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QR-313 for Dystrophic Epidermolysis Bullosa

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  1. PlayBall10 29 november 2017 13:25

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    ProQR Receives Orphan Drug Designation from EMA for Drug Candidate QR-313 for Dystrophic Epidermolysis Bullosa

    Key Updates

    •ProQR’s drug candidate, QR-313 for dystrophic epidermolysis bullosa (DEB) receives orphan drug designation in the EU from the EMA.
    •QR-313 also received U.S. ODD from the FDA in September 2017.
    •QR-313 represents the fifth candidate in the company’s pipeline to receive ODD in the U.S. and EU.
    •DEB is a severe genetic skin disease with no disease modifying treatments currently available.
    •QR-313 targets the most common mutations within DEB, which are mutations in exon 73 of the COL7A1 gene and is designed for topical administration.
    •QR-313 is expected to enter the clinic in 2018, with interim data also expected in 2018.

    LEIDEN, the Netherlands, Nov. 29, 2017 (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 including cystic fibrosis, Leber's congenital amaurosis 10 and dystrophic epidermolysis bullosa, today announced that investigational drug QR-313 for dystrophic epidermolysis bullosa (DEB) has received orphan drug designation (ODD) from the European Medicines Agency (EMA). QR-313 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause in dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. DEB is a rare genetic disease that can lead to severe blistering of the skin resulting in high treatment burden and poor quality of life for patients.

    In September 2017, QR-313 also received ODD from the FDA. This marks the fifth drug candidate in the company’s pipeline to receive ODD from the FDA and EMA. A first-in-human clinical trial of QR-313 is expected to be initiated in 2018, with interim data readout also expected in 2018.

    “We are pleased to have orphan drug designation for our QR-313 program targeting dystrophic epidermolysis bullosa in both the U.S. and Europe,” said David M. Rodman, MD, Chief Development Strategy Officer of ProQR. “This represents another milestone for our company and highlights the unmet need for patients with this devastating disease. Our goal is to develop and actively advance a pipeline of programs that can treat DEB mutations in a targeted manner.”

    About EMA Orphan Drug Designation (ODD)

    In Europe, to qualify for orphan drug designation, a medicine must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating, when the prevalence in the EU is not more than 5 in 10,000 (or it is unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development), and when no satisfactory method of diagnosis, prevention or treatment of the condition exist, or, if such method exists, the medicine will be of significant benefit to those affected by the condition. As incentives to encourage the development of orphan medicines, the EU offers protocol assistance specific for designated orphan medicines, 10 years of market exclusivity once the medicine is on the market, and fee reductions.

    About QR-313

    QR-313 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in this exon can cause loss of functional collagen type VII (C7) protein. Absence of C7 results in the loss of anchoring fibrils that normally link the dermal and epidermal layers of the skin together. QR-313 is designed to exclude exon 73 from the mRNA (exon skipping) and produce a functional C7 protein, thereby restoring functionality of the anchoring fibrils. www.ir.proqr.com/phoenix.zhtml?c=2537...

  3. [verwijderd] 29 november 2017 17:39

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    idd...maar zolang de emissie niet afgerond is blijft de koers speelbal van speculanten/hedgefunds en al wat dies meer zij...money rules...medische vooruitgang is mooi meegenomen is wellicht het adagio.

    Om fundamentele redenen lijkt PRQR genoeg te bieden om te blijven volgen. De ontwikkelingen hebben een positieve ondertoon (zachtjes uitgedrukt)
  4. forum rang 4 harvester 1 december 2017 23:19

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    quote:

    ivet schreef op 29 november 2017 17:39:

    idd...maar zolang de emissie niet afgerond is blijft de koers speelbal van speculanten/hedgefunds en al wat dies meer zij...money rules...medische vooruitgang is mooi meegenomen is wellicht het adagio.

    Om fundamentele redenen lijkt PRQR genoeg te bieden om te blijven volgen. De ontwikkelingen hebben een positieve ondertoon (zachtjes uitgedrukt)
    De emissie was toch underwritten en dus bij voorbaat succesvol?
  5. forum rang 6 Tom3 16 juni 2018 16:02

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    Volgens de EB Research Partnership zijn er alleen al in de VS 30.000 EB patiënten. Worldwide kom je dan al snel op 300.000 uit. De ziekte beperkt zich niet tot een bepaald ras en/of geslacht:

    www.ebresearch.org/

    Ook organen kunnen aangetast worden, het gaat niet alleen om de huid.
  8. [verwijderd] 18 juni 2018 13:30

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    ik vermoed dat de kosten tussen gentherapie en RNAtechnologie ook zeer uiteen kunnen lopen in voordeel van ProQR. Op lange termijn zal genthearpie steeds meer goede resultaten kunnen boeken, maar er is nog een kostbare lange weg te gaan denk ik.
  9. forum rang 6 Tom3 18 juni 2018 14:48

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    quote:

    ivet schreef op 18 juni 2018 13:30:

    ik vermoed dat de kosten tussen gentherapie en RNAtechnologie ook zeer uiteen kunnen lopen in voordeel van ProQR. Op lange termijn zal genthearpie steeds meer goede resultaten kunnen boeken, maar er is nog een kostbare lange weg te gaan denk ik.
    Toen Abeona in 2016 haar eerste studieresultaten bekend maakte (en ik kan geen vervolgdata ontwaren op hun site) ging haar beurskoers nagenoeg naar het nulpunt. Dat die koers is opgeveerd is kennelijk te danken aan andere indicaties.
  12. [verwijderd] 6 september 2018 08:47

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    resultaten van deze trial zouden eind 2018 moeten volgen. Indien positief, dan niet alleen mooi als er zicht is op de eindstreep, maar ook een soort van risicospreiding.

    QR-313 Clinical trial: WINGS
    Phase 1/2 "Safety and efficacy study"
    WINGS is an ongoing international Phase 1/2, double-blind, randomized, intra-subject placebo controlled clinical trial of QR-313 in patients with recessive dystrophic epidermolysis bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene. QR-313 is an investigational drug that is designed to improve the healing and integrity of skin in DEB patients.

    Up to 8 patients will be included in WINGS (over 6 years of age) that have RDEB due to mutation(s) in exon 73 of the COL7A1 gene. During the study, QR-313 or placebo formulated in a gel, will be topically applied to a patient’s wounds two to three times a week (depending on bandage change frequency) for up to four weeks with an eight week follow-up period. WINGS is planned to be conducted at specialized centers in the US and selected European countries.

    Primary objectives of WINGS are to test whether QR-313 is safe and tolerated and to evaluate proof of mechanism by testing, at a molecular level, whether QR-313 is able to exclude the mutation from the RNA (skipping of exon 73 from COL7A1 mRNA assessed by polymerase chain reaction, or PCR). Secondary objectives are to test the effects on wound healing, skin strength, the presence of collagen type VII protein and anchoring fibrils in the skin as well as systemic distribution of QR-313 after topical administration.

    We expect the first interim results from this study to be available during 2018.

    What is a Phase 1/2 trial?
    A trial may include more than one phase. A phase 1/2 clinical trial tests the safety of an investigational drug, as well as assesses signals of efficacy.

    Combining phases 1 and 2 may allow research questions to be answered more quickly or with fewer patients.

    www.proqr.com/qr-313-wings-phase-12-s...

  13. [verwijderd] 6 september 2018 08:48

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    en ook:

    ProQR Initiates Phase 1/2 Clinical Trial of QR-313 for Dystrophic Epidermolysis Bullosa
    28 June 2018 at 7:00 AM EDT
    The trial, called WINGS, will evaluate the safety and efficacy of QR-313 in subjects with recessive dystrophic epidermolysis bullosa due to mutations in exon 73 of the COL7A1 gene.

    QR-313 is a first-in-class potential therapeutic RNA molecule designed to improve the healing and integrity of skin in DEB patients

    Interim results are expected in late 2018; full data are expected in 2019

    LEIDEN, the Netherlands, June 28, 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 a Phase 1/2 clinical trial called “WINGS”, to evaluate the safety and efficacy of QR-313 in patients with recessive dystrophic epidermolysis bullosa (RDEB) is open for enrollment.

    “The initiation of our first human clinical trial for QR-313 is an exciting next step in the development of this novel investigational therapy for DEB. WINGS is designed to initially provide molecular proof of mechanism and subsequently clinical proof of concept for QR-313,” said David M. Rodman, MD, Executive Vice President of Research and Development at ProQR. “Now that the study is initiated we expect to dose the first set of adult and pediatric patients over the next few months and provide interim proof of mechanism results late this year.”

    Dystrophic epidermolysis bullosa is a severe blistering disease that causes fragile skin. People with DEB live with constant pain and have a high risk of malnutrition and infections. Symptoms of the disease include poorly healing wounds, skin infections, fusion of fingers and toes, anemia and gastrointestinal tract problems. Some patients develop very aggressive forms of skin cancer in adulthood. There are currently no approved treatment options available that target the underlying cause of DEB.

    David M. Rodman, MD continued: “The WINGS study marks the second of three clinical trials that we are conducting at ProQR this year. Beyond WINGS for DEB we have an ongoing QR-110 study in LCA 10 patients and plan to start a clinical trial of QR-421a in patients that suffer from Usher syndrome type 2.”

    About the WINGS Trial

    WINGS is a first-in-human Phase 1/2 double-blind, randomized, intra-subject placebo controlled clinical trial of QR-313 in approximately eight subjects (at least six years of age) that have RDEB due to mutation(s) in exon 73 of the COL7A1 gene.

    The primary objectives for the study are to evaluate the safety and tolerability of topically applied QR-313 and assess proof of mechanism (exclusion, or skipping, of exon 73 from COL7A1 mRNA assessed by polymerase chain reaction). The secondary objectives are to quantify blood levels of QR-313, assess effects on wound healing, skin strength, the presence of collagen type 7 protein and anchoring fibrils in the skin.

    During the study QR-313 or placebo formulated in a gel will be topically applied to a patient’s wounds approximately every-other-day for up to four weeks with a subsequent eight week observation period. Up to four small skin biopsies will be performed and tissue analyzed for molecular endpoints. The trial will be conducted at specialized centers in the U.S. and selected European countries.

    Interim proof of mechanism results from the trial are expected in 2018. Full clinical proof of concept results from the study are expected in 2019. Depending on results of the interim analysis, the trial may be adapted to adjust the frequency and/or method of topical delivery to further enhance uptake and activity. It is planned that eight subjects will receive either active gel or placebo on two separate wounds. Clinical proof of concept will compare the rate, strength and stability of wounds treated with active gel to those treated with placebo.

    ir.proqr.com/news-releases/news-relea...
  16. forum rang 6 Tom3 9 september 2018 09:01

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    Spijt dat je een deeltje verkocht hebt? Ik voorspel een uitslaande brand aanstaande maandag ten gevolge van de geslaagde emissie. Mensen krijgen door dat De Boer c.s. bezig zijn met iets heel groots in wording. Nu blijkt dat hij geen bluffer is, alle ronkende taal in diverse geschriften over bijvoorbeeld Leber waren in feite understatements, stijgt deze club met stip. Genoemde hint op Twitter over DEB is weer een signaal dat de 2e therapie (in aanbouw) ook een winner is. In het abc stukje staat geschreven dat de ouders van Lizzy naar de DEBRA conferentie gaan. ProQR is daar natuurlijk ook. Tel uit je winst.
  17. [verwijderd] 9 september 2018 10:05

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    soms is iets hoger inkopen bij positieve bevestiging veiliger...potentieel blijft er genoeg over. Het is voortschrijdend inzicht.
    Met de kennis van nu had je maximale krediet opgenomen t.t.v. de koers van 3$ om in ProQR te steken. Ik heb voor mijn doen stevig ingezet en zoveel was dat niet meer t.o.v. wat ik in mijn verleden had.

    Evengoed blij dat het stevig de goede kant uit gaat!
  19. forum rang 6 Tom3 22 september 2018 11:07

    • Tom3

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    Faison heeft recent een ronkend artikel geschreven over Krystal. De beurskoers is in september sterk gedaald. Ik heb zo'n vermoeden dat ProQR daar de oorzaak van is. Faison rept er met geen woord over en spreekt over " buy the dip". Het wordt in ieder geval super spannend. Wel leuk is dat de DEB markt 2 keer zo groot is dan Proqr op haar site vermeldt.

    seekingalpha.com/article/4207507-krys...
  20. forum rang 6 Tom3 22 september 2018 11:19

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    Mij ervaringen met Molmed hebben mij geleerd dat het werken met het herpes simplex virus, zoals Krystal doet, nogal kostbaar en tijdrovend is. Is het te transporteren gen niet te groot voor deze drager? Enfin zowel Proqr als Krystal komen in Q4 met fase 1/2 trial dat op de proppen.
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