NASDAQ OMX

ProQR's Drug Candidate QRX-411 for Usher Syndrome Receives Orphan Drug Designation from FDA and EMA

Dela


Key Updates

  • ProQR's QRX-411 receives Orphan Drug Designation by the FDA and EMA for the treatment of retinitis pigmentosa, including Usher syndrome, the subtype targeted by QRX-411. Usher syndrome is an inherited condition that is characterized by combined deafness and blindness.
  • QRX-411 targets the pseudo-exon 40 (PE-40) mutation in the USH2A gene and currently there are no therapies commercially available or in clinical development for the vision loss associated with this disease.
  • QRX-411 has shown promising preclinical data in both patient fibroblasts and the optic cup model for mRNA restoration, which was presented at the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in May 2017.
  • A lead candidate has been selected for this program and is currently ready for IND-enabling studies.
  • QRX-411 is part of ProQR's ophthalmology pipeline that currently also includes one clinical compound, QR-110 for Leber's Congenital Amaurosis Type 10, and three preclinical programs, QRX-421 for Usher syndrome, QRX-1011 for Stargardt's disease and QRX-504 for Fuchs endothelial corneal dystrophy.

LEIDEN, the Netherlands, July 05, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR) today announced that the company's investigational drug QRX-411 has received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the treatment of retinitis pigmentosa, including Usher syndrome, the subtype targeted by QRX-411. Usher syndrome is the leading cause of combined deafness and blindness due to genetic defects in the Usher gene.

ODD in the U.S. and European Union provides a special status for investigational drugs being developed for rare diseases.  The ODD programs offer development program tax benefits and a waiver of the NDA application user fee, as well as market exclusivity for up to seven years in the U.S., and ten years in the European Union following market approval.

"We are pleased with the progress we have made to date with our novel RNA based therapeutic ophthalmology pipeline for patients suffering from genetic eye diseases.  Securing orphan drug designations from the FDA and EMA for QRX-411 is a milestone for the program and highlights the importance of addressing the unmet need of this debilitating disease," said Daniel A. de Boer, CEO of ProQR,  "The severe genetic retinal diseases we are targeting do not have any available therapies, especially disease modifying therapies focused on restoring vision or impeding progression of the disease.  We believe our novel RNA oligonucleotide approach has the potential to make a meaningful impact in the lives of Usher syndrome patients and others with rare genetic eye diseases."

Chief Development Strategy Officer, David M. Rodman, MD, notes, "At ProQR we have a unique opportunity to combine the flexibility of our oligonucleotide drug discovery platform with accelerated drug development strategies for rare diseases.  Orphan drug designation is an important step in rapidly bringing transformational precision medicines to patients with Usher syndrome and many other genetic causes of blindness in children and adults."

ProQR's growing ophthalmology portfolio includes:

  • QR-110 for Leber's congenital amaurosis Type 10 (LCA 10) due to the p.Cys998X mutation, which received IND and CTA clearance and is in clinical development (PQ-110-001 Phase 1/2 safety and efficacy study).  QR-110 was also granted Fast Track designation by the FDA and Orphan Drug designation by the FDA and EMA.
  • QRX-411 for Usher syndrome type II due to the PE-40 mutation in the USH2A gene, for which a clinical candidate has been selected and is ready for IND enabling development studies.
  • QRX-421 for Usher syndrome type II due to Exon 13 mutations in the USH2A gene, for which a clinical candidate has been selected and is ready for IND enabling development studies.
  • QRX-1011 for Stargardt's disease due to c.5461-10T>C mutations in the ABCA4 gene, which is in optimization phase.
  • QRX-504 for Fuchs endothelial corneal dystrophy (FECD), for which a clinical candidate has been selected and is ready for IND enabling development studies.

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 II is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene.

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 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*

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 QRX-411 and the clinical development and therapeutic potential thereof, statements regarding orphan drug designation, including the intended benefits of such status, statements regarding our ongoing and planned discovery and development of product candidates and the timing thereof, including those in our ophthalmology portfolio, and statements regarding our oligonucleotide drug discovery platform.  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. 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:
Bonnie Ortega
Director, Investor Relations
T: +1 858 245 3983
ir@proqr.com




This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: ProQR Therapeutics N.V. via Globenewswire

Om

NASDAQ OMX
NASDAQ OMX



Följ NASDAQ OMX

Abonnera på våra pressmeddelanden.

Senaste pressmeddelandena från NASDAQ OMX

CrownBio Supports Excellence in Translational Research with Grant Award that Funds Innovative PDX Model Development in Immuno-Oncology19.7.2018 15:03Pressmeddelande

"Cancer Research Center of Lyon iPS novel PDX models formulate new immune cell focused strategies" SAN DIEGO, July 19, 2018 (GLOBE NEWSWIRE) -- Crown Bioscience, a global drug discovery and development services company providing translational platforms to advance oncology, inflammation, cardiovascular and metabolic disease research, is proud to announce the award of its Patient-Derived Xenograft (PDX) grant program to doctors Fabrice Lavial, Christophe Caux and Bertrand Dubois from the Cancer Research Center of Lyon (CRCL). The grant program was established to support oncology researchers pursuing scientific advancements in cancer using PDX technology. Following an extensive review, CrownBio's Scientific Steering Committee selected Dr. Lavial and his colleagues at the CRCL due to their project's potential to create breakthroughs in preclinical oncology strategies. Their research combines PDX and iPS cell technologies to create patient-specific, humanized models. These unique models wil

Jumio Introduces Liveness Detection for Web to Better Deter Fraud During Account Onboarding19.7.2018 14:00Pressmeddelande

New identity proofing functionality ensures that the person behind a Web upload verification is present and the person actually creating a new online account PALO ALTO, Calif., July 19, 2018 (GLOBE NEWSWIRE) -- Jumio, the creator of Netverify(TM) Trusted Identity as a Service (TIaaS), today announced the addition of Liveness Detection for Web to better thwart fraudsters' evolving and sophisticated attempts to assume another individual's identity. With identity verification, Jumio's approach is to first verify the government-issued ID (e.g., driver's license, passport or ID card) and ensure that it is authentic and has not been doctored. For mobile SDK customers, Jumio then requires the user to take a selfie which enables the company to compare the person in the selfie to the person pictured in the ID document. Since Netverify is an omnichannel solution, Jumio also supports identity verification via desktop webcams. This channel enables our customers to reach large market segments who a

Northland Power Adds 17MW of Capacity to the Deutsche Bucht Offshore Wind Farm19.7.2018 12:33Pressmeddelande

TORONTO, July 19, 2018 (GLOBE NEWSWIRE) -- Northland Power Inc. ("Northland") (TSX:NPI) announced today that the Deutsche Bucht Mono Bucket pilot demonstrator ("Demo") project has reached financial close. All required funds for the Demo project have been contributed by Northland and committed by the project lenders. In May, the Federal Maritime and Hydrographic Agency of Germany (BSH) granted the Deutsche Bucht offshore wind project the permit required to erect two additional pilot wind turbines using Mono Bucket foundations. Deutsche Bucht will be the first offshore wind farm worldwide to test this new type of foundation structure under commercial operating conditions. As previously announced, the two turbines will contribute an additional 17 MW of capacity to the base 252 MW project for a total of 269 MW and bringing the total project cost to approximately €1.4 billion (CAD $2.0 billion) - up from €1.3 billion. Northland's corporate investment has increased from approximately $400 mi

Canadian Natural Resources Limited Announces 1 Billion Barrel Production Milestone at the Athabasca Oil Sands Project19.7.2018 11:00Pressmeddelande

CALGARY, Alberta, July 19, 2018 (GLOBE NEWSWIRE) -- Canadian Natural Resources Limited ("Canadian Natural"), as operator of the Jackpine and Muskeg River mines at the Athabasca Oil Sands Project ("AOSP"), is pleased to announce that the AOSP has achieved a mined production milestone of 1 billion barrels since operations began in 2003. Canadian Natural's President Tim McKay stated, "This is a significant milestone and demonstrates the size and scale of Canada's oil sands mining operations. The oil sands, through responsible development, provide sustainable long life low decline production and significant value for stakeholders. This milestone could not have been achieved without the dedication of the employees and contractors who focus on safe, reliable operations, while minimizing our environmental footprint to ensure long term sustainable development." Canadian Natural has a 70% working interest in the AOSP along with Chevron Canada Limited (20%) and Shell Canada Limited (10%). Canadi

ForgeRock Opens Up Open Banking19.7.2018 10:00Pressmeddelande

Delivers New, Open Reference Implementation and Sandbox Directory for Developers to Test Open Banking APIs in Five Minutes or Less LONDON, July 19, 2018 (GLOBE NEWSWIRE) -- ForgeRock®, the leading platform provider of digital identity management solutions, announced today the availability of the ForgeRock Open Banking Directory. Continuing its commitment to innovation and industry leadership, ForgeRock has delivered a reference bank implementation and directory, providing a technical sandbox for organizations looking to build and test Open Banking/PSD2 APIs. The lightweight and incredibly fast test platform has been acknowledged and celebrated by the team at UK Open Banking, the Implementation Entity set up by the UK Government's Competition and Markets Authority (CMA) in 2016, to deliver Open Banking. The ForgeRock development team has created an Open Banking Technical Solution Guide on the directory. Open Banking regulations, which began coming online in the UK earlier this year, for

TradeStation International Ltd to Offer Subscription Account Featuring TradeStation Technologies' Advanced Analytical Capabilities19.7.2018 10:00Pressmeddelande

LONDON, July 19, 2018 (GLOBE NEWSWIRE) -- TradeStation International Ltd, the London-based, United Kingdom subsidiary of TradeStation Group, Inc., today announced that it is now offering European traders, investors, advisors and market analysts access to TradeStation Analytics -- a non-brokerage subscription account featuring all of the advanced analytical capabilities of the TradeStation award-winning* platform. The software subscription account, which is provided to customers directly by affiliate TradeStation Technologies, Inc., offers market participants an opportunity to join the thousands of U.S.-based TradeStation subscribers - professionals and non-professionals alike - who trust TradeStation's cutting-edge technology to perform sophisticated market analysis, identify opportunities and stay one step ahead of today's rapidly changing financial markets. This TradeStation Analytics premium subscription package includes: Market Data delivered with speed, accuracy and reliability, w

I vårt pressrum kan du läsa de senaste pressmeddelandena, få tillgång till pressmaterial och hitta kontaktinformation.

Besök vårt pressrum