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Poster Networking

Platelet Disorders

PB0716 - VAYHIT: A clinical trial program evaluating the efficacy and safety of the anti-B-cell-activating factor receptor (BAFF-R)/B-cell-depleting monoclonal antibody ianalumab in patients with primary ITP

Monday, June 24, 2024
13:45 – 14:45 ICT
Room: Exhibition Hall

    Presenting Author(s)

  • Waleed Ghanima

    Head of Research and Consultant Hematologist
    Department of Medicine, Østfold Hospital Trust
    Kalnes, Ostfold, Norway

    • Co-Author(s)

  • Nichola Cooper

    Consultant Hematologist
    Hammersmith Hospital
    London, England, United Kingdom

  • FZ

    Francesco Zaja

    Associate Professor
    DSM, University of Trieste and Department of Hematology, Azienda Sanitaria Universitaria Giuliano-Isontina
    Trieste, Friuli-Venezia Giulia, Italy

  • Adam Cuker, MD, MS

    Department of Medicine and Department of Pathology and Laboratory Medicine
    Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
    Philadelphia, Pennsylvania, United States

  • MM

    Marc Michel

    Head of Department
    Department of Internal Medicine, Henri Mondor University Hospital, Assistance Publique-Hôpitaux de Paris, Paris-Est Créteil University
    Créteil, Ile-de-France, France

  • Raymond Wong, MD

    Clinical Associate Professor (honorary)
    The Chinese University of Hong Kong
    Shatin, Hong Kong

  • WB

    Wilma Barcellini

    Physician
    Department of Hematology, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
    Milan, Lombardia, Italy

  • FZ

    Fengkui Zhang

    Director
    State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Anemia Therapeutic Center, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
    Tianjin, Tianjin, China (People's Republic)

  • David J. Kuter, MD, DPhil

    Chief of Hematology
    Hematology Division, Massachusetts General Hospital, Harvard Medical School
    Boston, Massachusetts, United States

  • CB

    Charlotte A. Bradbury

    Consultant Hematologist
    Faculty of Health Sciences, University of Bristol
    Bristol, England, United Kingdom

  • TG

    Tomás José González-López

    Professor
    Department of Hematology, Hospital Universitario de Burgos
    Burgos, Castilla y Leon, Spain

  • PU

    Patrick Urban

    Global Program Clinical Head
    Novartis Pharma AG
    Basel, Basel-Stadt, Switzerland

  • AA

    Alex Allepuz

    Senior Clinical Development Medical Director
    Novartis Pharma AG
    Basel, Basel-Stadt, Switzerland

  • JF

    Justyna Fronczek-Sokol

    Senior Clinical Development Director
    Novartis Pharma AG
    Basel, Basel-Stadt, Switzerland

  • JH

    Jens Haenig

    Global Scientific Director
    Novartis Pharma AG
    Basel, Basel-Stadt, Switzerland

  • PE

    Pascal Edrich

    Global Program Biostatistics Head
    Novartis Pharma AG
    Basel, Basel-Stadt, Switzerland

  • NP

    Nadège Pfender

    Global Study Lead
    Novartis Pharma AG
    Basel, Basel-Stadt, Switzerland

  • HA

    Hanny Al-Samkari

    Classical Hematologist and Clinical Investigator
    Division of Hematology, Massachusetts General Hospital, Harvard Medical School
    Boston, Massachusetts, United States

Background: BAFF levels are elevated in ITP and correlate with disease activity. Ianalumab targets BAFF-R with a unique dual mechanism of action: blockade of BAFF-R-mediated signaling and direct B-cell depletion via enhanced antibody-dependent cellular cytotoxicity. Corticosteroids are first-line ITP treatments with high initial response rates; however, one-third or fewer patients attain sustained remission after discontinuation. Corticosteroids are also associated with significant morbidity. Thrombopoietin receptor agonists (TPO‑RAs) are second-line ITP treatments that often achieve durable responses. However, chronic administration is required in most patients: fewer than one-third maintain durable responses after discontinuation. Treatment after TPO-RAs is not well defined, and options become limited as patients progress through lines of therapy. An unmet need exists in ITP for well-tolerated treatments with disease-modifying potential that safely induce and maintain high response rates beyond treatment completion.

Aims: We report designs of the randomized, double-blind, three-arm Phase III VAYHIT1 (NCT05653349) and VAYHIT2 (NCT05653219) trials and the open-label, single-arm Phase II VAYHIT3 (NCT05885555) trial.

Methods: VAYHIT1 and VAYHIT2 will assess ianalumab versus placebo in addition to first-line corticosteroids and short-term second-line eltrombopag, respectively, in adults with primary ITP. In both trials, patients will be randomized 1:1:1 to intravenous low-dose ianalumab, high-dose ianalumab or placebo every 4 weeks (q4w) for 4 infusions in total. The primary endpoint in both trials is time from randomization to treatment failure (Table). VAYHIT3 will assess intravenous ianalumab q4w for 4 infusions in total in patients with primary ITP who previously received corticosteroid and TPO-RA treatment. The primary endpoint is confirmed response (Table).

Results: As of writing, the VAYHIT trials are recruiting.

Conclusion(s): The VAYHIT program is built on the hypothesis that a short therapy targeting BAFF-R and B cells will potentially modify disease and ameliorate ITP, particularly in earlier treatment lines. Ianalumab is expected to safely induce high response rates that are maintained beyond treatment completion.