BioWorld Today Contributing Writer

Alexion Pharmaceuticals Inc. will tackle a new category of disease following its acquisition of key technology assets from Germany-based Orphatec Pharmaceuticals GmbH. Alexion will take over development of an investigational therapy for molybdenum cofactor deficiency (MoCD) Type A, a rare disease that causes brain damage and death in infants. The move follows a solid earnings report, featuring Soliris sales of $156 million for the fourth quarter.

Assets from Orphatec and recently acquired Alexion are part of a strategy to diversify Alexion's early stage pipeline. "We selected Taligen and Orphatec for their close alignment with what we know well and do well with regard to technology, research and development, and our overall focus on life-transforming treatments for patients with life-threatening rare disorders," said CEO Leonard Bell during Thursday morning's conference call.

Alexion, of Cheshire, Conn., will acquire the technology for $3 million up front, with future regulatory and commercial success milestone payments.

Infants with MoCD Type A have a genetic deficiency of the protein cPMP. The result is a loss of molybdenum cofactor. Babies who suffer from MoCD generally live only weeks or months after birth as neurotoxin sulfites build up in their bodies, causing seizures followed by widespread neurologic damage, then death. There are no treatments on the market for the disorder.

Orphatec's investigational therapy seeks to replace the missing cPMP, and enable normal MoCD production. Early clinical results in Germany and Australia have been encouraging.

In a case study reported in the May 2010 issue of Pediatrics, researchers treated a 36-day old infant diagnosed with MoCD with 80 to 160 µg of cPMP per kg of body weight. Urinary markers of sulfite oxidase and xanthine oxidase deficiency disappeared within one to two weeks. Symptoms also faded, with return of normal rhythms on ECG.

Jefferies and Co. Inc. analyst Eun K. Yang noted, "Acquisitions bolster ALXN's early pipeline and diversify beyond Soliris; focus remains on ultra-orphan diseases." Jefferies, however, remains "on the sidelines" due to the already rich valuation of Alexion stock.

Alexion's product portfolio includes its flagship product Soliris (eculizumab) for paroxysmal nocturnal hemoglobinuria plus a pipeline of complement-targeted therapies.

Soliris was the first marketed complement inhibitor. According to Alexion, of Cheshire, Conn., the complement protein C5 is involved in mediating the immune response that can cause damaging inflammation. Soliris is a fully humanized monoclonal antibody targeted at C5 that has been approved for paroxysmal nocturnal hemoglobinuria.

Alexion is developing Soliris in other therapeutic areas including atypical hemolytic uremic syndrome, kidney transplant rejection and dense deposit disease.

In hematology, the company is working on catastrophic anti-phospholipid syndrome and cold agglutinin disease. And in the neurology space, Alexion is testing Soliris in myasthenia gravis.

Another of Alexion's pipeline products, anti-CD200 antibody (samalizumab) is in clinical trials for B-chronic lymphocytic leukemia. At the American Society of Hematology meeting in December, Alexion reported that samalizumab was well tolerated at all doses and exhibited dose-dependent biological and pharmacokinetic responses with initial evidence of antitumor activity.

Alexion also reported its fourth quarter and year-end earnings Thursday. Soliris sales for the fourth quarter of 2010 were $156 million, up from $110.6 million in 2009, and for all of 2010, sales were $541 million, up from $387 million.

Alexion attributed the increase in sales to increasing penetration in its core markets of the U.S., Europe and Japan, as new patients responded to awareness campaigns for earlier identification and treatment of PNH.

Alexion's non-GAAP the fourth quarter net income was $48.6 million, 51 cents per share, with operating expenses of $82.8 million, including a $5.1 million increase in research and development expenses over the same period in 2009. The company said that the increase reflected expansions in clinical trial programs.

Its non-GAAP income for all of 2010 was $167.3 million, or $1.78 per share, compared to $108.4 million the previous year. Alexion closed 2010 with $361.6 million in cash, cash equivalents and securities, up from $176.2 million on Dec. 31, 2009. It spent $114 million of its cash on the acquisition of Taligen Therapeutics Inc. and Orphatec in January and February of 2011. (See BioWorld Today, Feb. 1, 2011.)

Alexion's non-GAAP results exclude share-based compensation and taxes that are not payable in cash as well as expenses related to the acquisitions of Orphatec and Taligen.

On Jan. 31, Alexion acquired Taligen for $111 million up front with additional contingent payments based on clinical and regulatory milestones.

Taligen's lead compound TT30 for wet age-related macular degeneration (AMD) targets complement receptor 2 and factor H. TT30 is set to become a follow-on to Soliris as well as rounding out Alexion's assets in the area of complement inhibitors.