Takeda reveals PhII fail for rare genetic condition, looks to new product pipeline to bolster growth

Take­da’s Phase II pro­gram in a rare hered­i­tary dis­ease called metachro­mat­ic leukody­s­tro­phy (MLD) is “like­ly to be dis­con­tin­ued” af­ter fail­ing its pri­ma­ry and sec­ondary end­points, the com­pa­ny re­vealed Thurs­day in its lat­est quar­ter­ly earn­ings re­port.

MLD is a fast-pro­gress­ing and fa­tal neu­rode­gen­er­a­tive con­di­tion that can af­fect pa­tients’ move­ment, speech, abil­i­ty to walk, and even be­hav­ior. It’s com­mon­ly di­ag­nosed in child­hood, and caused by the de­fi­cien­cy of an en­zyme called aryl­sul­fa­tase A. Take­da’s can­di­date, TAK-611, is an en­zyme re­place­ment ther­a­py that makes use of tech­nol­o­gy li­censed from Bio­Marin. How­ev­er, the com­pa­ny not­ed Thurs­day that the can­di­date “did not meet pri­ma­ry and sec­ondary end­points” in a Phase II study.

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