A partnership—and potential purchase

NEW HAVEN, Conn. & AMSTERDAM—Alexion Pharmaceuticals Inc. and Complement Pharma have inked an agreement under which the companies will co-develop the latter's CP010, a preclinical C6 complement inhibitor for neurodegenerative disorders. Per the terms of the agreement, Complement Pharma stands to receive up to €14 million in milestone-dependent payments through Phase 1b development of CP010. The companies will work together to advance CP010, with Complement Pharma being responsible for manufacturing the compound and conducting preclinical and Phase 1 studies. This deal extends through the completion of Phase 1b development, and Alexion will have an option to acquire Complement during the duration of their partnership.

“Alexion has been a pioneer in complement biology since the development of Soliris, the world’s first complement inhibitor, which has demonstrated the significant impact that C5 inhibition can have on several diseases caused by uncontrolled complement activation,” Dr. John Orloff, executive vice president and head of Research & Development at Alexion, said in a press release. “This collaboration provides an exciting opportunity to expand on our more than two decades of complement expertise to potentially treat additional diseases by targeting C6, a different component of the complement system.”

CP010, a humanized monoclonal antibody, binds to C6 in circulation to inhibit its activity in the body. By blocking C6, the formation of the destructive membrane attack complex (MAC), a complex of terminal complement proteins that mediate cellular injury in the wake of complement activation, is also blocked. As MAC has been implicated in neurodegeneration and CP010 has been able to inhibit C6 in vitro and in vivo, it is thought that this approach could potentially treat a number of central nervous system disorders.

“Our understanding of the role of complement in many disorders continues to increase and many neurodegenerative diseases show activation of the complement system, even in early stages of disease,” said Dr. Frank Baas, chief scientific officer at Complement Pharma. “We believe that C6 inhibition has the potential to treat multiple neurodegenerative disorders, and we look forward to working with Alexion, a partner with a worldwide leading position in complement biology, to realize the possibilities of this approach.”

And moving from news of a potential acquisition to a confirmed one, Alexion announced on May 25 that its offer for all outstanding shares of Wilson Therapeutics has been accepted by the latter's shareholders—to the tune of 97.7 percent of the total shares and votes in the company. More than 90 percent of the total shares were tendered to the offer, and in light of that fact, and the fulfillment of all offer conditions and regulatory approval, the offer has been declared unconditional, with settlement slated to take place on May 30. Alexion extended the acceptance period until June 8 to give shareholders extra time to tender their shares to the offer, and settlement for those who do so is expected on June 15.

Alexion announced its plans to acquire the Stockholm-based company on April 11 with the news that it had made a recommended public cash offer to Wilson Therapeutics' shareholders through a wholly owned subsidiary. The offer's total equity value comes to SEK 7,100 million, based on outstanding shares on a fully diluted basis, or approximately $855 million. Wilson Therapeutics focuses on the development of novel therapies for individuals with rare copper-mediated disorders, including WTX101, a Phase 3 compound being advanced as a treatment for Wilson disease. Wilson disease is a rare genetic disorder that drastically affects the liver and central nervous system.

“Alexion is a global leader in rare diseases with a proven record of developing and commercializing therapies for patients with rare diseases, making them a great partner to make WTX101 available to Wilson disease patients worldwide,” said Jonas Hansson, CEO of Wilson Therapeutics, at the time of the initial announcement.

“The acquisition of Wilson Therapeutics is a key first step in rebuilding our pipeline,” Ludwig Hantson, CEO of Alexion, commented in a statement. “We look forward to using our significant experience in rare metabolic and neurological diseases to improve treatment for patients with Wilson disease through the development of WTX101, which has the potential to be the first new treatment in more than 20 years and become the new standard of care for Wilson disease.”