Company to progress to second dose cohort in the MARVEL-1 study immediately, with first patient dosing expected in mid-2022
LONDON, March 24, 2022 (GLOBE NEWSWIRE) – Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the “Company” or “Freeline”), today announced an updated clinical development plan and timelines for FLT190 for people with Fabry disease, a rare, inherited lysosomal storage disorder resulting in cell abnormalities and organ dysfunction that affect small blood vessels, tissues and organs including skin, kidneys, heart, and the nervous system. The Company will proceed immediately to the second dose cohort (1.5e12 vg/kg) in the MARVEL-1 dose escalation study, with the first patient expected to be dosed in mid-2022.
Previous plans to dose an additional patient in the first cohort (7.5e11 vg/kg) have been revised based on a thorough review of the pre-clinical data and the clinical efficacy and safety data for the first and second patients in the MARVEL-1 study, which now extend two years and nine months, respectively. Freeline presented the results of this data review to the independent Data Monitoring Committee (DMC) of the MARVEL-1 study, which supports accelerated progression to the second dose cohort.
“The consistency of the data, including the additional safety and efficacy data collected since our last update, and the feedback from DMC and regulatory authorities reinforce our confidence about proceeding to the second dose level without dosing a third patient at the lowest dose. We are also exploring potential options to further accelerate dosing based on the safety and efficacy data from this next patient,” said Pamela Foulds, MD, Chief Medical Officer of Freeline. “We are very excited about the opportunity to advance the development of our potentially life-changing gene therapy for people living with Fabry disease.”
“Strong execution on our clinical programs has been a key pillar of delivering on our commitment to bring life-changing medicines to patients,” said Michael Parini, Chief Executive Officer of Freeline. “The progress being made in our FLT190 program under the leadership of our Chief Medical Officer Dr. Pamela Foulds is energizing and consistent with the culture we have instilled in this new version of Freeline to unlock the value of our promising clinical candidates.”
Freeline plans to provide an update on its Fabry program in the second half of 2022. The Company will report full-year 2021 financial results and provide a more complete update on business highlights on March 31, 2022.
About Freeline Therapeutics Holdings plc
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 the missing or dysfunctional 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. Freeline is headquartered in the UK and has operations in Germany and the US.
About Fabry Disease
Fabry disease is a genetic lysosomal disease that leads to a deficiency in α-galactosidase A (α-Gal A), which is a key enzyme needed to break down a fatty substance called globotriaosylceramide (Gb3) and lyso-Gb3. Without the enzyme, this fatty substance builds up throughout the body, affecting tissues and organs including skin, kidneys, heart, and the nervous system. Freeline is currently focused on classic Fabry disease where patients have little to no functional α-Gal A enzyme. The current standard of care is lifelong intravenous infusions of enzyme replacement therapy (ERT) or pharmacological chaperone therapy (PCT). Certain treatments can carry a significant burden on the patient. The aim of Freeline’s investigational gene therapy program is to deliver a one-time treatment of a long-lasting gene therapy that will provide a sustained, therapeutically relevant level of α-Gal A that the Company believes would eliminate the need for ERT or PCT.
About FLT190 for Fabry Disease
FLT190 is an investigational AAV gene therapy in development as a potential treatment for patients with Fabry disease. FLT190 consists of a potent, rationally designed capsid (AAVS3) containing an expression cassette with a codon-optimized human α-Gal A cDNA under the control of a liver-specific promoter. The Company’s current MARVEL-1 Phase 1/2 dose-finding trial is evaluating the potential safety and efficacy of FLT190 in Fabry patients, who often have pre-existing cardiac manifestations due to underlying substrate accumulation and disease progression in the heart. 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.
This press release may contain 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. Examples include, among other topics, statements that the first patient in the second dose cohort of the Company’s Phase 1/2 MARVEL-1 clinical trial of FLT190 is expected to be dosed in mid-2022, regarding the potential to further accelerate dosing based on the safety and efficacy data from this next patient, regarding FLT190’s potential to be a life-changing gene therapy for people living with Fabry disease and that the Company plans to provide an update on its Fabry program in the second half of 2022. 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Arne Naeveke, PhD
Vice President, Head of Corporate Communications
+1 617 312 2521