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Osaka, Japan - Saturday, July 10, 2021[Przelew AETOS]

(business wire) --Takeda Pharmaceutical Co., Ltd.(TSE: 4502 / NYSE: TAK)("Takeda") today announced the results of two final phase 3 analyzes of the Hereditary Angioedema Long-Term Prevention (HELP) study^™An open-label (OLE) extension evaluating 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 (minimum, maximum) reduction in Aggression Index was 87.4% (-100; 852.8) and the median reduction was 97.7% compared to baseline observed in the study population (N=212) %, the mean (standard deviation) duration of treatment patients received was 29.6 (8.2) months.¹At steady-state, from day 70 to the end of the treatment period, aggression indices further decreased to an average of 92.4% and a median reduction to 98.2%.^2,3Another analysis further showed that TAKHZYRO was a well-tolerated drug that prevented HAE episodes in specific subgroups of HAE patients and disease characteristics over an extended 132-week treatment period.³The data will be presented at the Mixed Congress of European Allergy and Clinical Immunology (EAACI) 2021 in Krakow, Poland and Madrid, Spain.

"Hereditary angioedema is a lifelong condition, and research has shown that the fear of another attack can limit a patient's lifestyle," said Markus Magerl, MD, Department of Dermatology, Venereology and Allergology, Charité Medical University Berlin, Germany, said the professor. "The effectiveness of TAKHZYRO in the long-term prevention of HAE episodes will be an important consideration for patients and physicians when developing treatment plans for patients aimed at reducing the number of HAE episodes."

The original Phase 3 HELP study, conducted in 125 patients aged 12 years and over for 26 weeks, is the largest randomized controlled trial of HAE prevention to date and the longest active treatment to date.⁴The HELP OLE study was designed to evaluate the long-term safety (primary endpoint) and efficacy of TAKHZYRO for up to 2.5 years. Full results are based on data collected between May 2016 and October 2019 and include 109 patients with relapse initially assessed in the HELP study and 103 eligible non-transfer patients who did not participate in the initial study but experienced at least one HAE . The patient has had seizures in the last 12 weeks.¹

Seizure-Free Status During Stable Lanadelumab Treatment in Patients with Hereditary Angioedema: Results of the HELP Open-Label Extension Study (ePoster Presentation: Abstract #342)

The results of the HELP OLE study showed that TAKHZYRO was consistently effective in preventing HAE attacks by reducing the frequency of attacks over a treatment period of up to 132 weeks. TAKHZYRO has a half-life of approximately 14 days and is expected to reach steady state in approximately 70 days. The steady-state seizure-free analysis of the HELP OLE study showed that the efficacy of TAKHZYRO 300 mg subcutaneously every two weeks in rolling patients was consistent with the preliminary results of the HELP study.²

Compared to the baseline observed in the study population (N=212), the mean (minimal, maximal) reduction in aggression index was 87.4% (-100; 852.8) and the median reduction was 97.7% (98.0 % reversed, 96.9% irreversible).^1,2Steady-state attack rate is further reduced to an average of 92.4% (-100, 0.44), which includes 92.7% rolls (-100, -40.9) and 91.9% no-rolls (-100 , 0.44), with a median number reduced by 98.2% (98.4% knocked down, 97.3% not knocked down). Within the first six months of treatment after day 70 of the steady-state phase, 58.6% of patients (n=119) were seizure-free, 54.7% relapsed, and 62.9% were seizure-free. The longest duration of seizure-free periods after day 70 was ≥ 6 months in 83.7% and ≥ 12 months in 70%. The median and longest seizure-free periods at steady-state were 14.8 and 18.6 months, respectively, with a maximum seizure-free period of more than 12 months in 70.0% of patients (n=142).²

Long-term prevention of exacerbations in a subgroup of patients with hereditary angioedema (HAE) with lanadelumab: final results of the open-label extension study HELP (oral presentation: abstract #392)

In the OLE analysis of the follow-up HELP study, treatment with TAKHZYRO 300 mg every two weeks was well tolerated and effective over the extended treatment period in reducing HAE type, seizure quality index, history of long-term prophylaxis use, and baseline morbidity.³

The safety profile of TAKHZYRO was comparable across all subgroups evaluated, with 54.7% of patients (n=116) experiencing treatment-related TEAEs, most commonly injection site pain.³

"For over a decade, we've been listening to the HAE community to better understand the need for long-term, targeted preventive therapies and dedicating our resources to developing treatment options," said Neil Inhaber, M.D., vice president, Global Medical, Takeda (Takeda) Director of HAE and Transplantation. "These analyzes further confirm that TAKHZYRO can play an important role in the lives of HAE patients."

O TAKHZYRO® (lanadelumab)

TAKHZYRO® (lanadelumab) is indicated for the routine prophylaxis of recurrent HAE episodes in patients 12 years of age and older. TAKHZYRO is a fully human monoclonal antibody that specifically binds to plasma kallikrein and reduces its activity. TAKHZYRO is produced in Chinese Hamster Ovary (CHO) cells by recombinant DNA technology. 9

TAKHZYRO is for subcutaneous use and has a half-life of approximately two weeks in HAE patients. TAKHZYRO is indicated for self-administration or administration by a healthcare professional only after training by a healthcare professional. 9

TAKHZYRO is not indicated for the treatment of acute episodes of HAE. 9

TAKHZYRO is available in 300 mg vials or pre-filled syringes under regional marketing authorisation. Please check your local prescribing information for more information.

TAKHZYRO European Safety Information

Please refer to the Summary of Product Characteristics (SmPC) of TAKHZYRO before prescribing.

TAKHZYRO treatment should be initiated under the supervision of a physician experienced in the management of patients with hereditary angioedema (HAE). TAKHZYRO should only be self-administered or administered by a caregiver after training by a healthcare professional in the subcutaneous injection technique. 9

Contraindications

Hypersensitivity to the active substance or to any of the excipients. 9

Warnings and measures

Traceability: To improve the traceability of biopharmaceutical products, names and batch numbers of managed products should be clearly recorded. 9

Hypersensitivity reactions have been observed. If severe hypersensitivity reactions occur, TAKHZYRO should be discontinued immediately and appropriate therapy initiated. 9

General: TAKHZYRO is not indicated for the treatment of acute episodes of HAE. In the event of a breakthrough HAE episode, individualized treatment with approved rescue medications should be started. There are no clinical data available on the use of lanadelumab in HAE patients with normal C1-INH activity. 9

Effects on coagulation test results: Due to the interaction of lanadelumab with the aPTT assay, lanadelumab may increase activated partial thromboplastin time (aPTT). The reagent used in the aPTT laboratory test initiates internal coagulation by activating plasma kallikrein in the contact system. Inhibition of plasma kallikrein by lanadelumab increases aPTT in this assay. In patients treated with TAKHZYRO, no increase in APTT was associated with abnormal bleeding adverse events. The international normalized ratio (INR) did not differ between the treatment groups. 9

Sodium content: This medicine contains less than 1 mmol (23 mg) sodium per vial, that is to say essentially sodium free. 9

interactive

No specific drug interaction studies have been performed. Based on the properties of lanadelumab, no pharmacokinetic interactions with co-administered drugs are expected. 9

As expected, based on the mechanism of action (MOA) of lanadelumab and C1 esterase inhibitors, co-administration of the rescue drug C1 esterase inhibitor had an additive effect on lanadelumab-cHMWK response. 9

Immunogenicity

Treatment with lanadelumab was associated with treatment-emergent anti-drug antibodies (ADA) in 11.9% (10/84) of patients. All antibody titers were low. ADA reactions were transient in 20% (2/10) of ADA positive patients. 2.4% (2/84) of patients treated with lanadelumab tested positive for neutralizing antibodies. 9

The development of ADA, including neutralizing antibodies to TAKHZYRO, does not appear to adversely affect the pharmacokinetic (PK) and pharmacodynamic (PD) profiles or clinical response. 9

Side effects

The most common adverse reactions (52.4%) associated with TAKHZYRO were injection site reactions (ISRs), including injection site pain, injection site erythema, and injection site bruising. Of these ISRs, 97% were mild, 90% resolved within 1 day of onset, with a median duration of 6 minutes. 9

Hypersensitivity reactions (mild to moderate itching, tongue discomfort and tingling) have been observed (1.2%)

very often

(incidence ≥ 1/10):

Injection site reactions*

Common

(≥1/100 do <1/10):

Hypersensitivity**, dizziness, maculopapular rash, myalgia, alanine aminotransferase increased, aspartate aminotransferase increased.

*Injection site reactions include: pain, erythema, bruising, discomfort, hematoma, haemorrhage, itching, swelling, induration, paraesthesia, reaction, fever, edema and rash.

** Hypersensitivity reactions include: itching, discomfort and tingling of the tongue.

The Summary of EU Product Characteristics is available at https://www.ema.europa.eu/en/documents/product-information/takhzyro-epar-product-information_en.pdf.

Full US prescribing information, including approved indications and important safety information, can be found at https://www.shirecontent.com/PI/PDFs/TAKHZYRO_USA_ENG.pdf.

O firmie Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TOKYO: 4502 / NYSE: TAK) is a global, values-driven, research and development-driven biopharmaceutical leader based in Japan, dedicated to discovering and delivering life-changing therapies in the spirit of what we do for our patients, our people and planet. Takeda focuses its research and development efforts on four therapeutic areas: oncology, rare genetics and hematology, neurology and gastroenterology (GI). We also make targeted investments in research and development for plasma-derived drugs and vaccines. We focus on developing highly innovative medicines that help transform people's lives by pushing the boundaries of new therapeutic options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees work to improve the quality of life for patients and work with our medical partners in approximately 80 countries. For more information, please visit https://www.takeda.com.

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Medical information

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1 Banerji A, Hao J, Ming Y et al. Long-term efficacy and safety of lanadelumab: final results of the open-label HELP extension study. ACAI 2020.
2 Magerl M, Johnston D et al. Seizure-free status on stable lanadelumab treatment in patients with hereditary angioedema: results of the open-label HELP extension study. EAACI 2021.
3 Maurer M, Bernstein J et al. Long-term seizure prophylaxis with lanadelumab in a subgroup of patients with hereditary angioedema (HAE): final results of the open-label HELP extension study. EAACI 2021.
4 Banerji A, Riedl MA, Bernstein JA, and others; For helping the investigators. Effect of lanadelumab versus placebo on preventing episodes of hereditary angioedema: a randomized clinical trial. Journal of the American Medical Association. 2018;320(20):2108-2121.
5 Cicardi M, Bork K, Caballero T, and others on behalf of HAWK (International Working Group on Hereditary Angioedema). Evidence-based recommendations for the treatment of angioedema due to congenital C1 inhibitor deficiency: an international working group consensus report. allergy. 2012;67(2):147-157.
6 Crane BL. Hereditary angioedema. N English J Med. 2008;359(10):1027-1036.
7 Banerji A. Burden of disease in patients with hereditary angioedema. Ann Allergy Asthma Immunization. 2013;111(5):329-336.
8 Longhurst HJ, Bork K. Hereditary angioedema: causes, symptoms and treatment. Br J Hosp Med. 2006;67(12):654-657.
9 Summary of the European Summary of Product Characteristics for TAKHZYRO (lanadelumab).

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