Freeline reports initial data from first cohort of Phase 1/2 GALILEO-1 trial

Freeline Therapeutics reported positive initial safety, tolerability and enzyme activity data from the ongoing Phase 1/2 GALILEO-1 trial evaluating FLT201, its adeno-associated virus gene therapy candidate, in Gaucher disease. In addition to demonstrating a favorable safety and tolerability profile, data from the first two patients in GALILEO-1 show that a single infusion of FLT201 led to several hundred-fold increases in GCase activity in plasma and normalization of GCase activity in leukocytes. Positive Initial Clinical Data for FLT201: The data reported today include assessments of safety, tolerability and GCase activity from the first two patients in GALILEO-1, which is a first-in-human, international, multicenter Phase 1/2 dose-finding study in people with Gaucher disease Type 1. Both patients were treated with a dose of 4.5×1011 vg/kg and have successfully come off their prior therapies. As of the September 27 data cutoff, the data demonstrated: Favorable safety and tolerability, with no infusion reactions and no serious adverse events as of 13 weeks post-dosing for patient 1 and six weeks post-dosing for patient 2. All treatment-related adverse events were Grade 1 and resolved without intervention. No elevations in liver transaminase levels during the same time periods. Alanine-transaminase and aspartate-transaminase levels remained in the normal range in both patients. Robust increases in plasma GCase levels. Patient 1 showed a nearly 700-fold increase over baseline to more than 70 mumol/L/h as of 12 weeks post-dosing. Patient 2 showed a similarly robust response, with a greater than 300-fold increase over baseline to approximately 30 mumol/L/h as of four weeks post-dosing. Normalization of leukocyte GCase activity, demonstrating cellular uptake of GCase from the plasma. Leukocyte GCase activity reached normal levels in patient 1 within four weeks of dosing and remained normal as of the last measurement. Similarly, leukocyte GCase activity in patient 2 reached normal levels within four weeks of dosing. Both patients had normal hemoglobin levels at baseline and have remained in the normal range at each weekly assessment, including those taken after coming off enzyme replacement therapy or substrate replacement therapy.