Shire to Highlight Advancements in Rare Genetic Diseases

CAMBRIDGE, Massachusetts, February 14, 2017 /PRNewswire/ --

14 presentations and 5 satellite symposia across 3 lysosomal storage disorders at important scientific meeting represent Shire's continued focus on the diagnosis, treatment, and management of these life-altering diseases  

Shire plc (LSE: SHP, NASDAQ: SHPG), the global leader in rare diseases, will be presenting 14 posters, including two late-breaking abstracts, at the 13th annual WORLDSymposium™ 2017 in San Diego, Calif., (February 13-17). Presentations are focused on data in lysosomal storage diseases (LSDs), including Hunter syndrome (also known as Mucopolysaccharidosis type II or MPS II), type 1 Gaucher disease, and Fabry disease, and highlight Shire's commitment to innovative research in genetic diseases.

"We are proud of our ongoing commitment to patients with rare genetic diseases and look forward to showcasing the depth and breadth of our scientific research across the LSDs," said Hartmann Wellhoefer, M.D., Vice President, Head of Medical Affairs, Genetic Diseases. "The data we will be presenting represent our continued focus on advancing the diagnosis, treatment, and management of life-altering diseases."

Each year, WORLDSymposium hosts a scientific meeting presenting the latest information from basic science, translational research, and clinical trials for lysosomal diseases. This symposium is designed to educate researchers and clinicians to better understand diagnostic and management options for patients with lysosomal diseases; identify areas requiring additional basic and clinical research, public policy and regulatory attention; and present the latest findings in the natural history of lysosomal diseases.

At the meeting, in addition to data presentations, Shire also will be sponsoring three booths (#12, 16, and 19) in the exhibition hall, as well as five satellite symposia. Following are the key Shire presentations at the meeting:

• A long-term extension study evaluating intrathecal idursulfase-IT in children with Hunter Syndrome and cognitive impairment (HIT-045/046)
  Poster #240, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom  
• Clinical outcomes after 3 years of idursulfase treatment in patients with MPS II: data from the Hunter Outcome Survey (HOS)
  Poster #114, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• The Challenge of using Hospital Episode Statistics to identify a Hunter Syndrome Cohort in the UK
  Poster #231, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• Health care resource utilization by patients with Hunter Syndrome in the UK Hospital Episode Statistics database
  Poster #230, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• Validation of the shortened Hunter Syndrome-functional outcomes for clinical understanding scale (HS-FOCUS) questionnaire
  Poster #220, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• Profile of patients with Mucopolysaccharidosis type II without cognitive impairment who started idursulfase treatment aged >20 years: data on late treatment initiation from the Hunter Outcome Survey (HOS)
  Poster #185, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• Characteristics of patients with Mucopolysaccharidosis type II diagnosed ≥5 years of age: data from the Hunter Outcome Survey (HOS)
  Poster #134, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• Intrafamilial variability in the clinical presentation of Mucopolysaccharidosis type II: data from the Hunter Outcome Survey (HOS)
  Poster #85, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• Urinary glycosaminoglycan levels in a Mucopolysaccharidosis type II pediatric population receiving idursulfase therapy: data from the Hunter Outcome Survey (HOS) for patients aged <18 months
  Poster #8, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• Medical histories in Gaucher disease: a descriptive analysis from 852 patients in the Gaucher Outcome Survey (GOS)
  Poster #82, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• Characteristics of 27 patients with type 3 Gaucher disease: a descriptive analysis from the Gaucher Outcome Survey
  Poster #303, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• Real-world treatment patterns from 647 patients with Gaucher disease: an analysis from the Gaucher Outcome Survey
  Poster #70, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• Outcomes of 453 pregnancies in patients with Gaucher disease: an analysis from the Gaucher Outcome Survey
  Poster #184, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• Prompt agalsidase alfa therapy initiation after symptom onset is associated with improved renal and cardiovascular outcomes in the Fabry Outcome Survey
  Poster #199, Tuesday, February 14, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• The Australian ATYOURSIDE patient support experience
  Poster #LB-09, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom 
• The Australian ATHOME™ infusion service experience
  Poster #LB-19, Wednesday, February 15, 2017 from 4:30-6:30 pm PT, Harbor Ballroom  

Shire is dedicated to addressing challenges in the LSD community, and will be sponsoring several symposia at the meeting:

Sponsored by Shire Commercial 

• On the Frontlines of Managing Patients with Lysosomal Diseases 
  Tues, February 14 from 6:30-7:30 am PT
  General Session Room (Seaport Ballroom D-H)
• Pioneers and Innovators in Gaucher Disease 
  Wed, Feb 15 from 11:45 am-12:45 pm PT
  General Session Room (Seaport Ballroom D-H)

Sponsored by Shire Medical 

• MPS II: One Family's Journey 
  Tues, February 14 from 11:45 am - 12:45 pm PT
  General Session Room (Seaport Ballroom D-H)
• Discovery and Research Platforms in Rare Disease 
  Tues, Feb 14 from 6:30-8:30 pm PT
  General Session Room (Seaport Ballroom D-H)
• MPS II with Cognitive Impairment: Approaches to Improving Identification and Assessment 
  Wed, Feb 15 from 6:30-7:30 am PT
  Seaport Ballroom A-C

About the Genetic Disease Franchise 

Shire is dedicated to helping patients with inherited illnesses. Shire's genetic disease franchise has a strong legacy in developing therapies for LSDs, with a portfolio that includes commercial products, late-stage investigational therapies, and pipeline candidates, as well as a robust R&D program.

Hunter Syndrome 

Hunter syndrome, or mucopolysaccharidosis II (MPS II), is a serious genetic disorder that primarily affects males. It interferes with the body's ability to break down and recycle specific mucopolysaccharides, also known as glycosaminoglycans or GAGs. Hunter syndrome is one of several related lysosomal storage diseases.

Type 1 Gaucher Disease 

Type 1 Gaucher disease is a rare, inherited metabolic condition, and the most common of a family of rare diseases known as lysosomal storage disorders (LSDs). It affects approximately 1 in 100,000 people in the general population, and 1 in 855 people in the Ashkenazi Jewish community. Patients with type 1 Gaucher disease may experience varying symptoms and degrees of disease severity, making type 1 Gaucher disease difficult to diagnose.

Fabry Disease 

Fabry disease is a lysosomal storage disorder (LSD) 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). Fabry disease affects an estimated 8,000 to 10,000 people worldwide.

NOTES TO EDITORS 

About Shire 

Shire is the leading global biotechnology company focused on serving people with rare diseases and other highly specialized conditions. We strive to develop best-in-class products, many of which are available in more than 100 countries, across core therapeutic areas including Hematology, Immunology, Neuroscience, Ophthalmics, Lysosomal Storage Disorders, Gastrointestinal / Internal Medicine / Endocrine and Hereditary Angioedema; and a growing franchise in Oncology.

Our employees come to work every day with a shared mission: to develop and deliver breakthrough therapies for the hundreds of millions of people in the world affected by rare diseases and other high-need conditions, and who lack effective therapies to live their lives to the fullest.

http://www.shire.com

Forward-Looking Statements 

Statements included herein that are not historical facts, including without limitation statements concerning future strategy, plans, objectives, expectations and intentions, the anticipated timing of clinical trials and approvals for, and the commercial potential of, inline or pipeline products are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, Shire's results could be materially adversely affected. The risks and uncertainties include, but are not limited to, the following:

• Shire's products may not be a commercial success;
• increased pricing pressures and limits on patient access as a result of governmental regulations and market developments may affect Shire's future revenues, financial condition, and results of operations;
• Shire conducts its own manufacturing operations for certain of its products and is reliant on third party contract manufacturers to manufacture other products and to provide goods and services.  Some of Shire's products or ingredients are only available from a single approved source for manufacture.  Any disruption to the supply chain for any of Shire's products may result in Shire being unable to continue marketing or developing a product or may result in Shire being unable to do so on a commercially viable basis for some period of time;
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• certain of Shire's therapies involve lengthy and complex processes, which may prevent Shire from timely responding to market forces and effectively managing its production capacity;
• Shire has a portfolio of products in various stages of research and development. The successful development of these products is highly uncertain and requires significant expenditures and time, and there is no guarantee that these products will receive regulatory approval;
• the actions of certain customers could affect Shire's ability to sell or market products profitably. Fluctuations in buying or distribution patterns by such customers can adversely affect Shire's revenues, financial conditions, or results of operations;
• Shire's products and product candidates face substantial competition in the product markets in which it operates, including competition from generics;
• adverse outcomes in legal matters, tax audits and other disputes, including Shire's ability to enforce and defend patents and other intellectual property rights required for its business, could have a material adverse effect on the combined company's revenues, financial condition, or results of operations;
• inability to successfully compete for highly qualified personnel from other companies and organizations;
• failure to achieve the strategic objectives with respect to Shire's acquisition of NPS Pharmaceuticals, Inc., Dyax Corp. ("Dyax") or Baxalta Inc. ("Baxalta") may adversely affect Shire's financial condition and results of operations;
• Shire's growth strategy depends in part upon its ability to expand its product portfolio through external collaborations, which, if unsuccessful, may adversely affect the development and sale of its products;
• a slowdown of global economic growth, or economic instability of countries in which Shire does business, as well as changes in foreign currency exchange rates and interest rates, that adversely impact the availability and cost of credit and customer purchasing and payment patterns, including the collectability of customer accounts receivable;
• failure of a marketed product to work effectively or if such a product is the cause of adverse side effects could result in damage to the Shire's reputation, the withdrawal of the product and legal action against Shire;
• investigations or enforcement action by regulatory authorities or law enforcement agencies relating to Shire's activities in the highly regulated markets in which it operates may result in significant legal costs and the payment of substantial compensation or fines;
• Shire is dependent on information technology and its systems and infrastructure face certain risks, including from service disruptions, the loss of sensitive or confidential information, cyber-attacks and other security breaches or data leakages that could have a material adverse effect on Shire's revenues, financial condition, or results of operations;
• Shire incurred substantial additional indebtedness to finance the Baxalta acquisition, which may decrease its business flexibility and increase borrowing costs;
• difficulties in integrating Dyax or Baxalta into Shire may lead to the combined company not being able to realize the expected operating efficiencies, cost savings, revenue enhancements, synergies or other benefits at the time anticipated or at all; and other risks and uncertainties detailed from time to time in Shire's filings with the Securities and Exchange Commission, including those risks outlined in "ITEM 1A: Risk Factors" in Shire's Quarterly Report on Form 10-Q for the quarter ended September 30, 2016.

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SOURCE Shire plc