16 November 2020 - - Japanese biopharmaceutical Takeda Pharmaceutical company Ltd. (TSX: 4502) (NYSE: TAK) has released the final results from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study Open-label Extension showing that Takhzyro (lanadelumab-flyo) helped prevent and reduce the frequency of hereditary angioedema attacks long term in patients 12 years of age and older who received treatment for a mean (standard deviation) duration of 29.6 months, the company said.

Results were consistent with the safety and efficacy of Takhzyro in the pivotal trial.

The mean (min, max) HAE attack rate was reduced by 87.4% (-100; 852.8) overall versus baseline (N=212) and in a pre-specified exploratory endpoint, nearly 70% (68.9%) of patients treated with Takhzyro 300 mg every two weeks experienced an attack-free period of more than 12 months (n=209).

The data are being presented at the 2020 American College of Allergy, Asthma and Immunology (ACAAI) Virtual Annual Scientific Meeting and will also be published in the November issue of ACAAI's journal Annals of Allergy, Asthma and Immunology.

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.

The complete results from the HELP Study OLE showed that the safety profile of Takhzyro was consistent with the original findings from the HELP Study, with treatment-related treatment emergent adverse events (TEAEs) occurring in 54.7% of patients (n=116) and the most common being injection-site pain, respiratory tract infection, or headache.

In addition, data from the HELP Study OLE showed that the efficacy of Takhzyro 300 mg administered subcutaneously every two weeks in rollover patients was consistent with the original findings from the HELP Study.

The mean (min, max) reduction in the attack rate compared to baseline observed in the study population (N=212) was of 87.4% (-100; 852.8), with approximately 93% of patients experiencing at least a 70% reduction of the attack rate.

Additional pre-specified exploratory endpoints measured attack-free periods.