Homology Medicines Presents Data Showing Single Dose HMI-102 Resulted in Long-Term Correction of PKU, Including Reduction of Phe and Increased Brain Neurotransmitters in Murine Model
- Phase 1/2 pheNIX Study of Gene Therapy Candidate HMI-102 is Underway and Initial Clinical Data is Anticipated by Year End -
“These findings are part of a larger body of preclinical data demonstrating the therapeutic potential and efficacy of HMI-102, which provided a strong foundation for the recent initiation of our Phase 1/2 pheNIX study, the first gene therapy candidate for PKU to enter clinical trials,” said
Highlights of the oral presentation at ESHG in
- Effective, sustained phenotype correction in PAHenu2 murine model on a normal protein diet;
- Decreased serum Phe, as well as normalization of serum tyrosine levels and a change in coat color, both indicating a restoration of Phe metabolism; and
- Reduced brain Phe and the neurotransmitter metabolite, 5-HIAA to normal levels.
Homology has received Fast Track Designation for HMI-102 from the
About the pheNIX Phase 1/2 Clinical Study in PKU
The pheNIX study is designed to evaluate the safety and efficacy of HMI-102 gene therapy in a randomized, concurrently-controlled, dose-escalation study. The pheNIX study is expected to enroll up to 21 patients, ages 18-55 years old, who have been diagnosed with classic PKU and will receive a single dose of HMI-102, which uses Homology’s AAVHSC15 vector. In addition to safety measures, the study will also evaluate reduction in serum Phe levels. The study design allows for expansion of the number of patients in any dose cohort as long as the dose selected has been deemed safe and effective by the Data Monitoring Committee and the Homology Review Team. A decision to expand a dose cohort would trigger the addition of a concurrent, randomized control arm consisting of classic PKU patients that will be monitored for Phe levels before they crossover into the treatment arm. Homology expects to report initial clinical data from the pheNIX study by the end of 2019. Additional information about the pheNIX study can be found at www.clinicaltrials.gov.
About Phenylketonuria (PKU)
PKU is a rare, inherited inborn error of metabolism caused by mutations in the PAH gene. The current standard of care is a highly restrictive diet, but it is not always effective, and there are currently no treatments available that address the genetic defect in PKU. If left untreated, PKU can result in progressive and severe neurological impairment. PKU affects approximately 16,500 people in the U.S., and an estimated 350 newborns are diagnosed each year.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding our expectations surrounding the design, initiation, enrollment and timing of the release of data for the pheNIX clinical trial in PKU; the potential, safety, efficacy, and regulatory and clinical progress of our product candidates; beliefs about preclinical data; and our position as a leader in the development of genetic medicines. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop or commercialize marketable products; the early stage of our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the capabilities and potential expansion of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; effects of significant competition; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; and risks relating to intellectual property. These and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended
|SVP, Corporate Communications
& Patient Advocacy
Source: Homology Medicines, Inc.