ProQR to Present Interim Phase 1/2 Results for QR-110 in Leber’s Congenital Amaurosis 10 at the Symposium for Retinal Degeneration on Sept. 5th

LEIDEN, The Netherlands and CAMBRIDGE, Mass., Aug. 15, 2018 (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 genetic rare diseases, today announced it will issue data from a planned interim analysis in the ongoing Phase 1/2 clinical trial of QR-110 in patients with Leber’s congenital amaurosis 10 (LCA10) due to the p.Cys998X mutation (the PQ-110-001 trial) at the 18th International Symposium on Retinal Degeneration (RD2018), which is being held on Sept. 3-8, 2018 in Killarney, Ireland.

Presentation Details:
Presentation title:    Intravitreal Antisense Oligonucleotide Therapy (QR‐110) for the Treatment of
Leber Congenital Amaurosis due to Photoreceptor Cilium Defect in Subjects
with the CEP290 p.Cys998X Mutation
Presenter:    Dr. Artur Cideciyan, Research Professor of Ophthalmology at the Scheie Eye Institute,
University of Pennsylvania and investigator of the PQ-110-001 trial
Date and Time:    Wednesday, Sept. 5, 2018 at 8:30 am GMT
Session Details:    Platform Session V: Drug Therapy

Investor conference call

Management will also host a conference call for its investors to discuss the presented interim Phase 1/2 data on Wednesday, Sept. 5, 2018 starting at 8:15 am ET. Details for the call will be included in a press release the same day and will be made available in the “Investors” section of ProQR’s website ( under “Events and Presentations”.

About PQ-110-001 trial

PQ-110-001 is an open-label trial that has been designed to enroll approximately six children (age 6 - 17 years) and six adults (≥ 18 years) who have LCA10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. Patients are receiving four intravitreal injections of QR-110 into one eye; once every three months. The trial is being conducted at three specialized centers with significant expertise in genetic retinal disease: the University of Iowa, Iowa City, IA, US, the Scheie Eye Institute at the University of Pennsylvania, Philadelphia, PA, US and the Ghent University Hospital, Ghent, Belgium.

The primary objectives of the trial are safety and tolerability. Secondary objectives include pharmacokinetics as well as restoration/improvement of visual function and retinal structure through ophthalmic endpoints such as visual acuity, full field stimulus testing (FST), optical coherence tomography (OCT), pupillary light reflex (PLR), mobility course and fixation stability. Changes in quality of life in the trial subjects are also being evaluated.

About Leber’s Congenital Amaurosis 10

Leber’s congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children and consists of a group of diseases of which LCA 10 is one of the more severe forms. LCA10 is caused by mutations in the CEP290 gene, of which the p.Cys998X mutation is the most common. LCA10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To date, there are no treatments approved or other products in clinical development that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA10 because of this mutation.

About QR-110

QR-110 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis 10 due to the p.Cys998X mutation in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. QR-110 is designed to restore wild-type CEP290 mRNA leading to the production of wild-type CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and fast track status by the FDA.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as Leber’s congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
*Since 2012*


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. Such statements include those relating to the RD2018 presentation, QR-110 and the PQ-110-001 trial, as well as our clinical development plans for and therapeutic potential of our product candidates. 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. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, 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, except as required by law.

ProQR Therapeutics N.V.:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431


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