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Aandeel Pharming Group AEX:PHARM.NL, NL0010391025

Laatste koers (eur) Verschil Volume
0,881   +0,025   (+2,86%) Dagrange 0,843 - 0,882 8.243.635   Gem. (3M) 6,4M

Pharming december 2024

2.971 Posts
Pagina: «« 1 ... 53 54 55 56 57 ... 149 »» | Laatste | Omlaag ↓
  1. forum rang 4 bammie 11 december 2024 09:45
    Pharming Group N.V. Announces Positive Topline Data in Pediatric Clinical Trial of Leniolisib
    December 11, 2024 at 01:00 am EST

    Pharming Group N.V. announced positive topline results of data from its Phase III clinical trial (NCT05438407) evaluating the investigational drug leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kd) inhibitor, in children aged 4 to 11 years with activated phosphoin depositide 3-kinase delta syndrome (APDS). Leniolisib, marketed under the brand name Joenja®? in the U.S., received approval from the U.S. Food and Drug Administration (FDA) for the treatment of APDS in adult and pediatric patients 12 years of age and older in March 2023.

    Pharming plans to include data from this 4-11-year-old trial in regulatory filings worldwide for the approval of leniolisib for pediatric patients with APDS, beginning in 2025. Two hallmarks of APDS, lymphoprol proliferation and abnormal immunophenotype, showed improvement from baseline to 12 weeks in this single arm study. The study enrolled 21 children with APDS ages 4 to 11 years at sites in the United States, Europe, and Japan.

    The single-arm, open-label clinical trial is evaluating the safety, tolerability, and efficacy of leniolisib. The study's primary efficacy endpoints are a reduction in index lymph node size and an increased proportion of passive B cells out of total B cells from baseline at 12 weeks. Eligible patients enrolled in this study are continuing to receive leniolisib for an additional year through an open-label extension trial to further evaluate the safety, tolerability,and efficacy in these patients.

    In addition, a separate Phase III clinical trial including children aged 1 to 6 years with APDS is ongoing to evaluate a new pediatric formulation of leniolisib in this younger population. APDS is a rare primary immunodeficiency that was first characterized in 2013. Results from a randomized, placebo-controlled Phase III clinical trial demonstrated statistically significant improvement in the coprimary endpoints, reflecting a favorable impact on the immune dysregulation and deficiency seen in these patients, and interim open label extension data has supported the safety and tolerability of long-term leniolisib administration.8,9 Leniolisib is currently under regulatory review in the European Economic Area, Canada and Australia for APDS, with plans to pursue further regulatory approvals in Japan and South Korea.

    Leniolisib is also being evaluated in two Phase III clinical trials in children with APDS and in a Phase II clinical trial in primary immunodeficiencies (PIDs) with immune dysregulation linked to altered PI3Kd signaling in lymphocytes. Examples of forward-looking statements may include statements with respect to timing and progress of Pharming's preclinical studies and clinical trials of its product candidates, Pharming's clinical and commercial prospects, and Pharming's expectations regarding its projected working capital requirements and cash resources, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Pharming's clinical trials and ramifications for the cost thereof; and clinical, scientific, commercial, competitive and technical developments.

    Bron:
    www.marketscreener.com/quote/stock/PH...
  2. forum rang 4 Marrr 11 december 2024 09:55
    quote:

    Anna91 schreef op 11 december 2024 09:42:

    [...]
    Dank!

    Vandaag lekker door rijden op de Pharming highway. Nog wel een lange weg te gaan om thuis te komen.
    Ik ben thuis bij hectometerpaaltje 0,93. Maar 't is en blijft Pharming, lidmaatschap Wegenwacht blijft nog steeds aan te raden. ;-)
  3. forum rang 4 MichelH 11 december 2024 10:03
    quote:

    Concrete schreef op 11 december 2024 09:20:

    Laat ik er nu net gisteravond 5000 verkocht hebben, iets wat ik zelden doe, dacht laat mij nu ook eens wat traden ...:)
    Wel erg zonde hoor. Een maand geleden vroeg je nog advies om te blijven in Pharming of over te stappen naar PostNL. Ik reageerde dat ik dat al vreemd vond omdat je zelf de voorspelling deed dat Pharming eind 2024 wel eens naar de €1,40 kon gaan. Maar het blijft een lastige opdracht om bij je standpunt te blijven zeker na een flinke koersstijging. Hoop dat je er wel wat rendement op hebt gemaakt.
2.971 Posts
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