New Data from the Phase 3 HELP Study™ Open-Label Extension Evaluating Safety and Efficacy of TAKHZYRO® (lanadelumab) for Hereditary Angioedema Patients to be Presented at European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress

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− Evaluation of Security and Efficacy for as much as 2.5 years with TAKHYZRO is In step with Preliminary Interval of Therapy, Constructing on Rising Physique of Proof on the Lengthy-term Security and Efficacy

− Remaining Affected person Subgroup Evaluation Suggests Reductions of HAE Assaults Throughout Vary of Affected person Demographics and Illness Traits with TAKHZYRO

OSAKA, Japan — Takeda Pharmaceutical Company Limited( TSE:4502/NYSE:TAK) (“Takeda”) right this moment introduced outcomes from two last analyses from the Part 3 HELP (Hereditary Angioedema Lengthy-term Prophylaxis) Research^™ Open-label Extension (OLE), which evaluated the long-term security (major endpoint) and efficacy of TAKHZYRO^® (lanadelumab) 300 mg each two weeks for as much as 2.5 years. Within the first evaluation, the imply (min, max) discount within the assault fee in comparison with baseline noticed within the examine inhabitants (N=212) was of 87.4 p.c (-100; 852.8), and the median discount was 97.7 p.c and sufferers acquired therapy for a imply (normal deviation) period of 29.6 (8.2) months.¹ At regular state – day 70 to the top of the therapy interval – assault charges had been additional decreased to a imply of 92.4 p.c and a median discount of 98.2 p.c.^2,3 A further evaluation additional suggests TAKHZYRO was a well-tolerated therapy that prevented HAE assaults over an prolonged deliberate 132 week therapy interval throughout particular HAE affected person demographic and illness attribute subgroups.³ These knowledge are being introduced on the 2021 European Academy of Allergy and Scientific Immunology (EAACI) Hybrid Congress in Krakow, Poland and Madrid, Spain.

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“Hereditary angioedema is a lifelong situation and analysis reveals that issues about one other assault can restrict the best way sufferers lead their lives,” stated Prof. Markus Magerl, M.D., Division of Dermatology, Venereology and Allergology, Charité – Universitätsmedizin in Berlin, Germany. “The efficacy of TAKHZYRO to stop HAE assaults over the long run will probably be an necessary consideration for sufferers and physicians as they develop a therapy plan for sufferers that’s targeted on lowering the variety of HAE assaults.”

The unique Part 3 HELP Research was performed in 125 sufferers aged 12 years and older over 26 weeks, making it the most important randomized, managed prevention examine in HAE, with the longest lively therapy period, so far.⁴ The HELP Research OLE was designed to guage the long-term security (major endpoint) and efficacy of TAKHZYRO for as much as 2.5 years. The entire outcomes had been based mostly on knowledge collected between Could 2016 and October 2019 and included 109 rollover sufferers who had been initially evaluated within the HELP Research, and 103 eligible non-rollover sufferers who didn’t take part within the preliminary examine however had skilled at the least one HAE assault throughout the earlier 12 weeks.¹

Assault-free standing throughout regular state of lanadelumab therapy in sufferers with hereditary angioedema: findings from the HELP open-label extension examine (Digital Poster Presentation: Summary #342)

Outcomes from the HELP Research OLE discovered that TAKHZYRO sustained efficacy within the prevention of HAE assaults by lowering assault charges in a therapy interval of as much as 132 weeks. TAKHZYRO, which has a half-life of roughly 14 days, is predicted to achieve regular state at roughly 70 days. The HELP Research OLE evaluation of attack-free standing throughout the regular state interval confirmed that the efficacy of TAKHZYRO 300 mg administered subcutaneously each two weeks in rollover sufferers was per the unique findings from the HELP Research.²

The imply (min, max) discount within the assault fee in comparison with baseline noticed within the examine inhabitants (N=212) was of 87.4 p.c (-100; 852.8), and the median discount was 97.7 p.c (98.0 p.c rollovers, 96.9 p.c non-rollovers).^1,2 At regular state, assault charges had been additional decreased to a imply of 92.4 p.c (-100, 0.44) comprised of 92.7 p.c rollovers (-100, -40.9) and 91.9 p.c non-rollovers (-100, 0.44) and a median discount of 98.2 p.c (98.4 p.c rollovers, 97.3 p.c non-rollovers). Through the first six months of therapy after day 70 throughout the steady-state interval, 58.6 p.c of sufferers (n=119) had been attack-free, 54.7 p.c rollovers and 62.9 p.c non-rollovers. The utmost period of attack-free interval after day 70 ≥ 6 months was 83.7 p.c and ≥ 12 months was 70 p.c. The technique of the common and most period of attack-free interval throughout regular state had been 14.8 and 18.6 months, respectively, with 70.0 p.c of sufferers (n=142) having a most period of attack-free interval better than 12 months.²

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Lengthy-term prevention of assaults with lanadelumab throughout subgroups of sufferers with hereditary angioedema (HAE): last outcomes from the HELP open-label extension examine (Oral Presentation: Summary #392)

In an extra HELP Research OLE evaluation, therapy with TAKHZYRO 300 mg each two weeks was well-tolerated and successfully decreased assault charges over an prolonged therapy interval throughout completely different affected person demographic and illness traits, together with affected person age, gender, race, HAE sort, physique mass index, historical past of long-term prophylaxis use, and baseline assault fee.³

The security profile of TAKHZYRO was comparable throughout all evaluated subgroups with treatment-related TEAEs occurring in 54.7 p.c of sufferers (n=116) and the commonest being injection-site ache.³

“For greater than a decade, we’ve listened to the HAE neighborhood to additional perceive the necessity for long-term, preventive focused therapies and have dedicated our sources to growing therapy choices,” stated Neil Inhaber, M.D., Vice President, World Medical Head, HAE and Transplant at Takeda. “These analyses additional assert the necessary position TAKHZYRO can play within the lives of people that stay with HAE.”

About The HELP Research^™ Open-label Extension

The HELP (Hereditary Angioedema Lengthy-term Prophylaxis) Research^™ Open-label Extension (OLE) is an analysis of the long-term efficacy and security of TAKHZYRO in hereditary angioedema (HAE) sufferers of at the least 12 years of age and older. 2 hundred and twelve sufferers acquired therapy with TAKHZYRO at the beginning of the OLE Research (109 rollover sufferers initially evaluated within the HELP Research and who continued into the OLE, and 103 eligible sufferers who didn’t take part within the HELP Research however who had skilled at the least one assault within the final 12 weeks). Rollover sufferers acquired a dose of 300 mg TAKHZYRO on Day 0 after which each two weeks after their first assault. Non-rollover sufferers had been handled with one 300 mg dose each two weeks, starting on Day 0. 100 and ninety-six individuals accomplished at the least 12 months of therapy and 173 individuals accomplished at the least 30 months of therapy.¹

About Hereditary Angioedema

Hereditary angioedema (HAE) is a uncommon genetic dysfunction that ends in recurring assaults of oedema – swelling – in numerous components of the physique, together with the stomach, face, toes, genitals, palms and throat. The swelling might be debilitating and painful.^5,6,7 Assaults that impede the airways may cause asphyxiation and are probably life threatening.^7,8 HAE impacts an estimated 1 in 50,000 folks worldwide. It’s typically beneath acknowledged, beneath recognized and beneath handled.^5,7,8

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Takeda in Hereditary Angioedema

Hereditary Angioedema (HAE), like so many different uncommon ailments, is very advanced, and sufferers, their households and caregivers typically endure years of pressure attempting to know their illness, get a definitive prognosis and achieve entry to the medicines they want. At Takeda we’re a dedicated champion for the sufferers we serve. Each particular person residing with HAE is exclusive and by listening and reacting to their wants, we translate the insights we achieve into revolutionary options – from prognosis to ongoing administration. Advancing the science is essential to the best way we function and we’re daring in our mission to speed up prognosis and develop therapies that can make a distinction to the lives of HAE sufferers, their help networks and people medical professionals who take care of them.

About TAKHZYRO^® (lanadelumab)

TAKHZYRO^® (lanadelumab) is indicated for routine prevention of recurrent assaults HAE in sufferers aged 12 years and older. TAKHZYRO is a completely human monoclonal antibody that particularly binds and reduces plasma kallikrein exercise. TAKHZYRO is produced in Chinese language Hamster Ovary (CHO) cells by recombinant DNA know-how.⁹

TAKHZYRO is formulated for subcutaneous administration and has a half-life of roughly two weeks in sufferers with HAE. TAKHZYRO is meant for the self-administration or administration by a caregiver, solely after coaching by a healthcare skilled.⁹

TAKHZYRO just isn’t supposed for therapy of acute HAE assaults.⁹

Relying on regional advertising and marketing authorization, TAKHZYRO is on the market as a 300 mg dose in a vial or pre-filled syringe. Please seek the advice of native prescribing info for extra info.

TAKHZYRO Security Info for Europe

Please seek the advice of the TAKHZYRO Summary Product Characteristics (SmPC) earlier than prescribing.

TAKHZYRO therapy must be initiated beneath the supervision of a doctor skilled within the administration of sufferers with hereditary angioedema (HAE). TAKHZYRO could also be self-administered or administered by a caregiver solely after coaching on SC injection approach by a healthcare skilled.⁹

Contraindication

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

Warnings and Precautions

Traceability: As a way to enhance the traceability of organic medicinal merchandise, the identify and the batch variety of the administered product must be clearly recorded.⁹

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Hypersensitivity reactions have been noticed. In case of a extreme hypersensitivity response, administration of TAKHZYRO should be stopped instantly and applicable therapy should be initiated.⁹

Normal: TAKHZYRO just isn’t supposed for therapy of acute HAE assaults. In case of a breakthrough HAE assault, individualized therapy must be initiated with an authorized rescue medicine. There are not any accessible medical knowledge on the usage of lanadelumab in HAE sufferers with regular C1-INH exercise.⁹

Interference with coagulation take a look at: Lanadelumab can improve activated partial thromboplastin time (aPTT) because of an interplay of lanadelumab with the aPTT assay. The reagents used within the aPTT laboratory take a look at provoke intrinsic coagulation by way of the activation of plasma kallikrein within the contact system. Inhibition of plasma kallikrein by lanadelumab can improve aPTT on this assay. Not one of the will increase in aPTT in sufferers handled with TAKHZYRO had been related to irregular bleeding antagonistic occasions. There have been no variations in worldwide normalised ratio (INR) between therapy teams.⁹

Sodium content material: This medicinal product incorporates lower than 1 mmol sodium (23 mg) per vial, that’s to say primarily ‘sodium-free’.⁹

Interactions

No devoted drug-drug interplay research have been performed. Based mostly on the traits of lanadelumab, no pharmacokinetic interactions with co-administered medicinal merchandise is predicted.⁹

As anticipated, concomitant use of the rescue medicine C1 esterase inhibitor ends in an additive impact on lanadelumab-cHMWK response based mostly on the mechanism of motion (MOA) of lanadelumab and C1 esterase inhibitor.⁹

Immunogenicity

Therapy with lanadelumab has been related to growth of therapy emergent anti-drug antibodies (ADA) in 11.9% (10/84) of topics. All antibody titres had been low. The ADA response was transient in 20% (2/10) of ADA constructive topics. 2.4% (2/84) of lanadelumab-treated topics examined constructive for neutralizing antibodies.⁹

The event of ADA together with neutralising antibodies towards TAKHZYRO didn’t seem to adversely have an effect on the pharmacokinetic (PK) and pharmacodynamics (PD) profiles or medical response.⁹

Hostile Reactions

Essentially the most generally noticed antagonistic response (52.4%) related to TAKHZYRO was injection website reactions (ISR) together with injection website ache, injection website erythema and injection website bruising. Of those ISRs, 97% had been of delicate depth, 90% resolved inside 1 day after onset with a median period of 6 minutes.⁹

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Hypersensitivity response (delicate and reasonable pruritus, discomfort and tingling of tongue) was noticed (1.2%)

For European Union Abstract of Product Traits, please go to https://www.ema.europa.eu/en/documents/product-information/takhzyro-epar-product-information_en.pdf.

For full U.S. Prescribing Info, together with the authorized indication and necessary security info, please go tohttps://www.shirecontent.com/PI/PDFs/TAKHZYRO_USA_ENG.pdf.

About Takeda Pharmaceutical Firm Restricted

Takeda Pharmaceutical Firm Restricted ( TSE: 4502/NYSE: TAK) is a world, values-based, R&D-driven biopharmaceutical chief headquartered in Japan, dedicated to find and ship life-transforming therapies, guided by our dedication to sufferers, our folks and the planet. Takeda focuses its R&D efforts on 4 therapeutic areas: Oncology, Uncommon Genetics and Hematology, Neuroscience, and Gastroenterology (GI). We additionally make focused R&D investments in Plasma-Derived Therapies and Vaccines. We’re specializing in growing extremely revolutionary medicines that contribute to creating a distinction in folks’s lives by advancing the frontier of latest therapy choices and leveraging our enhanced collaborative R&D engine and capabilities to create a strong, modality-diverse pipeline. Our workers are dedicated to enhancing high quality of life for sufferers and to working with our companions in well being care in roughly 80 international locations. For extra info, go to https://www.takeda.com.

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¹ Banerji A, Hao J, Ming Y et al; Lengthy-Time period Efficacy and Security of Lanadelumab: Remaining Outcomes from the HELP Open-Label Extension Research. ACAAI 2020.
² Magerl M, Johnston D, et al. Assault-free standing throughout regular state of lanadelumab therapy in sufferers with hereditary angioedema: findings from the HELP open-label extension examine.EAACI 2021.
³ Maurer M, Bernstein J, et al. Lengthy-term prevention of assaults with lanadelumab throughout subgroups of sufferers with hereditary angioedema (HAE): last outcomes from the HELP open-label extension examine. EAACI 2021.
⁴ Banerji A, Riedl MA, Bernstein JA, et al; for the HELP Investigators. Impact of lanadelumab in contrast with placebo on prevention of hereditary angioedema assaults: a randomized medical trial. JAMA. 2018;320(20):2108-2121.
⁵ Cicardi M, Bork Ok, Caballero T, et al; on behalf of HAWK (Hereditary Angioedema Worldwide Working Group). Proof-based suggestions for the therapeutic administration of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an Worldwide Working Group. Allergy. 2012; 67(2):147-157.
⁶ Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.
⁷ Banerji A. The burden of sickness in sufferers with hereditary angioedema. Ann Allergy Bronchial asthma Immunol. 2013;111(5):329-336.
⁸ Longhurst HJ, Bork Ok. Hereditary angioedema: causes, manifestations, and therapy. Br J Hosp Med. 2006;67(12):654-657.
⁹ TAKHZYRO (lanadelumab) European Abstract of Product Traits.

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