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Takeda to Highlight Advancements in Rare Lysosomal Storage Disorders at Global Scientific Meeting

Takeda to Highlight Advancements in Rare Lysosomal Storage Disorders at Global Scientific Meeting − 12 presentations will be presented across 3 lysosomal storage disorders, underscoring Takeda's commitment to patients with rare diseases

PR Newswire

OSAKA, Japan, Feb. 4, 2019

OSAKA, Japan, Feb. 4, 2019 /PRNewswire/ -- Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) ("Takeda") today announced that it will feature 12 presentations, including 11 posters and one oral presentation, at the 15th annual WORLDSymposium™ 2019 in Orlando, Florida, February 4-8. Presentations and other company activities will focus on its research and development efforts in lysosomal storage disorders (LSDs) including Hunter syndrome (also known as Mucopolysaccharidosis type II or MPS II), type 1 Gaucher disease, Fabry disease and metachromatic leukodystrophy (also known as MLD).

The company will also sponsor a satellite symposium on the role of biomarkers in clinical management of lysosomal diseases, and a booth (Booth #105) in the WORLDSymposium exhibit hall.

"This year we are pleased to demonstrate that Takeda is committed to supporting the lysosomal storage disorder community and to advancing treatment in areas of high unmet need," said Hartmann Wellhoefer, M.D., Vice President, Medical Affairs, Rare Disease and Internal Medicine, Global R&D, Takeda. "We look forward to continuing to participate annually in this unique congress which gives us the opportunity to reconnect and engage with the world's leading LSD experts and patient organizations, who all share our passion for supporting patients affected by rare disease."

The upcoming WORLDSymposium annual meeting focuses on the latest scientific and clinical research on LSDs, a collection of some 50 disorders caused by specific enzyme deficiencies and leading to significant disability and disease burden.1

Takeda's presence at the meeting includes the following presentations, which are intended for scientific discussion only:

  • Intrathecally administered recombinant human arylsulfatase A in patients with late-infantile metachromatic leukodystrophy: Phase 2b clinical trial design
    Oral presentation, Wednesday, February 6, 2019 at 3:45 pm ET Regency Ballroom U
  • Clinical characteristics of patients with neuronopathic and non-neuronopathic Mucopolysaccharidosis type II: Data from the Hunter Outcome Survey (HOS) 
    Poster #210, Tuesday, February 5, 2019 from 4:30-6:30 pm ET, Regency Ballroom R
  • Gaucher disease (GD)-specific patient-reported outcome (PRO) measures for clinical monitoring and for clinical trials
    Poster #101, Tuesday, February 5, 2019 from 4:30-6:30 pm ET, Regency Ballroom R
  • Long-term analysis of velaglucerase alfa-treated patients with Gaucher disease who entered the Gaucher Outcome Survey real-life registry (GOS)
    Poster #209, Tuesday, February 5, 2019 from 4:30-6:30 pm ET, Regency Ballroom R
  • Characteristics of patients with Mucopolysaccharidosis type II who have received a bone marrow transplant: data from the Hunter Outcome Survey (HOS)
    Poster #246, Wednesday, February 6, 2019 from 4:30-6:30 pm ET, Regency Ballroom R
  • Evaluation of the long-term treatment effects of idursulfase using statistical modelling: Data from the Hunter Outcome Survey (HOS)
    Poster #245, Wednesday, February 6, 2019 from 4:30-6:30 pm ET, Regency Ballroom R
  • Neurodevelopmental status and adaptive behaviour of pediatric patients with Hunter syndrome: A longitudinal observational study
    Poster #244, Wednesday, February 6, 2019 from 4:30-6:30 pm ET, Regency Ballroom R
  • Analysis of cognitive ability and adaptive behaviour assessment tools used in an observational study of patients with Hunter syndrome
    Poster #387, Wednesday, February 6, 2019 from 4:30-6:30 pm ET, Regency Ballroom R
  • Study to determine predictive potential of an algorithm for earlier diagnosis of Gaucher disease: Retrospective biobank study utilizing real-world data available in Finland
    Poster #316, Wednesday, February 6, 2019 from 4:30-6:30 pm ET, Regency Ballroom R
  • A charitable access program for underserved lysosomal disease patients worldwide
    Poster #LB-34, Wednesday, February 6, 2019 from 4:30-6:30 pm ET, Regency Ballroom R
  • Renoprotective effect of agalsidase alfa: 12-year follow-up of male Fabry patients 
    Poster #LB-53, Wednesday, February 6, 2019 from 4:30-6:30 pm ET, Regency Ballroom R
  • Classification of genetic variants in patients with Fabry disease enrolled in the Fabry Outcome Survey (FOS)
    Poster #LB-20, Wednesday, February 6, 2019 from 4:30-6:30 pm ET, Regency Ballroom R

The company will also be sponsoring a lunch symposium, which is also intended for scientific discussion only at the meeting:

  • Biomarkers: Ready for Prime Time in the Clinical Management of Lysosomal Diseases?
    Tuesday, February 5, 2019, 11:45 am-12:45 pm ET, Windermere Ballroom WX

Fabry Disease
Fabry disease is a lysosomal disease affecting both males and females that interferes with the body's ability to break down a specific fatty substance (globotriaosylceramide or Gb3) which accumulates within the body due to deficiency of a specific enzyme (α-galactosidase A).[2] Fabry disease affects an estimated 1 in 117,000 live births.[3]

Hunter Syndrome
Hunter syndrome is a severely debilitating, rare lysosomal disease caused by a deficiency of iduronate-2-sulfatase, an enzyme that is needed to break down substances in the body called glycosaminoglycans (GAGs).[4] Without this enzyme, GAGs can build up, causing a range of disease-related signs and symptoms.4,[5] Roughly two of every three patients with Hunter syndrome are also affected with progressive cognitive decline.[6] Hunter syndrome affects 1 in 162,000 total live births, and almost exclusively males.3

Type 1 Gaucher Disease
Type 1 Gaucher disease is a rare, inherited metabolic condition, and the most common lysosomal disease. It affects approximately 1 in 100,000 people in the general population, and 1 in 855 people in the Ashkenazi Jewish community.[7] Patients with type 1 Gaucher disease may experience varying symptoms and degrees of disease severity, making type 1 Gaucher disease difficult to diagnose.[8]

Metachromatic Leukodystrophy
Metachromatic Leukodystrophy (MLD) is a rare, inherited disorder that affects the central nervous system (CNS). MLD is a fatal, progressive demyelinating lysosomal disease caused by the deficiency of Arylsufatase-A (ARSA) leading to a toxic accumulation of sulfatides in the CNS and/or PNS.[9]   

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, Gastroenterology (GI), Neuroscience, and Rare Diseases. 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.

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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 the words such as "targets", "plans", "believes", "hopes", "continues", "expects", "aims", "intends", "will", "may", "should", "would", "could" "anticipates", "estimates", "projects" or words or terms of similar substance or the negative thereof. Any forward-looking statements in this document are based on the current assumptions and beliefs of Takeda in light of the information currently available to it. Such forward-looking statements do not represent any guarantee by Takeda or its management of future performance and involve known and unknown risks, uncertainties and other factors, including but not limited to: the economic circumstances surrounding Takeda's business, including general economic conditions in Japan, the United States and worldwide; competitive pressures and developments; applicable laws and regulations; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; changes in exchange rates; claims or concerns regarding the safety or efficacy of marketed products or products candidates; and post-merger integration with acquired companies, any of which may cause Takeda's actual results, performance, achievements or financial position to be materially different from any future results, performance, achievements or financial position expressed or implied by such forward-looking statements. For more information on these and other factors which may affect Takeda's results, performance, achievements, or financial position, see "Item 3. Key Information—D. Risk Factors" in Takeda's Registration Statement on Form 20-F filed with the U.S. Securities and Exchange Commission, available on Takeda's website at:  or at Neither Takeda nor its management gives any assurances that the expectations expressed in these forward-looking statements will turn out to be correct, and actual results, performance or achievements could materially differ from expectations. Persons receiving this press release should not place undue reliance on forward looking statements. Takeda undertakes no obligation to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make. Past performance is not an indicator of future results and the results of Takeda in this press release may not be indicative of, and are not an estimate, forecast or projection of Takeda's future results.

While Takeda plans to announce an earnings forecast which includes an estimated financial impact of the Shire acquisition once a reasonable financial estimate is determined, the consideration of the asset valuation as well as purchase price allocation, schedule and manner of amortization and depreciation for the business combination accounting will require more time. It is also difficult to estimate the effect on profit and loss since the completion of the acquisition to the end of the consolidated accounting period, nor the acquisition related costs for the full fiscal year with a reasonable level of accuracy at this time. Considering the sizable effect on the business results due to the acquisition, Takeda is not furnishing a new consolidated forecast in a provisional or partial way at this time. It is our objective to disclose a Shire acquisition post-close consolidated business forecast for the fiscal year once a holistic and reasonable earnings forecast can be determined.

1 Fuller M, Meike PJ, Hopwood JJ. Epidemiology of lysosomal storage diseases: an overview. In: Mehta A, Beck M, Sunder-Plassmann G, editors. Fabry Disease: Perspectives from 5 Years of FOS. Oxford: Oxford PharmaGenesis; 2006. Chapter 2.
2 Fabry Disease. National Organization for Rare Disorders. Accessed January 2018. Available at:
3 Meikle PJ et al. Prevalence of Lysosomal Storage Disorders. JAMA. 1999. 281(3):249-54.
4 Wraith JE et al. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur J Pediatr. 2008. 167(3):267-77.
5 Martin R. Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome). PEDIATRICS. Volume 121, Number 2, February 2008.
6 Young I. A clinical and genetic study of Hunter's syndrome. 2 Differences between the mild and severe forms. Journal of Medical Genetics, 1982, 19, 408-411.
7 Guggenbuhl P, Grosbois B, Chalès G. Gaucher disease. Joint Bone Spine. 2008; 75(2):116-124.
8 Grabowski GA. Phenotype, diagnosis, and treatment of Gaucher's disease. Lancet. 2008; 372:1263-71.
9 Rosenberg JB, Kaminsky SM, Aubourg P, Crystal RG, Sondhi D. Gene therapy for metachromatic leukodystrophy. J Neuroscience Res. 2016;94(11);1169-79.

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Last Updated: 04-Feb-2019