Ontvang nu dagelijks onze kooptips!

word abonnee
Van beleggers
voor beleggers
desktop iconMarkt Monitor

Aandeel Pharming Group AEX:PHARM.NL, NL0010391025

Laatste koers (eur) Verschil Volume
0,927   -0,002   (-0,22%) Dagrange 0,918 - 0,945 3.918.696   Gem. (3M) 6,7M

Prophylactic possibilities

68 Posts
Pagina: 1 2 3 4 »» | Laatste | Omlaag ↓
  1. jurpsy 1 december 2010 18:28
    1 December 2010

    Data from a small, open label Ruconest (conestat alfa) study (OPERA) testing
    prophylactic therapy has reported. Due to the short half-life, we assumed that Ruconest had no prophylactic efficacy. Surprisingly, attack frequency dropped from 0.6 attacks per week to 0.25, a c 50% reduction. The data is indicative only as this was not a controlled study. It opens the way for Pharming and its partners to run formal prophylactic studies. This could target the current c $165m Cinryze market.

    Trial design

    This was an open-label study in 25 high attack frequency patients. Baseline attackfrequency was assessed on patient medical history over two years. Patients received 50U/kg of Ruconest once weekly for eight weeks.

    Efficacy seen

    From the few reported statistics, all patients had at least one attack every two weeks with a few patients having extraordinarily high attack rates: every few days. Themedian number of attacks per week dropped from 0.6 (averaged over two years) to 0.25 (averaged over eight weeks). Even though the study was observational with a historic baseline and the treated period was short, there seems to be a two-fold effect in reducing attack frequency. This is surprising as the Ruconest half life in
    plasma is only a few hours. One hypothesis is that Ruconest binds to endothelial cell proteins in the capillary walls conferring longer-lasting protection.

    Next stage

    The higher dose of Ruconest – 2,100U per vial vs 1,000U for Cinryze – may have a greater prophylactic advantage; Cinryze is given twice per week. This would need a randomised, controlled cross-over study. Such a trial would have to be co-funded by
    US partner Santarus (in the US) or by sobi (in the EU). Full development of this indication is unlikely in 2011. However, in the EU Berinert is used off-label for prophylaxis. In the US, twice a week Cinryze is approved only for prophylaxis. The reduction in attacks seen is comparable with the results of Pharming’s OPERA study.

    Valuation:

    Encouraging data, unchanged valuation
    Our risk-adjusted NPV indicates €0.71 per share, perhaps €0.56 fully diluted.

    Update

    Balance Sheet as at 31 December 2010e
    Debt/Equity* (%) 65%
    NAV per share**(c) 5.6
    Net cash (€m) 8.4
    * Bonds of €10.9m repaid October leaving loans of
    €13m compared to €20m est year-end equity.
    ** Adjusted for shares in issue 26 August 2010.
    Business
    Pharming, a Dutch company listed on
    Euronext, has focused on Ruconest /
    Rhucin for angioedema, a rare hereditary
    disease. Ruconest is now EU approved
    and will be marketed by sobi and Esteve.
    Kidney transplant trials could start soon.
    The DNage 51% owned subsidiary is
    being divested.

    .
    Exhibit 1: Financials

    Note: Many balance sheet items are carried at fair value, which is discounted with adjustments being taken through the P&L. The
    remaining 2007 bonds current face value of €10.9m plus €0.4m in additional interest was fully redeemed at investor request in October
    2010. We assume €5m is raised during 2010 from the SEDA that, plus €12m equity in H1 and €7.5m from converted private bonds,
    makes €24.5m for 2010; €21.2m of the SEDA currently remains. There are 12.2m cashless warrants (c 6.5m shares) to be converted.
    Ruconest and Rhucin upfront payments (total €14.8m to date) are spread over 10 years, but cash was received in 2010. The sobi
    regulatory milestone is assumed to be fully recognised in 2010. We assume that the $5m (€3.8m) Santarus regulatory milestone in
    respect of BLA acceptance by the FDA will be received and fully recognised in Q111.

    www.edisoninvestmentresearch.co.uk/re...

    suc6,

    jurps
  2. [verwijderd] 1 december 2010 19:20
    Surprisingly, attack frequency dropped …….

    Dat dank je de koekoek.

    Wordt het niet eens tijd dat de Firma Pharming en de door haar betaalde Firma Edison openlijk gaan erkennen dat Ruconest en Cinryze moleculair gelijkwaardig zijn.

    En dat het dan dus helemaal niet Surprisingly is dat Ruconest deze effecten vertoond.

    Sijmen de Vries heeft begin dit jaar gezegd dat als Ruconest als eerste op de Europese markt ging komen, dat Cinryze het wel kon vergeten wegens de Orphan Drug status. MAW Het gaat hier wel degelijk om C1 esterase inhibitor .

    Een misselijkmakende PR dus.

    Zoals ik eerder heb gezegd, Pharming heeft op het verkeerde paard gewed met het acute. Patiënten zijn veel meer gebaat bij het prophylactische, kunnen ze thuis of waar dan ook zelf toedienen.

    En nu probeert Pharming een inhaalslag te maken ?

    groet Camobs
    AB Jurpsy
  3. jurpsy 1 december 2010 19:36
    Na het PB. van 29/11 (Pharming Announces Topline Study Results On Prophylactic Use Of Ruconest In Hereditary Angioedema)heb ik Pharming gevraagd wat uiteindelijk hun bedoeling is met het behaalde inzicht van deze (niet gekontroleerde) studie.

    Dit was het antwoord :

    ................Wij zijn ons op dit moment aan 't beraden wat de meest efficiente strategie is, zowel qua kosten als tijd, voor de verdere ontwikkeling van Ruconest/Rhucin voor profylactisch gebruik. In Europa is het langdurig profylactisch gebruik beperkt tot de meest ernstige gevallen, en Berinert wordt als profylaxis gebruikt "zonder formele indikatie". Het is dus niet zeker of een dure registratie studie de juiste aanpak is voor "Europa".........

    Opmerkelijk feit is toch wel het volgende:

    .... Even though the study was observational with a historic baseline and the treated period was short, there seems to be a two-fold effect in reducing attack frequency. This is surprising as the Ruconest half life in plasma is only a few hours. One hypothesis is that Ruconest binds to endothelial cell proteins in the capillary walls conferring longer-lasting protection......

    jurpsy
  4. [verwijderd] 1 december 2010 20:48
    there seems to be a two-fold effect in reducing attack frequency. This is surprising as the Ruconest half life in plasma is only a few hours. One hypothesis is that Ruconest binds to endothelial cell proteins in the capillary walls conferring longer-lasting protection......

    mooi nieuws jurpsy
    ab!
  5. [verwijderd] 1 december 2010 21:03
    quote:

    ruconestor schreef:

    there seems to be a two-fold effect in reducing attack frequency. This is surprising as the Ruconest half life in plasma is only a few hours. One hypothesis is that Ruconest binds to endothelial cell proteins in the capillary walls conferring longer-lasting protection......

    mooi nieuws jurpsy
    ab!
    En dit vind jij mooi nieuws ?

    Voor iemand die “ moleculair identiek my ass! “ bazuint op dit forum, zou ik mij nog even achter de oren krabben.

    Ter info Cinryze half life is 55 uur.

    Dus als het werkelijk "my ass" is, dan is dit eigenlijk minder mooi nieuws.

    groet Camobs
68 Posts
Pagina: 1 2 3 4 »» | Laatste |Omhoog ↑

Meedoen aan de discussie?

Word nu gratis lid of log in met je emailadres en wachtwoord.