Final Results from the Longest Hereditary Angioedema Study of Active Treatment Duration Conducted to Date Support the Sustained Safety and Efficacy of TAKHZYRO (lanadelumab) Injection for Long-Term Prevention of Hereditary Angioedema Attacks

Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”) today announced the publication of the final results from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study Open-label Extension (OLE) designed to evaluate the long-term safety (primary endpoint) and efficacy of TAKHZYRO (lanadelumab) 300mg every two weeks for up to 2.5 years. The study results show that preventative treatment with TAKHZYRO markedly reduced the frequency of hereditary angioedema (HAE) attacks in patients 12 years of age and older who received treatment for a mean duration of almost 2.5 years (29.6 months; 8.2 standard deviation).¹ The data were published online this month in the journal Allergy.

Secondary endpoints of the study showed the mean (min;max) HAE attack rate observed in the study population (N=209) was reduced by 87.4 percent (-100; 852.8) overall versus baseline, and attacks requiring acute treatment (N=106) were reduced by 93.4 percent (-100; -52.8).¹ Reductions were also shown (N=209) in the rate of moderate or severe attacks (84.3 percent). Patients treated with TAKHZYRO 300 mg every two weeks reduced the HAE disease burden by being attack-free for a mean (SD) of 97.7 percent (6.0 percent) of days during treatment, and the average duration of the attack-free period was 14.8 months. Nearly 7 out of 10 patients (68.9 percent) experienced an attack-free period of more than 12 months (n=209). Treatment-related treatment-emergent adverse events (TEAEs) were reported by 54.7 percent of patients (N=116), most commonly injection site reactions. There were no reports of serious treatment-related TEAEs.

The validated Angioedema Quality of Life Questionnaire (AE-QoL) was among the patient-reported outcome tools used to evaluate patients’ quality of life (QoL). Both rollovers and non-rollovers achieved the minimal clinically important difference for the AE-QoL total score: the mean (SD) change in total score from baseline was –10.2 (17.9) and –19.5 (21.3) for rollovers and non-rollovers, respectively. Most of the improvements in AE-QoL scores were observed during the early follow-up period (from day 0 to 56), before reaching a plateau, and scores were generally maintained during subsequent visits.¹