Did Sarep­ta re­al­ly score a suc­cess in the lat­est cut of their next-gen Duchenne MD drug? As usu­al, that's a lit­tle com­pli­cat­ed

Sarep­ta boost­ers have been keep­ing their fin­gers crossed that the read­out on their new-and-im­proved 30 mg/kg ap­proach to ex­on-skip­ping for Duchenne mus­cu­lar dy­s­tro­phy pa­tients will do much bet­ter than the low­er dose they start­ed out with. And this morn­ing they were re­ward­ed with a ba­sic out­line of suc­cess for the part A seg­ment of their MO­MEN­TUM study — though you can ex­pect plen­ty of ques­tions about its ex­plo­ration with a tiny group of 4 pa­tients, a con­tin­ued re­liance on a ques­tion­able bio­mark­er with no ac­tu­al proof of ef­fi­ca­cy, ev­i­dence of se­ri­ous tox­i­c­i­ty with sev­er­al ad­verse events and a cu­ri­ous case of pre­dic­tive mod­el­ing to achieve the kind of suc­cess some an­a­lysts have been look­ing for.

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.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER