In a release on
The year-on-year increase in Q4 net product sales of 38 percent reflected steady additions of new patients with paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome commencing Soliris treatment.
Soliris is approved in nearly 50 countries for the treatment of patients with PNH, including
Full Year 2013 Non-GAAP Financial Results
Alexion's non-GAAP operating expenses for the full year 2013 were
Full Year 2013 GAAP Financial Results Alexion reported GAAP net income of
Alexion's GAAP operating expenses for the full year 2013 were
Fourth Quarter Non-GAAP Financial Results
Alexion's non-GAAP operating expenses for Q4 2013 were
Fourth Quarter GAAP Financial Results Alexion reported a GAAP net loss of
On a GAAP basis, operating expenses for Q4 2013 were
Balance Sheet As of
"In 2013, we provided Soliris to an increasing number of patients with PNH and aHUS worldwide. We demonstrated steady growth in PNH, grew steadily the number of new patients with aHUS receiving Soliris in the U.S. and the first countries of
Research and Development Progress Alexion currently has development programs underway with eculizumab (Soliris) and additional therapeutic candidates.
Ultra-Rare Disease Programs With Eculizumab
-Transplant: Antibody-Mediated Rejection - Enrollment is ongoing in the Company-sponsored, multinational living-donor kidney transplant trial in patients at elevated risk of AMR and in the expanded Company-sponsored, multinational deceased-donor kidney transplant trial in patients at elevated risk of AMR.
-Transplant: Delayed Graft Function - Alexion is planning to commence a single, multinational registration trial for the prevention of delayed graft function in renal transplant patients. Earlier this month, eculizumab received an orphan drug designation from the
-Neurology: Neuromyelitis Optica - Alexion is planning to commence a single, multinational, placebo-controlled, registration trial in relapsing NMO.
-Neurology: Myasthenia Gravis - Alexion is planning to commence a single, multinational, placebo-controlled, registration trial in severe, refractory MG.
Ultra-Rare Disease Programs
-Asfotase Alfa: Alexion is developing asfotase alfa as a treatment for patients with pediatric-onset hypophosphatasia, an ultra-rare, inherited and life-threatening metabolic disease. The Company received Breakthrough Therapy designation for asfotase alfa in pediatric-onset HPP in Q2 2013. Alexion completed the initial analysis of its natural history study in infants with HPP and has now initiated a natural history study in juveniles with HPP.
-cPMP Replacement Therapy (ALXN 1101): Alexion is developing cPMP as a treatment for patients with Molybdenum Cofactor Deficiency Type A, a severe, ultra-rare and genetic metabolic disorder that causes catastrophic and irreversible neurologic damage within the first few weeks of life. The Company received Breakthrough Therapy designation for cPMP replacement therapy for patients with MoCD Type A in Q3 2013. A natural history study in MoCD patients is ongoing and Alexion plans to initiate a synthetic cPMP bridging study.
-ALXN1007: Alexion is preparing to commence two Phase 2 proof-of- concept studies of ALXN1007, a novel anti-inflammatory antibody, in severe and life-threatening ultra-rare disorders.
Establishment of mRNA Research Capabilities Beyond its current development programs, the Company announced on
2014 Financial Guidance
In 2014, worldwide net product sales are expected to be within a range of
Alexion is a biopharmaceutical company.
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In a release on