Alex­ion gam­bles on an ear­ly-stage drug for rare dis­eases, bag­ging Ap­ple Tree-backed Syn­tim­mune in $1.2B buy­out

Alex­ion Phar­ma­ceu­ti­cals is adding a clin­i­cal-stage as­set to the pipeline, buy­ing Syn­tim­mune for $400 mil­lion up­front with an­oth­er $800 mil­lion on the ta­ble for mile­stones.

The big prize here is the an­ti­body SYNT001, which is be­ing stud­ied for rare IgG-me­di­at­ed dis­eases. The lead pro­gram is a Phase Ib/IIa study that is ex­am­in­ing its ef­fi­ca­cy in warm au­toim­mune he­molyt­ic ane­mia (WAI­HA) and in pa­tients with pem­phi­gus vul­garis (PV) or pem­phi­gus fo­li­aceus. 

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