• Phase II data demonstrate that rozanolixizumab was well tolerated by patients with primary ITP across all dose groups • Clinically relevant improvements in platelet count and decrease in immunoglobin G (IgG) levels were observed in all dose groups • Safety, tolerability and efficacy data support Phase III development of rozanolixizumab for primary ITP • Rozanolixizumab’s subcutaneous route of administration could provide a new treatment option for patients with primary ITP
Brussels, Belgium, Atlanta Georgia – 9 December 2019, 1:45 AM CEST – UCB, a global biopharmaceutical company, today announced positive results from a Phase II study (TP0001; NCT02718716) of its novel, first-in-class subcutaneous (SC, under the skin) monoclonal antibody, rozanolixizumab, in patients with primary immune thrombocytopenia (ITP). The data were presented during an oral presentation today at the 61st American Society of Hematology (ASH) Annual Meeting & Exposition in Orlando, Florida.
The Phase II TP0001 study to assess the safety, tolerability and efficacy of rozanolixizumab was designed to explore a multiple dose regimen in order to inform the dosing strategy for further development in ITP. Sixty-six 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 weekly) of SC rozanolixizumab. The total weekly dose was similar in all treatment groups, ranging from 15 to 21 mg/kg.
Clinically relevant improvements (i.e., reaching ≥50x109/L) in platelet count and decreases in immunoglobin G (IgG) levels were observed across all dose groups, with higher response rate (55–67% in 1 x 15 mg/kg and 1 x 20 mg/kg dose groups vs 36–45% in 5 x 4 mg/kg, 3 x 7 mg/kg and 2 x 10 mg/kg dose groups) and shorter time to response achieved by the 1 x 15 mg/kg and 1 x 20 mg/kg rozanolixizumab dose groups.
Results confirm that rozanolixizumab was well tolerated across all dose groups,1 consistent with previous rozanolixizumab studies. The most commonly reported adverse event was headache, with mild-to-moderate headaches seen at higher doses; other reported adverse events included diarrhea and vomiting. These events were usually of short duration and the majority of events resolved without treatment. No patient discontinued the study due to side effects.
“ITP is a severe, often chronic disease that can have a significant, long-term impact on people’s health and quality of life. Despite approved therapies, there is still an urgent need for new treatment options that are well tolerated and provide a sustained increase in platelet count,” said Professor Tadeusz Robak, Professor of Hematology at the Medical University of Lodz, Poland. “These Phase II results for rozanolixizumab suggest the treatment could reduce IgG autoantibody levels and improve platelet count for people living with primary ITP.”
Individuals living with ITP experience unpredictable and debilitating symptoms including spontaneous bruising, bleeding and fatigue that can greatly impact their activities of daily life.4 Additionally, the limited current treatment options for people with ITP can be time-consuming and invasive. There is a need to discover new solutions that have the potential to improve patients’ health outcomes and quality of life. Rozanolixizumab is an advanced SC anti-neonatal Fc receptor (FcRn) therapy currently in clinical development and has the potential to provide a targeted, convenient option to optimize individualized patient care.
“Our research has enabled us to better understand rare, IgG autoantibody-mediated diseases such as ITP, including where gaps exist within the treatment paradigm and the overall patient experience,” said Dr. Iris Loew-Friedrich, Chief Medical Officer, Executive Vice-President, UCB. “These results reaffirm our belief that targeting the neonatal Fc receptor pathway could have the potential to transform the treatment experience for people with ITP. We look forward to expanding this knowledge in Phase III trials in ITP and other patient populations.”