PTC whiffs on an­oth­er Duchenne study, plans to go to FDA again any­way

Once again PTC Ther­a­peu­tics’ Duchenne mus­cu­lar dy­s­tro­phy drug has failed a clin­i­cal tri­al. And once again Stu­art Peltz and his crew are try­ing to use the da­ta to prove why the FDA should ap­prove it.

PTC an­nounced late Thurs­day that in its lat­est Phase II study, their flag­ship drug Translar­na failed to sig­nif­i­cant­ly im­prove dy­s­trophin ex­pres­sion across 20 pa­tients when mea­sured by ei­ther of the as­says used for the tri­al. The tri­al, known as Study 045, joins larg­er Phase IIb and Phase III Duchenne stud­ies and one Phase III cys­tic fi­bro­sis tri­al on the list of piv­otal fail­ures for a drug that first en­tered the clin­ic near­ly two decades ago and has thrice been bat­ted down by the FDA.

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