Achillion is mainly focused on the advancement of its oral small molecule complement inhibitors into late-stage development and commercialisation.
The deal adds Alexion’s product portfolio with two clinical-stage oral small molecule Factor D inhibitors. It will also serve as the foundation and enhance its expertise for a wider oral Factor D inhibition development platform to treat multiple additional complement-mediated diseases.
Factor D, a key serine protease, is a major control point in the alternative pathway (AP) of the complement system.
Alexion said that it will continue development of Achillion’s oral Factor D inhibitor portfolio, which includes two clinical-stage medicines-in-development such as danicopan (ACH-4471) and ACH-5228, as well as multiple compounds in preclinical development.
Danicopan’s phase 3 development is being commenced as an add-on therapy for paroxysmal nocturnal hemoglobinuria (PNH) patients with extravascular hemolysis (EVH).
Danicopan is also under Phase 2 development for C3G, while ACH-5228 is in phase 2 development for PNH.
PNH is a chronic, progressive, and ultra-rare blood disorder characterized by hemolysis, while C3G is an ultra-rare kidney disease that is not having approved treatment.
Achillion secured a breakthrough therapy designation for danicopan in combination with a C5 monoclonal antibody to treat patients with PNH who are sub-optimal responders to a C5 inhibitor alone.
Alexion CEO Dr Ludwig Hantson said: “The acquisition of Achillion adds two clinical-stage Factor D inhibitors to our growing pipeline, representing important continued momentum in expanding and diversifying our portfolio and advancing our mission of transforming the lives of people with rare diseases.
“We believe oral Factor D inhibition holds great promise in treating people with multiple rare, complement-mediated diseases, providing the opportunity to significantly expand our portfolio into new therapeutic areas and to help many more patients.”
In November 2018, Alexion Pharmaceuticals completed its previously announced acquisition of US-based clinical-stage biotechnology firm Syntimmune in a deal worth $1.2bn.