Clinical Trial Updates (SCD)

Ferriprox® (deferiprone)

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  • Product Information

    Product Information

    Scientific name: Deferiprone
    Brand name: Ferriprox®
    RESPONSIBLE: Disc Medicine Inc and Mabwell Therapeutics

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Last update: 22/6/2023

    Trial Name: N/A
    Code: N/A (Τo be initiated in second half of 2023)
    Phase: 1 (Proof-of Mechanism)
    Eligible patient diagnosis: Healthy Volunteers
    No. of Patients enrolled: N/A
    Study Sites: N/A Sites per country

    Anticipated completion date: 2024
    Scope of the Study / Aim: Targets TMPRSS6 with an antibody to enable the increase of hepcidin and reduction of iron

  • Regulatory Information

    Regulatory Information

    Status: Not Authorised

    Additional notable points:

    • FDA: Received acceptance of an Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) in November 2022.

Update: 30 June 2023

  • Phase 1 trial to begin in second half of 2023 with a focus on polycythemia vera to prove therapeutic hypothesis and assess safety. Subsequently Phase 1b proof-of-concept studies can be undertaken in thalassaemia.

Sources:https://ir.discmedicine.com/static-files/549caf12-e7be-45ff-8667-86908e4e6bdd

Update: 31 March 2023

  • Disc Medicine Inc and Mabwell Therapeutics have entered into an agreement to obtain an exclusive license to a portfolio of monoclonal antibodies targeting TMPRSS6 (Transmembrane Serine Protease 6, also known as Matriptase-2) including the phase 1-ready drug candidate MWTX-003.
  • Disc plans to initiate a phase 1 trial in healthy volunteers in the second half of 2023. MWTX-003 has the potential to address a wide range of hematologic disorders including polycythemia vera and beta-thalassemia by controlling iron homeostasis. Genetic studies show that TMPRSS6 affects red blood cell formation by controlling the level of iron that is available for erythropoiesis.
  • Clinical and non-clinical evidence has shown that increasing hepcidin and reducing iron levels by inhibiting TMPRSS6 has potential to treat hematologic disorders.
  • MWTX-003 is phase 1-ready and received acceptance of an Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) in November 2022.
    Sources:
    https://www.globenewswire.com/news-release/2023/01/20/2592372/0/en/Disc-
    Medicine-Announces-Exclusive-Licensing-Agreement-with-Mabwell-Therapeutics-
    for-Novel-Anti-TMPRSS6-Monoclonal-Antibodies-to-Modulate-Iron-
    Homeostasis.html
    https://ir.discmedicine.com/news-releases/news-release-details/disc-medicine-
    reports-first-quarter-2023-financial-results-and
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