Homology Medicines Initiates Phase 1/2 Study for HMI-102 Gene Therapy Candidate for Adults with PKU

Homology Medicines (NASDAQ:FIXX) has announced it has started enrolling of its Phase 1/2 pheNIX trial for HMI-102, a gene therapy candidate to treat adult patients with phenylketonuria (PKU).

As quoted in the press release:

The pheNIX study is designed to evaluate the safety and efficacy of the investigational gene therapy in a randomized, concurrently-controlled, dose-escalation study. PKU is an inborn error of metabolism caused by a mutation in the PAH gene that results in a potentially toxic buildup of phenylalanine (Phe), an essential amino acid derived primarily from dietary protein.

“Our early work with the clinical sites has enabled us to move expeditiously from receiving IND clearance to enrolling patients,” said Arthur Tzianabos, Ph.D., President and Chief Executive Officer of Homology Medicines. “We now have both external and internal operational GMP capabilities utilizing our proprietary process development and commercial manufacturing platform that can supply HMI-102 for the pheNIX trial all the way through to commercial scale. Our early investment in manufacturing is a key strategic advantage for Homology that has allowed us to progress this program rapidly to the clinic.”

The pheNIX trial 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. 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. Homology expects to report initial clinical data from the pheNIX trial by the end of 2019. Additional information about the pheNIX trial can be found at www.clinicaltrials.gov.

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