Lack of ear­ly ef­fi­ca­cy for Trans­late Bio's mR­NA-based cys­tic fi­bro­sis drug rais­es some big ques­tions

Look­ing to bring mR­NA ther­a­peu­tics to cys­tic fi­bro­sis, Trans­late Bio re­vealed its sec­ond in­ter­im analy­sis in a Phase I/II study show­ing their ex­per­i­men­tal drug to be large­ly safe and tol­er­a­ble. But ques­tions re­main whether or not the pro­gram, dubbed MRT5005, will prove ef­fi­ca­cious with a top an­a­lyst all but de­clar­ing an end to the pro­gram.

Trans­late said that da­ta from 12 pa­tients across three co­horts demon­strate that mul­ti­ple dos­es of their in­haled cys­tic fi­bro­sis treat­ment sug­gest the ther­a­py is safe. The mR­NA-cen­tric pro­gram al­so ap­peared safe in an ad­di­tion­al co­hort with 4 pa­tients in a sin­gle-dose co­hort.

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