Homology Medicines provides updates on pheEDIT, juMPStart trials

Homology Medicines announced enrollment and site status updates from the pheEDIT Phase 1 gene editing trial with HMI-103 for phenylketonuria, or PKU, and the juMPStart Phase 1 gene therapy trial with HMI-203 for Hunter syndrome, or MPS II. The first participant was recently dosed in the pheEDIT trial, with additional participants in screening. Homology expects to provide initial data from the trial mid-year 2023. There are nine active clinical trial sites with more expected to be initiated throughout 2023. The juMPStart trial has five clinical sites in the U.S. and Canada with more expected to be initiated, and initial data is expected in the second half of 2023. Homology shared new preclinical data today supporting the immunosuppression regimen incorporated in both the pheEDIT and juMPStart trials. In non-human primates, or NHPs, use of a prophylactic T-cell inhibitor combined with steroids reduced the neutralizing antibody, or nAb, response to the AAVHSC vector and increased mRNA expression, compared to NHPs not receiving the regimen and to those receiving each agent alone. Homology plans to present these data at an upcoming scientific conference. Building on the unique mechanism of action of gene editing candidate HMI-103, Homology shared preclinical potency data. HMI-103 is designed to use homologous recombination to integrate the PAH gene and a liver-specific promoter into the genome and to maximize PAH expression in all transduced liver cells. In the preclinical PKU model, the murine surrogate of HMI-103 was ten times more potent than non-integrating gene therapy vector HMI-102. In 2023, Homology plans to progress its pipeline of genetic medicines, including conducting IND-enabling studies of HMI-104, a one-time GTx-mAb development candidate for the treatment of paroxysmal nocturnal hemoglobinuria, or PNH. The company is also focused on efforts to partner the optimized HMI-204 gene therapy candidate for metachromatic leukodystrophy, or MLD, with near-term plans to present preclinical data from the program for the first time. The company will continue to work with Oxford Biomedica Solutions, the AAV manufacturing and innovation business Homology established with Oxford Biomedica and that supplies Homology’s programs. Oxford Biomedica Solutions recently announced that its platform has produced high-quality titers of E15 vg/L and achieved over 90% fully intact vector.