Homology Medicines Inc. (NASDAQ: FIXX) has commenced the Phase 1 study for HMI-203 for Hunter syndrome or mucopolysaccharidosis Type II treatment in adults. HMI-203 is an in-vivo gene therapy product.
juMPStart trial to evaluate safety and efficacy of HMI-203
The juMPStart study is an open-label, dose-escalation trial evaluating the efficacy and safety of a single HMI-203 intravenous IV injection. Hunter syndrome is a lysosomal storage condition resulting from changes in the iduronate 2-sulfatase (IDS) gene, which causes I2S enzyme activity to be missing or inadequate, resulting in a toxic lysosomal buildup of glycosaminoglycans (GAGs). Despite the availability of enzyme replacement therapy, there are currently no treatments targeting the condition’s peripheral organ and cognitive manifestations.
CEO Arthur Tzianabos said, “We are excited to start the Phase 1 trial for HMI-203, our investigational gene therapy for Hunter syndrome, as our team remains dedicated to improving outcomes for the patient community since our prior work developing ERT. With today’s milestone, we have also accomplished our goal of having three clinical programs by the end of 2021, with our gene therapy and gene editing clinical trials for PKU and the initiation of the juMPStart trial for Hunter syndrome. We are executing on this and our other programs, and we look forward to our continued collaboration with the MPS II community.”
Homology Medicine transitioning HMI-203 to clinical trials
Homology Chief Scientific Officer Albert Seymour said, “We are pleased to be transitioning HMI-203 into the clinic for patients, building on the positive preclinical data we presented at scientific conferences, including the upcoming ASHG meeting. These data showed long-term expression of I2S in multiple organs, sustained secretion in the serum, reduced GAGs in all tissues tested as well as the cerebrospinal fluid, and phenotypic correction in the Hunter disease murine model following a one-time administration. We appreciated the insights from the Hunter syndrome community as we designed our clinical program.”