GlobeNewswire Inc | Sep 9, 2021 07:00AM EDT

September 09, 2021

LONDON, Sept. 09, 2021 (GLOBE NEWSWIRE) -- 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 that both the U.S. Food and Drug Administration (FDA) and European Commission (EC) have granted Orphan Drug Designation (ODD) for FLT201 for the treatment of Gaucher disease.

Effectively advancing our core programs is priority one in our refreshed strategic plan, said Michael Parini, Chief Executive Officer of Freeline. Receiving orphan drug designation for FLT201 is an important regulatory milestone in delivering on the promise of our Gaucher program to address the urgent, unmet need of patients living with Gaucher disease Type 1.

Our robust preclinical data suggest that the proprietary engineered, more stable GCase variant in FLT201 has the potential to deliver sustained levels of -glucocerebrosidase and penetrate target tissues in Gaucher disease. We believe our approach may offer significant improvements in clinical outcomes over the existing standard of care, particularly in terms of GCase uptake into difficult-to-reach tissues, said Mr. Parini.

The Company remains on track to initiate our Phase 1/2 dose-finding trial in Gaucher disease Type 1 before year end, continued Mr. Parini. We are enthusiastic about advancing this program, and we plan to share clinical data from the dose-finding trial at the right time in 2022.

Orphan Drug Designation in the United States is granted by the FDA Office of Orphan Products Development to advance the evaluation and development of safe and effective therapies for the treatment of rare diseases or conditions affecting fewer than 200,000 individuals in the United States. Under the Orphan Drug Act, the FDA may provide grant funding toward clinical trial costs, tax credits, FDA user-fee benefits and seven years of market exclusivity in the U.S. following marketing approval.

Orphan Drug Designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP). It is assigned to medicines intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating and affects fewer than five in 10,000 people in the EU. The designation could allow regulatory and financial incentives for companies to develop and market therapies, including market exclusivity, protocol assistance and fee reductions.

About Gaucher Disease

Gaucher disease is a genetic disorder in which a fatty substance called glucosylceramide accumulates in macrophages in certain organs due to the lack of functional GCase. The disorder is hereditary and presents in various subtypes. Freeline is currently focused on Gaucher disease Type 1, the most common type, which impacts the health of the spleen, liver, blood system and bones. The current standard of care is intravenous infusion of ERT every two weeks, which is a significant treatment burden on the patient. The aim of Freelines 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 endogenous GCase, thus eliminating the need for ERT.

About FLT201 for Gaucher Disease

FLT201 is an investigational liver-directed AAV gene therapy in preclinical development for the treatment of Gaucher disease Type 1. FLT201 contains a liver-specific promoter and a GBA1 sequence that expresses Freelines novel, proprietary GCase variant 85, which has a 20-fold longer half-life at lysosomal pH conditions than wild-type GCase protein. Freelines high-transducing AAVS3 capsid advances our goal to address unmet needs for those affected by Gaucher disease by enabling sustained, endogenous production of GCase following one-time intravenous infusion. To our knowledge, Freeline is the only company to date that has announced a program for the development of an AAV gene therapy for the treatment of Gaucher disease Type 1 and plans to file an IND for this program in 2021.

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 the missing or dysfunctional protein into the patients bloodstream. The Companys integrated gene therapy platform includes in-house capabilities in research, clinical development, manufacturing and commercialization. The Company has clinical programs in HemophiliaB and Fabry disease, as well as preclinical programs in Gaucher disease Type 1 and HemophiliaA. Freeline is headquartered in the UK and has operations in Germany and the US.

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 Companys 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 regarding the initiation, timing, progress and results of the Companys preclinical studies and clinical trials, including commencement of the Phase 1/2 dose-finding clinical trial of FLT201 and data readouts from that trial; and manufacturing, research, pipeline, and clinical trial plans, including anticipated clinical development milestones for the Companys product candidates. 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 managements 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 Companys recurring losses from operations; the uncertainties inherent in research and development of the Companys product candidates, including statements regarding the timing of initiation, completion and the outcome of clinical studies or trials and related preparatory work and regulatory review, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with preclinical and clinical data, including the possibility of unfavorable new preclinical, clinical or safety data and further analyses of existing preclinical, clinical or safety data; the Companys ability to design and implement successful clinical trials for its product candidates; the recent departures of a number of executive officers of the Company, and the Companys ability to fill open positions, implement an orderly transition process and retain key talent; whether the Companys cash resources will be sufficient to fund the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements for the Companys expected timeline; the potential for a pandemic, epidemic or outbreak of infectious diseases in the US, UK or EU, including the COVID-19 pandemic, to disrupt and delay the Companys clinical trial pipeline; the Companys 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 Companys ability to enroll patients in clinical trials for its product candidates; the possibility that one or more of the Companys 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 Companys ability to obtain and maintain regulatory approval of its product candidates; the Companys limited manufacturing experience which could result in delays in the development, regulatory approval or commercialization of its product candidates; and the Companys 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. We cannot guarantee that any forward-looking statement will be realized. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in the Companys Annual Report on Form 20-F for the fiscal year ended December 31, 2020 and in subsequent reports on Form 6-K, in each case including in the sections thereof captioned Cautionary Statement Regarding Forward-Looking Statements and Item 3.D. Risk factors. Many of these risks are outside of the Companys 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 Companys reports and documents filed with theU.S. Securities and Exchange Commission (the SEC). You may review these documents by visiting EDGAR on theSECwebsite atwww.sec.gov.

Contact

David S. ArringtonVice President Investor Relations & Corporate CommunicationsFreeline Therapeuticsdavid.arrington@freeline.life+1 (646) 668 6947