Presentation at the 18th International Symposium on Retinal Degeneration (RD2018) at
Investor conference call to be held at
|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
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
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.
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.
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
Chief Financial Officer
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Source: ProQR Therapeutics N.V.