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  1. Lingus 29 november 2023 19:28
    quote:

    todra schreef op 29 november 2023 18:30:

    [quote alias=mercurius-adept id=15100344 date=202311282032]
    dit is dan weer ff schrikken:

    US FDA investigating safety risk of CAR-T cancer therapies

    @mercurius Het betreft alleen allogene CAR-T ( donor cellen ) , niet de Autogene CART-T ( eigen patient cellen )
    Denk het niet:

    De FDA heeft vastgesteld dat het risico op T-celmaligniteiten van toepassing is op alle momenteel goedgekeurde BCMA-gerichte en CD19-gerichte genetisch gemodificeerde autologe CAR T-cel-immunotherapieën. T-celmaligniteiten zijn opgetreden bij patiënten die werden behandeld met verschillende producten uit deze klasse.

    Bron (automatisch vertaald): www.fda.gov/vaccines-blood-biologics/...
  2. forum rang 6 mercurius-adept 29 november 2023 20:58
    Leerink analyst:

    Currently, secondary malignancies are listed in the “warnings and precautions” section of every CAR-T therapy’s label. Should the FDA take regulatory action, the Leerink team argued the agency would most likely:

    1. flag the risk in a black-box warning, which is the highest level of a safety alert on a medication’s label. That wouldn’t be too big a problem, given CAR-Ts already have several boxed warnings, including for potentially life-threatening cytokine release syndrome and neuro toxicities.

    2. Alternatively, the FDA could decide to restrict CAR-T use to high-risk patients or to later-line treatment settings, the Leerink analysts said. Such actions would limit these products’ commercial potential in a meaningful way.

    3. “A worst-case scenario is that today’s FDA announcement portends increased scrutiny and higher risk/benefit bar for CAR-T in earlier lines and settings beyond oncology,” the Leerink analysts wrote in their note Tuesday

    www.fiercepharma.com/pharma/fda-inspe...
  3. todra 30 november 2023 10:23
    quote:

    mercurius-adept schreef op 29 november 2023 20:58:

    Leerink analyst:

    Currently, secondary malignancies are listed in the “warnings and precautions” section of every CAR-T therapy’s label. Should the FDA take regulatory action, the Leerink team argued the agency would most likely:

    1. flag the risk in a black-box warning, which is the highest level of a safety alert on a medication’s label. That wouldn’t be too big a problem, given CAR-Ts already have several boxed warnings, including for potentially life-threatening cytokine release syndrome and neuro toxicities.

    2. Alternatively, the FDA could decide to restrict CAR-T use to high-risk patients or to later-line treatment settings, the Leerink analysts said. Such actions would limit these products’ commercial potential in a meaningful way.

    3. “A worst-case scenario is that today’s FDA announcement portends increased scrutiny and higher risk/benefit bar for CAR-T in earlier lines and settings beyond oncology,” the Leerink analysts wrote in their note Tuesday

    www.fiercepharma.com/pharma/fda-inspe...
    @mercurius

    Dit concludeert de FDA : "The FDA also said the benefits of these CAR-T products continue to outweigh their risks, but the agency is weighing potential regulatory actions."

    Dit plaatst dit artikel in het juiste perspectief
  4. Lingus 30 november 2023 11:20
    quote:

    todra schreef op 30 november 2023 10:23:

    [...]

    @mercurius

    Dit concludeert de FDA : "The FDA also said the benefits of these CAR-T products continue to outweigh their risks, but the agency is weighing potential regulatory actions."

    Dit plaatst dit artikel in het juiste perspectief
    Dat geldt voor de CAR-T producten die al op de markt zijn, voor oncologische indicaties. Cabaletta onderzoekt de inzet van CAR-T voor B-cel gemedieerde autoimmuunziektes. Zoals de commentaren aangeven: het kan zijn dat de safety lat dan wat hoger komt te liggen. Ik vind de onderzoeken en de voorlopige resultaten van Cabaletta razend interessant en veelbelovend, maar zolang dit boven de markt hangt blijft er onzekerheid. Misschien overdrijf ik en loop een toch stijgende koers mis, dat is dan maar zo. Succes allen!
  5. Chasse_Patate 4 januari 2024 17:59
    quote:

    Lingus schreef op 20 december 2022 16:35:

    .....

    Gisteren werd een langlopende order op Sellas (SLS) aangetikt; ik heb ze voor $2,00. In augustus op de watchlist gezet, in november liep de fase 3 studie van leidend product galinpepimut-S in acute myeloïde leukemie vertraging van bijna een jaar op. De resultaten worden nu rond de jaarwisseling 2023/2024 verwacht. De reden van de vertraging kan positief uitgelegd worden: de vroege gegevens wezen op een mediane totale overleving (OS) die twee keer langer is dan de gepoolde onderzoekspopulatie. Dat geeft een langere doorlooptijd van de studie. De gegevens zijn geblindeerd, waardoor de langere OS kan liggen aan verbeterde zorg in de controle-arm, óf het ligt aan de doeltreffendheid van galinpepimut-S. Ik hoop natuurlijk op het laatste, en dat zou een bevestiging zijn van de fase 2 resultaten. Extra feitje: CEO en oprichter dr. Angelos Stergiou is tevens mede-oprichter van Anavex. Misschien aardig om een stuk van zijn levensverhaal te lezen: medium.com/authority-magazine/dr-ange...

    .....

    Resultaten staan nu voor Q1.
    Vandaag een duik naar 60 cent na een grote offering.
    Voor wie er nog vertrouwen in heeft een koopkans.
    Maar nog altijd groot risico, doe uw eigen onderzoek.
  6. forum rang 4 Wall Street Trader 14 januari 2024 15:47
    For members who are still interested in Zevra (NASDAQ: ZVRA), here is the latest analyst rating.

    CANTOR Fitzgerald

    EQUITY RESEARCH January 2, 2024
    Price: $6.55
    Price Target: NA
    Rating: Overweight

    Large Cap Pharma, Biopharma, & Biotech

    Zevra Therapeutics, Inc. (ZVRA)

    Company Update

    Don't Miss This One On The Way Up, Why We Like The Stock Heading Into 2024

    We see three things this year that could drive ZVRA's stock to re-rate.

    1. We expect the approval and launch of arimoclomol in 2H24 for the treatment of Niemann-Pick disease type C (NPC), an ultra-rare, genetic, progressive and fatal neurological disease.

    ZVRA is prepared for an AdCom, but it is not certain one will be required. See more details on arimoclomol's resubmission here.
    The advocacy community plans to send a petition to the FDA in support of arimoclomol's approval. See details here.
    If approved, ZVRA expects a bolus of patients, ~70, in its early access program to be converted into commercial patients.

    2. ZVRA could report better than expected sales for OLPRUVA, which is indicated for the treatment of urea cycle disorders (UCDs).

    This product comes from the acquisition of Acer. ZVRA will finish creating a commercial infrastructure, in January, for a previously un-promoted drug which should accelerate sales, in our view. See more details here.

    The integration of Acer is important for arimoclomol because there is an 80-90% overlap between UCD and NPC. ZVRA is also deciding whether or not to move forward with other products in Acer's pipeline, some of which have not been disclosed yet.

    3. We expect ZVRA to report positive topline data from its Phase 2 Idiopathic Hypersomnia (IH) study for KP1077. The Street is underestimating the market opportunity for KP1077, in our view. Notably, KP1077:

    Should receive better scheduling than other stimulants, making it more accessible; physicians we have spoken with think that this is a meaningful competitive advantage for the drug
    Its smooth PK profile should result in fewer CV events allowing higher doses to be given, hence increasing efficacy without compromising safety
    There is a large unmet need for the treatment of IH which opens a meaningful market opportunity for KP1077, if the data are positive.
    ZVRA is also looking at KP1077 for the established indication of narcolepsy as well.

    There is no financing overhang. When ZVRA reported 3023 results it noted that as of 9/30/23 it had $83.4MM in cash, cash equivalents, and investments which supports a cash runway into 2026. Notably, the cash runway forecast does not include commercial revenue from arimoclomol following potential FDA approval 2H24, or the potential sale of the Priority Review Voucher (PRV), which could be received upon approval (could be worth ~$100MM), or any revenue from sales of OLPRUVA.
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