Following the release of preliminary results from a clinical trial, Homology Medicines Inc. (NASD: FIXX) saw a 3.08% increase to $1.34 in after-hours trading on Wednesday.
What information has FIXX offered?
pheEDIT Phase 1 gene editing experiment with HMI-103 for phenylketonuria (PKU) and juMPStart Phase 1 gene therapy trial with HMI-203 for Hunter syndrome enrollment and site status updates were released by Homology Medicines (FIXX) (MPS II).
Homology Medicines just administered the first dosage to the trial’s first participant, pheEDIT, and began screening other patients. Midway through 2023, FIXX anticipates releasing the trial’s initial data. Nine clinical trial locations are now operational, and more are anticipated to be up during 2023. Initial findings from the juMPStart trial are anticipated in the second half of 2023. There are now five study sites in the United States and Canada with plans to open additional ones.
While HMI-103 and HMI-203 offer distinct one-time treatments to PKU and MPS II, respectively, designed to address important unmet medical needs, FIXX’s focus is and will remain concentrated on clinical trial implementation. FIXX will advance these products based on the interest of the medical and patient groups. It’s significant to note that FIXX intends to publish preliminary clinical findings from both studies in 2023, including the first results from its human nuclease-free gene editing method.
FIXX provided preclinical potency data, building on the distinctive method of action of gene editing candidate HMI-103. The PAH gene and a liver-specific promoter will be integrated into the genome of HMI-103 via homologous recombination in order to enhance PAH production in all transduced liver cells. The murine surrogate of HMI-103 was ten times more effective than the non-integrating gene therapy vector HMI-102 in the preclinical PKU model.
FIXX believes that HMI-103, by enhancing PAH through genome integration and episomal expression, has the potential to cure PKU in both children and adults. This is what you want from one-time genetic therapies, therefore you optimized HMI-103 by including vector design, a potent liver-specific promoter, and improved packaging.
What did FIXX find in the data?
New preclinical information was released by Homology Medicines (FIXX) in support of the immunosuppressive regimen used in the pheEDIT and juMPStart studies. In comparison to NHPs not receiving the regimen and to those receiving each treatment alone, the administration of a prophylactic T-cell inhibitor coupled with steroids decreased the neutralizing antibody (nAb) response to the AAVHSC vector and boosted mRNA expression. These findings will be presented by FIXX at a forthcoming scientific meeting.