AstraZeneca fleshes out pivotal data on $930M rare blood disorder drug it wants to pair with Soliris, Ultomiris

As­traZeneca says its add-on pill for a rare blood dis­or­der known as parox­ys­mal noc­tur­nal he­mo­glo­bin­uria, or PNH, helped pa­tients al­ready tak­ing Ul­tomiris or Soliris pro­duce more he­mo­glo­bin than ei­ther ap­proved drug alone.

In an in­ter­im cut of da­ta of the AL­PHA study, 63 pa­tients with PNH who ex­pe­ri­ence symp­toms re­lat­ed to ex­travas­cu­lar he­mol­y­sis — when blood cells are de­stroyed out­side blood ves­sels — saw their he­mo­glo­bin lev­els in­crease by an av­er­age of 2.94 g/dL on dan­i­co­pan and ei­ther Ul­tomiris or Soliris com­pared to 0.50 g/dL in those pa­tients tak­ing Ul­tomiris or Soliris and place­bo at 12 weeks.

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