- Data Package Supported Initiation of Ongoing pheNIX Clinical Trial for Adults with PKU -
BEDFORD, Mass., March 16, 2020 – Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today the peer-reviewed publication of preclinical data that supports Homology’s HMI-102 investigational gene therapy program for the treatment of adults with phenylketonuria (PKU). HMI-102 is currently being evaluated in the pheNIX Phase 1/2 clinical trial, and the Company plans to provide an update on the trial when selecting the dose for the expansion part, which is currently anticipated in mid-2020.
The published data shows that a single administration of HMI-102 (AAVHSC15-PAH) produced a sustained reduction in phenylalanine (Phe), the key biomarker in the diagnosis and management of PKU, for the lifespan of the established murine model for PKU. The data also demonstrated a concomitant increase in tyrosine (Tyr), a metabolite of Phe and precursor to neurotransmitters, indicating enzymatic activity. Additionally, brain levels of Phe, 5-HIAA (downstream serotonin metabolite) and coat color were normalized, further indicating restoration of the Phe metabolic pathway.
“We developed a robust preclinical data package for our investigational HMI-102 gene therapy, which supported the initiation of our ongoing Phase 1/2 pheNIX clinical trial for adults with PKU,” stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology Medicines. “These published data demonstrated that a single dose of HMI-102 was able to restore the normal biochemical pathway in the established PKU model on normal protein diet. Initial data from the pheNIX trial suggests that the increased PAH enzymatic activity after administration of HMI-102 seen in the preclinical model was also observed in the clinical study.”
Key data in the publication include:
• A single IV administration of HMI-102 reduced serum Phe concentrations to normal levels within one week in the PKU murine model, and mean levels remained below 120 µM (the normal range) over the course of the 48-week study.
• A corresponding increase in Tyr concentration was also observed, indicating restoration of the Phe/Tyr metabolic pathway.
• HMI-102 administration was also associated with an increase in 5-HIAA and a decrease in Phe in the brain to normal levels.
The publication, “Sustained Correction of a Murine Model of Phenylketonuria Following a Single Intravenous Administration of AAVHSC15-PAH,” was peer-reviewed and published in the journal Molecular Therapy: Methods & Clinical Development. For more information, please visit www.homologymedicines.com/publications.
About the Phase 1/2 pheNIX Clinical Trial in Phenylketonuria (PKU)
The pheNIX trial is the first gene therapy clinical trial ever conducted for people with PKU. pheNIX is designed to evaluate the safety and efficacy of a single intravenous administration of HMI-102 in adult patients with PKU aged 18-55. The study design allows for expansion of the number of patients in any dose cohort pending review by the Data Monitoring Committee and the Homology Internal Data Review Team. A decision to expand would trigger the addition of the randomized, concurrently controlled Part B of the trial, which has the potential to be converted to a registrational trial. The primary efficacy endpoint of the expansion part is incidence of sustained plasma Phe concentration ≤360 μmol/L as demonstrated by two measurements ≤360 μmol/L between 16 and 24 weeks.
About Homology Medicines, Inc.
Homology Medicines, Inc. is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology’s proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities and intellectual property position it as a leader in the development of genetic medicines. For more information, please visit www.homologymedicines.com.
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