Shire's SHP626 (Volixibat) Receives FDA Fast Track Designation for an Investigational Treatment for Adults Who Have Nonalcoholic Steatohepatitis (NASH) With Liver Fibrosis

LEXINGTON, Massachusetts, July 29, 2016 /PRNewswire/ —

Shire plc (LSE: SHP, NASDAQ: SHPG) today announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation for SHP626 (volixibat) for an investigational treatment of adults who have nonalcoholic steatohepatitis (NASH) with liver fibrosis. Shire is developing SHP626 as a once daily, orally-administered inhibitor of the apical sodium dependent bile acid transporter (ASBT), a protein which is primarily responsible for recycling bile acids from the intestine to the liver. NASH is a serious, chronic liver disease for which there are currently no approved drugs.

“Shire’s development plan for SHP626 is designed to address the unmet need in the treatment of adult patients who have NASH with liver fibrosis,” said Philip J. Vickers, Ph.D., Head of R&D, Shire. “This Fast Track designation is further recognition of the critical need to develop new, effective therapeutic options for patients with this serious condition.”

The FDA Fast Track Designation for SHP626 in NASH was supported by preclinical and Phase 1 studies. The FDA’s Fast Track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. However, it does not guarantee that the FDA will ultimately approve SHP626 for NASH or the timing of any such approval.

Shire will initiate its Phase 2 trial with SHP626 as a randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability and efficacy of three doses of volixibat over 48-weeks in adult patients with NASH. The Phase 2 study will be conducted in the U.S., Canada and the United Kingdom.

Additional information on the SHP626 Phase 2 study can be found on clinicaltrials.gov:

https://clinicaltrials.gov/ct2/show/NCT02787304?term=volixibat&rank=1

SHP626 has been evaluated in preclinical and Phase 1 studies, in which the safety, tolerability and preliminary activity of SHP626 compared to placebo in healthy volunteers, as well as in overweight and obese volunteers, was assessed. The most common adverse events occurring in Phase 1 trials of SHP626 were gastrointestinal in nature, predominantly diarrhea. While this occurred in most patients, it was not considered serious. There was one serious adverse event reported that was considered related to SHP626, alanine aminotransferase elevation, that led to discontinuation of drug.

About Nonalcoholic Steatohepatitis (NASH) 

NASH is a type of nonalcoholic fatty liver disease (NAFLD), characterized by inflammation and the accumulation of fat in the liver, for which there are currently no approved drugs. It can be severe and lead to fibrosis, cirrhosis, liver failure and liver cancer. There is a steady rise in the prevalence of NASH in the U.S. and globally, and the disease is typically associated with obesity, type 2 diabetes, hypertension, high cholesterol and triglycerides. NASH is currently the second leading cause of liver transplantation in adults in the U.S., and is estimated to become the leading cause for liver transplantation if the current trajectory continues.

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:

All forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof. Except to the extent otherwise required by applicable law, we do not undertake any obligation to republish revised forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.

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INTSP/EXA/GBU/16/001

For further information please contact: 

Investor Relations
Sarah Elton-Farr, seltonfarr@shire.com , +44-1256-894157
Ian Karp, ikarp@shire.com, +1-781-482-9018
Robert Coates, rcoates@shire.com, +44-1256-894874

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