Pri­vate start­up claims a PhI­Ia win in sub­set of ALS pa­tients, but ques­tions re­main

A pri­vate Con­necti­cut biotech is claim­ing a win from a small Phase IIa tri­al in ALS pa­tients with a spe­cif­ic ge­net­ic mu­ta­tion.

AI Ther­a­peu­tics said Wednes­day a study of its lead com­pound, known as AIT-101, met its pri­ma­ry safe­ty and tol­er­a­bil­i­ty goals while al­so low­er­ing a dis­ease-re­lat­ed bio­mark­er af­ter 12 weeks. Re­searchers saw a 73% re­duc­tion in the bio­mark­er, which is re­ferred to as a tox­ic pro­tein ag­gre­gate, or poly(GP).

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