Disc Medicine blames ‘outsized’ placebo response on mixed Phase 2 data in rare blood disorder

De­spite dri­ving re­duc­tions in a key bio­mark­er, Disc Med­i­cine’s drug for a rare blood dis­or­der flunked on ef­fi­ca­cy in a Phase 2 study. The com­pa­ny at­trib­uted the neg­a­tive read­out to an “out­sized place­bo re­sponse,” but that wasn’t enough to stop a rout on its stock.

Disc set out to see if its drug can help pa­tients with ery­thro­poi­et­ic pro­to­por­phyr­ia (a con­di­tion that ren­ders them ex­treme­ly sen­si­tive to sun­light, of­ten ex­pe­ri­enc­ing acute pain), low­er lev­els of a tox­ic metabo­lite un­der­ly­ing the dis­ease, and spend more time un­der the sun with­out pain.

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