Final Results from the Phase 3 HELP Study ™ Open-Label Extension Support TAKHZYRO ® (lanadelumab-flyo) Injection as a Long-term Preventive Treatment Option in Patients with Hereditary Angioedema
Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) today announced the final results from the Phase 3 HELP (Hereditary Angioedema Long-term Prophylaxis) Study™ Open-label Extension (OLE) showing that TAKHZYRO® (lanadelumab-flyo) helped prevent and reduce the frequency of hereditary angioedema (HAE) attacks long term in patients 12 years of age and older who received treatment for a mean (standard deviation) duration of 29.6 (8.2) months.1 Results were consistent with the safety and efficacy of TAKHZYRO in the pivotal trial. The mean (min, max) HAE attack rate was reduced by 87.4% (-100; 852.8) overall versus baseline (N=212) and in a pre-specified exploratory endpoint, nearly 70% (68.9%) of patients treated with TAKHZYRO 300 mg every two weeks experienced an attack-free period of more than 12 months (n=209).1, 2 The data are being presented at the 2020 American College of Allergy, Asthma and Immunology (ACAAI) Virtual Annual Scientific Meeting and will also be published in the November issue of ACAAI’s journal Annals of Allergy, Asthma & Immunology.
“The unpredictability of HAE attacks has a significant impact on the lives of HAE patients. HAE is a lifelong condition, so reducing the frequency and severity of attacks is an important therapeutic goal for many individuals living with HAE,” said Marc A. Riedl, M.D., investigator in the HELP Study OLE and Professor of Medicine and Clinical Director, U.S. Hereditary Angioedema Association Center at the University of California, San Diego. “The original placebo-controlled HELP Study demonstrated the efficacy and safety of TAKHZYRO over 26 weeks. The results from the open-label extension study are encouraging as they show that TAKHZYRO may help prevent attacks over the long term with continued treatment.”
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.3 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
“A significant amount of progress has been made in advancing the science to better understand and treat HAE over recent years. In 2018, we received the first regulatory approvals for TAKHZYRO as a first-of-its-kind monoclonal antibody preventive therapy in HAE, and we have already seen the difference it has made by preventing attacks in many patients around the world,” said Donatello Crocetta, M.D., Global Medical Head, Rare Immunology and Metabolic Diseases, Chief Medical Office, Takeda. “Continued research such as the HELP Study OLE is critical to further build our understanding of the potential of TAKHZYRO as a long-term preventive treatment option for those living with HAE.”
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 treatment emergent adverse events (TEAEs) occurring in 54.7% of patients (n=116) and the most common being injection-site pain, respiratory tract infection, or headache.1 In addition, data from the HELP Study OLE 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. The mean (min, max) reduction in the attack rate compared to baseline observed in the study population (N=212) was of 87.4% (-100; 852.8), with approximately 93% of patients experiencing at least a 70% reduction of the attack rate.1 Additional pre-specified exploratory endpoints measured attack-free periods.2
The abstracts being presented on the HELP Study OLE, available via the ACAAI meeting website, are as follows:
- Long-Term Efficacy and Safety of Lanadelumab: Final Results from the HELP Open-Label Extension Study (Poster #P150)
- Attack-Free Status During Extended Treatment with Lanadelumab for Hereditary Angioedema: HELP OLE Study Final Results (Poster #P157)
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. One hundred and ninety-six participants completed at least 12 months of treatment and 173 participants completed at least 30 months of treatment.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.4-6 Attacks that obstruct the airways can cause asphyxiation and are potentially life threatening.6,7 HAE affects an estimated 1 in 50,000 people worldwide. It is often under recognized, under diagnosed and under treated.4, 6, 7
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 unafraid to push at the boundaries of success in our mission to accelerate diagnosis and develop transformative and sustainable 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-flyo) 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.8 TAKHZYRO is intended for self-administration or administration by a caregiver. The patient or caregiver should be trained by a healthcare professional.8
U.S. Indication and Important Safety Information
TAKHZYRO (lanadelumab-flyo) is indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in patients ≥12 years of age.8
IMPORTANT SAFETY INFORMATION
Hypersensitivity reactions have been observed. In case of a severe hypersensitivity reaction, discontinue TAKHZYRO administration and institute appropriate treatment.8
Adverse Reactions: The most commonly observed adverse reactions (≥10% and higher than placebo) associated with TAKHZYRO were injection site reactions consisting mainly of pain, erythema, and bruising at the injection site; upper respiratory infection; headache; rash; myalgia; dizziness; and diarrhea. Less common adverse reactions observed included elevated levels of transaminases; one patient discontinued the trial for elevated transaminases.3, 8
Use in Specific Populations: The safety and efficacy of TAKHZYRO in pediatric patients <12 years of age have not been established.8
No data are available on TAKHZYRO in pregnant women. No data are available on the presence of lanadelumab in human milk or its effects on breastfed infants or milk production.8
To report SUSPECTED ADVERSE REACTIONS, contact Dyax Corp., a Takeda company, at 1-800-828-2088, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
For U.S. audiences, please see the full Prescribing Information including Medication Guide for TAKHZYRO®.
For EU audiences, please see the Summary of Product Characteristics (SmPC) for TAKHZYRO®.
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 bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, 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.
For more information, visit https://www.takeda.com.
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.
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; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; 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/reports/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.
1 Banerji A, Hao J, Ming Y et al; Long-Term Efficacy and Safety of Lanadelumab: Final Results from the HELP Open-Label Extension Study. ACAAI 2020.
2 Riedl MA, Johnston DT, Lumry WR et al; Attack-Free Status During Extended Treatment with Lanadelumab for Hereditary Angioedema: HELP OLE Study Final Results. ACAAI 2020.
3 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.
4 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.
5 Zuraw BL. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-1036.
6 Banerji A. The burden of illness in patients with hereditary angioedema. Ann Allergy Asthma Immunol. 2013;111(5):329-336.
7 Longhurst HJ, Bork K. Hereditary angioedema: causes, manifestations, and treatment. Br J Hosp Med. 2006;67(12):654-657.
8 TAKHZYRO® (lanadelumab-flyo) injection Prescribing Information.
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