, Industrial Leadership and Societal Challenges.
• ProQR received an additional tranche of €1.2 million under the Innovation credit program or "Innovatiekrediet" by the Dutch government, through its agency RVO (previously: "AgentschapNL") of the Ministry of Economic Affairs, for the cystic fibrosis development program. The credit covers 35% of the costs incurred for the program up to an initial maximum of € 5.0 million through November 30, 2015. The credit is interest-bearing at a rate of 10% per annum. The credit, including accrued interest, is repayable in three instalments on January 31, 2017, January 31, 2018 and January 31, 2019, depending on the technical success of the program.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe diseases such as cystic fibrosis and Leber's congenital amaurosis. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Since 2012.

FORWARD-LOOKING STATEMENTS

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding the initiation of clinical trials of QR-010 and QR-110, statements regarding our ongoing and planned discovery and development of product candidates, statements regarding the timing and availability of funding under the Horizon 2020 program and statements regarding the terms and conditions of funding under the Innovation credit program. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.