Freeline Therapeutics – Focusing On Life-changing TherapiesNovember 12, 2021
Shares of Freeline Therapeutics Holdings plc (FRLN) have lost nearly 83% year-to-date and trade around $3.
Freeline is a clinical-stage biotechnology company developing next-generation gene therapy for patients suffering from inherited systemic debilitating diseases like Haemophilia, Fabry disease, and Gaucher disease.
The company’s lead product candidate is FLT180a, a next-generation AAV gene therapy for the treatment of Haemophilia B.
FLT180a has completed a phase I/II dose-finding trial in patients with Haemophilia B, dubbed B-AMAZE.
Hemophilia is a genetic bleeding disorder where a protein made by the body to help make blood clot, known as the clotting factor, is either partly or completely missing. Hemophilia B is caused due to deficiency of the clotting factor IX (nine) while hemophilia A is the result of the deficiency of clotting factor VIII (eight).
Study results from the B-AMAZE trial, reported last December, showed durable Factor IX (“FIX”) activity up to a period of nearly three years, with no bleeds reported requiring FIX supplementation. The four-year durability data from the B-AMAZE trial is expected to be presented at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition next month.
A run-in study to collect data on patients’ FIX activity levels, bleed frequency, and eligibility for gene therapy clinical trials, dubbed ECLIPSE, is underway.
A six-month phase I/II dose-confirmation trial of FLT180a is expected to be initiated by year end 2021. This trial is designed to confirm the dose and immune management regimen for the proposed phase III pivotal trial of FLT180a, which is planned for initiation by mid-2023.
Next in the pipeline is FLT190, an investigational AAV gene therapy for treatment of Fabry disease, under a phase I/II trial, dubbed MARVEL-1.
Fabry disease is a genetic lysosomal storage disorder that leads to buildup of a fatty substance called globotriaosylceramide (“Gb3”) throughout the body, affecting tissues and organs. This disorder is caused by a deficiency of an enzyme alpha-galactosidase A.
The MARVEL-1 trial, initiated in July 2019, is designed to enroll 15 participants, with study completion expected by December 2022.
Freeline is also developing an AAV-mediated gene therapy for the treatment of Gaucher disease Type 1, which goes by the name FLT201.
Gaucher disease, caused due to deficiency of an enzyme called glucocerebrosidase (GCase), is a genetic disorder in which a fatty substance called glucosylceramide accumulates in macrophages in certain organs. Gaucher disease Type 1, the most common type, impacts the health of spleen, liver, blood system and bones.
FLT201 is expected to advance into a phase I/II dose-finding trial for the treatment of Gaucher disease Type 1 by the end of this year.
There is one more product candidate in the pipeline – FLT210 – a second generation AAV-hFVIII vector, for the treatment of Hemophilia A.
The company expects to complete preclinical IND/CTA enabling studies of FLT210 by the end of this year.
The company’s cash and cash equivalents were $164.7 million as of June 30, 2021.
Freeline Therapeutics, headquartered in the UK, made its debut on the Nasdaq Global Select Market on August 7, 2020, pricing its IPO at $18 per American Depositary Share (ADS).
FRLN has traded in a range of $2.91 to $18.88 in the last 1 year. The stock closed Thursday’s trading at $3.17, up 0.63%.
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