Jonathan Rothberg, AI Therapeutics founder (Stuart Ramson/AP Images for Detect, Inc.)

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).

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