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LONDON, June 24, 2022 (GLOBE NEWSWIRE) — Freeline Therapeutics Holdings plc (FRLN) today announced the upcoming presentation of important new clinical data for its AAVS3-based gene therapy candidate FLT180a to the International Society of Thrombosis and haemostasis (ISTH) Congress to be held in London, July 9-13, 2022.
Presentation will provide initial safety and efficacy data from the first cohort of the Phase 1/2 B-LIEVE trial, which aims to confirm the FLT180a dosing and immune management regimen for the pivotal Phase trial 3 scheduled for 2023. The treatment goal with FLT180a is to provide a functional cure for people with hemophilia B by providing predictable and sustained normalization of coagulation factor IX levels with a good safety profile.
The poster (PB0213) will be presented between 6:30 and 7:30 BST on Sunday July 10, 2022. The abstract has been published on the ISTH website:
The presentation poster will be available in the Investors section of the Freeline website after its presentation at the ISTH congress.
About the B-LIEVE Dose Confirmation Trial
B-LIEVE is a Phase 1/2 dose-confirmation trial of FLT180a using short-term immune prophylaxis with the goal of normalizing FIX levels in patients with severe and moderately severe hemophilia B. The starting dose of 7.7e11 vg/kg was selected based on the results of the Phase 1/2 B-AMAZE dose-finding trial and multiple modeling approaches. Freeline expects the B-LIEVE trial to finalize a dose for the pivotal Phase 3 trial intended to enable predictable and sustained expression of factor IX (FIX) within the normal range for people with hemophilia B .
About FLT180a for people with hemophilia B
Freeline’s FLT180a candidate uses a potent and rationally designed AAVS3 capsid containing an expression cassette encoding a gain in the Padua variant of human factor IX (FIX) function. FLT180a was studied in B-AMAZE, a Phase 1/2 dose-finding trial in patients with severe and moderately severe hemophilia B with the aim of normalizing FIX activity in patients with hemophilia B moderate and severe. Patients treated in B-AMAZE are followed in a long-term follow-up study. A Phase 1/2 dose confirmation trial of FLT180a called B-LIEVE to finalize a dose for a pivotal Phase 3 trial is underway.
About hemophilia B
Hemophilia B is a rare and debilitating inherited bleeding disorder caused by a defect in the clotting factor IX (FIX) gene. Hemophilia B is linked to the X chromosome and mainly affects boys and men; however, women who carry an affected copy of the clotting factor gene may also experience symptoms. Hemophilia B is classified as mild, moderate or severe, depending on the level of FIX in the blood, and is diagnosed by blood tests. The World Federation of Hemophilia’s 2020 Annual Global Survey estimated that there are approximately 15,000 patients with hemophilia B in the United States, Europe and Japan. A meta-analysis using national registries in Australia, Canada, France, Italy, New Zealand and the United Kingdom estimated a prevalence in men of 3.8 in 100,000, or about 1 in 30,000 .1
About Freeline Therapeutics
Freeline is a clinical-stage biotechnology company developing transformative systemic gene therapies mediated by adeno-associated virus (AAV). The company is dedicated to improving the lives of patients through innovative, timely treatments that can provide functional cures for debilitating inherited systemic 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 in the patient’s blood circulation. The Company’s integrated gene therapy platform includes in-house research, clinical development and commercialization capabilities. The company has clinical programs in hemophilia B, Fabry disease and Gaucher disease type 1. Freeline is headquartered in the UK with operations in Germany and the United States.
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 opinions, expectations, beliefs, plans, Freeline Therapeutics’ goals, assumptions or projections. Holdings plc (the “Company”) regarding future events or future results, as opposed to statements that reflect historical facts. In some cases, you can identify these forward-looking statements by terminology such as “anticipate”, “intend”, “believe”, “estimate”, “plan”, “seek”, “project” or “will”. expect”, “could”, “will”, “would”, “could” or “should”, the negative of these terms or similar expressions. Forward-looking statements are based on management’s current beliefs and assumptions and on information currently available to the Company, and you should not place undue reliance on such statements. Forward-looking statements are subject to numerous risks and uncertainties. These risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. We cannot guarantee that any forward-looking statement will materialize. Should known or unknown risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results could differ materially from past results and from those anticipated, estimated or projected. Investors are cautioned not to place undue reliance on forward-looking statements. A more detailed list and description of the risks, uncertainties and other matters can be found in the Company’s Annual Report on Form 20-F for the year ended December 31, 2021 and in subsequent reports on Form 6- K, in each case, including in the sections entitled “Caution Regarding Forward-Looking Statements” and “Item 3.D. Risk Factors.” Many of these risks are beyond the Company’s control and could cause its actual results to differ materially from those it believed would occur. The forward-looking statements included in this press release are made only as of the date hereof. The Company does not undertake, and specifically disclaims, any obligation to update these statements or to publicly announce the results of any revisions to these statements to reflect future events or developments, unless the required by law. For more information, please refer to the Company’s reports and documents filed with the United States Securities and Exchange Commission (the “SEC”). You may view these documents by visiting EDGAR at SEC website at www.sec.gov.
Arne Naeveke, PhD
Vice President, Head of Corporate Communications
+1 617 312 2521
1 Iorio A et al. Annals of Internal Medicine 2019;171(8):540-7