Takeda Pharmaceutical Company Limited announces the final results from the Phase 3 HELP Study Open-label Extension (OLE) showing that Takhzyro (lanadelumab-flyo) helped prevent and reduce the frequency of HAE attacks long term in patients 12 years of age and older who received treatment for a mean (standard deviation) duration of 29.6 months.
Results are consistent with the safety and efficacy of Takhzyro in the pivotal trial. The mean HAE attack rate was reduced by 87.4% overall versus baseline and in a pre-specified exploratory endpoint, nearly 70% of patients treated with Takhzyro 300 mg every two weeks experienced an attack-free period of more than 12 months.
“The unpredictability of HAE attacks has a significant impact on the lives of HAE patients. HAE is a lifelong condition, so reducing the frequency and severity of attacks is an important therapeutic goal for many individuals living with HAE,” says Marc A. Riedl, M.D., investigator in the HELP Study OLE and Professor of Medicine and Clinical Director, U.S. HAEA Center at the University of California, San Diego. “The original placebo-controlled HELP Study demonstrated the efficacy and safety of Takhzyro over 26 weeks. The results from the open-label extension study are encouraging as they show that Takhzyro may help prevent attacks over the long term with continued treatment.”
The original Phase 3 HELP Study was conducted in 125 patients aged 12 years and older over 26 weeks, making it the largest randomized, controlled prevention study in HAE, with the longest active treatment duration, to date. The HELP Study OLE was designed to evaluate the long-term safety (primary endpoint) and efficacy of Takhzyro for up to 2.5 years. The complete results were based on data collected between May 2016 and October 2019 and included 109 rollover patients who were originally evaluated in the HELP Study, and 103 eligible non-rollover patients who did not participate in the initial study but had experienced at least one HAE attack in 12 weeks.
“A significant amount of progress has been made in advancing the science to better understand and treat HAE over recent years. In 2018, we received the first regulatory approvals for Takhzyro as a first-of-its-kind monoclonal antibody preventive therapy in HAE, and we have already seen the difference it has made by preventing attacks in many patients around the world,” says Donatello Crocetta, M.D., Global Medical Head, Rare Immunology and Metabolic Diseases, Chief Medical Office, Takeda. “Continued research such as the HELP Study OLE is critical to further build our understanding of the potential of Takhzyro as a long-term preventive treatment option for those living with HAE.”
The complete results from the HELP Study OLE show that the safety profile of Takhzyro is consistent with the original findings from the HELP Study, with treatment-related treatment emergent adverse events (TEAEs) occurring in 54.7% of patients and the most common being injection-site pain, respiratory tract infection, or headache. In addition, data from the HELP Study OLE show that the efficacy of Takhzyro 300 mg administered subcutaneously every two weeks in rollover patients is consistent with the original findings from the HELP Study. The mean reduction in the attack rate compared to baseline observed in the study population is of 87.4%, with approximately 93% of patients experiencing at least a 70% reduction of the attack rate. Additional pre-specified exploratory endpoints measured attack-free periods.