FIXX - FDA lifts clinical hold on Homology Medicines' phenylketonuria trial

Homology Medicines (NASDAQ:FIXX) on Monday said that the U.S. Food and Drug Administration (FDA) had lifted a clinical hold on the company's pheNIX gene therapy clinical trial with HMI-102 for adults with phenylketonuria (PKU). FIXX stock +2.8% to $2.21 in premarket trading. PKU is a rare inherited disorder which causes an amino acid called phenylalanine to build up in the body. If untreated, it can lead to brain damage, intellectual disabilities or seizures. The FDA had imposed the clinical hold in February, citing the need to modify the trial's risk mitigation measures in response to elevated liver function tests in patients in the study. FIXX on Monday said that it had responded to the FDA's concerns by making changes to the study protocol, which include a new steroid-sparing immunosuppression regimen that incorporates a T-cell inhibitor and a shorter course of steroids. FIXX said it plans to share an update on the pheNIX

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FDA lifts clinical hold on Homology Medicines' phenylketonuria trial