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Home  /   Forum   /   ProQR Therapeutics   /   Nieuws media financieel ProQR Therapeutics NV

Aandeel ProQR Therapeutics NV OTC:PRQR.Q, NL0010872495

Vertraagde koers (usd) Verschil Volume
2,655   +0,065   (+2,51%) Dagrange 2,475 - 2,705 718.766   Gem. (3M) 1,5M

Nieuws media financieel ProQR Therapeutics NV

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31 Posts
Pagina: «« 1 2 | Laatste | Omlaag ↓
  1. [verwijderd] 10 december 2018 13:30
    quote:

    ivet schreef op 10 december 2018 13:19:

    The credit, including accrued interest, is repayable depending on obtaining market approval.

    Je denkt toch zeker niet dat onze regering over 1 nacht ijs gaat en dit geld geeft zonder dat ze dit terug gaan krijgen? Zij zien het dus ook helemaal zitten met ProQR :)
  2. [verwijderd] 29 januari 2019 15:05

    Di 29-1-2019, 13:31

    ProQR Announces “ProQR Vision 2023” Strategy at its Annual R&D Day

    LEIDEN, the Netherlands & CAMBRIDGE, Mass., Jan. 29, 2019 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines, today announced its “ProQR Vision 2023” strategy, which is focused on the development and commercialization of RNA medicines for inherited retinal diseases (IRDs). The management team will elaborate on this strategy and present advancements across its broader clinical development pipeline during an R&D day event being held today in New York.
    ProQR plans to independently advance its pipeline of RNA medicines to establish a multi-product, platform company in IRD. By 2023, the company expects its pipeline to have at least two commercial products, and at least three late-stage and seven early-stage programs in development. In parallel, the company will continue to expand its RNA platform capabilities in other therapeutic areas, and plans to selectively partner programs in non-core therapeutic areas and non-rare diseases.

    “The ‘ProQR Vision 2023’ strategy presents a clear path for ProQR to become a fully-integrated company that is independently developing and commercializing innovative RNA medicines for patients who suffer from IRDs. Given the high unmet need across many of the 300 IRDs known today, we believe this integrated approach will allow us to more efficiently turn our scientific innovation into multiple potentially life-changing medicines for patients,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “With our experienced team, predictive translational models and capabilities in precision medicine, we are well-positioned to execute on this long-term strategy and advance the field of medicines for inherited blindness.”

    “ProQR Vision 2023” strategy and key goals

    The “ProQR Vision 2023” strategy provides a clear path forward to develop the company’s platform of RNA medicines for IRD patients in need. The programs in ProQR’s pipeline utilize the RNA oligonucleotide technology platform that repairs the genetic defect in the RNA to address the underlying cause of genetic diseases. The product candidates in ProQR’s pipeline target diseases with a well-understood genetic cause where rational drug design can be applied to yield RNA molecules with therapeutic potential. One notable differentiator for ProQR’s therapeutic candidates for IRDs is that they are designed for intravitreal delivery. This design is intended to promote rapid delivery of the molecules to the target cells across the entire retina, which we believe is an advantage in the development of medicines for retinal disease.

    The key deliverables for “ProQR Vision 2023” include:

    Sepofarsen (formerly QR-110) for Leber's congenital amaurosis 10 (LCA10): Complete pivotal program around year-end 2020 for submission of a New Drug Application (NDA) in the U.S. and a Marketing Authorization Application (MAA) in Europe in 2021
    QR-421a for Usher syndrome Exon 13: Start the Phase 1/2 STELLAR proof-of-concept clinical trial, with interim data in mid-2019 and initiate an adaptive multiple-dose trial with projected readout in 2021
    QR-1123 for P23H autosomal dominant retinitis pigmentosa (adRP): Initiate a Phase 1/2 proof-of-concept clinical trial in 2019, with data expected in 2020
    QR-411a for Usher syndrome PE40: Conduct Investigational New Drug (IND)-enabling work in 2019 to start a proof-of-concept clinical trial in 2020
    QR-504 for Fuchs endothelial corneal dystrophy (FECD): Complete IND-enabling activities in 2019 to start a proof-of-concept clinical trial in 2020
    Accelerate discovery: Expand efforts to generate additional programs for inherited retinal diseases amenable to RNA oligo treatment with the goal of establishing at least seven new programs for development
    Selectively explore and expand platform: Expand platform to develop medicines in new therapeutic areas and create opportunities to build businesses in other therapeutic areas
    Key 2019 business goals

    In pursuit of the “ProQR 2023 Vision” strategy, ProQR will focus on critical success factors in 2019:

    Clinical trial execution: Expand our clinical trial operational infrastructure to support enrollment of more than four clinical programs, including the execution of a pivotal Phase 2/3 trial in LCA10, while also preparing the next wave of ophthalmology drugs through IND-enabling activities and advancing to the clinic
    Start building commercial capabilities: Build a commercial infrastructure in preparation for a potential commercial launch of sepofarsen in 2021 and QR-421a in North America and Europe in 2023, if these candidates are approved
    Expand pipeline for inherited blindness: Scale up discovery and lead development efforts to expand the IRD pipeline into other forms of genetic blindness
    Validate and expand Axiomer®: Further validate and expand ProQR's proprietary Axiomer® RNA editing platform through first development candidate selection and potential additional alliances
    Selectively partner non-core programs: Explore alliances based on the technology platform and pipeline programs outside of IRDs
    Financial discipline: Maintain strong financial discipline by operating the business in a capital efficient way
    “Our ‘ProQR Vision 2023’ strategy presents a long-term plan that allows us to focus on our most promising assets and leverage this progress to expand our business in order to create the most value for patients and other stakeholders, including investors,” said Smital Shah, Chief Business and Financial Officer at ProQR. “Clinical data for our lead program, sepofarsen, has presented strong potential for this medicine to be a first-in-class and first-to-market medicine for LCA10. We are putting together a commercial strategy for sepofarsen to launch in 2021, subject to regulatory approvals and other factors. While we are building towards that goal, we are focused on execution and financial discipline throughout our operations.”
  3. [verwijderd] 29 januari 2019 15:06
    ProQR R&D day details

    ProQR is holding an R&D day today in New York. The event will be webcast live and is accessible from the Investor Relations section of ProQR’s website (www.proqr.com) under Events and Presentations. An archived recording of the event will be available via webcast for 90 days following the presentation date.
  4. [verwijderd] 8 mei 2019 13:05
    ProQR Announces Financial Results for the First Quarter of 2019

    LEIDEN, Netherlands & CAMBRIDGE, Mass., May 08, 2019 (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, today reported its financial results for the first quarter ended March 31, 2019.
    “We made significant progress in 2019 thus far with the initiation of both the Phase 2/3 trial for sepofarsen and the proof of concept trial for QR-421a in Usher syndrome as we focus on our mission to become a fully-integrated company developing and commercializing RNA medicines for patients with inherited retinal diseases,” said Daniel A. de Boer, CEO of ProQR. “We expect 2019 to be a transformative year for ProQR as we execute on these trials and advance three additional programs towards the clinic in our effort to bring more innovative medicines for inherited retinal diseases to patients.”
    Corporate Highlights and Business Update
    Sepofarsen (formerly QR-110) for LCA10
    • In April 2019, the first patient was dosed in the Phase 2/3 ILLUMINATE trial in patients with sepofarsen in Leber’s Congenital Amaurosis 10 (LCA10) with data expected around the end of 2020. The protocol included the feedback from the U.S. Food and Drug Administration (FDA) as well as the European Medicines Agency (EMA).
    QR-421a for Usher syndrome type 2
    • In March 2019, the first patient was dosed in the Phase 1/2 STELLAR clinical trial for QR-421a in patients with Usher syndrome type 2 or non-syndromic retinitis pigmentosa (RP). Interim data from the study are expected to be announced mid-2019. Earlier, the FDA granted Fast Track designation for QR-421a for Usher syndrome type 2 and non-syndromic RP due to mutations in exon 13 of the USH2A gene.
    QR-313 for dystrophic epidermolysis bullosa
    • In March 2019, ProQR announced the strategic spin out of the Dystrophic Epidermolysis Bullosa (DEB) activities into the newly formed company, Wings Therapeutics. Wings Therapeutics will be led by interim CEO Mark de Souza, PhD, former CEO of Lotus Tissue Repair and Hal Landy, MD, former medical advisor to Lotus Tissue Repair and CMO of Enobia. Wings Therapeutics will focus on developing therapies for Dystrophic Epidermolysis Bullosa and continue to conduct clinical trials with QR-313 in exon 73 as well as progress other RNA molecules that are designed for other mutations that cause DEB. ProQR will have a minority stake in Wings Therapeutics and will be eligible for milestone and royalty payments on sales of commercial products, if any. Financial details were not disclosed.
    Scientific Updates
    • Data on the ProQR portfolio were presented at three scientific conferences in April and May. One abstract was presented at the Retinal Cell & Gene Therapy Innovation Summit, three abstracts were presented at the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) and one abstract was presented at the annual meeting of the American Society for Gene and Cell Therapy (ASGCT).
    Business Updates
    • In April 2019, ProQR nominated Bart Filius, Chief Operating Officer (COO) and Chief Financial Officer (CFO) at Galapagos NV and Theresa Heggie, Senior Vice President, Head of Canada, Europe, Middle East and Africa (CEMEA) at Alnylam Pharmaceuticals to join the Supervisory Board, subject to approval by shareholders at the Annual Meeting of Shareholders. Mr. Filius will also become chair of the Board Audit Committee upon his election to the board.
    Financial highlights
    At March 31, 2019, ProQR held cash and cash equivalents of €94.1 million, compared to €105.6 million at December 31, 2018. Net cash used in operating activities during the three-month period ended March 31, 2019 was €12.4 million, compared to €9.7 million for the same period last year.
    Research and development (R&D) costs increased to €12.0 million for the quarter ended March 31, 2019 from €7.7 million for the same period last year and comprised of allocated employee costs including share-based payments, the costs of materials and laboratory consumables, outsourced activities, license and intellectual property costs and other allocated costs. The increase in R&D expenses was primarily due to initiation of the clinical trials for sepofarsen and QR-421a.
    General and administrative costs increased to €3.2 million for the quarter ended March 31, 2019 from €2.7 million for the same period last year.
    Net loss for the three-month period ended March 31, 2019 was €14.2 million, or €0.36 per diluted share, compared to a €10.7 million loss, or €0.33 per diluted share, for the same period last year. For further financial information for the period ending March 31, 2019, please refer to the financial statements appearing at the end of this release.
  5. [verwijderd] 8 mei 2019 13:05
    About sepofarsen
    Sepofarsen (formerly named 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 (also known as the c.2991+1655A>G 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. Sepofarsen is designed to restore normal (wild-type) CEP290 mRNA, leading to the production of normal CEP290 protein by binding to the mutated location in the pre-mRNA, causing normal splicing of the pre-mRNA. Sepofarsen 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 received fast-track designation from the FDA.
    About Leber’s Congenital Amaurosis
    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 LCA10 is the most frequent and 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 that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA10 because of this mutation.
    About QR-421a
    QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of vision loss in Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional Usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a 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 received fast-track designation from the FDA.
    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. Usher syndrome type 2 is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene. To date, there are no approved treatments or products in clinical development that treat the vision loss associated with Usher syndrome type 2.
    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 Leber’s congenital amaurosis 10 and Usher syndrome type 2. 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 our product candidates, including sepofarsen and QR-421a, and the clinical development and the therapeutic potential thereof, statements regarding our ongoing and planned discovery and development of product candidates and the timing thereof, including our plans for advancing our development programs into the clinic, statements regarding our strategic spinout of Wings Therapeutics, and statements regarding the appointment of new members to our Supervisory Board. 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, 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.
    Investor Contact:
    Hans Vitzthum
    LifeSci Advisors
    T: +1 617-535-7743
    hans@lifesciadvisors.com
    Media Contact:
    Sara Zelkovic
    LifeSci Public Relations
    T: +1 646 876 4933
    Sara@lifescipublicrelations.com

  9. [verwijderd] 7 augustus 2019 13:27
    ir.proqr.com/news-releases/news-relea...

    ProQR Announces Financial Results for the Second Quarter of 2019
    7 August 2019 at 7:00 AM EDT
    LEIDEN, Netherlands and CAMBRIDGE, Mass., Aug. 07, 2019 (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, today reported its financial results for the second quarter ended June 30, 2019.

    “In the first half of 2019, we made tremendous progress towards accomplishing the goals set out in ProQR’s Vision 2023 strategy, which includes starting the Phase 2/3 trial for sepofarsen in LCA10 patients, initiating the first-in-human trial for QR-421a for usher syndrome type 2 and advancing QR-1123 further towards the clinic,” said Daniel A. de Boer, CEO of ProQR. “In line with our strategy, we also announced adding two new targets to our growing pipeline in ophthalmology and we are making progress in other areas, including increasing our efforts to develop our retinoid models and advancing commercial planning.”

    Corporate Highlights and Business Update

    Sepofarsen (formerly QR-110) for LCA10

    The first patient was dosed in the Phase 2/3 ILLUMINATE trial in patients with sepofarsen in Leber’s Congenital Amaurosis 10 (LCA10) at the beginning of the second quarter of 2019. This trial is being conducted globally at 16 sites in seven countries. QR-110 has received Fast Track designation from FDA for the treatment of LCA10.

    In July 2019, the Company was granted access to the PRIority MEdicines (PRIME) program by the European Medicines Agency (EMA) for sepofarsen. The PRIME program is particularly focused on medicines that may provide a therapeutic advantage over existing treatments or that are for indications that currently have no treatment options. To be eligible and accepted for PRIME, a medicine has to demonstrate its potential to benefit patients with unmet medical needs based on early clinical data coupled with non-clinical data. Through PRIME, the EMA offers additional support to medicine developers including early interaction and dialogue. The program is intended to optimize development plans and expedite the review and approval process so that these medicines may reach patients as early as possible. As of June 2019, less than 30% (54 out of 181) of applications to the PRIME program have been granted access, and only 20% (one out five) of ophthalmology applications have been granted access.
    QR-421a for Usher syndrome type 2

    The current ongoing trial (Phase 1/2 STELLAR) for QR-421a in patients with syndromic (Usher syndrome type 2) and non-syndromic retinitis pigmentosa (RP) is being conducted at seven sites across North America and Europe. Interim data from the first two planned cohorts is expected in Q1 2020.

    The FDA has granted Fast Track designation for QR-421a for Usher syndrome type 2 and non-syndromic RP due to mutations in exon 13 of the USH2A gene.
    QR-1123 for autosomal dominant retinitis pigmentosa (adRP)

    During the second quarter, the Company held a meeting with the FDA regarding the clinical development of QR-1123 for autosomal dominant RP (adRP) due to the P23H mutation, based on which the Phase 1/2 proof-of-concept clinical trial is expected to start in 2019. The Company in-licensed this program from Ionis Pharmaceuticals in October 2018.
    Scientific Updates

    During the second quarter of 2019, the Company presented new data on the ProQR portfolio at three scientific conferences, including the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) and the annual meeting of the American Society for Gene and Cell Therapy (ASGCT). Subsequently, the Company presented at the 2019 annual Usher connections conference in July 2019.
    Business Updates

    In May 2019, shareholders approved the appointments of Bart Filius and Theresa Heggie to the Supervisory Board at the 2019 Annual Meeting of Shareholders, with the appointments effective July 1, 2019. Mr. Filius was also appointed chair of the Board Audit Committee following his election to the board. Mr. Filius currently serves as the Chief Operating Officer (COO) and Chief Financial Officer (CFO) at Galapagos NV. Ms. Heggie currently serves as the Senior Vice President, Head of Canada, Europe, Middle East and Africa (CEMEA) at Alnylam Pharmaceuticals.
    Financial highlights

    At June 30, 2019, ProQR held cash and cash equivalents of € 82.5 million, compared to €105.6 million at December 31, 2018. Net cash used in operating activities during the three-month period ended June 30, 2019 was €10.9 million, compared to €5.4 million for the same period last year.

    Research and development costs totaled €9.5 million for the quarter ended June 30, 2019 compared to €6.0 million for the same period last year and are comprised of allocated employee costs, including share-based payments, the costs of materials and laboratory consumables, outsourced activities, license and intellectual property costs and other allocated costs. The increase in R&D expenses was primarily due to initiation of the clinical trials for sepofarsen and QR421a in 2019 and completion of the clinical trial for eluforsen in 2018.

    General and administrative costs increased to €2.9 million for the quarter ended June 30, 2019 compared to €2.6 million for the quarter ended June 30, 2018.

    Net loss for the three-month period ended June 30, 2019 was €11.7 million or €0.30 per share, compared to a €7.4 million loss or €0.23 per share for the same period last year. For further financial information for the period ended June 30, 2019, please refer to the financial statements appearing at the end of this release.
  10. [verwijderd] 12 augustus 2019 13:04
    ir.proqr.com/news-releases/news-relea...

    ProQR Announces Clearance of IND to Start Clinical Trial of QR-1123 in Patients with Autosomal Dominant Retinitis Pigmentosa (adRP)
    12 August 2019 at 7:00 AM EDT
    LEIDEN, Netherlands and CAMBRIDGE, Mass., Aug. 12, 2019 (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, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for QR-1123. ProQR plans to start enrolling patients in a Phase 1/2 trial for QR-1123 in 2019.

    QR-1123 is a first-in-class investigational oligonucleotide designed to address the underlying cause of the vision loss associated with autosomal dominant retinitis pigmentosa (adRP) due to the P23H mutation in the rhodopsin (RHO) gene.

    P23H is the most prevalent mutation associated with adRP in the U.S. This disease causes progressive vision loss in approximately 2,500 patients in the United States, leading to blindness in mid-adulthood. There are no approved therapies for adRP and QR-1123 is the first investigational medicine to be developed for patients that suffer from this disease.

    “We are pleased to have an open IND for QR-1123, based on which we will be advancing our next inherited retinal disease program into the clinic this year,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “This represents our fifth IND in less than five years and our third clinical program for severe genetic eye diseases. With a strong in vitro and in vivo proof-of-concept, we are excited about the potential of this medicine to make a positive impact on the lives of patients with adRP.”

    About QR-1123

    QR-1123 is a first-in-class investigational antisense oligonucleotide that was discovered and developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology for the treatment of adRP due to the P23H mutation in the RHO gene. The therapy aims to inhibit the formation of the mutated toxic version of the rhodopsin protein by specifically binding the mutated RHO mRNA. Binding of QR-1123 causes allele specific knockdown of the mutant mRNA by a mechanism called RNase H mediated cleavage without affecting the normal RHO mRNA. QR-1123 is intended to be administered through intravitreal injections in the eye. QR-1123 was in-licensed from Ionis Pharmaceuticals in 2018.

    About the Phase 1/2 trial for adRP

    PQ-1123-001, is a first-in-human study that will initially include up to 12 adults with adRP due to the P23H mutation in in the rhodopsin (RHO) gene. The trial will include a single-dose escalation (open label) arm and a multiple-dose (double-masked) arm in which a single intravitreal injection of QR-1123 or sham procedure will be given in one eye. The objectives of the trial will include evaluation of safety, tolerability, pharmacokinetics and efficacy, as measured by restoration or improvement of visual function and retinal structure through ophthalmic endpoints such as visual acuity (BCVA), visual field and optical coherence tomography (OCT). Changes in quality of life as reported by trial subjects in patient reported outcomes, or PROs, will also be evaluated. Patients completing this trial will be able to participate in an extension study if eligible. The trial is designed to be conducted at expert sites in North America and is expected to start in 2019.

    About adRP

    Autosomal dominant retinitis pigmentosa, or adRP, is a severe and rare genetic disease that causes progressive reduction in night and peripheral vision during childhood and frequently leads to blindness in mid adulthood. In the United States, the most prevalent mutation associated with adRP is the P23H point mutation (also known as the c.68C>A mutation) in the rhodopsin (RHO) gene and affects approximately 2,500 people. This gain of function mutation causes misfolding of the rhodopsin protein that becomes toxic to the photoreceptor cells in the retina. Over time the cells die and vision is progressively lost. There are currently no therapies approved or in clinical development for P23H adRP. A natural history study in patients with P23H adRP has been conducted.
  11. [verwijderd] 9 september 2019 13:04
    ProQR Receives Fast Track Designation from FDA for QR-1123 for Autosomal Dominant Retinitis Pigmentosa

    LEIDEN, Netherlands and CAMBRIDGE, Mass., Sept. 09, 2019 (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, today announced that it received Fast Track designation from the Food and Drug Administration (FDA) for QR-1123.
    QR-1123 is a first-in-class investigational antisense oligonucleotide designed to address the underlying cause of vision loss associated with autosomal dominant retinitis pigmentosa (adRP) due to the P23H mutation in the rhodopsin (RHO) gene.

    Fast Track designation is granted by the FDA to drugs in development for serious conditions with the potential to fulfill an unmet medical need. It was established with the intention to bring promising drugs to patients sooner by facilitating development with more frequent FDA interactions and expediting the review process.

    “We are very pleased to receive Fast Track designation by the FDA for QR-1123. There are no current treatment options available for patients to improve vision or prevent vision loss due to adRP. Further, this designation emphasizes the high unmet need in this disease,” said Daniel de Boer, Chief Executive Officer of ProQR. “We look forward to beginning enrollment in the Phase 1/2 (Aurora) clinical trial for QR-1123 in the coming months.”

    About QR-1123

    QR-1123 is a first-in-class investigational antisense oligonucleotide that was discovered and developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology for the treatment of adRP due to the P23H mutation in the RHO gene. The therapy aims to inhibit the formation of the mutated toxic version of the rhodopsin protein by specifically binding the mutated RHO mRNA. Binding of QR-1123 causes allele specific knockdown of the mutant mRNA by a mechanism called RNase H mediated cleavage without affecting the normal RHO mRNA. QR-1123 is intended to be administered through intravitreal injections in the eye. QR-1123 was in-licensed from Ionis Pharmaceuticals in 2018, and subsequently received IND clearance in August 2019.

    About the Phase 1/2 Aurora trial

    Aurora, or PQ-1123-001 trial, is a first-in-human study that will initially include up to 35 adults with adRP due to the P23H mutation in the rhodopsin (RHO) gene. The trial will include a single-dose escalation (open label) arm and a multiple-dose (double-masked) arm in which intravitreal injections of QR-1123 or sham procedure will be given in one eye. The objectives of the trial will include evaluation of safety, tolerability, pharmacokinetics and efficacy, as measured by restoration or improvement of visual function and retinal structure through ophthalmic endpoints such as visual acuity (BCVA), visual field (VF) and optical coherence tomography (OCT). Changes in quality of life (patient reported outcomes), or PROs, will also be evaluated. The trial will be conducted at expert sites in North America and is expected to start in 2019.

    About adRP

    Autosomal dominant retinitis pigmentosa, or adRP, is a severe and rare genetic disease that causes progressive reduction in night vision during childhood, leading to visual field loss and frequently ending in blindness in mid adulthood. In the United States, the most prevalent mutation associated with adRP is the P23H point mutation (also known as the c.68C>A mutation) in the rhodopsin (RHO) gene and affects approximately 2,500 people. This gain of function mutation causes misfolding of the rhodopsin protein that becomes toxic to the photoreceptor cells in the retina. Over time the cells die and vision is progressively lost. There are currently no therapies approved or in clinical development for P23H adRP. A natural history study in patients with P23H adRP has been conducted.

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