Three years in­to a PhI­II pro­gram for a failed Duchenne MD drug, Cataba­sis hauls down the flag and ad­mits de­feat

Three years ago, Cataba­sis CEO Jill Milne and the crew in­sist­ed they had found good rea­son for great cheer once they plumbed the da­ta from their failed study for the Duchenne MD drug edasa­lonex­ent. Plung­ing in­to the ex­tend­ed open-la­bel da­ta, they said, you could find sol­id ev­i­dence of ef­fi­ca­cy. And that jus­ti­fied a try in Phase III.

But they were wrong.

Mon­day, af­ter the bell, the lit­tle biotech ac­knowl­edged that their piv­otal at­tempt fol­low­ing the mid-stage flop was an­oth­er fail­ure. The pri­ma­ry, change in base­line on the North Star Am­bu­la­to­ry As­sess­ment, and the sec­ondary on timed func­tion tests both came up short of sta­tis­ti­cal sig­nif­i­cance.

Endpoints News

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.