Shares in US genetic medicines company Homology Medicines (Nasdaq: FIXX) were set to open 37% lower on Monday following the release of bad news since Friday’s close.
The US Food and Drug Administration (FDA) has notified the company that its pheNIX gene therapy trial of HMI-102 in adults with phenylketonuria (PKU) has been placed on clinical hold due to the need to modify risk mitigation measures in the study in response to observations of elevated liver function tests.
PKU is an inherited disorder that increases the levels of phenylalanine in the blood. Currently, there are no treatment options that target the underlying genetic cause of the disease. According to the National PKU Alliance, PKU affects nearly 16,500 people in the USA with approximately 350 newborns diagnosed each year. The worldwide prevalence is estimated to be 50,000 people.
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