Homology Medicines Inc. (FIXX) Announces First Presentation of Data with HMI-203 In Vivo Gene Therapy Development Candidate for Hunter Syndrome

February 8, 2021 2:51 PM EST

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Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic medicines company, announced today the first scientific presentation of data from IND-enabling studies with its HMI-203 gene therapy development candidate for Hunter syndrome (MPS II). The results from Homology’s in vivo approach demonstrated the potential of HMI-203 to address peripheral manifestations in the murine disease model and showed that HMI-203 crossed the blood-brain-barrier following a single I.V. administration. These data will be presented during a poster presentation at the 17th Annual WORLDSymposium™ Meeting, where Homology will also present long-term data from its HMI-202 in vivo gene therapy program for metachromatic leukodystrophy (MLD).

“By leveraging our family of AAVHSC vectors to target both peripheral organs and the central and peripheral nervous systems in preclinical models of Hunter syndrome and MLD, debilitating diseases that have high unmet medical need, we also demonstrated the continued expansion of our CNS platform,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “This is our first presentation of preclinical data from our Hunter syndrome gene therapy program, and these IND-enabling studies showed that a single I.V. administration produced high levels of enzymatic expression across disease-relevant tissues and achieved phenotypic correction of skeletal deformities. These results support our plans to move this program forward into a Phase 1/2 clinical trial this year.”

In the poster titled, “HMI-203: Investigational Gene Therapy for Mucopolysaccharidosis Type II (MPS II), or Hunter Syndrome,” a single I.V. administration of HMI-203 in the adult murine model:

  • Led to robust biodistribution and sustained human I2S (hI2S) enzyme expression, which resulted in:
    • Significant reductions in key Hunter syndrome biomarkers of heparin sulfate glycosaminoglycans (GAGs) and lysosomal-associated membrane protein 1 (LAMP-1) in the brain, liver, heart, spleen, lung and kidneys compared with vehicle.
    • Significant reductions in heparan sulfate GAGs in the cerebrospinal fluid (CSF) compared with vehicle.
  • Ameliorated paw deformities, as shown by significant changes in measurements of ankle depth, paw width, paw depth and ankle width compared with vehicle.
  • Led to uptake of hI2S from the serum of the HMI-203-treated model in human cell lines, demonstrating potential for cell cross-correction.

In an additional poster titled, “HMI-202: Gene Therapy Development Candidate for Metachromatic Leukodystrophy (MLD),” a single I.V. administration of HMI-202:

  • Crossed the blood-brain-barrier and blood-nerve-barrier in the murine MLD disease model and in non-human primates (NHPs), with human ARSA (hARSA) detected in neuronal and glial cells.
  • Showed durable hARSA activity in the central nervous system of the disease model, with distribution levels resembling those of Arsa in normal age-matched controls.
  • Demonstrated significant changes in key MLD biomarkers of LAMP-1, glial fibrillary acidic protein (GFAP), MAL transcript and neuronal sulfatides in the disease model compared with vehicle, similar to age-matched wild type controls.

Homology’s e-poster presentations will take place on February 11, 2021 at 2:30 p.m. ET. For more information, visit www.homologymedicines.com/publications.

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