PTC claims PhI­II win in rare ge­net­ic dis­ease

A PTC Ther­a­peu­tics treat­ment for phenylke­tonuria, a rare dis­ease that can cause a tox­ic build-up of an amino acid, met the pri­ma­ry end­point in a Phase III tri­al.

The biotech said Wednes­day that the drug, sepi­apterin, re­duced blood lev­els of amino acid pheny­lala­nine by an av­er­age of 63% in the pri­ma­ry analy­sis pop­u­la­tion. In a sub­set of pa­tients with clas­sic PKU — the most se­vere form of the dis­ease — the av­er­age re­duc­tion was 69%. The tri­al has en­rolled 156 adults and chil­dren; 98 pa­tients are in the pri­ma­ry analy­sis pop­u­la­tion.

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