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Hemofilie B; nog steeds on track?

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  1. Fartknock 11 december 2017 13:06

    • Fartknock

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    tools.eurolandir.com/tools/Pressrelea...

    uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

    - Clinical Benefit Maintained in All Patients, with Durable Increases in FIX Clotting Activity at Up to Two Years of Follow-Up -

    - Second Dose Cohort Demonstrated 89% Reduction in Spontaneous Bleeds, with No Confirmed Bleeds Reported in Last 12 Months -

    - AAV5-Based Gene Therapy Continues to Show Favorable Safety Profile, with No Immune Responses or Loss of FIX Activity in Any Patient -

    - Company Preparing to Initiate Pivotal Study with the FIX-Padua Modification (AMT-061) in 2018 -

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Dec. 11, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its investigational gene therapy in patients with severe hemophilia B. The data includes up to two years of follow-up from the low-dose cohort and up to 18 months of follow-up from the second, higher-dose cohort.

    The AAV5-based AMT-060 remains safe and well-tolerated with up to two years of follow-up, with no new serious adverse events and no development of inhibitors. No patient in the study has had any loss of Factor IX (FIX) activity or capsid-specific, T-cell-mediated immune response.

    Eighteen-month follow-up data from the second-dose cohort continue to show stable FIX activity with substantial improvement in disease state in all five patients, including the discontinuation of routine prophylactic FIX infusions in all patients that previously required chronic replacement therapy. The annualized spontaneous bleeding rate for the second dose cohort declined 89% to a mean of 0.3 bleeds after gene transfer. In the last year of follow-up, no patient in the second cohort has reported any spontaneous bleeds.

    These clinical data were presented this morning in an oral presentation at the 58th American Society of Hematology (ASH) Annual Meeting taking place in Atlanta, Georgia.

    "We continue to observe a therapeutic benefit from AMT-060 that is clearly superior to patients' previous prophylactic FIX replacement therapy regimen, with stable elevations in Factor IX levels and a cessation of spontaneous bleeds," stated Professor Frank W.G. Leebeek, M.D. Ph.D. of the Erasmus University Medical Center in Rotterdam, the Netherlands.

    "Most importantly, the AAV5-based AMT-060 remains safe and well-tolerated, with no loss of FIX activity, no activation of T-cell response and no development of inhibitors for any of the 10 patients in the study, up to two years after treatment. The safety profile observed in this study continues to suggest that the AAV5 vector offers long-term safety, efficacy and the potential for broad application in hemophilia B patients."

    uniQure announced in October that, following meetings with the FDA and EMA, it plans to initiate a pivotal study in 2018 with AMT-061, which combines an AAV5 vector with the FIX-Padua mutant. AMT-061 and AMT-060 are identical in structure apart from two nucleotide substitutions in the coding sequence for FIX. The gene variant, referred to as FIX-Padua, has been reported in multiple preclinical and nonclinical studies to provide an approximate 8 to 9-fold increase in FIX clotting activity compared to the wild-type FIX gene. All other critical quality attributes of AMT-061 are expected to be comparable to those of AMT-060, as AMT-061 utilizes the same AAV5 capsid and proprietary insect cell-based manufacturing platform.

    "These data give us continued confidence that our AAV5-based gene therapies offer multi-year durability, superior safety and broad applicability as a result of a favorable immunogenicity profile compared to other AAV vectors," stated Matthew Kapusta, chief executive officer of uniQure. "We believe AMT-061 has the potential to provide curative benefits to nearly all hemophilia B patients, without the complications associated with capsid-related immune responses. Preparations for the pivotal study are underway and the manufacturing of AMT-061 for clinical use has been initiated."

    Phase 1/2 Trial Overview

    The AMT-060 gene therapy consists of a codon-optimized wild type FIX gene cassette, the LP1 liver promoter and an AAV5 viral vector manufactured by uniQure using its proprietary insect cell-based technology platform.

    •The Phase I/II, open-label, multi-center study includes 10 patients each receiving a one-time, 30-minute, intravenous administration of AMT-060, without the prophylactic use of corticosteroids.

    •The study includes two dose cohorts of five patients each, with the first cohort receiving 5x1012gc/kg and the second cohort receiving 2x1013 gc/kg.

    •Nine patients in the trial were classified as having severe (<1% FIX activity) hemophilia. One patient in the low-dose cohort had a moderate/severe (1.5% FIX activity) phenotype.

    Data Update from Phase I/II Clinical Trial of AMT-060 in Hemophilia B Patients

    Data as of October 26, 2017:

    •All 10 patients in the study have demonstrated improvements in their disease state as measured by reduced FIX replacement therapy and bleeding frequency.


    •In the second-dose cohort, no spontaneous bleeds have been reported in the last year of follow-up, with a reduction in the annualized spontaneous bleed rate of 89% compared to the one-year period prior to administration of AMT-060. Total bleeds were reduced by 75%.

    •As previously announced, eight of the nine patients that required chronic FIX infusions prior to administration of AMT-060 have discontinued prophylaxis after treatment. All eight patients remained prophylaxis-free at the last follow-up.

    •Across both dose cohorts, cumulative annualized FIX consumption decreased by 84%, from 2.64 million to 428,554 IU.

    •Through up to 18 months of follow-up among the five patients in the second-dose cohort, the mean steady-state FIX activity persisted at approximately 7% of normal. The mean FIX activity at the last follow-up (18 months) was 8.1%, ranging from 4.2% to 11.1%.
  2. flosz 11 december 2017 13:36

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    $QURE Stable elevations in FIX activity and reductions in
    annualized bleeding rate over up to 2 years of follow-up of adults with severe or moderate-severe hemophilia B treated with AMT-060 (AAV5-hFIX) gene therapy
    uniqure.com/investors-newsroom/ASH%20...,%202017.pdf
    Linkje "doet t niet", link via twitter werkt prima: twitter.com/floszcrxl/status/94019730...
  3. Fartknock 18 december 2017 13:24

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    tools.eurolandir.com/tools/Pressrelea...

    uniQure Announces Publication in the Journal Blood of Clinical Data from Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

    Data Demonstrate Clinical Effectiveness and Superior Immunogenicity Profile of AAV5 Gene Therapy in Severe and Moderate-Severe Hemophilia B Patients

    LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Dec. 18, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the online publication in Blood of clinical data from the ongoing Phase I/II trial of AMT-060 in patients with severe hemophilia B. The manuscript reports data on up to one year of follow-up from the low-dose cohort of patients in the trial and up to six months of follow-up on patients in the higher-dose cohort.

    The published manuscript, entitled "Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B," shows that AAV5 liver-directed wildtype hFIX gene transfer was well tolerated and clinically effective in patients with severe and moderate-severe hemophilia B. No cellular immune responses to the AAV5 vector were detected and Factor IX (FIX) expression levels were stable over the entire observation period. The manuscript is available online today and will be included in a future print edition of Blood.

    The study included ten adults with hemophilia B and severe-bleeding phenotype. A single infusion of AMT-060 had a favorable safety profile and resulted in stable and clinically-important FIX activity increases, along with a marked reduction in spontaneous bleeds and FIX concentrate use, without detectable cellular-immune responses against capsids. In the higher-dose cohort, mean FIX activity increased to 6.9% of normal. Annualized FIX use decreased by 73%, and mean annual bleeding rate declined by 70%, from 3.0 to 0.9. FIX activity was stable in both cohorts and eight of nine participants receiving FIX at study entry stopped prophylaxis.

    "Blood's publication of this manuscript is a testament to the quality of the study and its significance to the advancement of gene therapy in hemophilia," stated Professor Wolfgang Miesbach, M.D., of the University Hospital Frankfurt, Germany. "We're very pleased to have these data published in a journal as highly regarded as Blood."

    Additional follow-up on the patients from this study was presented on December 11, 2017, at the American Society of Hematology (ASH) Annual Meeting. The AAV5-based AMT-060 remains safe and well-tolerated with now up to two years of follow-up in the low-dose cohort and 1.5 years in the higher-dose cohort. There were no new serious adverse events and no development of inhibitors. To date, no patient in the study has had any loss of Factor IX activity or encountered a capsid-specific, T-cell-mediated immune response.

    uniQure is preparing to initiate a pivotal study in 2018 with AMT-061, which combines an AAV5 vector with the FIX-Padua mutant. AMT-061 and AMT-060 are identical in structure apart from two nucleotide substitutions in the coding sequence for FIX. The gene variant, referred to as FIX-Padua, has been reported in multiple preclinical and nonclinical studies to provide an approximate 8 to 9-fold increase in FIX clotting activity compared to the wild-type FIX gene. All other critical quality attributes of AMT-061 are expected to be comparable to those of AMT-060, as AMT-061 utilizes the same AAV5 capsid and proprietary insect cell-based manufacturing platform.

    "The data from the Phase I/II trial show that our AAV5-based gene therapies offer multi-year durability and a favorable immunogenicity profile, enabling hemophilia B patients to discontinue frequent infusions of FIX replacement therapy and to reduce the risk of spontaneous bleeding," stated Dr. Steven Zelenkofske, chief medical officer of uniQure. "With AAV5 emerging as a potential best-in-class vector for systemic administration to the liver, we look forward to advancing AAV5-FIX-Padua (AMT-061) into a pivotal trial in 2018."
  4. Tex Mex 18 december 2017 20:24

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    Publicatie in Blood. Netjes gedaan. Zeker niet het minste tijdschrift in de medische literatuur!
  6. [verwijderd] 21 december 2017 21:38

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    Ik bedoel, in het bericht op nu.nl wordt jammer genoeg niet vermeld dat deze gentherapie door Uniqure is ontwikkeld.
    In het bericht op de website van ErasmusMC staat dit natuurlijk wél:
    "Gentherapie vond plaats door gebruik te maken van een aangepast, niet-ziekmakend virus waarin het factor IX gen is ingebouwd. In de studie zijn twee verschillende doseringen toegediend van een AAV-5 virus-FIX gen product (vector), ontwikkeld door uniQure Biopharma te Amsterdam. De toediening gebeurde door een éénmalige infusie van dertig minuten bij tien patiënten."
  12. flosz 11 januari 2018 17:35

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    Rare Disease Report (RDR) sat down with Matthew Kapusta, uniQure’s CEO, to discuss AMT-061, the excitement surrounding it, and what he expects to see from the drug in 2018.

    RDR: What’s the background of AMT-061? How long has it been in development?

    Kapusta: We’ve been developing, for a number of years, a gene therapy targeting hemophilia B, and we have one that is an AAV5-delivering Padua-FIX construct. It is administered through a simple IV infusion that is typically infused over 30 minutes, and ultimately, I think the vision is that, commercially, this will be a fairly simple outpatient procedure. We did a Phase 1/2 study on the first generation of the product that was using the wild-type gene cassette, and at the most recent ASH meeting in December, we presented 2-year follow-up data that showed a dramatic clinical effect on the patients. What we decided to do last year, though, was to make a slight modification to the trans gene that increases the potency of the Factor IX protein that is actually produced and we demonstrated that this slight modification will increase the clotting activity of each protein molecule by about 6-8 times.

    RDR: What were the driving forces behind the update to the compound?

    Kapusta: One of the things that we were hearing from the key opinion leaders was that they wanted to see higher levels of Factor IX activity, and curative levels of Factor IX are generally considered to be above 40-50%. With this new potency change, we think we can get patients into the 30-50% range of normal Factor IX activity level, so near curative levels. At those levels, the expectation is that patients will no longer require Factor IX replacement therapy, and that there will be a near cessation of bleeding. The potential that the long-term prognosis of their joint health will be significantly benefitted. It’s really, really exciting stuff. The data that we presented at ASH in December showed remarkably durable levels of Factor IX activity up to 2 years post-treatment. The patients, really, were not bleeding, and if you look at the presentation, you’ll see actual images of the patients who were in the study. We’ve met these patients and their lives have been remarkably transformed. We’re taking this construct into a pivotal study. We expect to initiate this study in the third quarter of this year. The pivotal study will include approximately 40 patients who will service their own control. We will be administering the gene therapy to these patients and then following them up for about 52 weeks. We think that this single registration study will provide sufficient enough evidence for us to submit a BLA (Biologics License Application).

    RDR: How have people within the hemophilia community been reacting to the potential of AMT-061?

    Kapusta: The reception has been really very exciting. There’s been a lot of media coverage, and the hemophilia community, not unlike a lot of these other rare and orphan disease communities, are very tight-knit. There’s been a growing level of excitement about gene therapy. I think, initially, there might have been some pretty reasonable anxiety about what to expect as it pertained to safety and how long the treatment would last, but the first gene therapy clinical studies were done more than 8 years ago. All of the clinical work that has been done in hemophilia – and there have been multiple studies conducted – have shown well-tolerated, highly safe gene therapy with the ability to have a dramatic impact on the lives of patients.

    I think there’s a growing level of excitement. This is a patient population that has become increasingly cautious over the years, and they have gotten used to taking replacement therapy. If you think about what is happening to these patients, some hemophiliacs are taking 3+ infusions per week, and if you multiply that out, you’re talking about 150-200 infusions per year for the rest of their lives. The mere thought of being able to take a 30-minute IV infusion that eliminates the need to do that, and not only eliminate the need to do it, but that it might completely eliminate the propensity for bleeding, it’s pretty remarkable. Even the payers are getting greater appreciation and excitement for it. Not only is it 150-200 infusions per year, but the cost of this could be $300-500 per patient per year for the rest of their lives. It’s really millions of dollars per patient, and the potential to have a one-time curative therapy is very exciting even to the payers. We’re very pleased to be in the middle of this, and in a position to begin a registrational study. We’re excited to potentially be first-to-market with a transformative therapy.

    RDR: What kinds of milestones is uniQure hoping to hit in 2018?

    Kapusta: We have a number of important milestones. I would say that the key ones are we expect to file our IND (investigational new drug) application with the FDA this quarter. We expect to initiate the treatment of patients in our program in the third quarter of this year. We expect to release data from those initial patients before the end of the year. Those are the key milestones on the hemophilia B program.

    RDR: There are a lot of other players in the gene therapy space. What makes uniQure stand out?

    Kapusta: One of the most critical things about bringing the gene therapies to market successfully is going to be the ability to manufacture these products. One of the differentiator points for UniQure is that we’ve been in the field of gene therapy for more than 20 years, and we’ve spent more than 10 years refining and optimizing our manufacturing capabilities. There aren’t many other players in the gene therapy space that can boast like that.
    www.raredr.com/news/uniqure-ceo-speak...
  13. QLVT 12 januari 2018 22:57

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    Slechte beleggingsweek voor mij ,het enige is dat het mooi weer is op curacau ,waar ik op vakantie ben,Ik dacht ik verdien mijn vakantie wel terug maar valt nu meer dan 250% duurder uit. Volgende week maar weer afzien en dan over 3 week naar pharming.....
  14. T. Montana 13 januari 2018 01:05

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    quote:

    cqtvld schreef op 12 januari 2018 22:57:

    Slechte beleggingsweek voor mij ,het enige is dat het mooi weer is op curacau ,waar ik op vakantie ben,Ik dacht ik verdien mijn vakantie wel terug maar valt nu meer dan 250% duurder uit. Volgende week maar weer afzien en dan over 3 week naar pharming.....
    Het zware leven van de retail belegger in een notendop :)
  15. flosz 24 maart 2018 10:53

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    Practical Implications of Factor IX Gene Transfer for Individuals with #HemophiliaB: A Clinical Perspective www.liebertpub.com/doi/pdf/10.1089/hu...

    GeneTherapy with adeno-associated virus vector 5–human factor IX in adults with #HemophiliaB www.ncbi.nlm.nih.gov/pmc/articles/PMC...
    Bijlage:
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