Researchers to Present New Data on Asfotase Alfa in Infants and Juveniles with Hypophosphatasia at the Joint Meeting of the

  Researchers to Present New Data on Asfotase Alfa in Infants and Juveniles
  with Hypophosphatasia at the Joint Meeting of the Pediatric Academic
  Societies and the Asian Society for Pediatric Research

-- New data from a natural history study in infants with hypophosphatasia also
                              to be presented --

Business Wire

CHESHIRE, Conn. -- April 11, 2014

Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that researchers
are scheduled to present new data from the extension phase of two clinical
studies examining the long-term efficacy and safety of asfotase alfa in
infants and juveniles with hypophosphatasia (HPP) at the joint meeting of the
Pediatric Academic Societies (PAS) and the Asian Society for Pediatric
Research, which takes place May 3-6, 2014 in Vancouver, B.C., Canada. The
meeting will also feature the presentation of results from a retrospective
natural history study of patients with perinatal and infantile HPP.

HPP is an inherited, ultra-rare metabolic disorder that can lead to
progressive damage to multiple vital organs, destruction and deformity of
bones, and death. The U.S. Food and Drug Administration (FDA) granted
Breakthrough Therapy designation for asfotase alfa in pediatric-onset HPP,
defined as patients whose first signs or symptoms of HPP occurred prior to 18
years of age, including perinatal-, infantile-, and juvenile-onset forms of
the disease.

Abstracts summarizing the asfotase alfa studies and the HPP natural history
study were published today on the PAS website and are available to conference
registrants at:

The following abstract will be presented in a poster session on May 4, 2014
from 4:15pm – 7:30pm Pacific Daylight Time (PDT):

  *Abstract 752396: “Asfotase Alfa: Sustained Efficacy and Tolerability in
    Infants and Young Children with Life-Threatening Hypophosphatasia,” Whyte,
    et al.

The following abstracts will be presented in a poster session on May 5, 2014
from 4:15pm – 7:30pm PDT:

  *Abstract 752577: “Asfotase Alfa: Long-Term Safety and Efficacy in Children
    with Hypophosphatasia,” Madson, et al.
  *Abstract 752416: “Hypophosphatasia: A Retrospective Natural History Study
    of the Severe Perinatal and Infantile Forms,” Whyte, et al.

About Hypophosphatasia (HPP)

Hypophosphatasia (HPP) is a chronic, life-threatening, genetic, and ultra-rare
metabolic disease characterized by defective bone mineralization that can lead
to destruction and deformity of bones, profound muscle weakness, seizures, and
respiratory failure.^1-4

HPP is caused by a genetic deficiency of an enzyme known as tissue
non-specific alkaline phosphatase (TNSALP).^1

The genetic deficiency in HPP can affect people of all ages.^1HPP is
traditionally classified by the age of the patient at the onset of symptoms of
the disease. Patients with perinatal-onset HPP manifest their first signs of
disease in utero or at birth. This form of the disease often leads to death at
or soon after birth. Those patients who survive birth often have severe
rickets and severely compromised respiratory function.^5

Patients with infantile-onset HPP develop their first signs or symptoms of HPP
before 6 months of age. Individuals with this form of disease develop rickets,
skeletal abnormalities, fractures, failure to thrive and respiratory failure.
The prognosis of these patients may be poor with mortality estimated to be as
high as at 50%.^1

Patients with juvenile-onset HPP exhibit their first signs or symptoms of HPP
after 6 months of age and before 18 years of age.Individuals with this form
of the disease are at risk for rickets, skeletal complications including
fractures, and can have delayed acquisition of age-appropriate motor skills
due to skeletal hypomineralization and muscle weakness leading to the need for
walking assistance; some may never walk.^1

About Asfotase Alfa

Asfotase alfa is an investigational, highly innovative, first-in-class
targeted enzyme replacement therapy. Asfotase alfa is designed to address the
underlying cause of HPP by normalizing the genetically defective metabolic
process, and preventing or reversing the severe and potentially
life-threatening complications of life-long dysregulated mineral metabolism.

According to the FDA, a Breakthrough Therapy designation is designed to
expedite the development of a drug to treat a serious or life-threatening
disease when preliminary clinical evidence indicates that the drug may
demonstrate substantial improvement over existing therapies on one or more
clinically significant endpoints. The Breakthrough Therapy designation is part
of the FDA Safety and Innovation Act (FDASIA) of 2012.^6

About Alexion

Alexion is a biopharmaceutical company focused on serving patients with severe
and rare disorders through the innovation, development and commercialization
of life-transforming therapeutic products. Alexion is the global leader in
complement inhibition and has developed and markets Soliris® (eculizumab) as a
treatment for patients with PNH and aHUS, two debilitating, ultra-rare and
life-threatening disorders caused by chronic uncontrolled complement
activation. Soliris is currently approved in nearly 50 countries for the
treatment of PNH, and in the United States, European Union, Japan and other
countries for the treatment of aHUS. Alexion is evaluating other potential
indications for Soliris in additional severe and ultra-rare disorders beyond
PNH and aHUS, and is developing other highly innovative biotechnology product
candidates across multiple therapeutic areas. This press release and further
information about Alexion can be found at:



1. Whyte MP. Hypophosphatasia. In: Glorieux FH, Jueppner H, Pettifor J, eds.
Pediatric bone: biology and diseases. 3rd ed. San Diego, CA: Academic Press,
2012: 771-94.

2. Seshia SS, Derbyshire G, Haworth JC, Hoogstraten J. Myopathy with
Hypophosphatasia. Arch Dis Child. 1990; 65(1):130-1.

3. Whyte MP. Hypophosphatasia: Nature's window on alkaline phosphatase
function in humans. In: Principles of Bone Biology, 3rd Ed. Part II, Chapter
73: Molecular Mechanisms of Metabolic Bone Disease, Academic Press, 2008:

4. Silver MM, Vilos GA, Milne KJ. Pulmonary hypoplasia in neonatal
hypophosphatasia. Pediatr Pathol. 1998; 8:483-93.

5. Whyte MP. Hypophosphatasia and the extracellular metabolism of inorganic
pyrophosphate: Clinical and laboratory aspects. Crit Rev Clin Lab Sci. 1991;

6. Public Law 112-144. U.S. Government Printing Office, July 9,


Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Executive Director, Corporate Communications
Kim Diamond, 203-439-9600
Senior Director, Corporate Communications
Rx Communications
Rhonda Chiger, 917-322-2569
Press spacebar to pause and continue. Press esc to stop.