Lowest dose cohort complete and study positioned for dose escalation
Company to present FLT190 data by year-end
LONDON, June 16, 2021 (GLOBE NEWSWIRE) — Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the “Company” or “Freeline”), a clinical-stage biotechnology company developing transformative AAV-mediated gene therapies for patients suffering from inherited systemic debilitating diseases, today announced dosing of the second patient in the ongoing Phase 1/2 MARVEL-1 clinical trial of FLT190, its liver-directed AAV gene therapy candidate for Fabry disease.
“Dosing the second patient in the MARVEL-1 study is an important milestone for Freeline and evidence of progress in our Fabry program,” said Theresa Heggie, Chief Executive Officer of Freeline. “Easing of COVID-19 restrictions, together with geographic expansion of study sites, should enable continued enrollment as we work to make FLT190 available to the Fabry disease patient community. With dosing complete in our first cohort, we look forward to advancing through the next cohorts in our dose-finding study. We plan to present data later this year to demonstrate the potential for FLT190 to be a transformative treatment for patients with Fabry disease.”
MARVEL-1 is a multicenter, international, Phase 1/2 dose-finding trial in adult males with classic Fabry disease being conducted in two parts: in previously treated patients and in previously untreated patients. The study is evaluating the safety and efficacy of FLT190 across four dose levels in approximately 10 patients and monitoring patients for nine months after dosing. Patients will be eligible to participate in a long-term follow-up study for further evaluation of the safety and efficacy of FLT190. In addition to safety, efficacy endpoints include levels of αGLA production, clearance of globotriaosylceramide (“Gb3”) and globotriaosylsphingosine (“LysoGb3”) from the plasma, changes in renal and skin biopsies, renal and cardiac function, αGLA immune response, and quality of life.
