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.