Business
Homology Medicines Reports Second Quarter 2020 Financial Results and Provides Business Update
Three Cohorts Enrolled in Phase 1/2 pheNIX Gene Therapy Trial for PKU Progressed Into Later Stages of IND-Enabling Studies for MLD Gene Therapy and PKU Gene
About this update from Q32 Bio Inc.
[{"type":"text","content":"Three Cohorts Enrolled in Phase 1/2 pheNIX Gene Therapy Trial for PKU\n Progressed Into Later Stages of IND-Enabling Studies for MLD Gene Therapy and PKU Gene Editing Programs and Published Key Data Successfully Executed Multiple Internal 2,000-Liter Bioreactor Runs Using Commercial Process and Platform BEDFORD, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today financial results for the second quarter ended June 30, 2020, highlighted recent accomplishments and provided a business update. “The pheNIX trial for adult patients with PKU is ongoing, and we continue to be encouraged by both the clinical data suggesting enzymatic activity and the safety profile of HMI-102. We have dosed patients in three cohorts in the dose-escalation phase of our trial, and this has taken more time than we anticipated due to the pandemic. We continue to assess the data, which includes a recently dosed patient, before we select the dose for our expansion phase. As a result, we are extending our timeline and still expect to provide an update once we have selected a dose for the expansion phase, which has the potential to be a registrational trial.” “In the second quarter, we achieved a milestone in our nuclease-free gene editing program with the peer-reviewed publication of the quantitative molecular methods that we used to show the precision and efficiency of our technology in preclinical studies of PKU. We also announced longer-term data demonstrating that our MLD gene therapy candidate produced durable human ARSA protein at normal levels in advanced IND-enabling studies. Additionally, we demonstrated that our commercial manufacturing process in a 2,000-liter bioreactor resulted in high quality and productivity, and that our AAVHSC vectors packaged more efficiently than non-Clade F AAV5,” concluded Dr. Tzianabos. Second Quarter 2020 and Recent Accomplishments Announced today an update to the Phase 1/2 pheNIX gene therapy clinical trial with HMI-102 for adults with phenylketonuria (PKU): Since the Company’s initial trial data reported in December 2019: Homology continued to observe encouraging clinical data that suggest PAH enzymatic activity.High-dose Cohort 3 patients were enrolled and dosed.No treatment-related serious adverse events were reported. Homology is extending the...