Freeline Doses Second Patient of Phase 1/2 MARVEL-1 Clinical Trial for Fabry Disease
LONDON, June 16, 2021 – 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.
- Lowest dose cohort complete and study positioned for dose escalation
- Company to present FLT190 data by year-end
“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.
FLT190 is an investigational AAV gene therapy in development as a potential treatment for patients with Fabry disease that is currently being studied in a Phase 1/2 clinical trial. FLT190 consists of our next generation potent AAVS3 engineered capsid, containing an expression cassette with a codon-optimized human GLA cDNA under the control of the liver-specific promoter FRE1. The treatment is administered by intravenous infusion that lasts approximately one hour and does not require the patient to undergo stem cell harvest or conditioning with chemotherapy.
About Fabry Disease
Fabry disease is a genetic lysosomal storage disorder that leads to the deficiency of a key enzyme needed to break down a fatty substance called globotriaosylceramide (“Gb3”). Without the enzyme, this fatty substance builds up throughout the body, affecting tissues and organs. Fabry disease occurs in all ethnic groups and is estimated to affect one in every 40,000 people.
About Freeline Therapeutics
Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (“AAV”) vector-mediated systemic gene therapies. The Company is dedicated to improving patient lives through innovative, one-time treatments that provide functional cures for inherited systemic debilitating diseases. Freeline uses its proprietary, rationally-designed AAV vector, along with novel promoters and transgenes, to deliver a functional copy of a therapeutic gene into human liver cells, thereby expressing a persistent functional level of a missing protein into the patient’s bloodstream. The Company’s integrated gene therapy platform includes in-house capabilities in research, clinical development, manufacturing and commercialization. The Company has clinical programs in Hemophilia B, Fabry disease and Gaucher disease Type 1, as well as a preclinical program in Hemophilia A. Freeline is headquartered in the UK and has operations in Germany and the US.
This press release contains statements that constitute “forward-looking statements” as that term is defined in the United States Private Securities Litigation Reform Act of 1995, including statements that express the Company’s opinions, expectations, beliefs, plans, objectives, assumptions or projections regarding future events or future results, in contrast with statements that reflect historical facts. 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