Analysts’ fears have been confirmed as a US FDA demand for clinical outcomes data has forced Applied Therapeutics to delay a planned filing for its lead product candidate, AT-007, in the rare disease galactosemia based on biomarker data alone.

The diuretic bumetanide had shown potential to improve the core symptoms of children and adolescents with autism in small studies but has failed to demonstrate any sign of effectiveness in two late-stage trials.

Taysha gains control of GAN candidate and program; BI licenses COMT inhibitors from Lieber Institute; plus three new gene therapy-driven tie-ups between biopharma and academia.