Business Wire

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

Share

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

“Effective prevention backed by clinical evidence is critical for healthcare professionals who treat patients with HAE,” said Aleena Banerji, M.D., Division of Rheumatology, Allergy and Immunology, Department of Medicine, Massachusetts General Hospital, Harvard Medical School and principal investigator for the HELP Study. “The potential to be attack-free for periods of time can help to provide an additional sense of assurance for those living with this chronic and unpredictable disease."

The original Phase 3 HELP Study was conducted in 125 patients aged 12 years and older over 26 weeks, making it one of the largest randomized, controlled prevention studies in HAE, with the longest active treatment duration, to date.2,3,4,5,6 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 in 12 weeks.1 The complete 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 TEAEs occurring in 54.7 percent of patients (n=116) and the most common being injection-site pain, upper respiratory tract infection, or headache.1

“This study supports the use of TAKHZYRO as a long-term preventative treatment option for those 12 years of age and older living with HAE who are seeking a preventative treatment option that is proven to reduce HAE attacks,” said Neil Inhaber, M.D., Vice President, Global Medical Heald, HAE and Transplant at Takeda. “Takeda has more than 10 years supporting people with this rare disease across the HAE portfolio and we are committed to providing these patients with effective treatment options that may help them experience periods of time without attacks. Our legacy and dedication to HAE hopefully empowers patients to confidently navigate their HAE journey.”

About the HELP Study™ Open-label Extension

The HELP (Hereditary Angioedema Long-term Prophylaxis) Study™ Open-label Extension (OLE) is an evaluation of the long-term efficacy and safety of TAKHZYRO in hereditary angioedema (HAE) patients of at least 12 years of age and older. Two hundred and twelve patients received treatment with TAKHZYRO at the start of the OLE Study (109 rollover patients originally evaluated in the HELP Study and who continued into the OLE, and 103 eligible patients who did not participate in the HELP Study but who had experienced at least one attack in the last 12 weeks). Rollover patients received a dose of 300 mg TAKHZYRO on Day 0 and then every two weeks after their first attack. Non-rollover patients were treated with one 300 mg dose every two weeks, beginning on Day 0. The majority of patients (92.5%; N=196) completed at least 12 months in the study, and 81.6% (N=173) completed at least 30 months.1

About Hereditary Angioedema

Hereditary angioedema (HAE) is a rare genetic disorder that results in recurring attacks of oedema – swelling – in various parts of the body, including the abdomen, face, feet, genitals, hands and throat. The swelling can be debilitating and painful.7,8,9 Attacks that obstruct the airways can cause asphyxiation and are potentially life threatening.9,10 HAE affects an estimated 1 in 50,000 people worldwide. It is often under recognized, under diagnosed and under treated.7,9,10

Takeda in Hereditary Angioedema

Hereditary Angioedema (HAE), like so many other rare diseases, is highly complex, and patients, their families and caregivers often undergo years of strain trying to understand their disease, get a definitive diagnosis and gain access to the medicines they need. At Takeda we are committed to be a champion for the patients we serve. Every individual living with HAE is unique and by listening and reacting to their needs, we translate the insights we gain into innovative solutions – from diagnosis to ongoing management. Advancing the science is crucial to the way we operate and we are bold in our mission to accelerate diagnosis and develop treatments that will make a difference to the lives of HAE patients, their support networks and those medical professionals who care for them.

About TAKHZYRO® (lanadelumab) Injection

TAKHZYRO is a fully human monoclonal antibody that specifically binds and decreases plasma kallikrein and is indicated for prophylaxis to prevent HAE attacks in patients 12 years and older. TAKHZYRO is formulated for subcutaneous administration and has a half-life of approximately two weeks.3 TAKHZYRO is intended for self-administration or administration by a caregiver. The patient or caregiver should be trained by a healthcare professional.7

TAKHZYRO Safety Information for Europe

Please consult the TAKHZYRO Summary Product Characteristics (SmPC) before prescribing.

TAKHZYRO treatment should be initiated under the supervision of a physician experienced in the management of patients with hereditary angioedema (HAE). TAKHZYRO may be self-administered or administered by a caregiver only after training on SC injection technique by a healthcare professional.3

Contraindication

Hypersensitivity to the active substance or to any of the excipients.3

Warnings and Precautions

Traceability: In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded.3

Hypersensitivity reactions have been observed. In case of a severe hypersensitivity reaction, administration of TAKHZYRO must be stopped immediately and appropriate treatment must be initiated.3

General: TAKHZYRO is not intended for treatment of acute HAE attacks. In case of a breakthrough HAE attack, individualized treatment should be initiated with an approved rescue medication. There are no available clinical data on the use of lanadelumab in HAE patients with normal C1-INH activity.3

Interference with coagulation test: Lanadelumab can increase activated partial thromboplastin time (aPTT) due to an interaction of lanadelumab with the aPTT assay. The reagents used in the aPTT laboratory test initiate intrinsic coagulation through the activation of plasma kallikrein in the contact system. Inhibition of plasma kallikrein by lanadelumab can increase aPTT in this assay. None of the increases in aPTT in patients treated with TAKHZYRO were associated with abnormal bleeding adverse events. There were no differences in international normalised ratio (INR) between treatment groups.3

Sodium content: This medicinal product contains less than 1 mmol sodium (23 mg) per vial, that is to say essentially 'sodium-free'.3

Interactions

No dedicated drug-drug interaction studies have been conducted. Based on the characteristics of lanadelumab, no pharmacokinetic interactions with co-administered medicinal products is expected.3

As expected, concomitant use of the rescue medication C1 esterase inhibitor results in an additive effect on lanadelumab-cHMWK response based on the mechanism of action (MOA) of lanadelumab and C1 esterase inhibitor.3

Immunogenicity

Treatment with lanadelumab has been associated with development of treatment emergent anti-drug antibodies (ADA) in 11.9% (10/84) of subjects. All antibody titres were low. The ADA response was transient in 20% (2/10) of ADA positive subjects. 2.4% (2/84) of lanadelumab-treated subjects tested positive for neutralizing antibodies.3

The development of ADA including neutralising antibodies against TAKHZYRO did not appear to adversely affect the pharmacokinetic (PK) and pharmacodynamics (PD) profiles or clinical response.3

Adverse Reactions

The most commonly observed adverse reaction (52.4%) associated with TAKHZYRO was injection site reactions (ISR) including injection site pain, injection site erythema and injection site bruising. Of these ISRs, 97% were of mild intensity, 90% resolved within 1 day after onset with a median duration of 6 minutes.3

Hypersensitivity reaction (mild and moderate pruritus, discomfort and tingling of tongue) was observed (1.2%)

Very common

(frequency ≥1/10):

Injection site reactions*

Common

(≥1/100 to <1/10):

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

*Injection site reactions include: pain, erythema, bruising, discomfort, haematoma, haemorrhage, pruritus, swelling, induration, paraesthesia, reaction, warmth, oedema and rash.

** Hypersensitivity includes: pruritus, discomfort and tingling of tongue.

For European Union Summary of Product Characteristics, please visit https://www.ema.europa.eu/en/documents/product-information/takhzyro-epar-product-information_en.pdf.

For full U.S. Prescribing Information, including the approved indication and important safety information, please visithttps://www.shirecontent.com/PI/PDFs/TAKHZYRO_USA_ENG.pdf.

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people’s lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions. For more information, visit https://www.takeda.com.

Important Notice

For the purposes of this notice, “press release” means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (“Takeda”) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws.

The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, “Takeda” is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words “we”, “us” and “our” are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.

Forward-Looking Statements

This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takeda’s future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as “targets”, “plans”, “believes”, “hopes”, “continues”, “expects”, “aims”, “intends”, “ensures”, “will”, “may”, “should”, “would”, “could” “anticipates”, “estimates”, “projects” or similar expressions or the negative thereof. These forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding Takeda’s global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations, including global health care reforms; challenges inherent in new product development, including uncertainty of clinical success and decisions of regulatory authorities and the timing thereof; uncertainty of commercial success for new and existing products; manufacturing difficulties or delays; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on Takeda and its customers and suppliers, including foreign governments in countries in which Takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to Takeda’s operations and the timing of any such divestment(s); and other factors identified in Takeda’s most recent Annual Report on Form 20-F and Takeda’s other reports filed with the U.S. Securities and Exchange Commission, available on Takeda’s website at: https://www.takeda.com/investors/sec-filings/ or at www.sec.gov. Takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results or statements of Takeda in this press release may not be indicative of, and are not an estimate, forecast, guarantee or projection of Takeda’s future results.

References


1 Banerji A, Bernstein JA, Johnston DT, et al; for HELP OLE Investigators. Long-term prevention of hereditary angioedema attacks with lanadelumab: the HELP OLE Study. Allergy. 2021 Jul 21. doi: 10.1111/all.15011. Epub ahead of print.

2 Banerji A, Riedl MA, Bernstein JA, et al; for the HELP Investigators. Effect of lanadelumab compared with placebo on prevention of hereditary angioedema attacks: a randomized clinical trial. JAMA. 2018;320(20):2108-2121.

3 TAKHZYRO (lanadelumab) European Summary of Product Characteristics.

4 CINRYZE (C1 esterase inhibitor [human]) [prescribing information]. Lexington, MA: Shire ViroPharma Incorporated; 2018.

5 HAEGARDA [prescribing information]. Kankakee, IL: CSL Behring LLC; 2017.

6 Craig T, Zuraw B, Longhurst H, et al. Long-term outcomes with subcutaneous C1-inhibitor replacement therapy for prevention of hereditary angioedema attacks. J Allergy Clin Immunol Pract. 2019;7(6):1793-1802.

7 Cicardi M, Bork K, Caballero T, et al; on behalf of HAWK (Hereditary Angioedema International Working Group). Evidence-based recommendations for the therapeutic management of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an International Working Group. Allergy. 2012; 67(2):147-157.

8 Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.

9 Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.

10 Longhurst HJ, Bork K. Hereditary angioedema: causes, manifestations, and treatment. Br J Hosp Med. 2006;67(12):654-657.

PromoMats job code: C-ANPROM/INT/TAKH/0019
Date of preparation: July 2021

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Media Contacts:
Japanese Media
Ryoko Matsumoto
ryoko.matsumoto@takeda.com
+81 (0) 3-3278-3414

Media outside Japan
Linda Calandra
Linda.calandra1@takeda.com
+1 617 301 2092

About Business Wire

Business Wire
Business Wire



Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

Leaders in Cloud Adoption Approach Cloud Differently and Achieve 10x Greater ROI, Says Research by Wipro FullStride Cloud Services22.9.2021 18:24:00 CEST | Press release

Wipro Limited (NYSE: WIT, BSE: 507685, NSE: WIPRO), a leading global information technology, consulting and business process services company, today released a report indicating that leaders in cloud adoption approach their cloud journey differently than beginners, enabling them to achieve a 10x greater annualized return on investment. Wipro’s report, “Making Business Thrive: A Cloud Leader Roadmap for Achieving 10x ROI,” outlines these best practices for enterprises beginning their cloud journey or seeking to accelerate their results. The report also identifies dozens of key cloud-adoption metrics for multiple industries and countries. Wipro FullStride Cloud Services developed the report based on a survey of 1,300 global C-level executives and key decision-makers from 11 industries and six countries (see “Methodology” below). Wipro considered leaders to be the top 19% of respondents based on cloud maturity, while beginners represented the lowest 32%; this was not based on revenue. Thi

Nathan's Famous Continues International Expansion with European Push to Offer Hot Dogs in France22.9.2021 17:57:00 CEST | Press release

Nathan's Famous, Inc., the American tradition serving New York favorites for more than 100 years, announces a new collaboration with Tommy's Diner, a 50's throwback American diner based in France, to offer its hot dogs in all locations across the country, making it the first time Nathan's Famous is served in France. This relationship continues Nathan's Famous growth in Europe and beyond. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210922005823/en/ Nathan's Famous Menu. (Photo: Business Wire) "Nathan's Famous is known the world over, which is why we've had such great success in recent years growing in the Middle East, Asia, and Great Britain," states James Walker, Senior Vice President, Restaurants. "This new relationship with Tommy's is a big step for us as we look to see that growth spread throughout Europe, and we're excited that they are our first French outpost and will be one of the first internationally to have our

IHMA Opens Microbiology Reference Laboratory in Shanghai, China to Further Support Global Anti-Infective Product Discovery and Development22.9.2021 16:30:00 CEST | Press release

IHMA has opened a microbiology reference laboratory in Shanghai, China in February of 2021. This facility will join IHMA locations in the USA, Switzerland and India to allow the company to provide more robust global support for anti-infective product discovery and development. The China facility will provide microbiology testing to support profiling of new antimicrobial products, antimicrobial resistance surveillance, molecular characterization of resistant organisms, and serve as a central laboratory for clinical trials conducted in China. “We have been working for almost 30 years to maximize our in vitro support for clients who are developing anti-infective products globally. Our facility in China will greatly enhance that effort and support clients wishing to conduct studies in this important market,” said Jack L. Johnson, President/CEO of IHMA. About IHMA: As the leading global microbiology service provider to the biopharmaceutical industry, IHMA utilizes its extensive laboratory a

Workiva Named a Leader Among Governance, Risk, and Compliance Platforms by Independent Research Firm22.9.2021 16:24:00 CEST | Press release

Workiva Inc. (NYSE:WK), today announced that it was classified as a Leader in The Forrester Wave™: Governance, Risk, and Compliance (GRC) Platforms Q3 2021 report. The company’s connected cloud platform enables collaboration and deep integration into existing systems and workstreams to simplify audit, risk and compliance, operational, financial, and non-financial reporting for companies across the globe. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210922005704/en/ Workiva Named a Leader Among Governance, Risk, and Compliance Platforms by Independent Research Firm (Graphic: Business Wire) Evaluating GRC solutions against 25 criteria, Forrester identified 15 vendors available today, and proceeded to research, analyze, and score them. The report summarizes how each provider measures up, and ranks their solutions based on how well they satisfy the current and future needs of audit, risk, and compliance professionals. Workiva

The Scottish National Blood Transfusion Service (SNBTS) to Deploy Biolog-id Smart Storage RFID Kits Nationwide22.9.2021 15:50:00 CEST | Press release

The Scottish National Blood Transfusion Service (SNBTS) – the specialist provider of transfusion medicine in Scotland – will implement the biolog-id blood inventory devices to optimize the management of its blood products across the country. Through this deployment, biolog-id’s cutting-edge technology will streamline the complex inventory and distribution processes of the red blood cells to distant sites, enhancing the visibility and management of those lifesaving products between the Scottish blood centre and each remote blood bank. Designed for healthcare professionals specialized in the transfusion field, the Biolog Transfusion Solution combines RFID enabled devices powered by a proprietary IT engine and electronic labels. Biolog-id’s contactless devices are integrated into SNBTS’ IT transfusion management solution (MAK-SYSTEM – e-TRACELINE), offering real-time visibility of each component at each step of the supply chain. The corner-stone devices of this solution are the RFID Smart

Let’s Build a Digital Future Together: Join us at Dahua Partner Day 2021!22.9.2021 15:45:00 CEST | Press release

Dahua Technology, a world-leading video-centric smart IoT solution and service provider, will hold its 2nd online Dahua Technology Partner Day on October 13th - 14th with the theme “Building a digital future”. Adhering to its strategy of open cooperation, Dahua Technology is committed to creating a win-win ecosystem with leading technology partners around the world. This year, Dahua Technology will join hands with its 22 global partners to discuss interoperability, share industry trends, and explore the future of digitalization and intelligence together. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210922005395/en/ (Photo: Business Wire) “We are excited to host our second virtual technology partner day. Technologies have evolved fast in the past year, and the needs of digitization and diversified challenges facing different industries today are driving the integration of security companies,” says Mr. Jiaqi Gao, Overseas Ma

Takeda Selects Four New Partners for Annual Global Corporate Social Responsibility (CSR) Program to Help Strengthen Health Systems in Low- and Middle-Income Countries22.9.2021 15:30:00 CEST | Press release

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced the addition of four partnerships to its Global Corporate Social Responsibility (CSR) Program, which makes long-term commitments to strengthen health systems and improve access to healthcare for all in developing and emerging countries. As part of an annual decision-making process, Takeda employees worldwide voted to add IntraHealth International, Jhpiego, Pathfinder International and the World Food Programme(WFP) as new partners for the Global CSR Program, which now supports 20 programs in 72 countries. Takeda’s commitments to these new partners in FY2021 include: JPY 953 million to IntraHealth International to work with 12 private schools in Mali, Senegal and Niger to increase the number of qualified, trained nurses who can serve rural communities facing a critical shortage of health workers in these countries. Over the next five years, the project will help private schools partner with the public se

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom