FIXX - Homology Medicines slumps on gene therapy data in inherited metabolic disorder

Homology Medicines plunges ([[FIXX]] -21.4%) on high volume as investors appear underwhelmed with results from dose-escalation portion of the Phase 1/2 study evaluating its gene therapy (HMI-102) for phenylketonuria ((PKU)), a rare inherited disorder characterized by an inability to break down phenylalanine ((Phe)), an amino acid that is commonly found in many foods, which accumulates in the body to toxic levels.Alanine aminotransferases ((ALTs)) elevations were observed in cohorts 2 & 3 (mid- and high-dose), and were managed with increased steroids when necessary. High levels of ALT may indicate liver damage.Through 52 weeks, patients in Cohort 1 (low dose) continued to show no meaningful reductions in Phe.Through 48 weeks, one patient in Cohort 2 had Phe levels of <360 ?mol/L and/or <600 ?mol/L at multiple timepoints and had reached a minimum Phe level of 42 ?mol/L, compared with a baseline level of 1,010 ?mol/L.Through 13 weeks, one patient in Cohort 3 had a

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Homology Medicines slumps on gene therapy data in inherited metabolic disorder