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Nieuws media financieel ProQR Therapeutics NV

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Frenky_Tornado
0
quote:

ivet schreef op 10 dec 2018 om 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 :)
ivet
1

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.”
ivet
0
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.
Frenky_Tornado
1
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.
Frenky_Tornado
1
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

Frenky_Tornado
1
Helaas lukt het me niet de balans in een fatsoenlijke tabelvorm hier in te plakken. Ik heb een poging gewaagd om er een pdf van te maken.
Bijlage:
nmgn
0
Geen verrassingen. 2019 wordt een belangrijk jaar, maar dat hadden wij zelf ook al wel door :-)
Tom3
0
De kas is nog rijkelijk gevuld en De Boer is geen big spender. Op naar de AVA en de Usher read out!
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