Research and Development Progress:
Alexion currently has development programs underway with its five highly innovative therapeutic candidates: eculizumab (Soliris) and four additional novel therapeutic candidates beyond eculizumab that have the potential to become first-in-class therapies for patients with other severe and ultra-rare disorders.
Ultra-Rare Disease Programs With Eculizumab
- Nephrology- STEC-HUS: Data from the full cohort of 198 enrolled patients in the Company-sponsored Shiga-toxin-producing E. coli hemolytic uremic syndrome (STEC-HUS) trial were presented at the American Society of Nephrology (ASN) meeting. Preliminary findings from an exploratory post hoc, matched-control analysis of patients with severe STEC-HUS receiving eculizumab versus other patients who received only best supportive care during the German epidemic were also reported at ASN.
- Nephrology- Kidney Transplant: Eculizumab is now being evaluated in two different potential kidney transplant indications. Enrollment is ongoing in Company-sponsored, multi-national, living-donor and deceased-donor kidney transplant trials in patients at elevated risk of Acute Humoral Rejection (AHR), also known as antibody mediated rejection. Alexion is also expanding its kidney transplant program to include a delayed-graft function (DGF) clinical trial.
- Neurology- NMO: The Company has commenced discussions with regulators in both the United States and Europe to discuss plans for a Company-sponsored multi-national, placebo-controlled, registration trial in relapsing neuromyelitis optica (NMO).
- Neurology- MG: Alexion continues to work with investigators to design the next clinical trial to evaluate eculizumab as a treatment for patients with severe myasthenia gravis (MG).
Ultra-Rare Disease Programs With Highly Innovative Therapeutic Candidates Beyond Eculizumab
- Asfotase Alfa: A natural history study is ongoing in infants with hypophosphatasia (HPP), an ultra-rare, inherited and life-threatening metabolic disease. The Company is also completing optimization of the manufacturing process for asfotase alfa.
- cPMP Replacement Therapy: Alexion is developing a cPMP replacement therapy for the treatment of patients with Molybdenum Cofactor Deficiency Type A, a severe, ultra-rare and genetic metabolic disorder that is fatal in newborns. The Company continues to accelerate the regulatory and manufacturing processes for this therapeutic candidate and expects to initiate clinical studies in mid-2013.
- ALXN1102/ALXN1103: Enrollment continues in a Phase I study to characterize the mechanism of action and develop initial safety data for ALXN1102 and ALXN1103, intravenous and sub-cutaneous versions, respectively, of one of Alexion’s novel complement inhibitors.
- ALXN1007: The Company has completed dosing in a Phase I study of ALXN1007, a novel anti-inflammatory antibody, to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of this therapeutic candidate in healthy volunteers.
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