Preliminary data from a Phase 1/2 clinical trial, pheNIX, evaluating Homology Medicines’ (NASDAQ:FIXX) gene therapy HMI-102 in patients with phenylketonuria (PKU) showed encouraging action.
The first patient dosed experienced a 35% reduction from baseline in phenylalanine at week 1 and 48% at week 4. Tyrosine levels increased 72% and 85%, respectively, at each time point.
On the safety front, no treatment-emergent adverse events were observed.
Management will host a conference call today at 4:30 pm ET to discuss the data.
Shares up 1% after hours.
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