Ul­tragenyx shares surge as block­buster hope­ful buro­sum­ab con­tin­ues to per­form in piv­otal study

Just in time for its R&D day to­day, Uly­tragenyx $RARE and its part­ners at Ky­owa Hakko Kirin out­lined up­dat­ed 48 week da­ta on buro­sum­ab, which is on track to get a like­ly FDA ap­proval in the spring.

Mov­ing the goal line out to 48 weeks, from 24, re­searchers say that 84% of pa­tients suf­fer­ing from X-linked hy­pophos­phatemia — or XLH — and on the drug from the start of the tri­al hit their goal on serum phos­pho­rus lev­els, which was the pri­ma­ry end­point. And 89% of the switchovers from the place­bo group al­so hit their marks on the bio­mark­er for ef­fi­ca­cy.

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Life Sciences Senior Associate

General Catalyst

San Francisco, CA, USA