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Ablynx

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  1. [verwijderd] 2 december 2014 16:04
    Ablynx Demonstrates Bioequivalence Between Liquid And Lyophilised Formulations Of Its Anti-Vwf Nanobody, Caplacizumab


    inShare


    12/1/2014 10:14:27 AM

    Caplacizumab has first-in-class potential for the treatment of thrombotic thrombocytopenic purpura (TTP)

    On track to start Phase III study in patients with acquired TTP in mid-2015

    GHENT, Belgium, Nov. 26, 2014 (GLOBE NEWSWIRE) -- Ablynx [Euronext Brussels: ABLX] today announced positive results from the Phase I trial to demonstrate bioequivalence between the liquid and lyophilised formulations of caplacizumab, Ablynx's anti-von Willebrand factor Nanobody developed for the treatment of acquired TTP.

    The liquid formulation has been used in clinical trials to date but the lyophilised form is more stable and can be stored and transported at 5°C, which is much more convenient than the liquid formulation, which has to be stored and shipped frozen. The lyophilised form will now be used in the forthcoming Phase III trial and would be the formulation of choice for commercialisation.

    The Phase I trial involved a single-centre, open-label, randomised, single dose cross-over study in 24 healthy male subjects to evaluate the bioequivalence and tolerability of liquid and reconstituted lyophilised formulations of caplacizumab administered subcutaneously. The bioequivalence criteria were based on the evaluation of the "area under the plasma concentration versus time curve (AUC)" and the "maximum observed plasma concentration (Cmax)".

    The results from this study demonstrate that the lyophilised and liquid formulations of caplacizumab administered subcutaneously are bioequivalent based on the pharmacokinetic parameters as described above. In addition, both formulations were well tolerated and no treatment emergent serious adverse events were reported.

    Dr Edwin Moses, CEO of Ablynx, commented:

    "We are very pleased to show bioequivalence between the liquid and lyophilised formulations of caplacizumab, which is a very important milestone in the development of the product. We believe that this lyophilised form has a number of advantages and will allow for convenient self-administration in the home setting. We are on track to starting the Phase III study with this new lyophilised formulation in patients with acquired TTP in mid-2015."

    About caplacizumab

    Caplacizumab is a bivalent anti-vWF Nanobody which is highly potent and selective. It received Orphan Drug Designation in the US and EU in 2009 and could be the first drug specifically approved for the treatment of acquired TTP as an adjunct to plasma exchange and immunosuppressants. Efficacy and safety were demonstrated for caplacizumab in a worldwide Phase II study in patients with acquired TTP, demonstrating that it could act as a protective agent in the acute phase of the disease, preventing microthrombi and having the potential to reduce morbidity due to organ damage as well as preventing exacerbations. In addition, in this Phase II study, caplacizumab was generally well-tolerated and had a manageable bleeding side effect profile.

    Von Willebrand factor (vWF) is a blood glycoprotein involved in haemostasis, a complex process that causes the bleeding process to stop. vWF's primary function is to bind to other proteins, including glycoprotein Ib in the initiation of platelet adhesion. vWF is implicated in TTP where ultra-large, multimeric precursors of vWF (UL-vWF) are present in the blood of patients leading to unwanted blood clot formation. UL-vWF can readily bind platelets leading to the formation of characteristic string-like clots in small blood vessels. Caplacizumab inhibits platelet binding to UL-vWF and thus has the potential to prevent the formation of these string-like clots in the blood of patients with acquired TTP.

    About thrombotic thrombocytopenic purpura (TTP)

    TTP is a rare disorder of the blood coagulation system that causes extensive microscopic thromboses in small blood vessels throughout the body. It is a potentially life-threatening disorder characterised by thrombocytopenia, haemolytic anaemia and microvascular thrombosis causing variable degrees of tissue ischemia and infarction. TTP exists in two forms: a congenital and an acquired form, with the latter accounting for >90% of the patients. There are currently no drugs specifically approved for the treatment of TTP. The standard of care for the acquired form of TTP is multiple daily plasma exchanges until confirmed platelet normalisation which occurs when the patient's platelet count returns to normal. This treatment requires lengthy hospital stays and may be associated with clinical complications. Additionally, a significant number of patients will subsequently suffer either an exacerbation of the TTP episode or a later relapse after recovering from a first TTP episode. There are believed to be approximately 10,000 TTP-related events in the US and top 15 European markets per year.

    About Ablynx

    Ablynx is a biopharmaceutical company engaged in the development of Nanobodies®, proprietary therapeutic proteins based on single-domain antibody fragments, which combine the advantages of conventional antibody drugs with some of the features of small-molecule drugs. Ablynx is dedicated to creating new medicines which will make a real difference to society. Today, the Company has more than 30 proprietary and partnered programmes in development in therapeutic areas including inflammation, haematology, oncology and respiratory disease. The Company has collaborations and significant partnerships with pharmaceutical companies including AbbVie, Boehringer Ingelheim, Merck & Co, Merck Serono and Novartis. The Company is headquartered in Ghent, Belgium. More information can be found on www.ablynx.com.

    For more information, please contact

    Ablynx:

    Dr Edwin Moses
    CEO

    www.biospace.com/news/ablynx-demonstr...
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  3. Acht 8 december 2014 12:44
    Met dank aan NLFB op Beursig.nl

    Petercam verhoogt zijn advies voor de biotechgroep Ablynx van Add naar Buy, met een hoger koersdoel van 13 euro. Ablynx geeft zichzelf 50 procent kans dat caplacizumab, een veelbelovend middel tegen de bloedziekte TTP, effectief in de winkelrekken komt. Maar volgens Petercam is Ablynx iets te voorzichtig. De voorlopige testresultaten waren erg positief, er zijn geen gelijkaardige medicijnen tegen TTP op de markt en het 'veiligheidsprofiel' van het middel is ook behoorlijk, klinkt het. Ablynx wint een klein procent op 9,20 euro.

    Bron: De Tijd
  4. Cees01 8 december 2014 16:14
    PERSBERICHT: PRESENTATIE VAN BIJKOMENDE KLINISCHE DATA VAN DE FASE II STUDIE MET ABLYNX' ANTI-vWF NANOBODY, CAPLACIZUMAB, TIJDENS DE AMERICAN SOCIETY HEMATOLOGY ANNUAL MEETING

    -- Caplacizumab is een mogelijk eerste-in-zijn-klasse geneesmiddel met
    status van weesgeneesmiddel

    -- Caplacizumab, als aanvulling op plasmaferese en immunosuppressieve
    behandeling, heeft het potentieel om een belangrijk nieuw middel te
    worden voor de behandeling van verworven trombotische trombocytopenische
    purpura (TTP)

    -- Bijkomende data stelt dat caplacizumab de consumptie van bloedplaatjes
    verhindert, waardoor patiënten beschermd zijn tegen verdere vorming
    van microvasculaire tromboses

    -- Sterke Fase II klinische proof-of-concept data gepubliceerd:
    hugin.info/137912/R/1793635/617555.pdf in juni 2014

    Bijkomende Fase II resultaten zullen op donderdag 11 december om 15u
    Belgische tijd tijdens een webcastpresentatie worden toegelicht.

    De webcast is toegankelijk via de home pagina van Ablynx' website
    www.ablynx.com
  5. [verwijderd] 10 december 2014 10:34
    www.bolero.be/nl/analyse-en-inzicht/t...

    De Top Picks voor 2015 van KBC SecuritiesDeel deze pagina:
    Hier is ie dan. Het favorietenlijstje van KBC Securities voor 2015. Op www.bolero.be kunt zoals altijd tekst en uitleg vinden, maar de namen krijgt u van ons wel al cadeau.







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    Naar overzicht
  6. forum rang 5 K. Wiebes 11 december 2014 08:22
    Woensdag het PB en vandaag pas de WC.

    Voelen we hier nu - enigszins bombastisch - de hand van de nieuwe commerciële man, of hebben ze de tijd genomen om vandaag eens groot uit te pakken?

    Wanneer zijn de afspraken met de autoriteiten v.w.b. de conditional approval en - belangrijker nog - wanneer zijn hun uitspraken te verwachten ?

    Komt er duidelijkheid t.a.v. de omvang van de markt voor Capla, zoals zij die zien ?
    Zowel qua verwachting van het aantal behandelingen per jaar als qua omzet; en dan - voor de afwisseling - niét met ranges die 50% uit elkaar liggen. Gezien het beperkte aantal patiënten zou dat toch moeten kunnen.

    Gaat het onder eigen beheer gebeuren of heeft er zich toch, nadrukkelijk
    een partner gemeld ?

    Ik bén benieuwd.
  7. [verwijderd] 11 december 2014 11:37
    Bijkomende Fase II resultaten zullen op donderdag 11 december om 15u
    Belgische tijd tijdens een webcastpresentatie worden toegelicht.

    Vreemd dat de koers nu daalt . Zou verwachten dat we de lucht in zouden schieten .
    Misschien na 3 uur .

    www.bolero.be/nl/analyse-en-inzicht/t...

    Ik ga er van uit dat het komende jaar(en) een top jaar gaat worden .
3.291 Posts
Pagina: «« 1 ... 93 94 95 96 97 ... 165 »» | Laatste |Omhoog ↑

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