ProQR Announces Presentations and Investor & Analyst Event at the North American Cystic Fibrosis Conference

LEIDEN, the Netherlands, Oct. 24, 2017 (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 including cystic fibrosis, Leber's congenital amaurosis 10 and dystrophic epidermolysis bullosa, today announced details about upcoming presentations and an investor & analyst event at the North American Cystic Fibrosis Conference being held November 2 – 4, 2017 in Indianapolis, Indiana. 

Oral and Poster Presentations  

J. Stuart Elborn, Clinical Chair in Respiratory Medicine at Imperial College, Consultant at Royal Brompton Hospital, and immediate past-president of the European Cystic Fibrosis Society, will give an oral presentation titled: “A first-in-human, Phase 1B, dose-escalation study of QR-010, a novel antisense oligonucleotide administered in subjects with cystic fibrosis homozygous for the F508del CFTR mutation.”  The presentation will take place during the Workshop: “Toward the Goal of a One-Time Cure: Challenges & Opportunities” taking place in Room 140 on November 4 at 10:30am – 11:50am ET.

The Company will also present poster #206 with the same title during Poster Session I on November 2 at 11:15am – 1:45pm ET.  NACFC abstracts are now in the online edition of Pediatric Pulmonology.  

Investor and Analyst Event

ProQR management will host an investor & analyst event in Indianapolis on November 2 to further discuss the data from the Phase 1b study of the QR-010 candidate, opportunities to target stop-codon (or Class I) mutations in the CFTR gene using the Company’s Axiomer® technology platform, and provide an update on other product candidates in the pipeline.  The event begins at 8:30pm ET, with registration starting at 8:00pm ET.  Kindly RSVP to Jennifer Porcelli at to reserve a seat and for additional information. 

About Cystic Fibrosis

Cystic fibrosis (CF) is the most common fatal inherited disease in the Western world and affects over 75,000 patients worldwide. In people with CF, a defective CFTR gene causes a thick buildup of mucus in the lungs, pancreas and other organs. In the lungs, the mucus clogs the airways and traps bacteria leading to infections, extensive lung damage and eventually, respiratory failure. There is no cure for CF.  Disease manifestations lead to a shortened life expectancy with a median age of death of 30 years.  Although over 1,900 CF-causing gene mutations have been identified, approximately 85% of all CF patients are affected by the F508del mutation. Among all CF patients, approximately 50% are homozygous for the F508del mutation.

About QR-010

QR-010 is a first-in-class 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 technology was exclusively licensed from Massachusetts General Hospital. The F508del mutation results in the production of a misfolded CFTR protein that does not function normally. QR-010 is a single agent 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 received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 633545.

About the PQ-010-001 Trial

The Phase 1b study was a trial designed to assess safety, tolerability and pharmacokinetics of QR-010. A number of exploratory efficacy endpoints were assessed in the multiple dose groups. A total of 4 dose levels were studied: 6.25, 12.5, 25 and 50 mg of QR-010 in solution per dose administered via inhalation using the PARI eFlow® nebulizer. Subjects eligible to participate were males and females of 18 years and over with a ppFEV1 of ≥70% at time of inclusion, homozygous for the F508del mutation, and not taking CFTR modulator drugs. The study design planned to enroll 8 cohorts of 8 subjects (6 receiving QR-010, 2 receiving placebo). In cohorts 1-4, a single dose of QR-010 was administered, and in cohorts 5-8 twelve doses of QR-010 were administered over a 4-week period.

Recently announced preliminary top-line results from the trial showed that QR-010 was observed to be safe and well-tolerated across all dose levels and demonstrated that people with CF can benefit from taking QR-010. Most subjects in the trial reported having a reduction in CF symptoms after receiving QR-010 (as measured by an increase in the Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score, or CFQ-R RSS) compared to placebo. A supportive trend was observed in improved lung function (as measured by percent predicted forced expiratory volume in 1 second, or ppFEV1) compared to placebo. Subjects that received placebo did not report this reduction in CF symptoms or improvement in lung function. No change was observed on sweat chloride and weight gain. 

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 cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. 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. 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 QR-010, including its clinical development and therapeutic potential, and future development plans and potential product candidates. 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, including that positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, that a Fast Track designation by the FDA may not actually lead to a faster development, regulatory review or approval process, and that we may not be able to realize the potential benefits of orphan drug exclusivity, 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, except as required by law.

ProQR Therapeutics N.V.:

Media and Investor Contact:
Bonnie Ortega
T: +1 858 245 3983

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