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SHIRE PLC - Shire Reports Topline Results - Phase 2 IMAGO Study.

  Shire Reports Topline Results from First of Three
Placebo-Controlled Phase 2
          Studies of SHP625 (LUM001) in Children with Alagille Syndrome
Lexington, Mass. - April 09, 2015 - Shire plc (LSE: SHP, NASDAQ: SHPG)
today announced that the 13-week Phase 2 IMAGO trial of its
investigational compound SHP625 (LUM001) did not meet the primary or
secondary endpoints in the study of 20 pediatric patients with Alagille
syndrome (ALGS), a rare, life-threatening genetic disorder that presents
with chronic cholestasis (accumulation of bile acids in the liver) and
severe pruritus (itching). The primary endpoint was the change from
baseline in serum bile acid levels as compared to placebo. The secondary
endpoint of pruritus was assessed using the novel ItchRO(TM) instrument.
Mean serum bile acid levels and pruritus at the end of the study were
lower in both SHP625 and placebo treated groups as compared to baseline.
However, in a post-hoc analysis, a positive correlation between percent
changes from baseline in serum bile acid levels and pruritis was
observed in the SHP625 treated group. The number of patients in the
placebo treated group was too small to make an accurate assessment of
this relationship. There were no treatment emergent serious adverse
events in this study. As expected, the most common adverse events were
diarrhea and abdominal pain, which were more frequent with SHP625 than
with placebo. "We have gained important insights from these first
results from one of several phase 2 studies in the SHP625 development
program," said Philip J. Vickers, Ph.D., Head of Research and
Development, Shire. "We remain committed to continuing the ongoing
studies of SHP625 in ALGS and other indications." In addition to
IMAGO, two larger placebo-controlled phase 2 studies in ALGS are in
progress, one of which has pruritus as the primary endpoint. SHP625 is
also being studied in progressive familial intrahepatic cholestasis,
primary biliary cirrhosis and primary sclerosing cholangitis. ABOUT THE
IMAGO STUDY IMAGO is a phase 2 multicenter, randomized,
placebo-controlled study of children with ALGS, 1-18 years of age,
conducted in the United Kingdom. Subjects (n=20) were randomized to
receive oral, once-daily SHP625, 140 og/kg/ day (n=6) or 280 og/kg/day
(n=8) or placebo (n=6). The primary endpoint was change in fasting serum
bile acid levels from baseline to Week 13. Serum bile acid level
reductions from baseline were -66.1 omol/L and -42.1 omol/L (p=0.69) in
the SHP625 and in the placebo treated groups, respectively. For the
secondary endpoint of pruritus, the change from baseline for the
ItchRO(Obs) scores was -0.61 and -0.59 (p=0.95) in the SHP625 and the
placebo treated groups, respectively. Additional secondary endpoints
included change in liver enzymes. The most common adverse events with
SHP625 vs placebo were diarrhea (64.3% vs 33.3%) and abdominal pain
(42.9% vs 16.7%). ABOUT SHP625 SHP625 is being studied in several rare
cholestatic liver diseases for both pediatric and adult populations.
Preclinical models demonstrate that SHP625 is a potent, selective
minimally absorbed inhibitor of the apical sodium-dependent bile acid
transporter. It blocks bile acid reabsorption in the terminal ileum and
increases fecal bile acid excretion, thereby reducing recirculation of
bile acids to the liver. ABOUT ALAGILLE SYNDROME Alagille syndrome
(ALGS) is a rare, life-threatening genetic disorder occurring in
approximately one in 30,000 live births that affects the liver, heart,
kidney and other systems of the body. Problems associated with the
disorder generally become evident in infancy or early childhood. A
person with Alagille syndrome has fewer than the normal number of bile
ducts inside the liver. This causes bile to build up in the liver, a
condition called cholestasis, and leads to liver damage. In addition, a
buildup of bile acids in the blood also can cause pruritus, or itching,
which can be severe. There are no approved medical therapies for ALGS,
but some treatments can address its symptoms. Ten to 30 percent of
people with ALGS will require a liver transplant. For further
information please contact: Investor Relations Sarah Elton-Farr +44 1256 894157 Media Stephanie Fagan     +1 201 572 9581 Gwen Fisher    +1 484 595 9836 NOTES TO EDITORS Shire enables
people with life-altering conditions to lead better lives. Our strategy
is to focus on developing and marketing innovative specialty medicines
to meet significant unmet patient needs. We focus on providing
treatments in Rare Diseases, Neuroscience, Gastrointestinal and Internal
Medicine and we are developing treatments for symptomatic conditions
treated by specialist physicians in other targeted therapeutic areas,
such as Ophthalmics. THE "SAFE HARBOR" STATEMENT
included in this announcement that are not historical facts 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, that:
  * Shire's products may not be a commercial success;
  * product sales from ADDERALL XR and INTUNIV are subject to generic
  * the failure to obtain and maintain reimbursement, or an adequate
level of
    reimbursement, by third-party payers in a timely manner for
    products may affect future revenues, financial condition and results
  * Shire conducts its own manufacturing operations for certain of its
    and is reliant on third party contract manufacturers to manufacture
    products and to provide goods and services. Some of the Shire's
products or
    ingredients are only available from a single approved source for
    manufacture. Any disruption to the supply chain for any of the
    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
    commercially viable basis for some period of time;
  * the manufacture of Shire's products is subject to extensive
oversight by
    various regulatory agencies. Regulatory approvals or interventions
    associated with changes to manufacturing sites, ingredients or
    manufacturing processes could lead to significant delays, an
increase in
    operating costs, lost product sales, an interruption of research
    or the delay of new product launches;
  * 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
    by such customers can adversely affect Shire's revenues,
    conditions or results of operations;
  * investigations or enforcement action by regulatory authorities or
    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;
  * adverse outcomes in legal matters and other disputes, including
    ability to enforce and defend patents and other intellectual
    rights required for its business, could have a material adverse
effect on
    Shire's revenues, financial condition or results of operations;
  * Shire faces intense competition for highly qualified personnel from
    companies and organizations. Shire is undergoing a corporate
    and was the subject of an unsuccessful acquisition proposal and the
    consequent uncertainty could adversely affect Shire's ability
to attract
    and/or retain the highly skilled personnel needed for Shire to meet
    strategic objectives;
  * failure to achieve Shire's strategic objectives with respect to
    acquisition of NPS Pharmaceuticals, Inc. may adversely affect
    financial condition and results of operations; and other risks and
uncertainties detailed from time to time in Shire's filings with
the US Securities and Exchange Commission, including its most recent
Annual Report on Form 10-K. Press Release
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Date:Apr 9, 2015
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