Homology Medicines Announces FDA Fast Track Designation for HMI-102 Gene Therapy Development Candidate for Adults with PKU
- Company Provides Details of pheNIX, the First Gene Therapy Trial for PKU, and Remains on Track to Report Initial Clinical Results from the Phase 1/2 Study in 2019 -
“We believe FDA’s decision to grant Fast Track designation is a positive step forward for the development of HMI-102, which is designed to treat the underlying cause of this disease and allow freedom from a restrictive diet, the current standard of care,” said
FDA’s Fast Track process is intended to facilitate the development process and expedite review of drugs that are designed to treat serious conditions and fill an unmet medical need to get them to patients earlier. A drug with Fast Track designation can be eligible for more frequent meetings with and written communications from
pheNIX, the first gene therapy clinical trial in PKU, is an open-label, randomized, concurrently controlled, dose-escalation study designed to evaluate the safety and efficacy of HMI-102 in adults, aged 18-55 with classic PKU. HMI-102 is designed to deliver a functional copy of the PAH gene to liver cells. In addition to safety measures, the trial 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.
About Phenylketonuria (PKU)
PKU is a rare, inherited inborn error of metabolism caused by a mutation 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, including without limitation HMI-102; advancing our novel gene therapy and gene editing technology platform and pipeline; our goal of delivering potential cures to patients with PKU and other rare genetic diseases; 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: the fact that 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 marketable products; the early stage of our development efforts; our failure or the failure of our collaborators to successfully develop and commercialize drug candidates; 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; the inability to obtain orphan drug exclusivity; failure to obtain U.S. or international marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property; the price of our common stock may be volatile; significant costs as a result of operating as a public company; and any securities class action litigation. These and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended
1 “Fast Track.”
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Source: Homology Medicines, Inc.