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

February 4, 2019

- 12 presentations will be presented across 3 lysosomal storage disorders, underscoring Takeda’s commitment to patients with rare diseases


Cambridge, Mass. and Osaka,JAPAN,February 4,2019Takeda    Pharmaceutical    Company    Limited (T SE: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, Head of Rare Diseases and Internal Medicine, Global Medical Affairs, 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 burden1.

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

  • 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  (T SE: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.
For more information, visit https:// www.takeda.com

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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: https://rarediseases.org/rare-diseases/fabry-disease/.

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.