Freeline announces supply agreement for haemophilia B program

Published 23 Sep 2020

Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the “Company” or “Freeline”), a clinical stage, fully-integrated, next generation, systemic, AAV-based gene therapy company with the ambition of transforming the lives of patients suffering from inherited systemic debilitating diseases, today announces their supply agreement with Thermo Fisher Scientific Inc. (“Thermo Fisher”).

This agreement secures dedicated production capacity and resources for Freeline from 2021 to 2027, inclusive. This will provide capacity for the planned Phase 2b/3 pivotal clinical trial and potential commercialisation of Freeline’s haemophilia B program, FLT180a, using the Company’s proprietary manufacturing platform and processes. 

Freeline has established the manufacturing process for FLT180a at Thermo Fisher’s Cambridge facility over the past two years, most recently initiating clinical supply production there for its planned Phase 2b/3 pivotal clinical trial of FLT180a.

“I am delighted to announce this very significant extension of our partnership with Thermo Fisher,” said Jan Thirkettle, Chief Development Officer at Freeline. “The investment in, and commitment to, Thermo Fisher’s viral vector services operation gives us great confidence that they are the right partner to enable us to commercialise our haemophilia B therapy, if approved.”

“As demand for new gene therapies continues to grow, we have made strategic investments to expand our viral vector commercial manufacturing capabilities so we can provide our customers with an uninterrupted pathway from development to commercialisation. A great example of this is the work we are doing with Freeline to support its program to develop a novel treatment for haemophilia B and we are pleased to partner with them on this,” said Chris Murphy, vice president and general manager for Thermo Fisher’s viral vector services business.

About Freeline

Freeline is a clinical-stage biotechnology company focused on AAV-based gene therapy targeting the liver. Its vision is to create better lives for people suffering from chronic, systemic diseases using the potential of gene therapy as a one-time treatment to provide a potential functional cure. Freeline is headquartered in the U.K. and has operations in Germany and the U.S.

About Haemophilia

Haemophilia is a genetic bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing. This protein is called a clotting factor. In haemophilia A, there is a deficiency of the clotting factor VIII (eight) protein and in haemophilia B, there is a deficiency of the clotting factor IX (nine) protein. Haemophilia mainly affects boys and men; however, women can be ‘carriers’ of the affected gene and may experience symptoms. Haemophilia A is the most common type of haemophilia affecting about one in every 5,000 males, while haemophilia B affects about one in every 30,000 males. Haemophilia is classified as mild, moderate or severe, depending on the level of clotting factor VIII or IX in the blood and is diagnosed through blood tests.

About FLT180a

The Freeline haemophilia B program, FLT180a, uses a synthetic AAVS3 capsid and a gain of function variant of human factor IX (FIX). The therapy is currently being studied in a Phase 1/2 trial with the goal of normalising FIX activity in patients with moderate and severe haemophilia.

- Ends -

Forward-Looking Statements

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. Examples include discussion of the Company’s strategies, financing plans, and clinical trial plans. In some cases, you can identify such forward-looking statements by terminology such as “anticipate,” “intend,” “believe,” “estimate,” “plan,” “seek,” “project” or “expect,” “may,” “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 many risks and uncertainties, including the Company’s recurring losses from operations; the development of the Company’s product candidates, including statements regarding the timing of initiation, completion and the outcome of clinical studies or trials and related preparatory work; the Company’s ability to design and implement successful clinical trials for its product candidates; the potential for a pandemic, epidemic or outbreak of an infectious diseases in the U.S., U.K. or EU, including the COVID-19 pandemic, to disrupt the Company’s clinical trial pipeline; the Company’s failure to demonstrate the safety and efficacy of its product candidates; the fact that results obtained in earlier stage clinical testing may not be indicative of results in future clinical trials; the Company’s ability to enrol patients in clinical trials for its product candidates; the possibility that one or more of the Company’s product candidates may cause serious adverse, undesirable or unacceptable side effects or have other properties that could delay or prevent their regulatory approval or limit their commercial potential; the Company’s ability to obtain and maintain regulatory approval of its product candidates; the Company’s limited manufacturing experience which could result in delays in the development or commercialization of its product candidates; and the Company’s ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates. Such risks and uncertainties may cause the statements to be inaccurate and readers are cautioned not to place undue reliance on such statements. Many of these risks are outside of the Company’s control and could cause its actual results to differ materially from those it thought 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 declines, any obligation to update any such statements or to publicly announce the results of any revisions to any such statements to reflect future events or developments, except as required by law.

For further information, please reference the Company’s reports and documents filed with the U.S. Securities and Exchange Commission. You may get these documents by visiting EDGAR on the SEC website at www.sec.gov.