Homology Therapeutics Inc. (NASDAQ: FIXX) stock gained by 1.23% at last close while the FIXX stock price surges by 3.5% in the after-hours trading session. Homology Therapeutics, Inc. is a clinical-stage genetic medicines business focused on improving the lives of people with rare diseases by addressing the disease’s underlying cause.
FIXX stock’ Important Development
The pheEDIT Phase 1 clinical study for HMI-103 was announced today by Homology Medicines. It’s a one-time, in vivo product option for phenylketonuria (PKU) that uses a gene-editing method, according to the FDA’s clearance of an Investigational New Drug Application (IND) (FDA). From Homology’s dual gene therapy and gene editing technology platform, HMI-103 will be the world’s first gene-editing option for PKU to begin clinical trials. With the advent of pheEDIT, however, Homology gets closer to its aim of providing treatments for both adults and children with PKU.
Up to nine individuals between the ages of 18 and 55 who have been identified with PKU owing to PAH deficiency will be included in the HMI-103 pheEDIT study. Homology intends to recruit younger individuals in clinical studies after favorable safety and effectiveness outcomes have been demonstrated in the adult population. The goal of the Phase 1 dose-escalation experiment is to examine three doses of HMI-103 in order to establish the best dose(s) for a future trial. The experiment will track changes in serum phenylalanine (Phe) in addition to safety objectives.
Following the required Institutional Biosafety Committee and Institutional Review Board clearances at the clinical sites, Homology plans to administer the first patient in the pheEDIT clinical study. Before the administration of HMI-103, the study will involve an 82-day screening/run-in phase.
Homology also provided an update on the results of its continuing Phase 2 pheNIX clinical study, which is testing HMI-102 gene therapy in adults with PKU. Both dosages in the experiment were generally well tolerated and showed indications of biological action as of September 30, 2021, including clinically significant reductions in Phe levels, elevations in Tyr, and decreases in the Phe-to-Tyr ratio.
A total of 13 additional clinical trial locations have recently been introduced to pheNIX, with more sites likely to be added soon. Despite the increasing interest, enrolment is slower than expected, owing to the revival of COVID-19, and the Company plans to provide a more thorough pheNIX report in mid-2022 when it expects to have a bigger dataset.