Alexion-Complement Pharma Tie Up To Treat Neuro Disorders

 | Jun 11, 2018 09:16PM ET

Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced a partnership with Complement Pharma to co-develop the preclinical C6 complement inhibitor, CP010, for neurodegenerative disorders.

CP010 is a humanized monoclonal antibody in preclinical stages that binds to C6 in circulation to inhibit its function throughout the body by preventing MAC formation in both the periphery and the central nervous system.

Terms of the Partnership

Per the terms, Alexion and Complement Pharma will collaborate on the development program for CP010. Complement Pharma will be responsible for conducting preclinical and phase I studies as well as manufacturing CP010. In exchange, Alexion will provide Complement Pharma with up to €14 million in milestone-dependent payments through phase Ib development.

The phased agreement extends through the completion of phase Ib development. Moreover, Alexion has the option to acquire Complement Pharma during the term of the agreement.

Rationale of the Partnership

Alexion is looking to diversify its portfolio beyond its key growth driver, Soliris. Soliris is approved for the treatment of two severe and ultra-rare disorders resulting from chronic uncontrolled activation of the complement component of the immune system – paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS).

Since Soliris, a C5 inhibitor, has demonstrated the significant impact that C5 inhibition can have on various diseases caused by uncontrolled complement activation, the company plans to use its expertise to potentially treat additional diseases by targeting C6, a different component of the complement system.

The company’s other pipeline candidate, ALXN1210 is an innovative, long-acting C5 inhibitor that works by inhibiting the C5 protein in the terminal complement cascade.

Our Take

While the underlying growth of Soliris has been robust, Alexion is looking to develop its pipeline and reduce dependence on the former. The partnership with Complement Pharma is a step in the same direction and will also augment Alexion’s C5 franchise.

Earlier, Alexion announced that it will acquire Sweden-based Wilson Therapeutics for $855 million. The acquisition will add a late-stage candidate, WTX101, to Alexion’s pipeline. The candidate is currently in phase III for the treatment of Wilson disease, a rare genetic disorder. The candidate is a first-in-class oral copper-binding agent with a unique mechanism of action and ability to access and bind copper from serum and promote its removal from the liver.

The company also plans to use Halozyme's (NASDAQ:HALO) ENHANZE drug-delivery technology to develop a next-generation subcutaneous formulation of ALXN1210 to potentially further extend the dosing interval to once every two weeks or once per month.