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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