ProQR to Present its Ophthalmology Pipeline at ARVO, Highlighting Programs in Leber’s Congenital Amaurosis Type 10 (LCA 10) and Usher Syndrome

Key updates

• At ARVO ProQR will present additional positive pre-clinical proof-of-concept (PoC) data for QR-110 targeting LCA 10. The company has recently received IND clearance to start clinical trials for this program in both adult and pediatric patients.
• ProQR will also present data on its other candidates in the ophthalmology pipeline targeting Usher syndrome, QRX-411 and QRX-421.

LEIDEN, the Netherlands, May 01, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that the Company will present three abstracts during the ARVO conference, including additional positive pre-clinical PoC data for QR-110 in LCA 10.

“RNA based therapeutics for patients suffering from genetic eye diseases are a real opportunity to address an unmet need and we are now at the forefront of this with QR-110 advancing into the clinic. We’re also proud to share data that supports other promising candidates in our ophthalmology pipeline at ARVO”, said Noreen R. Henig, Chief Medical Officer at ProQR. “These programs have many common elements with QR-110 and we hope to advance these rapidly with the pre-clinical and clinical experience in LCA 10.”

ARVO Conference
During the 2017 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) to be held on May 7 – 11, 2017 in Baltimore, MD, USA, the Company will present three abstracts reporting pre-clinical data for three programs targeting forms of genetic retinopathy resulting in blindness.

For ProQR’s lead ophthalmic program, QR-110 for Leber’s congenital amaurosis Type 10 (LCA 10), a poster (#249 - B0280) with additional in vitro pharmacodynamic data will be presented on Sunday, May 7 from 8:30 – 10:15am ET. ProQR has received IND clearance to start a clinical trial for QR-110 in LCA 10 patients. Children and adults in the trial will be treated for a full year with QR-110 to assess safety and efficacy.

Next in ProQR’s ophthalmology pipeline are two programs each targeting specific mutations that result in Usher syndrome. QRX-411 and QRX-421 are candidate molecules that will be presented in two oral presentations. QRX-411 for the c.7595-2144A>G mutation will be presented on Monday, May 8, 2017 from 8:45 – 9:00am ET. QRX-421 for Exon 13 mutations will be presented on Tuesday, May 9, 2017 from 11:45am – 12:00pm.

Abstract titles:

• QR-110 Treatment for Leber’s Congenital Amaurosis Type 10: Restoration of CEP290 mRNA Levels and Ciliation in LCA 10 iPSC-Derived Optic Cups
• QRX-411, an RNA Oligonucleotide (AON) Directing a Splice Correction in USH2A mRNA Caused by the Frequent Deep-Intronic c.7595-2144A>G Mutation Associated with Retinitis Pigmentosa in Usher Syndrome Type 2
• QRX-421, an RNA Oligonucleotide (AON) Targeting Mutations in Exon 13 of USH2A, Associated with Retinitis Pigmentosa in Usher Syndrome Type 2, is Effective in Skipping Exon 13 in the USH2A mRNA of Patients Fibroblasts and Patient-Derived Optic Cups

The abstracts are published on the ARVO 2017 annual meeting website.

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 Type 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* 

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 Type 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.

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

About QRX-411
QRX-411 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the USH2A gene. The mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional or absence of USH2A protein. QRX-411 is designed to restore wild-type USH2A mRNA leading to the production of wild-type USH2A protein by binding the mutated pre-mRNA causing normal splicing of the pre-mRNA.

About QRX-421
QRX-421 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to mutations in exon 13 of the USH2A gene. Mutations in this exon can cause loss of functional USH2A protein that causes the disease. QRX-421 is designed to exclude exon 13 from the mRNA (exon skipping) and produce truncated but functional USH2A protein, thereby modifying the underlying disease.

About Usher syndrome
Usher syndrome is the leading cause of combined deafness and blindness. Patients with this syndrome generally progress to a stage in which they have very limited central vision and moderate to severe deafness. To date, there are no treatments approved or products in clinical development that treat the vision loss associated with the disease. Usher syndrome Type 2 is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene.

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 our plans to present data at ARVO, statements regarding QR-110, including the clinical development and therapeutic potential thereof, statements regarding the PQ-110-001 trial, including the study design and study endpoints, statements regarding our ongoing and planned discovery and development of existing and future product candidates, including QRX-411 and QRX-421 and statements regarding our RNA approach to treating diseases. 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 for the year ended December 31, 2016. 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.

Contact:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431
ir@proqr.com