Final Patient Subgroup Analysis Suggests Reductions of HAE Attacks Across Range of Patient Demographics and Disease Characteristics with TAKHZYRO

OSAKA, JAPAN-- July 12, 2021 -- Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced results from two final analyses from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study™ Open-label Extension (OLE), which evaluated the long-term safety (primary endpoint) and efficacy of TAKHZYRO® (lanadelumab) 300 mg every two weeks for up to 2.5 years. In the first analysis, the mean (min, max) reduction in the attack rate compared to baseline observed in the study population (N=212) was of 87.4 percent (-100; 852.8), and the median reduction was 97.7 percent and patients received treatment for a mean (standard deviation) duration of 29.6 (8.2) months.[1] At steady state - day 70 to the end of the treatment period - attack rates were further reduced to a mean of 92.4 percent and a median reduction of 98.2 percent.[2,3] An additional analysis further suggests TAKHZYRO was a well-tolerated treatment that prevented HAE attacks over an extended planned 132 week treatment period across specific HAE patient demographic and disease characteristic subgroups.[3] These data are being presented at the 2021 European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress in Krakow, Poland and Madrid, Spain.

“Hereditary angioedema is a lifelong condition and research shows that concerns about another attack can limit the way patients lead their lives,” said Prof. Markus Magerl, M.D., Department of Dermatology, Venereology and Allergology, Charité - Universitätsmedizin in Berlin, Germany. “The efficacy of TAKHZYRO to prevent HAE attacks over the long term will be an important consideration for patients and physicians as they develop a treatment plan for patients that is focused on reducing the number of HAE attacks.”

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.[4] 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 within the previous 12 weeks.[1]

Attack-free status during steady state of lanadelumab treatment in patients with hereditary angioedema: findings from the HELP open-label extension study (Electronic Poster Presentation: Abstract #342)

Results from the HELP Study OLE found that TAKHZYRO sustained efficacy in the prevention of HAE attacks by reducing attack rates in a treatment period of up to 132 weeks. TAKHZYRO, which has a half-life of approximately 14 days, is expected to reach steady state at approximately 70 days. The HELP Study OLE analysis of attack-free status during the steady state period 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.[2]

The mean (min, max) reduction in the attack rate compared to baseline observed in the study population (N=212) was of 87.4 percent (-100; 852.8), and the median reduction was 97.7 percent (98.0 percent rollovers, 96.9 percent non-rollovers).[1,2] At steady state, attack rates were further reduced to a mean of 92.4 percent (-100, 0.44) comprised of 92.7 percent rollovers (-100, -40.9) and 91.9 percent non-rollovers (-100, 0.44) and a median reduction of 98.2 percent (98.4 percent rollovers, 97.3 percent non-rollovers). During the first six months of treatment after day 70 during the steady-state period, 58.6 percent of patients (n=119) were attack-free, 54.7 percent rollovers and 62.9 percent non-rollovers. The maximum duration of attack-free period after day 70 ≥ 6 months was 83.7 percent and ≥ 12 months was 70 percent. The means of the average and maximum duration of attack-free period during steady state were 14.8 and 18.6 months, respectively, with 70.0 percent of patients (n=142) having a maximum duration of attack-free period greater than 12 months.[2]

Long-term prevention of attacks with lanadelumab across subgroups of patients with hereditary angioedema (HAE): final results from the HELP open-label extension study (Oral Presentation: Abstract #392)

In a further HELP Study OLE 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, including patient age, gender, race, HAE type, body mass index, history of long-term prophylaxis use, and baseline attack rate.[3]

The safety profile of TAKHZYRO was comparable across all evaluated subgroups with treatment-related TEAEs occurring in 54.7 percent of patients (n=116) and the most common being injection-site pain.[3]

“For more than a decade, we’ve listened to the HAE community to further understand the need for long-term, preventive targeted therapies and have committed our resources to developing treatment options,” said Neil Inhaber, M.D., Vice President, Global Medical Head, HAE and Transplant at Takeda. “These analyses further assert the important role TAKHZYRO can play in the lives of people who live with HAE.”