Homology Medicines Pauses Enrollment For Rare Inherited Disorder Study

  • Homology Medicines Inc FIXX paused enrollment in the pheNIX gene therapy of HMI-102 for adults with phenylketonuria (PKU) to free up cash.
  • PKU is a rare inherited disorder that causes an amino acid called phenylalanine to build up in the body that is toxic to the brain and nervous system.
  • In June, the FDA lifted the clinical hold previously placed on the pheNIX gene therapy clinical.
  • The company began the phase 1/2 study of HMI-102 in 2019. The FDA held back the study over elevated liver function tests by a few months this year.
  • Now Homology has voluntarily paused enrollment for financial reasons.
  • Also Read: Homology Medicines Announces Preclinical Gene Therapy Candidate For Genetic Storage Disorder. 
  • “Prioritization offers potential to generate data, which includes a new immunosuppression regimen with a shorter course of steroids and a T-cell inhibitor as part of the pheEDIT protocol, sooner than would be possible to resume enrollment at pheNIX trial sites,” the company said.
  • The company expects its cash reserves, which totaled $225.5 million at the end of June, to last into Q4 of 2024.
  • The phase 1 pheEDIT study in adults with PKU kicked off in June to evaluate HMI-103.
  • Price Action: FIXX shares traded 0.69% lower at $2.87 on the last check Tuesday.
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