Forum: ProQR Therapeutics » QRX-411 for Usher Syndrome | Belegger.nl

QRX-411 for Usher Syndrome

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PlayBall10
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ProQR's Drug Candidate QRX-411 for Usher Syndrome Receives Orphan Drug Designation from FDA and EMA
(GLOBE NEWSWIRE via COMTEX) --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.
ir.proqr-tx.com/phoenix.zhtml?c=25370...
TheAdmiral
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Frenky_Tornado
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quote:

PlayBall10 schreef op 5 jul 2017 om 13:33:



- A lead candidate has been selected for this program and is currently ready for IND-enabling studies.



Met name dit stukje in het bericht biedt hoop voor de patienten en beleggers. Ready to go! :)
ivet
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ProQR’s Drug Candidate QRX-421 for Usher Syndrome Receives Orphan Drug Designation from FDA and EMA

GlobeNewswire•September 5, 2017

Key Updates

ProQR’s drug candidate QRX-421 for Usher syndrome receives orphan drug designation from the FDA and EMA, representing the third candidate in the company’s ophthalmology pipeline and the fourth in the broader pipeline to receive ODD in the U.S. and EU.
There are currently no therapies commercially available or in clinical development for the vision loss associated with Usher syndrome type 2.
QR-421 is part of the company’s growing ophthalmology pipeline which also includes lead candidate, QR-110 for Leber’s congenital amaurosis 10 currently in clinical trials, and three additional pipeline programs, QRX-411 that addresses another genetic mutation resulting in Usher syndrome, QRX-1011 for Stargardt’s disease, and QRX-504 for Fuchs endothelial corneal dystrophy.
Promising QRX-421 pre-clinical data in both patient fibroblasts and the optic cup model for mRNA restoration were presented at the May 2017 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Baltimore, MD.
In July 2017, QRX-411, ProQR’s second candidate for Usher syndrome received ODD from the FDA and EMA.
LEIDEN, the Netherlands, Sept. 05, 2017 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (PRQR) today announced that investigational drug QRX-421 for Usher syndrome has received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA). This marks the third drug candidate in the company’s ophthalmology pipeline and the fourth drug in the broader pipeline to receive ODD from the FDA and EMA. QR-421 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to mutations in exon 13 of the USH2A gene. Usher syndrome is the leading cause of combined deafness and blindness.

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 to have ODD designation for both our programs targeting Usher syndrome in the U.S. and EU, representing yet another important milestone for our company and highlighting the unmet need for patients in this disease,” said David M. Rodman, MD, Chief Development Strategy Officer of ProQR. “At ProQR, we are focused on designing accelerated development strategies that capitalize on our oligonucleotide approach to potentially bring our novel medicines to patients quicker and receiving ODD designations for these is an important step towards this goal.”

ProQR’s ophthalmology pipeline includes the following:

QR-110 for Leber’s congenital amaurosis 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 ODD designation by the FDA and EMA.
QRX-421 for Usher syndrome type 2 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-411 for Usher syndrome type 2 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-411 also received ODD designation by the FDA and EMA.
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 QRX-421

QRX-421 is a first-in-class investigational 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 Usher syndrome type 2. Usher syndrome type 2 is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene.
ivet
0
ProQR's QRX-421 Gets Orphan Drug Designation from FDA and EMA

Zacks
Zacks Equity Research
ZacksSeptember 7, 2017Comment
ProQR Therapeutics N.V. PRQR announced that its pipeline candidate, QRX-421, has received orphan drug designation (ODD) from the FDA and European Medicines Agency (“EMA”) for Usher syndrome. In fact, QRX-421 is the third candidate in the company’s ophthalmology pipeline and the fourth drug in the broader pipeline to receive ODD from the FDA and EMA.
Currently, QRX-421 is being evaluated for the treatment of Usher syndrome due to mutations in exon 13 of the USH2A gene.

ProQR’s shares have outperformed the industry year to date. The stock has increased 10.2% compared with the industry’s gain of 9.9% in the same time frame.


We note that the FDA, through its Office of Orphan Products Development, usually grants ODD to those drugs and biologics that are being developed for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders affecting less than 200,000 people in the United States.

Benefits and incentives include a period of marketing exclusivity of seven years in the United States and ten years in the European Union upon regulatory approval for the designated indication.

Notably, ProQR has a growing ophthalmology pipeline and also includes QR-110 that is being developed for the treatment of Leber’s congenital amaurosis 10 in clinical studies (phase I/II).

Additionally, the pipeline includes three more candidates, QRX-411, which is being developed to treat another genetic mutation resulting in Usher syndrome. Markedly, QRX-411 received ODD from the FDA and EMA in July. The other two candidates are QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.

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finance.yahoo.com/news/proqr-apos-qrx...
Tom3
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In februari van dit jaar heeft Labiotech.eu een informatief redactioneel stuk gewijd aan het Usher therapie van ProQR naar aanleiding van de verkregen grant uit de VS:

labiotech.eu/blindness-deafness-usher...
Tom3
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Dit stuk mag ook niet ontbreken op het Usher draadje:

www.ushersyndroom.nl/samenwerking-rad...

Ook hier is het Radboud MC betrokken bij de ontwikkeling, de samenwerking is nu 2 jaar geleden gestart.
Tom3
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Goed ook om te lezen dat Usher 2a zich niet goed leent voor dna therapie omdat het gen in kwestie te groot is om door een virus op zijn plek te worden gebracht. Bij LCA bestaat die optie kennelijk wel. Spannend om te volgen wat de beste methode zal zijn.
Tom3
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Ik werd door een Amerikaan erop attent gemaakt dat Editas naast Leber ook Usher 2a in het vizier heeft. Dit moeten we natuurlijk secuur in de gaten houden:

ir.editasmedicine.com/phoenix.zhtml?c...

Frenky_Tornado
1
ProQR Announces Clearance of IND to Start Clinical Trial of QR-421a in Usher Syndrome Type 2 Patients


LEIDEN, Netherlands & CAMBRIDGE, Mass., Dec. 04, 2018 (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-421a. QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of the vision loss associated with Usher syndrome type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
Usher syndrome is the leading cause of combined deafness and blindness. Exon 13 mutations in the USH2A gene targeted by QR-421a cause vision loss in approximately 16,000 individuals in the Western world. ProQR plans to start enrolling patients in a Phase 1/2 trial named STELLAR in the coming months with preliminary data expected in mid-2019.
“We are pleased to be advancing QR-421a, our second therapy for an inherited retinal disease, into the clinic and continuing the development of our portfolio of transformative RNA medicines for severe genetic rare diseases,” said Daniel A. de Boer, Chief Executive Officer of ProQR. “QR-421a has shown promising activity in both the optic cup and zebra fish models and we are excited about the potential to make a meaningful impact for Usher syndrome patients.”
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. Mutations in this exon can cause loss of functional usherin protein that causes the disease. QR-421a is designed to exclude the genetic defect from the RNA in the eye, such that it leads to the expression of a shortened but functional usherin protein, thereby modifying the underlying disease. QR-421a has received orphan drug designation in the United States and the European Union.
About the Phase 1/2 “STELLAR” trial
STELLAR, or PQ-421a-001, is a first-in-human study that will initially include approximately 18 adults who have vision loss due to mutations in exon 13 of the USH2A gene and will be conducted at expert sites in the US and Europe. It will be a double-masked, randomized study exploring several dose levels and a control (sham injection), given as a single intravitreal injection of QR-421a into one eye. The first patient at each dose level will be dosed in an open-label manner. 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 in the trial subjects will also be evaluated
Data from the first-in-human study are expected in mid-2019. Patients completing this trial will be able to participate in an extension study if eligible. Results from the single dose trial will inform the next stage that will potentially include a seamless adaptive, multi-dose, controlled trial.
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, dystrophic epidermolysis bullosa and cystic fibrosis. 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 QR-421a and its clinical development and therapeutic potential, including commencement of the STELLAR trial, trial design and timing of results from this trial. 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, regulatory review or approval process, 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:
Lisa Hayes
Vice President of Investor Relations and Corporate Communications
T: +1 202 360 4855
lhayes@proqr.com
Media Contact:
Sara Zelkovic
LifeSci Public Relations
T: +1 646 876 4933
Sara@lifescipublicrelations.com
Tom3
0
De hamvraag nu is natuurlijk waarom ziet men na 3 maanden behandeling geen of weinig verandering in het gezichtsvermogen? Heeft Van Wijk iets over het hoofd gezien bij zijn speurtocht?
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