uniQure Terug naar discussie overzicht

Wat is de waarde van uniQure?

1. Izette 29 april 2019 15:41

   auteur info

   • Izette

     Lid sinds: 30 nov 2011

     Laatste bezoek: 08 feb 2025

   • Aanbevelingen

     Ontvangen: 47

     Gegeven: 7

   •    Aantal posts: 274

   
   AMSTERDAM (AP) _ UniQure NV (QURE) on Monday reported a loss of $27.8 million in its first quarter.
   
   The Amsterdam-based company said it had a loss of 74 cents per share.
   
   The results did not meet Wall Street expectations. The average estimate of six analysts surveyed by Zacks Investment Research was for a loss of 66 cents per share.
   
   The human gene therapy company posted revenue of $1.1 million in the period, also missing Street forecasts. Five analysts surveyed by Zacks expected $2 million.
   
   UniQure shares have risen 99% since the beginning of the year. The stock has risen 92% in the last 12 months.
   
2. Izette 29 april 2019 15:42

   auteur info

   • Izette

     Lid sinds: 30 nov 2011

     Laatste bezoek: 08 feb 2025

   • Aanbevelingen

     Ontvangen: 47

     Gegeven: 7

   •    Aantal posts: 274

   Presented Updated Clinical Data from Phase IIb Study of AMT-061 in Patients with Hemophilia B Demonstrating Increases in FIX Activity Sustained at up to 51% of Normal at 12 Weeks
   
   ~ Achieved IND Clearance and Fast Track Designation for AMT-130 in Huntington’s disease
   
   ~ Announced 6 Presentations at Upcoming ASGCT Meeting, Including Preclinical Data on Research Pipeline
   
   LEXINGTON, Mass. and AMSTERDAM, The Netherlands, April 29, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the first quarter of 2019 and highlighted recent progress across its business.
   
   "During the first quarter of 2019, we continued to make excellent progress across our portfolio of gene therapy product candidates,” stated Matt Kapusta, chief executive of uniQure. “We remain highly encouraged by the continued follow-up from our Phase IIb study of AMT-061, which shows the potential to normalize FIX activity in patients with hemophilia B. We are now focused on advancing our ongoing Phase III HOPE-B pivotal study of AMT-061 and have made significant progress activating sites and enrolling patients, with a goal of completing patient enrollment by the end of this year. We are also pleased to announce that AMT-061 received Orphan Drug Designation by the U.S. Food and Drug Administration, which, combined with our FIX-Padua intellectual property position and AAV5’s potentially favorable immunogenicity profile, may provide a meaningful first-mover advantage.”
   
   “With the clearance of our Investigational New Drug application for AMT-130 earlier this year, we are making headway in the preparations for our Phase I/II study of AMT-130 in Huntington’s disease. AMT-130, which recently received Fast Track Designation, is the world’s first one-time administered therapy for Huntington’s disease to enter clinical testing, and we continue to expect patient dosing in this landmark study to begin in the second half of the year.”
   
   Recent Company Progress
   
   — Advancing late-stage development of AMT-061 for the treatment of hemophilia B
   •Enrollment of patients in the global HOPE-B pivotal trial in hemophilia B is advancing and the Company currently is on track to complete enrollment by the end of 2019. In February, the Company presented updated clinical data on AMT-061 demonstrating sustained increases in Factor IX activity (FIX) up to 51% of normal and mean FIX activity for the three patients of 38% of normal at 12 weeks. None of the patients received Factor infusions, reported bleeding events or required immunosuppression over a combined 42 weeks of observation.
   
   •On April 17, 2019 the U.S. Food and Drug Administration (FDA) granted AMT-061 Orphan Drug Designation (ODD). ODD in the U.S. provides special status for investigational drugs being developed for rare diseases considered to affect only up to 200,000 people in the U.S. The ODD program offers product market exclusivity for up to seven years in the U.S. following regulatory approval, along with tax and financial incentives for companies developing medicines for such orphan indications.
   
   — Advancing AMT-130 into clinical development for the treatment of Huntington’s disease
   •In January 2019, the FDA declared effective the Company’s Investigational New Drug application (IND) for AMT-130. The Company expects to begin dosing patients in the second half of this year in its dose-escalating, randomized and controlled Phase I/II clinical study to assess the safety, tolerability and efficacy of a one-time treatment of AMT-130 in patients with Huntington’s disease, and is planning to announce initial safety data on the surgical procedure before the end of this year.
   
   •Earlier this month, the Company announced that the FDA has granted Fast Track designation for AMT-130. The Fast Track program is designed to facilitate the development of and expedite the review of therapies to treat serious conditions and fill an unmet medical need. A therapy granted Fast Track Designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if relevant criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a Biologics License Application (BLA) or New Drug Application (NDA).
   
   — Advancing robust pipeline of novel gene therapy research programs
   •The Company announced the acceptance of six data presentations, including multiple oral presentations featuring preclinical data for its gene therapy candidates in hemophilia A, Fabry disease and Huntington’s disease at the upcoming Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) on April 29- May 2, 2019.
   
   Upcoming Events
   
   •Present preclinical data on the Company’s pipeline and technology platform at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting, April 29- May 2, 2019.
   
   •Present preclinical data on AMT-150 for the treatment of Spinocerebellar Ataxia Type 3 (SCA3) at the 2019 American Academy of Neurology (AAN) Annual Meeting May 4-10, 2019.
   
   •Present 26 weeks of follow-up data on the three patients in the Phase IIb dose-confirmation study of AMT-061 in hemophilia B patients at the Hemostasis & Thrombosis Research Society (HTRS) 2019 Symposium on May 10, 2019.
   
   •Present preclinical data on AMT-130 for the treatment of Huntington’s disease at the 2019 Associazione Italiana Corea Di Huntington Roma Onlus Annual Meeting on May 18, 2019.
   
3. Izette 29 april 2019 15:43

   auteur info

   • Izette

     Lid sinds: 30 nov 2011

     Laatste bezoek: 08 feb 2025

   • Aanbevelingen

     Ontvangen: 47

     Gegeven: 7

   •    Aantal posts: 274

   Financial Highlights
   
   Cash Position: As of March 31, 2019, the Company held cash and cash equivalents of $208.8 million, compared to $234.9 million as of December 31, 2018. The Company currently expects cash and cash equivalents will be sufficient to fund operations into 2021.
   
   Revenues: Revenue for the three months ended March 31, 2019 was $1.1 million, compared to $3.5 million during the same period 2018. The decrease reflects the termination of activities associated with S100A1 in our collaboration with Bristol-Meyers Squibb in October 2018.
   
   R&D Expenses: Research and development expenses were $20.5 million for the three months ended March 31, 2019, compared to $17.1 million during the same period 2018. The change was primarily related to increased activities associated with our ongoing Phase III pivotal study of AMT-061 and planned Phase I/II study of AMT-130, increased share-based compensation and the hiring of additional clinical and operations staff at our Lexington site.
   
   SG&A Expenses: Selling, general and administrative expenses were $8.1 million for three months ended March 31, 2019, compared to $6.3 million during the same period 2018. The change was primarily related to increases in personnel and consulting expenses, professional fees and share-based compensation expenses.
   
   Net Loss: The net loss for the three months ended March 31, 2019 was $27.8 million, or $0.74 per share, compared to $18.8 million, or $0.59 per share during the same period 2018.
   
4. Prof. Dollar 30 april 2019 09:25

   auteur info

   • Prof. Dollar

     Lid sinds: 04 mrt 2011

     Laatste bezoek: 09 feb 2025

   • Aanbevelingen

     Ontvangen: 195

     Gegeven: 54

   •    Aantal posts: 2.291

   De datum en agenda van de Algemene Vergadering Aandeelhouders (AVA) is officieel bekend gemaakt.
   
   Dear Shareholder:
   
   On behalf of the Board of Directors of uniQure N.V. (the “Company”), I invite you to attend our 2019 Annual General Meeting of Shareholders (the “2019 Annual Meeting”). The 2019 Annual Meeting will be held on June 19, 2019, at 9:30 a.m., Central European Summer Time at the Company’s principal executive offices located at Paasheuvelweg 25a, 1105BP Amsterdam, the Netherlands.
   
   The matters to be voted upon at the 2019 Annual Meeting are listed in the Notice of the 2019 Annual Meeting and are more fully described in the proxy statement accompanying this letter (the “Proxy Statement”).
   
   At the 2019 Annual Meeting, you will be provided an opportunity to ask questions regarding the matters to be voted upon, gain an up-to-date perspective on the Company and its activities, and meet the directors of the Company.
   
   [..]
   
   Thank you for your continuing interest in the Company. We look forward to seeing you at the 2019 Annual Meeting.
   
   If you have any questions about the Proxy Statement, please contact investor relations at investors@uniQure.com.
   
   Sincerely,
   Matthew Kapusta
   
   De agenda
   

   1. Opening and announcements
   2. Report on the financial year 2018 (for discussion only)
   3. Explanation of the application of the remuneration policy (for discussion only)
   4. Adoption of the 2018 Dutch statutory annual accounts and treatment of the results (Voting Proposal No. 1)
   5. Discharge of liability for the members of the Board of Directors (Voting Proposal No. 2)
   6. Board Appointment: Re-election of Matthew Kapusta as executive director (Voting Proposal No. 3);
   7. Renew the designation of the Board as the competent body to issue Ordinary Shares and options (Voting Proposal No. 4)
   8. Reauthorize the Board to exclude or limit preemptive rights upon the issuance of Ordinary Shares (Voting Proposal No. 5)
   9. Reauthorize the Board to repurchase Ordinary Shares (Voting Proposal No. 6)
   10. Appointment of KPMG Accountants N.V. as external auditors of the Company for the financial year 2019 (Voting Proposal No. 7)
   11. To approve, on an advisory basis, the compensation of the named executive officers of the Company (Voting Proposal No. 8)
   12. To approve, on an advisory basis, the frequency of advisory votes on the compensation of the named executive officers of the Company (Voting Proposal No. 9)
   13. Any other business
   14. Closing of the meeting

   
   Het is een tamelijk 'routine matige agenda'. Waarde van het bezoek zit zoals altijd in het informele: sfeer proeven en gelegenheid vragen te stellen.
   
   Vraag 1: welke forumleden zijn voornemen de AVA te bezoeken?
   Vraag 2: welke vragen zouden gesteld moeten worden?
5. Prof. Dollar 2 mei 2019 13:32

   auteur info

   • Prof. Dollar

     Lid sinds: 04 mrt 2011

     Laatste bezoek: 09 feb 2025

   • Aanbevelingen

     Ontvangen: 195

     Gegeven: 54

   •    Aantal posts: 2.291

   Persbericht
   
   uniQure Announces New Preclinical Data in Hemophilia A and Fabry Disease in Oral Presentations at the 22nd ASGCT Annual Meeting
   
   LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 02, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, will present today new preclinical data on its gene therapy candidates AMT-180 for the treatment of hemophilia A and AMT-190 for the treatment of Fabry disease. These data will be featured today in back-to-back oral presentations at the 22nd American Society for Gene and Cell Therapy (ASGCT) Annual Meeting in Washington D.C.
   
   AMT-180 for Hemophilia A
   Hemophilia A is an X-linked bleeding disorder resulting from a deficiency in coagulation Factor VIII that serves as a cofactor for Factor IX in the activation of the coagulation cascade. About 30 percent of the hemophilia A patient population develops inhibitors to Factor VIII over the course of the disease.
   
   AMT-180 comprises a recombinant AAV5 vector incorporating a proprietary modified Factor IX hat, when activated through normal mechanisms, induces thrombin generation independently of Factor VIII.
   
   Data from multiple in vitro and in vivo studies show that a single intravenous administration of AMT-180 results in dose-dependent, therapeutically meaningful Factor VIII-independent activity as measured by thrombin generation and one-stage clotting assay. AMT-180 is a differentiated approach that is suggested to be hepatocyte-friendly and non-thrombogenic based on the studies conducted to date and is expected to reduce and potentially prevent bleedings in hemophilia A patients with and without inhibitors.
   
   AMT-180 Preclinical Data Findings
   Proof-of-concept for AMT-180 was established through studies across two different animal models. The oral presentation at ASGCT features the following data:
   

   • Preclinical studies in FVIII-depleted human plasma show that AMT-180 induced clinically relevant thrombin activation, and up to 29% of Factor VIII-independent activity, in plasma with and without inhibitors.
   
   
   • The mechanistic proof-of-concept of AMT-180 was demonstrated in a hemophilia A mouse model, where a single intravenous administration of AMT-180 resulted in sustained, dose-dependent hemostatic effect as measured by one-stage clotting assay.
   
   
   • The studies further demonstrate that AMT-180 shows activation kinetics similar to native FIX and is not hyperactive.
   
   
   • A pilot study in non-human primates demonstrated that a single administration of AMT-180 resulted in sufficient FIX protein expression that translates to clinically relevant Factor VIII-independent activity in humans. No elevation of coagulation activation markers or signs of thrombi formation were observed.

   
   “Data from these preclinical studies show the exciting potential of AMT-180 to provide clinically meaningful Factor VIII-independent activity after a one-time administration.” stated Sander van Deventer, M.D., Ph.D., chief scientific officer at uniQure. “We are particularly encouraged by the broad potential of AMT-180 in treating both patients with and without inhibitors, and the unique approach of AMT-180 that potentially circumvents durability issues because of its hepatocyte-friendly profile.”
   
   AMT-190 for Fabry Disease
   Fabry disease is an X-linked genetic disorder resulting from a deficiency of a-galactosidase A (a-gal or GLA). The current standard of care for Fabry disease is bi-weekly infusions of enzyme replacement therapy, a treatment that has shown not to be effective in many patients due to poor targeting of target organs such as the kidney and heart. In addition, a significant number of patients develop antibodies to the enzyme, a-gal or GLA.
   
   AMT-190 is a novel AAV5 gene therapy approach for Fabry disease that comprises a recombinant AAV5 vector incorporating a proprietary, exclusively licensed, modified NAGA (ModNAGA) variant. AMT-190 provides expression of ModNAGA, which shows a high structural resemblance to a-gal. This approach may have several advantages over a-gal therapies, including higher stability in blood, better biodistribution in the target organs, secondary toxic metabolite reduction and improved cross-correction of neighboring cells. ModNAGA is also effective in the presence of a-gal antibodies.
   
   Data from in vitro and in vivo studies show that AMT-190 has the potential to become a one-time treatment option that improves upon the enzyme replacement standard of care with more efficient uptake in the kidney and heart and an improved immunogenicity profile.
   
   AMT-190 Preclinical Data Findings
   Proof-of-concept for AMT-190 was established through multiple studies in wild-type and Fabry mice. The oral presentation at ASGCT features the following data:
   
   

   • Preclinical in vitro studies demonstrated that the expression of ModNAGA results in GLA activity in cells and suggest that uptake of ModNAGA is mediated by the Mannose-6-phosephase (M6P) receptor.
   
   
   • In vivo studies in wild-type mice show that a single intravenous administration of AMT-190 resulted in a ten- to twenty-fold higher GLA activity in the plasma compared to the control group, suggesting that AMT-190 has the potential to provide therapeutically relevant GLA activity in plasma and in target organs.
   
   
   • These results were underscored by a study in GLA knock-out mice, demonstrating significantly increased GLA activity in plasma and significantly reduced Lyso-Gb3 in the target organs after a single dose of AMT-190. In silico and in vitro studies also show that the modifications introduced into NAGA pose a very low immunogenicity risk.

   
   “These data show that AMT-190 has the potential to be a differentiated, one-time treatment option that could be used by all Fabry patients,” added Dr. van Deventer. “We will continue to advance our preclinical research toward our goal of developing a best-in-class gene therapy for Fabry disease.”
   
   An overview of the AMT-180 and AMT-190 preclinical data presented at ASGCT can be found on the Investor section of uniQure’s corporate website.
6. T. Montana 3 mei 2019 01:06

   auteur info

   • T. Montana

     Lid sinds: 23 okt 2015

     Laatste bezoek: 09 mei 2024

   • Aanbevelingen

     Ontvangen: 22

     Gegeven: 46

   •    Aantal posts: 390

   Hoe verhoudt 29% in dit preklinische model zich tot de competitie?
7. Prof. Dollar 3 mei 2019 10:40

   auteur info

   • Prof. Dollar

     Lid sinds: 04 mrt 2011

     Laatste bezoek: 09 feb 2025

   • Aanbevelingen

     Ontvangen: 195

     Gegeven: 54

   •    Aantal posts: 2.291

   quote:

   T. Montana schreef op 3 mei 2019 01:06:

   Hoe verhoudt 29% in dit preklinische model zich tot de competitie?
   

   Begin april heeft Sangamo in samenwerking met Pfizer Phase 1/2 Interim Data voor hun Hemophilia A gentherapie product bekendgemaakt. Zij claimen, afhankelijk van het meetinstrument, een expressie tussen de 94% en 140%. www.pfizer.com/news/press-release/pre...
   
   Op dit moment acht ik het product van QURE dus absoluut niet competitief, hooguit één van de toekomstige alternatieven voor die 30% van de patiëntenpopulatie die remmers (inhibitors) ontwikkelen.
   
   Er zijn twee mitsen die de toekomst moeten uitwijzen:
   

   • Mij is niet geheel duidelijk waar QURE haar 29% op baseert. Mogelijk dat men nu enkel het mechanisme heeft gepresenteerd (proof-of-concept van Super9; een factor 9 die geen factor 8 nodig heeft), maar dat men nog werkt aan specifieke doseringsstudies. Lees: finetuning met de nieuwe lever-specifieke promotor zodat ze die 29% opbeuren naar een stabiele 100% expressie. Dit zal dan het konijn uit de hoge hoed zijn.
   • Dan is er nog de benadering van Sangamo, die door middel van expressie van factor 8 in de lever de FIX maakt, waarvan QURE vermoed dat dit niet resulteert in een duurzame oplossing (met andere woorden de expressie zal naar verloop van tijd significant afnemen).
     

   Kortom, wat betreft ontwikkeling ligt QURE ogenschijnlijk achter en of ze in staat is de concurrentie 'rechts in te halen' ligt voorlopig besloten in de tijd.
8. T. Montana 3 mei 2019 19:36

   auteur info

   • T. Montana

     Lid sinds: 23 okt 2015

     Laatste bezoek: 09 mei 2024

   • Aanbevelingen

     Ontvangen: 22

     Gegeven: 46

   •    Aantal posts: 390

   quote:

   Prof. Dollar schreef op 3 mei 2019 10:40:

   [...]
   Begin april heeft Sangamo in samenwerking met Pfizer Phase 1/2 Interim Data voor hun Hemophilia A gentherapie product bekendgemaakt. Zij claimen, afhankelijk van het meetinstrument, een expressie tussen de 94% en 140%. www.pfizer.com/news/press-release/pre...
   
   Op dit moment acht ik het product van QURE dus absoluut niet competitief, hooguit één van de toekomstige alternatieven voor die 30% van de patiëntenpopulatie die remmers (inhibitors) ontwikkelen.
   
   Er zijn twee mitsen die de toekomst moeten uitwijzen:
   

   • Mij is niet geheel duidelijk waar QURE haar 29% op baseert. Mogelijk dat men nu enkel het mechanisme heeft gepresenteerd (proof-of-concept van Super9; een factor 9 die geen factor 8 nodig heeft), maar dat men nog werkt aan specifieke doseringsstudies. Lees: finetuning met de nieuwe lever-specifieke promotor zodat ze die 29% opbeuren naar een stabiele 100% expressie. Dit zal dan het konijn uit de hoge hoed zijn.
   • Dan is er nog de benadering van Sangamo, die door middel van expressie van factor 8 in de lever de FIX maakt, waarvan QURE vermoed dat dit niet resulteert in een duurzame oplossing (met andere woorden de expressie zal naar verloop van tijd significant afnemen).
     

   Kortom, wat betreft ontwikkeling ligt QURE ogenschijnlijk achter en of ze in staat is de concurrentie 'rechts in te halen' ligt voorlopig besloten in de tijd.
   

   Dank Prof!
9. Prof. Dollar 7 mei 2019 13:21

   auteur info

   • Prof. Dollar

     Lid sinds: 04 mrt 2011

     Laatste bezoek: 09 feb 2025

   • Aanbevelingen

     Ontvangen: 195

     Gegeven: 54

   •    Aantal posts: 2.291

   Persbericht
   
   uniQure Announces Featured Presentations of New Data on Spinocerebellar Ataxia Type 3 at the 2019 American Academy of Neurology Annual Meeting
   
   

   • Data Show AMT-150 Able to Significantly Lower Mutant Ataxin-3 Protein in SCA3 Disease Model
   
   
   • Provides Further Support of Proof-of-concept of Company’s Proprietary miQURE™ Gene Silencing Platform

   LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 07, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, will today present preclinical data on its gene therapy candidate, AMT-150, for the treatment of Spinocerebellar Ataxia 3 (SCA3). The abstract entitled, “Development of an AAV-based microRNA Gene Therapy for Treating Spinocerebellar Ataxia Type 3,” is being recognized by the American Academy of Neurology for dual oral and poster presentations during its annual meeting taking place this week in Philadelphia, PA.
   
   Spinocerebellar Ataxia Type 3, also known as Machado-Joseph disease, is caused by a CAG-repeat expansion in the ATXN3 gene that results in an abnormal form of the toxic protein ataxin-3, leading to brain degeneration that results in movement disorders, rigidity, muscular atrophy and paralysis. There are no disease-modifying treatments for patients with SCA3, or medications to slow the progressive course of the fatal disease.
   
   uniQure’s AMT-150 incorporates the Company’s proprietary miQURE™ gene silencing technology and comprises an AAV5 vector to deliver a microRNA that is designed to halt ataxia in early manifest SCA3 patients. AMT-150 is delivered by intra-cisterna magna injection into the cerebrospinal fluid.
   
   AMT-150 Preclinical Data Findings:
   Mechanistic proof-of-concept of the non-allele-specific ataxin-3 protein-silencing approach was demonstrated using artificial microRNA candidates engineered to target the ataxin-3 gene in a SCA3 knock-in mouse model. The 6-week proof-of-concept study demonstrated that a single AMT-150 injection in the cerebrospinal fluid resulted in strong AAV transduction and significant mutant ataxin-3 lowering for at least one microRNA candidate at each of the primary sites of disease neuropathology, namely the cerebellum (up to 53%) and brainstem (up to 65%).
   
   These results were corroborated by preclinical studies in human induced Pluripotent Stem Cell (iPSC)-derived neurons showing a dose-dependent lowering of ataxin-3 mRNA of up to 55%.
   
   These studies, along with our previously-reported data in Huntington’s disease, further demonstrate the potential utility and safety profile of the miQURE™ technology, the Company’s proprietary gene-silencing platform. “We believe that the data from these preclinical studies in the knock-in mouse model and in iPSC-derived neurons show the potential of AMT-150 to alter the course of this devastating disease after a single administration,” stated Sander van Deventer, M.D., Ph.D., chief scientific officer at uniQure. “We are very proud of our proprietary, in-house developed miQURE™ technology, and we believe that it has the potential to treat a wide range of polyglutamine diseases, including Huntington’s disease and SCA3. We will continue to conduct additional research on AMT-150 to advance it toward our goal of IND-enabling studies.”
   
   AMT-130 Data Presentations
   In addition to the highlighted presentations on AMT-150 in SCA3, two encore presentations on the development of AMT-130 in Huntington’s disease also were featured during the AAN conference. One presentation included findings from magnetic resonance imaging volumetric analysis in twenty early manifest Huntington’s disease patients to determine the safety of delivering gene therapy to the striatum, or deep structures of the brain. The second reported on the biodistribution and tolerability of AMT-130 after bilateral intra-striatal delivery to non-human primates.
   
   An overview of the data presented at AAN can be found in the Investor section of uniQure’s corporate website.
10. Prof. Dollar 8 mei 2019 09:35

    auteur info

    • Prof. Dollar

      Lid sinds: 04 mrt 2011

      Laatste bezoek: 09 feb 2025

    • Aanbevelingen

      Ontvangen: 195

      Gegeven: 54

    •    Aantal posts: 2.291

    Het is stil rondom QURE's samenwerking met BMY. En niet zonder reden. Lees de quote uit het jaarverslag:
    
    "In February 2019, BMS requested a one-year extension of the research term. In April 2019, following an assessment of the progress of this collaboration and our expanding proprietary programs, we notified BMS that we do not intend to agree to an extension of the research term. Accordingly, we are currently in discussions with BMS to potentially amend the collaboration and license agreement and other related agreements following the expiration of the research term" www.iex.nl/Forum/Topic/1317038/4/Hart... en het complete verslag uniqure.gcs-web.com/static-files/26f2...
    
    Boeiende ontwikkeling! Beide hebben veel aan elkaar gehad: QURE kon het geld en de pr goed gebruiken en BMY kan de verkregen stukken nu voor meer geld verkopen (As of December 31, 2018, BMS held 6.4% of our outstanding ordinary shares). Het kan verkeren. En dan te bedenken dat QURE eerder Chiesi aan de kant heeft gezet om vervolgens op snelheid te komen met een nieuw hemofilie product. Lean and mean!
11. pim f 10 mei 2019 14:15

    auteur info

    • pim f

      Lid sinds: 29 sep 2004

      Laatste bezoek: 09 feb 2025

    • Aanbevelingen

      Ontvangen: 868

      Gegeven: 779

    •    Aantal posts: 4.103

    quote:

    Steve schreef op 29 april 2019 13:26:

    Thanks prof! 10 mei staat in de agenda genoteerd. Very exiting!
    

    op het programma kan ik ze niet terugvinden: www.eventscribe.com/2019/HTRS/agenda....
    
    misschien alleen een poster?
12. flosz 10 mei 2019 14:22

    auteur info

    • flosz

      Lid sinds: 13 feb 2008

      Laatste bezoek: 01 jul 2024

    • Aanbevelingen

      Ontvangen: 5298

      Gegeven: 2795

    •    Aantal posts: 7.005

    Poster(s) idd.
    www.eventscribe.com/2019/HTRS/postera... twitter.com/floszcrxl/status/11267815...
    
    47.1% mean FIX at 26 weeks
    www.iex.nl/Forum/Topic/1293267/14/Hem...
13. pim f 10 mei 2019 14:35

    auteur info

    • pim f

      Lid sinds: 29 sep 2004

      Laatste bezoek: 09 feb 2025

    • Aanbevelingen

      Ontvangen: 868

      Gegeven: 779

    •    Aantal posts: 4.103

    Dank u Flosz, ik heb de poster globaal doorgenomen en de stijging van FIX is minder sterk maar gaat wel door. In het licht van de posting van prof Dollar over de ontwikkelingen bij Pfizer/Sangamo, acht jij 47,1 een sterk cijfer of gemiddeld?
14. flosz 10 mei 2019 15:29

    auteur info

    • flosz

      Lid sinds: 13 feb 2008

      Laatste bezoek: 01 jul 2024

    • Aanbevelingen

      Ontvangen: 5298

      Gegeven: 2795

    •    Aantal posts: 7.005

    Hartstikke goed imho.
    Ook voorzichtig zijn in 1op1 vergelijken FIX-resultaten, gebruik steroïden,NAB’s, etc.etc.
    
    twitter.com/biotechobserver/status/10...
    twitter.com/profmakris/status/1069794...
15. Prof. Dollar 10 mei 2019 15:53

    auteur info

    • Prof. Dollar

      Lid sinds: 04 mrt 2011

      Laatste bezoek: 09 feb 2025

    • Aanbevelingen

      Ontvangen: 195

      Gegeven: 54

    •    Aantal posts: 2.291

    quote:

    pim f schreef op 10 mei 2019 14:35:

    Dank u Flosz, ik heb de poster globaal doorgenomen en de stijging van FIX is minder sterk maar gaat wel door. In het licht van de posting van prof Dollar over de ontwikkelingen bij Pfizer/Sangamo, acht jij 47,1 een sterk cijfer of gemiddeld?
    

    Let op, mijn eerdere bericht had betrekking op hemofilie A. Het persbericht van vandaag gaat over hemofilie B. Die resultaten zien er overigens keurig uit.
16. pim f 10 mei 2019 16:52

    auteur info

    • pim f

      Lid sinds: 29 sep 2004

      Laatste bezoek: 09 feb 2025

    • Aanbevelingen

      Ontvangen: 868

      Gegeven: 779

    •    Aantal posts: 4.103

    quote:

    Prof. Dollar schreef op 10 mei 2019 15:53:

    [...]
    Let op, mijn eerdere bericht had betrekking op hemofilie A. Het persbericht van vandaag gaat over hemofilie B. Die resultaten zien er overigens keurig uit.
    

    Oei, overheen gelezen. Merci voor uw aanvullende info
17. pim f 11 mei 2019 01:31

    auteur info

    • pim f

      Lid sinds: 29 sep 2004

      Laatste bezoek: 09 feb 2025

    • Aanbevelingen

      Ontvangen: 868

      Gegeven: 779

    •    Aantal posts: 4.103

    Disclaimer: Positie heden verder uitgebreid.
    
    Van het Yahoo-forum:
    
    Wow... interesting to learn that the Spark Therapeutics 122% takeover premium by Roche has the possibility of falling through, as investor lawsuits believe the price is too low, given gene therapy. This thing may crater unless there is a white knight. Here's more info from article below (can't post links in yahoo finance)-
    
    ------------------------------------------
    Roche’s $114.50-per-share offer for Spark Therapeutics represents a whopping 122% premium to the gene specialist’s closing price on the last trading day before the acquisition announcement. But some Spark investors still think the price is too low.
    
    So far, Spark investors have tendered only 29.4% of the shares outstanding, which doesn't exactly inspire confidence in the deal; Roche needs more than 50% to carry out the combo. The Swiss drugmaker said it's extending its tender offer to May 2 from the previous deadline of Wednesday.
    
    ----
    
    update Pim F:
    
    www.bizjournals.com/philadelphia/news...
    
    In dit artikel staat dat de deadline is opgerekt tot 3 juni en dat maar 26% van de aandelen is aangemeld...
18. Tex Mex 11 mei 2019 10:28

    auteur info

    • Tex Mex

      Lid sinds: 10 sep 2013

      Laatste bezoek: 05 feb 2025

    • Aanbevelingen

      Ontvangen: 26

      Gegeven: 8

    •    Aantal posts: 192

    quote:

    pim f schreef op 10 mei 2019 14:35:

    In het licht van de posting van prof Dollar over de ontwikkelingen bij Pfizer/Sangamo, acht jij 47,1 een sterk cijfer of gemiddeld?
    

    Prachtig resultaat. Ook knap dat de stijging precies lijkt te verlopen volgens het door uniQure gebruikte predictiemodel.
19. pim f 11 mei 2019 16:36

    auteur info

    • pim f

      Lid sinds: 29 sep 2004

      Laatste bezoek: 09 feb 2025

    • Aanbevelingen

      Ontvangen: 868

      Gegeven: 779

    •    Aantal posts: 4.103

    quote:

    Prof. Dollar schreef op 30 april 2019 09:25:

    On behalf of the Board of Directors of uniQure N.V. (the “Company”), I invite you to attend our 2019 Annual General Meeting of Shareholders (the “2019 Annual Meeting”). The 2019 Annual Meeting will be held on June 19, 2019, at 9:30 a.m., Central European Summer Time at the Company’s principal executive offices located at Paasheuvelweg 25a, 1105BP Amsterdam, the Netherlands.
    
    Vraag 1: welke forumleden zijn voornemen de AVA te bezoeken?
    Vraag 2: welke vragen zouden gesteld moeten worden?
    

    Ik ben van plan te gaan
20. Frontrunner 16 mei 2019 21:55

    auteur info

    • Frontrunner

      Lid sinds: 30 jul 2018

      Laatste bezoek: 07 feb 2025

    • Aanbevelingen

      Ontvangen: 13

      Gegeven: 0

    •    Aantal posts: 85

    quote:

    pim f schreef op 11 mei 2019 16:36:

    [...]
    
    Ik ben van plan te gaan
    

    Vraag 1: Ik ga niet. Ben ook niet uitgenodigd trouwens. Op basis waarvan wordt je eigenlijk uitgenodigd?
    Vraag 2: Ben wel benieuwd hoe ze tegen een overname aankijken? Zitten ze hierop te wachten of gaan ze liever door op eigen kracht? Vraag mij wel af of je hier een eerlijk antwoordt op gaat krijgen.