Tadeusz Robak MD, Ph.D. Professor of Hematology Medical University of Lodz, Poland

Rozanolixizumab, an Anti-FcRn Antibody for ITP: Primary Immune Thrombocytopenia

MedicalResearch.com Interview with:

Tadeusz Robak MD, Ph.D. Professor of Hematology Medical University of Lodz, Poland

Dr. Robak

Tadeusz Robak MD, Ph.D.
Professor of Hematology
Medical University of Lodz, Poland

MedicalResearch.com: What is the background for this study? What are the main findings?

  • Rozanolixizumab is an advanced SC anti-neonatal FcRn therapy currently in clinical development which has the potential to provide a targeted, convenient option to optimize individualized patient care.
  • The rozanolixizumab Phase II (TP0001) study was specifically designed to explore multiple dose regimens in order to inform the dosing strategy for further development in primary immune thrombocytopenia (ITP). Patients received either a single dose (1 x 15 mg/kg or 1 x 20 mg/kg) or multiple doses (5 x 4 mg/kg, 3 x 7 mg/kg, 2 x 10 mg/kg) of subcutaneous (SC) rozanolixizumab. The total dose was similar in all treatment groups, ranging from 15 to 21 mg/kg.
  • Rozanolixizumab was well tolerated by patients with primary ITP across all dose groups, consistent with previous rozanolixizumab studies. Additionally, improved platelet counts and reduced immunoglobin G (IgG) levels were seen at all doses and regimens of rozanolixizumab treatment, with higher response rate, higher percentage of responders and shorter time to response achieved by the 1 x 15 mg/kg, 1 x 20 mg/kg and the 3 x 7 mg/kg rozanolixizumab dose groups. These safety, tolerability and efficacy data support the Phase III development in patients with primary ITP.

 MedicalResearch.com: What should readers take away from your report?

  • Rozanolixizumab was shown to be well tolerated in patients with primary ITP, reducing IgG autoantibody levels and providing a sustained increase in platelet count.This is particularly promising for individuals living with ITP who experience severe symptoms including spontaneous bruising, bleeding and fatigue that greatly impact their daily life activities. There is a need for new treatment options, as some patients don’t respond sufficiently to the currently approved therapies and can experience burdensome side effects.
  • The positive Phase II data build on the growing body of evidence that suggest targeting the FcRn pathway could have the potential to transform the treatment experience for people with rare IgG autoantibody-mediated diseases such as primary ITP. 

MedicalResearch.com: What recommendations do you have for future research as a result of this work?

  • We look forward to expanding the knowledge gained from this study in further development of rozanolixizumab in ITP both for rescue and chronic therapy.
  • Rozanolixizumab is also currently being investigated in other rare IgG autoantibody-mediated autoimmune diseases, including myasthenia gravis (MG) and chronic inflammatory demyelinating polyneuropathy (CIDP).

Dr. Robak received research grant from UC

Citation: ASH abstract December 9, 2019

Rozanolixizumab, an Anti-FcRn Antibody: Final Results from a Phase II, Multiple-Dose Study in Patients with Primary Immune Thrombocytopenia 

Tadeusz Robak, MD1, Maciej Kaźmierczak2*, Isidro Jarque3*, Vasile Musteata, MD, PhD4*, Jacek Trelinski1*, Nichola Cooper, MD, MRCP5, Peter Kiessling6*, Ute Massow6*, Franz Woltering6*, Rose Snipes7*, Juan Ke8*, Grant Langdon9*, Birgit Haier6*, James B. Bussel, MD10 and Stephen Jolles11*



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Last Updated on December 19, 2019 by Marie Benz MD FAAD