PTC Ther­a­peu­tics re­ports sec­ond tri­al flop for late-stage pro­gram

PTC Ther­a­peu­tics an­nounced an­oth­er flop for its drug va­tiquinone a lit­tle over a month af­ter it failed to hit a pri­ma­ry end­point in a sep­a­rate Phase III tri­al.

The drug failed to show a re­duc­tion in ob­serv­able mo­tor seizures in the MIT-E tri­al test­ing out the drug for the treat­ment of mi­to­chon­dr­i­al dis­ease as­so­ci­at­ed seizures (MDAS) in chil­dren against a place­bo.

The Phase II/III study en­rolled 68 chil­dren with the dis­ease and though it didn’t hit its main goal, the study showed “ev­i­dence of treat­ment ef­fect” in the over­all study pop­u­la­tion in re­duc­ing the fre­quen­cy of seizures and in a sub­group of chil­dren with Leigh syn­drome. In the sub­group, there was “ben­e­fit” in the sec­ondary end­point, the oc­cur­rence of sta­tus epilep­ti­cus and dis­ease-re­lat­ed hos­pi­tal­iza­tions.

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