Ap­plied Ther­a­peu­tics says rare dis­ease drug fails PhI­II tri­al, still plans to seek ap­proval

Ap­plied Ther­a­peu­tics, which is de­vel­op­ing a treat­ment for a rare dis­ease that af­fects chil­dren who can’t process a sug­ar called galac­tose, missed the pri­ma­ry end­point in a Phase III study, but the com­pa­ny says its da­ta are strong enough to pur­sue reg­u­la­to­ry ap­proval in the US and in Eu­rope.

The New York-based biotech on Mon­day rolled out da­ta that as­sessed how AT-007, al­so known as gov­ore­stat, worked in 42 chil­dren be­tween the ages of 2 and 17 years over an 18-month pe­ri­od.

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