New Data from the Phase 3 HELP Study Open-Label Extension Evaluate the Long-Term Safety and Efficacy of TAKHZYRO® (lanadelumab) in Reducing Hereditary Angioedema Attacks

New Analyses Presented at the European Academy of Allergy and Clinical Immunology Digital Congress

OSAKA, Japan--(BUSINESS WIRE)-- Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced findings from two new interim analyses of data from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study™ Open-label Extension (OLE). The analyses suggest that TAKHZYRO® (lanadelumab) is well-tolerated and can prevent hereditary angioedema (HAE) attacks over an extended treatment period, with sustained and consistent reduction in monthly attack rate across a range of different patient subgroups. The data are being presented at the 2020 European Academy of Allergy and Clinical Immunology (EAACI) Digital Congress.

The original Phase 3 HELP Study was conducted in 125 patients aged 12 years and older over 26 weeks, making it the largest randomised, controlled prevention study in HAE, with the longest active treatment duration, to date.1 The HELP Study OLE was designed to evaluate the long-term safety (primary endpoint) and efficacy of TAKHZYRO for up to 2.5 years and was completed in November 2019. These interim analyses were based on data collected between May 2016 and August 2018 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. At the time of the interim analyses, patients received treatment for a mean duration of 19.7 months (0-26.1).2,3,4

“HAE attacks are unpredictable, often painful and can be debilitating for those living with the disease. These data are exciting as they help us better understand the potential of TAKHZYRO to provide sustained prevention and reduce attack frequency long-term, across a range of patient demographics,” said Donatello Crocetta, M.D., Global Medical Head, Rare Immunology and Metabolic Diseases, Chief Medical Office, Takeda. “The HELP Study OLE analyses add to the evidence that supports TAKHZYRO as a leading option in preventive HAE treatments.”

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 50% of patients (n=106).5 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. A sustained reduction in attack rate was observed in this group, with numerically lower mean monthly attack rates of 0.18 (n=25) during the extended treatment period of the HELP Study OLE and 0.26 (n=25) at the end of the HELP Study.6 The efficacy profile of non-rollover patients was similar to efficacy in rollover patients with two years of cumulative study experience in the HELP Study and HELP Study OLE. The median attack rate reduction was consistent across all subgroups, including patient sex, race, HAE type, age, BMI, history of prophylaxis use, history of laryngeal attacks, and baseline attack rate.7

In the study TEAEs occurred in ~95% of patients (N=212) and were mostly mild or moderate in severity. The TEAEs related to treatment that were reported in more than 5% of patients are injection site pain (33.9% of rollover patients [n=37] and 42.7% of non-rollover patients [n=44]), injection site erythema (11.9% of rollover patients [n=13] and 15.5% of non-rollover patients [n=16]) and injection site bruising (4.6% of rollover patients [n=5] and 9.7% of non-rollover patients [n=10]).5

Lanadelumab is well-tolerated and effective across patient subgroups: findings from the HELP open-label extension study; OAS 21 Recorded scientific content (#1287) In this interim analysis, treatment with TAKHZYRO 300 mg every two weeks was well-tolerated and effectively reduced attack rates over an extended treatment period across different patient demographic and disease characteristics.7

According to the interim analysis, the median attack rate reduction was consistent regardless of patient sex (98.6% reduction in males [n=69], 97.4% females [n=143]; race (97.8% white [n=198], 95.9% non-white [n=14]); HAE type (97.6% type 1 [n=189], 97.4% type 2 [n=21]); age (97.4%

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.