Homology’s PKU gene therapy gets FDA fast track designation

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Homology Medicines’ (NASDAQ:FIXX) received FDA fast track designation for HMI-102, a gene therapy for the treatment of adults with phenylketonuria (PKU), a rare genetic disease affecting metabolism.

Individuals with PKU lack the phenylalanine hydroxylase (PAH) enzyme that metabolizes phenylalanine. If left untreated, phenylalanine levels rise significantly, putting patients at risk of severe neurological impairment. HMI-102 is a one-time gene therapy that delivers a functional copy of the PAH gene to liver cells, enabling production of the enzyme.

“We believe FDA’s decision to grant fast track designation is a positive step forward for the development of HMI-102, which is designed to treat the underlying cause of this disease and allow freedom from a restrictive diet, the current standard of care,” Arthur Tzianabos, Homology’s president and CEO, said in a statement.

Homology is preparing to begin a Phase 1/2 trial, called pheNIX, in adult patients with PKU, and expects to report initial clinical data in 2019.

Jennifer Poland