Two clinical trials presented at the European Cystic Fibrosis Conference

ProQR Announces Results for the Second Quarter of 2017

Key updates
• Data from two clinical trials of QR-010 presented at the European Cystic Fibrosis Conference. Enrollment completed in the Phase 1b clinical trial in cystic fibrosis (CF) and top-line data expected to be announced in September.
• Two key patents granted protecting QR-010 for cystic fibrosis in the US and EU.
ProQR Therapeutics N.V. (Nasdaq:PRQR), today announced results for the second quarter of 2017.

“I am pleased with the numerous advancements our team has made this quarter in expanding the depth and breadth of our pipeline with the goal of developing medicines for patients in need,“ said Daniel de Boer, Chief Executive Officer of ProQR. “We have made significant progress in all of our programs as well as in our innovation efforts. Our QR-010 program for CF has completed enrollment of the Phase 1b trial and we look forward to announcing the results from this study in September. Furthermore, for the first program in our ophthalmology pipeline, QR-110 for LCA 10, the FDA and EMA have cleared the IND and CTA, enabling us to start a clinical trial in children and adults to study safety and efficacy. QR-110 was also granted Fast Track designation by the FDA underlining the need for therapeutics for these patients. Additionally, two other programs in our ophthalmology pipeline continue to generate exciting pre-clinical data in Usher syndrome including our QRX-411, which recently was granted orphan drug designation. Our QR-313 program for dystrophic epidermolysis bullosa (DEB) is also progressing in IND-enabling development, on track to start and complete a clinical trial in 2018. Beyond that, we are excited about the Axiomer® platform technology we announced at our R&D day. I’m proud of our scientific team to have invented such a groundbreaking scientific technology that holds the potential to bring a large number of new therapeutics to patients in need.”

Corporate Highlights
In April, ProQR received grants of two key patents protecting QR-010 in the US and EU which provides the Company with exclusive rights for QR-010 for the treatment of CF until at least July 2033. US patent no. 9,605,255 is directed to methods of targeting RNA for the most common mutation in CF, called F508del, using oligonucleotides to restore the function of the CFTR protein. In 2016, ProQR also received the grant of the equivalent European patent (EP 2 852 668 B1). Apart from these ProQR owned patents, the Company has an exclusive license to US patent no. 9,617,535 from Massachusetts General Hospital covering QR-010.
In June, the Company presented two abstracts at the European Cystic Fibrosis Conference (ECFS) in Seville, Spain. Steve Rowe, M.D., professor of Pulmonary, Allergy and Critical Care Medicine at University of Alabama and Director of the Gregory Fleming James Cystic Fibrosis Research Center, and director of the CFF Therapeutics Development Network gave an oral presentation on the final results of study PQ-010-002, a nasal potential difference proof-of-concept study (title “QR-010, an investigational RNA therapeutic, improves CFTR activity in cystic fibrosis subjects homozygous for the F508del mutation”). A poster was also presented on preliminary data from the single ascending dose cohorts of study PQ-010-001, an ongoing Phase 1b safety and tolerability trial (title “QR-010 via inhalation is safe, well-tolerated, and achieves systemic concentrations in a single ascending dose study in subjects with cystic fibrosis homozygous for the F508del CFTR mutation”).
In June, ProQR held its second annual Research & Development Day in New York featuring presentations by ProQR senior management and perspectives from several key opinion leaders on the Company’s development pipeline. During the event, the Company also introduced its next-generation Axiomer® RNA technology platform, which has the potential to yield a new class of medicines for genetic diseases. The archived webcast of the presentation is accessible from the ‘Investor Relations’ section of ProQR’s website ( under ‘Events and Presentations’.
About QR-010

QR-010 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of the disease by targeting the mRNA in CF patients that have the F508del mutation. The F508del mutation is a deletion of three of the coding base pairs, or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally. QR-010 is designed to bind to the defective CFTR mRNA and to restore CFTR function. QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a small, handheld aerosol delivery device which nebulizes QR-010 into a mist inhaled directly into the lungs. QR-010 has been granted orphan drug designation in the United States and the European Union and fast track status by the FDA. The QR-010 project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 633545.

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