Researchers to Present New Data at ENDO 2016 Advancing the Understanding of Strensiq® (asfotase alfa) in Infants, Children and Adult Patients with Hypophosphatasia (HPP)

CHESHIRE, Conn.–(BUSINESS WIRE)–Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
researchers will present new functional and quality-of-life data in
children with hypophosphatasia (HPP) treated with Strensiq® (asfotase
alfa) for at least five years, as well as new data on the biochemical
and physical function outcomes in juveniles and adults with HPP treated
with Strensiq for up to four years. Researchers will also present new
safety and efficacy data in infants with HPP treated with Strensiq for
up to three and a half years. Data will be presented in two oral
sessions and one poster presentation at the Endocrine Society’s 98th
Annual Meeting and Expo (ENDO), being held April 1-4, 2016, in Boston.

Strensiq is approved in the United States as a treatment for patients
with perinatal-, infantile- or juvenile-onset HPP. Strensiq is also
approved in the European Union, Japan and Canada.

HPP is a genetic and progressive metabolic disease in which patients
experience devastating effects on multiple systems of the body, leading
to debilitating or life-threatening complications. It is an ultra-rare
disease, which is defined as a disease that affects fewer than 20
patients per one million in the general population.1 HPP is
characterized by low alkaline phosphatase (ALP) activity and defective
bone mineralization that can lead to deformity of bones and other
skeletal abnormalities, as well as systemic complications such as
profound muscle weakness, seizures, pain and respiratory failure leading
to premature death in infants.2-6

Abstracts summarizing these studies were published on the ENDO website
and can be accessed using the links below.

The following abstracts will be presented in an oral session on Sunday,
April 3, 2016, from 11:45 a.m. to 1:15 p.m., Eastern Standard Time (EST):

  • Abstract OR26-2: “Reduction in Pain and Improved Function and
    Activities of Daily Living in Children with Hypophosphatasia Treated
    with Asfotase Alfa for 5 Years,” Phillips, et al.


  • Abstract OR26-3: “Biochemical and Physical Function Outcomes in
    Adolescents and Adults with Hypophosphatasia Treated with Asfotase
    Alfa for up to 4 Years: Interim Results from a Phase II Study,”
    Kishnani, et al.

    Accessible at:

The following abstract will be presented in a poster preview session on
Sunday, April 3, 2016, from 11:30 to 11:45 a.m., as well as a poster
session from 1:15 to 3:15 p.m., Eastern Standard Time (EST):

The following abstract will be presented in an oral session on Monday,
April 4, 2016, from 10:00 to 11:30 a.m., Eastern Standard Time (EST):

About Hypophosphatasia (HPP)

HPP is a genetic, chronic, progressive, and life-threatening ultra-rare
metabolic disease characterized by low alkaline phosphatase (ALP)
activity and defective bone mineralization that can lead to destruction
and deformity of bones and other skeletal abnormalities, as well as
systemic complications such as profound muscle weakness, seizures, pain,
and respiratory failure leading to premature death in infants.2-6

HPP is caused by mutations in the gene encoding an enzyme known as
tissue non-specific alkaline phosphatase (TNSALP).2,3 The
genetic deficiency in HPP can affect people of all ages.2 HPP
is traditionally classified by the age of the patient at the onset of
symptoms of the disease, with perinatal-, infantile- and juvenile-onset
HPP defined as patients who have their first symptom prior to 18 years
of age.

HPP can have devastating consequences for patients at any stage of life.2 In
a natural history study, infants who had their first symptom of HPP
within the first 6 months of life had high mortality, with an overall
mortality rate of 73 percent at 5 years.7 In these patients,
mortality is primarily due to respiratory failure.2,6,8 In
patients surviving and those with juvenile-onset HPP, long-term clinical
sequelae include recurrent and non-healing fractures, profound muscle
weakness, debilitating pain and the requirement for ambulatory assistive
devices such as wheelchairs, wheeled walkers and canes.2,5

About Strensiq® (asfotase alfa)

Strensiq® (asfotase alfa) is a highly innovative bone-targeted enzyme
replacement therapy that treats the underlying cause of HPP by replacing
the missing TNSALP enzyme. In clinical studies of patients with HPP who
had their first symptom prior to the age of 18, treatment with Strensiq
improved overall survival in infants, enhanced bone mineralization and
improved height, weight and mobility.

Strensiq is approved in the United States, European Union, Japan and

Important Safety Information

Hypersensitivity reactions have been reported in STRENSIQ-treated
patients. In clinical trials, 1 out of 99 treated patients (1%)
experienced signs and symptoms consistent with anaphylaxis.

Localized lipodystrophy, including lipoatrophy and lipohypertrophy, has
been reported at injection sites after several months in patients
treated with STRENSIQ.

Patients with HPP are at increased risk for developing ectopic
calcifications. In clinical trials, 14 cases (14%) of ectopic
calcification of the eye including the cornea and conjunctiva, and the
kidneys (nephrocalcinosis) were reported. There was insufficient
information to determine whether or not the reported events were
consistent with the disease or due to STRENSIQ. No visual changes or
changes in renal function were reported.

The most common adverse reactions reported were injection site
reactions, lipodystrophy, ectopic calcifications, and hypersensitivity

Please click
for the full Prescribing Information.

About Alexion

Alexion is a global biopharmaceutical company focused on developing and
delivering life-transforming therapies for patients with devastating and
rare disorders. Alexion developed and commercializes Soliris®
(eculizumab), the first and only approved complement inhibitor to treat
patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical
hemolytic uremic syndrome (aHUS), two life-threatening ultra-rare
disorders. As the global leader in complement inhibition, Alexion is
strengthening and broadening its portfolio of complement inhibitors,
including evaluating potential indications for eculizumab in additional
severe and ultra-rare disorders. Alexion’s metabolic franchise includes
two highly innovative enzyme replacement therapies for patients with
life-threatening and ultra-rare disorders, Strensiq® (asfotase alfa) to
treat patients with hypophosphatasia (HPP) and Kanuma™ (sebelipase alfa)
to treat patients with lysosomal acid lipase deficiency (LAL-D). In
addition, Alexion is advancing the most robust rare disease pipeline in
the biotech industry, with highly innovative product candidates in
multiple therapeutic areas. This press release and further information
about Alexion can be found at:




THE COUNCIL of 16 April 2014 on clinical trials on medicinal
products for human use, and repealing Directive 2001/20/EC.

2. Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev.
2013; 10(suppl 2):380-388.
3. Whyte MP. Hypophosphatasia: nature’s window on alkaline
phosphatase function in humans. In: Bilezikian JP, Raisz LG, Martin
TJ, eds. Principles of Bone Biology. Vol 1. 3rd ed. San Diego, CA:
Academic Press; 2008:1573-1598.
4. Whyte MP, Greenberg CR, Salman N, et al. Enzyme-replacement
therapy in life-threatening hypophosphatasia. N Engl J Med. 2012;
5. Seshia SS, Derbyshire G, Haworth JC, Hoogstraten J. Myopathy with
hypophosphatasia. Arch Dis Child. 1990; 65(1):130-131.
6. Baumgartner-Sigl S, Haberlandt E, Mumm S, et al.
Pyridoxine-responsive seizures as the first symptom of infantile
hypophosphatasia caused by two novel missense mutations (c.677T>C,
p.M226T; c.1112C>T, p.T371I) of the tissue-nonspecific alkaline
phosphatase gene. Bone. 2007; 40(6):1655-1661.
7. Whyte MP, Leung E, Wilcox W, et al. Hypophosphatasia: a
retrospective natural history study of the severe perinatal and
infantile forms. Poster presented at the 2014 Pediatric Academic
Societies and Asian Society for Pediatric Research Joint Meeting,
Vancouver, B.C., Canada, May 5, 2014. Abstract 752416.
8. Whyte MP, Rockman-Greenberg C, Hofmann C, et al. Improved
survival with asfotase alfa treatment in pediatric patients with
hypophosphatasia at high risk of death. Poster presented at the
American Society for Bone and Mineral Research (ASBMR) 2014 Annual
Meeting, Houston, September 14, 2014. Abstract 1097.


Alexion Pharmaceuticals, Inc.
Stephanie Fagan,
Senior Vice President, Corporate Communications
Diamond, 203-439-9600
Executive Director, Corporate Communications
Ridloff, CFA, 203-699-7722
Vice President, Investor Relations