- Late-Stage Investigational Enzyme Replacement Therapy for Ultra-Rare Life-Threatening Genetic Metabolic Disease -
- Conference Call Scheduled for Tomorrow Morning -
Enobia's lead product candidate ENB-0040 (asfotase alfa), is a human recombinant targeted alkaline phosphatase enzyme-replacement therapy for patients suffering with hypophosphatasia (HPP), an ultra-rare, life-threatening, genetic metabolic disease for which there are no approved treatment options. Alexion will acquire full worldwide development and commercial rights to asfotase alfa. Asfotase alfa was awarded orphan drug designation in the U.S. and EU in 2008 and Fast Track status in the U.S. in 2009, and is currently in Phase II clinical development.
"Hypophosphatasia is an ultra-rare and life-threatening disease, and
those patients who survive live with debilitating morbidities including
skeletal deformity, severe muscle weakness, and progressive damage to
vital organs," said
"Alexion has proven expertise in developing and commercializing
therapies to transform the lives of patients with severe and ultra-rare
disorders, making them the ideal partner to advance the work of the
Enobia team and bring asfotase alfa to HPP patients around the world,"
said
"Enobia and our scientific collaborators have developed an elegant
compound showing very promising clinical results to date," said Dr.
The Transaction
Alexion will acquire Enobia in an all-cash transaction. Under the terms
of the agreement, Alexion has agreed to pay
Alexion intends to finance the acquisition through cash on hand and
Goldman, Sachs & Co. is acting as financial advisor to Alexion.
About Hypophosphatasia (HPP)
HPP is an ultra-rare, genetic, and life-threatening metabolic disease characterized by defective bone mineralization and impaired phosphate and calcium regulation leading to progressive damage to multiple vital organs including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure.1,2,3,4 The severe manifestations of the genetic deficiency in HPP affect people of all ages, and approximately 50 percent of infants with the disease do not survive past one year of age.1
HPP is caused by a genetic deficiency of an enzyme known as tissue non-specific alkaline phosphatase (TNSALP), which causes life-long abnormalities in metabolism of the two vital minerals calcium and phosphate, leading directly to the debilitating morbidities and premature mortality of the disease.1 There are currently no therapies approved for HPP.1
About Asfotase Alfa
Asfotase alfa is an investigational, highly innovative, first-in-class recombinant protein that addresses the underlying cause of HPP by targeting replacement of the missing enzyme to the necessary body tissues. Asfotase alfa is designed to normalize the genetically defective metabolic process and prevent or reverse the severe and life-threatening complications of life-long dysregulated mineral metabolism in patients with HPP.
2012 Initial Financial Outlook
Alexion will provide 2012 financial guidance in February, including one-time expenses related to the Enobia acquisition. 2012 non-GAAP research and development expenses are expected to transiently rise to approximately 20 to 21% of sales, due to activities associated with Enobia's programs, and then to return to the Company's target of approximately 17% to 18% of sales in 2013. Non-GAAP selling, general and administrative expenses associated with the proposed acquisition are expected to have limited impact in 2012.
Alexion is reiterating all areas of 2011 guidance provided in its third quarter 2011 earnings announcement in October.
Conference Call Information
Alexion will host a conference call tomorrow,
About
About Alexion
[ALXN-G]
Safe Harbor Statement
This news release contains forward-looking statements, including
statements related to potential benefits from the acquisition of Enobia,
and its employees, technology and product candidate; statements related
to guidance regarding anticipated financial results for 2011 and 2012,
potential medical benefits for asfotase alfa for hypophosphotasia, and
assessment of the Company's financial position and commercialization
efforts. 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 Soliris
for its current or potential new indications, and a variety of other
risks set forth from time to time in Alexion's filings with the
| References | |
| 1. |
Mornet E. Review of Hypophosphatasia. |
| 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:
Molecular Mechanisms of Metabolic Bone Disease, Chapter 73:
1573-1598. |
| 4. | Silver MM, Vilos GA, Milne KJ. Pulmonary Hypoplasia in Neonatal Hypophosphatasia. Pediatr Pathol. 1998. 8:483-493. |
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