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MWTX-003

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  • Product Information

    Product Information

    Scientific name: MWTX-003
    Brand name: Ν/Α
    RESPONSIBLE: Disc Medicine Inc. and Mabwell Therapeutics

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Trial Name: N/A
    Code: N/A (Τo be initiated in the 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


    • Results & more information: Corporate Presentation (Sept 2025)

     

  • Clinical Trial/Study Information

    Clinical Trial/Study Information

    Trial Name: N/A
    Code: NCT07187973
    Phase: 1b
    Eligible patient diagnosis: Sickle Cell (HbSC or HbSS); age 18 years and older
    No. of Patients enrolled:
    Study Sites: 1 Sites per country

    Anticipated completion date: 2027
    Scope of the Study / Aim: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DISC-3405 in Participants with Sickle Cell Disease


  • Regulatory Information

    Regulatory Information

    Status: Not Authorised

    Additional notable points:

    • EMA: N/A
    • FDA: Received acceptance of an Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) (Nov 2022) / Orphan drug designation for PV (Feb 2025)
    • MHRA: N/A

Update: 22 December 2025

  • Progressing the ongoing Phase 2 study of DISC-3405 in patients with polycythemia vera, with initial data expected in 2026.
  • Initiated Phase 1b study of DISC-3405 in patients with SCD in October 2025, with initial data expected in 2026.

Sources: ASH 2025 Corporate Presentation
Disc Medicine Reports Third Quarter 2025 Financial Results and Provides Business Update – Disc Medicine Inc.
Disc Medicine Provides Update on Hematology Portfolio and Outlines Near-Term Business Objectives and Anticipated Milestones – Disc Medicine Inc.

 

Update: 30 September 2025

Phase 2 study in polycythemia vera initiated; initial data expected in 2026.

Source: https://ir.discmedicine.com/news-releases/news-release-details/disc-medicine-reports-second-quarter-2025-financial-results-and

 

Update: 30 June 2025

  • Data presented at the 30th EHA Annual Congress (12 – 15 June) in Milan, Italy, showed that DISC-3405 was well tolerated with no serious adverse events in a phase 1 study with healthy volunteers. Increases in hepcidin were observed as well as reductions in serum iron, supporting a once-a-month dosing regimen.
  • This data further supports initiating a phase 2 study in polycythemia vera.

Source: SINGLE- AND MULTIPLE-ASCENDING DOSES OF DISC-3405, A RECOMBINANT… – Carden M – EHA-1724 – Jun 14 2025

 

Update: 31 March 2025 

  • FDA has granted orphan drug designation to DISC-3405 for polycythemia vera
  • Phase 2 study in polycythemia vera expected to start in the first half of 2025
  • Preclinical data in sickle cell disease highlight the potential of DISC-3405 to benefit SCD by restricting iron

Sources: https://ir.discmedicine.com/news-releases/news-release-details/disc-medicine-reports-fourth-quarter-and-full-year-2024
https://www.pharmaceutical-technology.com/news/disc-wins-orphan-drug-tag-for-rare-blood-cancer/
https://ir.discmedicine.com/static-files/28eb4a9e-383f-4605-b904-b7cdecc2d19b

 

Update: 19 December 2024

New data

  • Data presented at the 66th ASH Annual Congress (7-10 December 2024) in San Diego (USA):
    • Preliminary findings in healthy volunteers in both single ascending dose (SAD) and multiple ascending dose (MAD) groups showed meaningful and sustained serum iron reductions, a safe and tolerable profile, and no serious adverse events.
    • These results support Disc’s plans to advance the DISC-3405 program into a Phase 2 study in polycythemia vera in 2025.

Source: Phase 1 Healthy Volunteer Study of DISC-3405, a Recombinant Humanized Antibody Targeting TMPRSS6
https://ir.discmedicine.com/news-releases/news-release-details/disc-medicine-presents-positive-clinical-and-translational-data

 

Update: 30 September 2024

  • Data from multiple ascending doses is expected by the end of the year.

Source: https://ir.discmedicine.com/news-releases/news-release-details/disc-medicine-reports-second-quarter-2024-financial-results-and

 

Update: 30 June 2024

  • Data presented at the 29th EHA Annual Congress (13 – 16 June 2024) in Madrid (Spain) showed that the Phase 1 trial in healthy volunteers demonstrated >50% serum iron suppression and sustained hepcidin induction, thus demonstrating clinical proof-of-mechanism as an iron restriction agent.

Source: https://www.stocktitan.net/news/IRON/disc-medicine-presents-positive-clinical-data-across-portfolio-at-7o4rwuahev00.html

 

Update: 31 March 2024

No update available.

 

Update: 20 December 2023

DISC-3405 (formerly MWTX-003) was in-licensed from Mabwell Therapeutics. It received IND approval from the US FDA in November 2022 and was fast-tracked by the FDA for polycythemia vera in September 2023.

Preclinical studies of DISC-3405 have demonstrated an increase in hepcidin production and suppression of serum iron levels in animal models of beta-thalassemia and polycythemia vera.

A phase 1 clinical study in healthy volunteers in October 2023 was initiated.

DISC-3405 could potentially treat multiple iron overload disorders, such a polycythemia vera, beta thalassemia, and other indications.

By inhibiting TMPRSS6, DISC-3405, an anti-TMPRSS6 antibody, is designed to increase the production of hepcidin to therapeutically reduce iron.

Sources: https://www.discmedicine.com/our-pipeline/mat-2-inhibitor/
https://ir.discmedicine.com/news-releases/news-release-details/disc-medicine-reports-third-quarter-2023-financial-results-and
https://ir.discmedicine.com/news-releases/news-release-details/disc-medicine-initiates-phase-1-study-disc-3405-anti-tmprss6-mab

 

Update: 30 September 2023

No update available.

 

Update: 30 June 2023

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

Source: 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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