FDA places gene therapy trial on hold
pharmafile | February 23, 2022 | News story | Research and Development |
Homology Medicines has shared that the FDA has placed its pheNIX gene therapy trial of HMI-102 in adults with phenylketonuria on clinical hold. This is due the observations of elevated liver function tests, and the need to modify the study’s risk mitigation measures to prevent any incidents which may arise.
“This hold on our PKU gene therapy trial is based on clinical observations in the pheNIX study and does not relate to CMC/manufacturing capabilities or Homology’s other clinical programs,” emphasised Arthur Tzianabos, president and CEO of Homology Medicines. “We plan to provide next steps once we have more information following our FDA interactions.”
Homology has two ongoing clinical programs, the pheEDIT gene-editing trial of HMI-103 for PKU and the juMPStart gene therapy trial of HMI-203 for Hunter syndrome, with program updates expected by the end of 2022.
The Phase I/II open-label, randomised, dose escalation study, is designed to evaluate the safety and efficacy of HMI-102 in adult patients with PKU, who can take oral steroids for a certain period. The study is not open to patients who have had histories of HIV, hepatitis B or C, and alcoholism or drug abuse. Those who suffer an underlying liver disease, and those who are pregnant or plan to be pregnant during the study period, will also not be eligible to participate.
Phenylketonuria (PKU) is a rare genetic condition which causes increased levels of phenylalanine, a kind of amino acid, in the body. People with PKU cannot break down phenylalanine, which as a result builds up in their blood and brain, and can cause brain damage and damage to the nervous system. Symptoms for PKU include behavioural difficulties, including difficulties with temper and episodes of self-harm; eczema; jerking movements in the arms and legs; tremors; and epilepsy.
Homology has shared that it expects to receive a formal letter from the FDA detailing the directive in the next 30 days. Participants to the study will receive a single intravenous administration of the gene therapy, and will be observed for 52 weeks, and will be monitored for a following four years to ensure stability.
Ana Ovey
