Eyes forward for AMD and more

Alexion acquires Taligen Therapeutics and creates Translational Medicine Group to accelerate development of expanded portfolio

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CHESHIRE, Conn.—With an upfront cash payment of $111 million for 100 percent of the company's equity interests, Alexion Pharmaceuticals Inc. recently acquired Taligen Therapeutics Inc., a Cambridge, Mass.-based, privately held development-stage biotechnology company.  

According to Dr. Leonard Bell, CEO and principal founder of Alexion, the acquisition broadens Alexion's portfolio of product candidates and expands Alexion's capabilities in translational medicine by bringing additional accomplished researchers to the company. Alexion feels that with the acquisition, the company is gaining promising preclinical compounds, including potential treatments for patients with ophthalmic diseases such as age-related macular degeneration (AMD), as well as other novel antibody and protein regulators of the complement inflammatory pathways—the complement system being a component of the innate immune system in which proteins circulating in the blood work to clear out pathogens.

In addition to the upfront payment, additional contingent payments would be earned upon reaching various clinical efficacy and product approval milestones in both the United States and European Union for up to six products.

While Taligen as a distinct company is no more, its former scientific staff will now form the core for Alexion's new Cambridge-based Translational Medicine Group, headed by former Taligen CEO Dr. Abbie Celniker, who is now head of translational medicine at Alexion. Celniker will report to Dr. Stephen Squinto, executive vice president and head of research and development at Alexion.

The new Translational Medicine Group will enable Alexion to accelerate Taligen's "highly innovative" product candidates, targeting patients with devastating and ultra-rare disorders, says David Hallal, senior vice president of global commercial operations for Alexion. "As a result of this acceleration, we expect to commence new clinical studies in rare disease with Taligen's novel first-in-class complement inhibitors within the next 12 to 24 months."

Hallal says that Alexion's ophthalmology program "has now been importantly expanded" with the acquisition of early-stage product candidates from Taligen, and pointed to progress on that front in particular with an ongoing proof-of-concept Phase II study being conducted with eculizumab in patients with the dry form of AMD. He says Alexion expects enrollment in that study to be completed in the first half of 2011, with preliminary data expected late in the year.

"With regard to Taligen's programs," Hallal says, "it is well-established that AMD is linked to uncontrolled activations of the alternative complement pathway; thus, by accelerating the investigation of Taligen's distinct and targeted alternative complement pathway inhibitors, while also completing our proof-of-concept studies with eculizumab, we are now best positioned to execute our plan to develop the optimal form of complement inhibition for local ophthalmic use."

Piper Jaffray analyst Ian Somaiya wrote in a research note about the acquisition: "We see this acquisition as a positive as it broadens Alexion's preclinical pipeline and limits potential competitors" and he called Taligen's intellectual property estate "compelling and unique."

"We believe this acquisition fits in well with Alexion's core biologic focus on complement mediators, but that given the early stage of Taligen's pipeline, it is unlikely to have a meaningful impact on Alexion in the near/medium term," noted Wells Fargo analyst Brian Abrahams in another research note.

Taligen's talented scientists and technology will enhance Alexion's existing staff and research and development programs, substantially increasing Alexion's ability to develop first-in-class therapies for patients with severe diseases, Alexion's CEO said in the news release about the deal, adding: "As product development opportunities continue to expand, we look forward to increasing the quality, speed, and throughput of our combined current and future development programs for the benefit of patients worldwide."

"Alexion has proven how highly innovative science can result in life-transforming therapies for patients with debilitating disorders," added Celniker in the same news release. "We are excited to be combining our research and development capabilities with Alexion's global team with the goal of accelerating the investigation of novel molecules from our combined portfolios and developing additional first-in-class compounds."

Alexion is a biopharmaceutical company working to develop and deliver life-changing drug therapies for patients with serious and life-threatening medical conditions, in many cases rare and ultra-rare diseases, in the arenas of hematologic and kidney diseases, neurologic disorders, transplants, inflammatory disorders and cancer. Soliris (eculizumab), Alexion's first marketed product, is approved in more than 35 countries as a therapy for patients with PNH, a debilitating and ultra-rare life-threatening blood disorder. Alexion is evaluating other potential indications for Soliris and is pursuing development of other innovative biotechnology product candidates in early stages of development.

Alexion acquires investigational therapy for infants suffering from ultra-rare genetic neurologic disorder

CHESHIRE, Conn.—Alexion's interest in rare disease treatments also served as the springboard for another announcement shortly after the Taligen acquisition, about the purchase of patents and assets from Cologne, Germany-based Orphatec Pharmaceuticals GmbH. Those assets are related to an investigational therapy for patients with molybdenum cofactor deficiency (MoCD) Type A, a devastating ultra-rare genetic disorder characterized by severe brain damage and rapid death in newborns. In addition to the intellectual property acquisition, Alexion has established a research collaboration with key MoCD researchers from Orphatec to accelerate development of the investigational therapy.

MoCD Type A involves a genetic deficiency of cPMP, causing a deficiency of molybdenum cofactor that in turn leads to catastrophic brain damage, with survival generally measured in weeks or months. Deficiency of the cofactor leads to accumulation of neurotoxic sulfite, resulting in uncontrollable seizures, severe and rapid neurological damage, and death. There are currently no treatment options for patients with MoCD Type A.

The investigational therapy is designed to replace the deficient cPMP, which enables MoCD production so that the infant's body can eliminate the toxic sulfite. Scientific discoveries underlying this highly innovative therapy were pioneered in Germany, and have led to encouraging early clinical experience with cPMP replacement therapy in several newborns. Investigators in Germany and Australia have reported clinically meaningful results in the first patient treated.

In Alexion's fourth quarter 2010 financial report, David Hallal, senior vice president of global commercial operations for Alexion, noted, after discussing aspects of the acquisition of Taligen earlier in the month: "We have further expanded our commitment to develop and deliver highly innovative treatments for patients and their families suffering from devastating, ultra-rare disorders with our acquisition this morning of Orphatec's candidate cPMP therapy for MoCD Type A. Given the extreme need for this potentially life-saving treatment, our new Translational Medicine Group [formerly the CEO and staff of Taligen] is accelerating this program. Our next steps are to generate cPMP clinical supplies and expedite IND-enabling studies. Our sense of urgency is driven by the absence of available treatments together with the very encouraging, positive early clinical experience with cPMP."

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