Homology Medicines Announces Encouraging Initial Data from First Dose Level in the pheEDIT Trial Evaluating Gene Editing Candidate HMI-103 in Adults with Classical PKU

Participant 1 Achieved Clinically Meaningful Reduction in Plasma Phe of Up to 99% Change from Baseline and Below the U.S. PKU Treatment Guideline Threshold (<360 μmol/L)^*; At 31 Weeks Post-Dose, Phe Level 319 μmol/L With a 59% Reduction from Baseline, Even After Dietary Protein Supplementation

Participant 2 Plasma Phe Level Reduction of 49% Change from Baseline at 17 Weeks Post-Dose

HMI-103 Has Been Generally Well-Tolerated

Webcast Scheduled for Today, July 27, 2023 at 4:30 p.m. ET

Excerpt from the Press Release:

BEDFORD, Mass., July 27, 2023 (GLOBE NEWSWIRE) — Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today encouraging initial clinical data from the first dose cohort in the pheEDIT Phase 1, dose-escalation trial evaluating gene editing candidate HMI-103 in adults with classical phenylketonuria (PKU), the most prevalent and severe form of the disease. As of the data cut-off date of July 26, 2023, HMI-103 has been generally well-tolerated in all three participants. Participant 1 experienced a reduction in plasma phenylalanine (Phe) levels to below the U.S. American College of Medical Genetics and Genomics (ACMG) PKU treatment guideline threshold of <360 μmol/L*, and the majority of Phe levels have been below 360 μmol/L through 31 weeks post-dose, including after the initiation of dietary protein supplementation. Participant 2 has experienced a meaningful plasma Phe reduction of 49% at 17 weeks post-dose. Participant 3 was recently dosed.

“We are pleased to report today that the first dose level in the pheEDIT gene editing trial for PKU was generally well-tolerated by all three participants, with Participant 1 achieving Phe levels below the U.S. PKU treatment guideline threshold even after the addition of dietary protein, and Participant 2 experiencing meaningful reductions in plasma Phe,” said Albert Seymour, Ph.D., President and Chief Executive Officer of Homology Medicines. “We believe these initial clinical data from the first cohort suggest that our one-time gene editing approach has the potential to restore the normal biochemical pathway and support our recommendation to dose-escalate to identify an optimal dose.”

HMI-103 is a nuclease-free gene editing candidate for PKU designed to harness the body’s natural DNA repair process of homologous recombination to insert a functional gene and liver-specific promoter, and to increase PAH with episomal expression in all transduced liver cells.

HMI-103 was administered to participants via a one-time intravenous (I.V.) infusion at a dose of 6E13 vg/kg**. As of the data cut-off date of July 26, 2023, HMI-103 has been generally well-tolerated by all three participants with no serious adverse events (SAEs), and the majority of treatment-related adverse events (AEs) have been mild.

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