ProQR's Drug Candidate QRX-411 for Usher Syndrome Receives Orphan Drug Designation from FDA and EMA
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.
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.
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.
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.
Director, Investor Relations
T: +1 858 245 3983
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
Följ NASDAQ OMX
Skriv in din e-postadress så får du ett mejl när vi har något nytt att berätta.
Senaste pressmeddelandena från NASDAQ OMX
Abiomed Receives FDA PMA Approval for Impella RP® for Right Heart Failure21.9.2017 00:23 | Pressmeddelande
DANVERS, Mass., Sept. 20, 2017 (GLOBE NEWSWIRE) -- Abiomed, Inc. (NASDAQ:ABMD), a leading provider of breakthrough heart support and recovery technologies, today announced it has received U.S. Food and Drug Administration (FDA) pre-market approval (PMA) for the Impella RP® heart pump. Culminating from five years of research, this approval follows the prior FDA Humanitarian Device Exemption (HDE) received in January 2015 and adds the Impella RP heart pump to Abiomed's platform of PMA approved devices. With this approval, the Impella RP heart pump is the only percutaneous temporary ventricular support device that is FDA-approved as safe and effective for right heart failure as stated in the indication: The Impella RP System is indicated for providing temporary right ventricular support for up to 14 days in patients with a body surface area greater than or equal to 1.5 m2, who develop acute right heart failure or decompensation following left ventricular assist device impl
Senegal and France to host Global Partnership for Education Financing Conference20.9.2017 23:00 | Pressmeddelande
NEW YORK, Sept. 20, 2017 (GLOBE NEWSWIRE) -- The Global Partnership for Education (GPE) is delighted that the governments of Senegal and France will co-host its financing conference, which will take place on February 8, 2018 in Dakar, Senegal. A photo accompanying this announcement is available at http://www.globenewswire.com/NewsRoom/AttachmentNg/d3003cff-ee20-409f-9729-9a9cef1d4d3e A video accompanying this announcement is available at http://www.globenewswire.com/NewsRoom/AttachmentNg/0a7eebb6-66b3-438b-904e-efeff83d94cf The announcement of the co-hosting was made by Presidents Macky Sall of Senegal and Emmanuel Macron of France at a high-level event on education financing held at the United Nations, which was attended by Secretary General António Guterres, several heads of state and leaders on global education. " The Global Partnership for Education has made substantial investments in education helpin
K2 Launches New Process Automation Platform Including New Cloud Service20.9.2017 18:00 | Pressmeddelande
K2's process automation platform allows businesses of all types around the globe to rapidly build and deploy automation across the business. BELLEVUE, Wash., Sept. 20, 2017 (GLOBE NEWSWIRE) -- K2 today announced the availability of its new low-code process automation platform - furthering innovation in the way organizations drive digital efficiencies. With a new managed cloud service, K2 Cloud, new on-premises version, K2 Five, and completely redesigned user interfaces and experiences, the platform helps businesses of all types around the globe rapidly deliver powerful process automation and realize business productivity gains. Automating business processes across an enterprise is crucial to the success of companies in the era of digital transformation. However, many initiatives are impacted by a lack of sufficient resources and the use of inefficient systems to manage processes. K2's low-code process automation platform solves these problems by enabling organ
8x8 and Aryaka Partner to Enhance Cloud Communications for Global Enterprises20.9.2017 16:43 | Pressmeddelande
Strategic Alliance will improve communications experience for business users worldwide SAN MATEO, Calif., Sept. 20, 2017 (GLOBE NEWSWIRE) -- Aryaka®, the leading global SD-WAN provider, and 8x8, Inc. (NASDAQ:EGHT), the world's first Communications Cloud provider, today announced that they are entering a strategic partnership that will improve the performance and reliability of business-critical cloud unified communications. This will enable global mid-market and enterprise companies to enhance business communications, collaboration and customer engagement for their employees, customers and partners around the world. Through this partnership, Aryaka's global SD-WAN solution will provide enterprise-grade private connectivity to further enhance the superior voice and video quality delivered by the 8x8 Communications Cloud(TM). When tested against several leading Unified Communications as a Service (UCaaS) providers, 8x8 was recently found by the Tolly Group to deliver the hi
NRT Technology Corp and Gaming Payments Innovator Sightline Payments Announce Merger20.9.2017 16:00 | Pressmeddelande
Combination Will Accelerate Innovation in Mobile, Data, Cashless Payments, and Provisional Credit Solutions to the Worldwide Integrated Resort Casino Industry LAS VEGAS, Sept. 20, 2017 (GLOBE NEWSWIRE) -- NRT Technology Corp. ("NRT") a leading provider of integrated ticket redemption and payment services to global casino operators, and Sightline Payments ("Sightline"), developers of the disruptive Play+ platform that's powering the next generation of cashless and mobile gaming payment solutions, announced today an agreement to merge. The company on closing will be named NRT Sightline and will form one of the largest pure play gaming payment technology companies in the world with an equity value of over $300 million. Following the closing of the transaction and subject to regulatory approvals, Sightline's CEO and Founder, Kirk Sanford, will assume the role of President and CEO of NRT Sightline. NRT's current Founder, P
BT and Hitachi Partner to Develop Industrial and Enterprise IOT Solutions20.9.2017 15:00 | Pressmeddelande
Initial Focus on Asset Intelligence and Predictive Maintenance Use Cases LAS VEGAS and LONDON, Sept. 20, 2017 (GLOBE NEWSWIRE) -- BT, one of the world's leading providers of communications services and solutions, and Hitachi Vantara, a wholly owned subsidiary of Hitachi Ltd. (TSE:6501) today announced a global partnership to co-create innovative solutions in the area of industrial and enterprise internet of things (IoT). The partnership aims to drive better business outcomes for global customers, including greater efficiency, productivity and cost savings. The companies will initially focus on exploring and designing asset intelligence and predictive maintenance solutions to deliver the desired outcomes for customers in industry sectors such as manufacturing and transportation. Announced at Hitachi's inaugural user conference, Hitachi NEXT 2017, in Las Vegas today, the partnership unites BT's global network infrastructure, cloud capabiliti
I vårt pressrum kan du läsa de senaste pressmeddelandena, få tillgång till pressmaterial och hitta kontaktinformation.Besök vårt pressrum