Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that
researchers will present new functional and quality-of-life data
from clinical studies in children with hypophosphatasia (HPP) who
were treated with asfotase alfa for at least three years at the
Endocrine Society’s 97th Annual Meeting and Expo (ENDO).
Researchers will also present data from two patient-reported
surveys characterizing the burden of HPP in adults. Abstracts
summarizing these studies were published on the ENDO website and
can be accessed using the links below. The ENDO annual meeting will
be held March 5-8, 2015, in San Diego.
The following abstracts will be presented in a poster session on
Friday, March 6, 2015, from 1:00 to 3:00 p.m., Pacific Standard
Time (PST):
- Abstract FRI-224: “Improved Activities
of Daily Living and Physical Function, with Decreased Pain, in
Children with Hypophosphatasia Treated for Three Years with
Asfotase Alfa: Results from the Childhood Health Assessment
Questionnaire and the Pediatric Outcomes Data Collection
Instrument,” Phillips, et al.Accessible at:
https://endo.confex.com/endo/2015endo/webprogram/Paper21021.html
- Abstract FRI-240: “Burden of Disease in
Adult Patients with Hypophosphatasia: Results from Patient-Reported
Outcome Surveys,” Weber, et al.Accessible at:
https://endo.confex.com/endo/2015endo/webprogram/Paper20871.html
The following abstract will be presented in an oral session on
Saturday, March 7, 2015, from 11:30 a.m. to 1:00 p.m., Pacific
Standard Time (PST):
- Abstract OR29-4: “Significantly
Improved Muscle Strength, Running Speed, and Agility in Children
with Hypophosphatasia Treated with Asfotase Alfa,” Phillips, et
al.Accessible at:
https://endo.confex.com/endo/2015endo/webprogram/Paper20906.html
About Hypophosphatasia (HPP)
HPP is a genetic, chronic and progressive ultra-rare metabolic
disease characterized by defective bone mineralization that can
lead to destruction and deformity of bones, profound muscle
weakness, seizures, respiratory failure and premature death.1-5
HPP is caused by mutations in the gene encoding an enzyme known
as tissue non-specific alkaline phosphatase (TNSALP).1,2 The
genetic deficiency in HPP can affect people of all ages.1 HPP is
classified by the age of the patient at the onset of symptoms of
the disease, and infantile- and juvenile-onset HPP is defined as
manifestation of the first symptom prior to 18 years of age.
HPP can have devastating consequences for patients at any stage
of life.1 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% at 5 years.6 In
these patients, mortality is primarily due to respiratory
failure.1,5,7 In patients surviving to adolescence and adulthood,
long-term clinical sequelae include recurrent and non-healing
fractures, debilitating weakness, severe pain and the requirement
for ambulatory assistive devices such as wheelchairs, wheeled
walkers and canes.1,4
About Asfotase Alfa
Asfotase alfa is an investigational, highly innovative,
first-in-class enzyme replacement therapy. Asfotase alfa is
designed to address the underlying cause of HPP by aiming to
restore the genetically defective metabolic process, thereby
preventing or reversing the severe and potentially life-threatening
complications of life-long dysregulated mineral metabolism.
In 2013, the FDA granted Breakthrough Therapy designation for
asfotase alfa. 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. In 2014, Alexion completed regulatory filings in the
U.S., Europe and Japan for asfotase alfa for the treatment of
HPP.
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 paroxysmal nocturnal hemoglobinuria (PNH) and
atypical hemolytic uremic syndrome (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 nearly 40
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, including
asfotase alfa, across multiple therapeutic areas. This press
release and further information about Alexion can be found at:
www.alexionpharma.com.
[ALXN-G]
Safe Harbor Statement
This news release contains forward-looking statements, including
statements related to potential medical benefits of asfotase alfa
for hypophosphatasia (HPP). Forward-looking statements are subject
to factors that may cause Alexion's results and plans to differ
from those expected, including, for example, decisions of
regulatory authorities regarding marketing approval or material
limitations on the marketing of asfotase alfa for HPP, delays in
arranging satisfactory manufacturing capabilities and establishing
commercial infrastructure for asfotase alfa for HPP, the
possibility that results of clinical trials are not predictive of
safety and efficacy results of asfotase alfa in broader or
different patient populations, the risk that third party payors
(including governmental agencies) will not reimburse for the use of
asfotase alfa (if approved) at acceptable rates or at all, the risk
that estimates regarding the number of patients with asfotase alfa
and observations regarding the natural history of patients with
asfotase alfa are inaccurate, and a variety of other risks set
forth from time to time in Alexion's filings with the Securities
and Exchange Commission, including but not limited to the risks
discussed in Alexion's Quarterly Report on Form 10-Q for the period
ended September 30, 2014. Alexion does not intend to update any of
these forward-looking statements to reflect events or circumstances
after the date hereof, except when a duty arises under law.
References
1.
Rockman-Greenberg C. Hypophosphatasia.
Pediatr Endocrinol Rev. 2013; 10(suppl 2):380-388.
2.
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.
3.
Whyte MP, Greenberg CR, Salman N, et al.
Enzyme-replacement therapy in life-threatening hypophosphatasia. N
Engl J Med. 2012; 366(10):904-913.
4.
Seshia SS, Derbyshire G, Haworth JC,
Hoogstraten J. Myopathy with hypophosphatasia. Arch Dis Child.
1990; 65(1):130-131.
5.
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.
6.
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.
7.
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:MediaIrving Adler, 203-271-8210Executive Director,
Corporate CommunicationsorKim Diamond, 203-439-9600Senior Director,
Corporate CommunicationsorInvestorsElena Ridloff, CFA,
203-699-7722Executive Director, Investor Relations
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