FRLN Freeline Therapeutics Holdings plc

4.43
-0.16  -3%
Previous Close 4.59
Open 4.6
52 Week Low 4.26
52 Week High 21.69
Market Cap $158,788,486
Shares 35,843,902
Float 35,843,902
Enterprise Value $-57,768,271
Volume 212,773
Av. Daily Volume 230,974
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Upcoming Catalysts

Drug Stage Catalyst Date
FLT190 (MARVEL-1)
Fabry disease
Phase 1/2
Phase 1/2
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FLT180a
Hemophilia B
Phase 1/2
Phase 1/2
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Drug Pipeline

Drug Stage Notes
FLT201
Gaucher disease
Phase 1
Phase 1
Phase 1 trial to be initiated late-2021.

Latest News

  1. CAMBRIDGE, England, July 5, 2021 /PRNewswire/ -- Transine Therapeutics, a biotechnology company developing a novel class of therapeutic RNAs based on its pioneering SINEUP® platform technology, today announced the appointment of Jan Thirkettle, Ph.D. as its Chief Executive Officer. He joins Transine's team at a time of rapid growth following the Company's recent successful £9.1 million extended Seed financing, which was co-led by Takeda Ventures, Inc. and the Dementia Discovery Fund.

    Dr Thirkettle has close to 25 years' experience of building cross-functional teams to drive delivery of innovative technologies. Jan has built platforms and played a leadership role in the commercialisation of multiple therapeutics across small molecule, biopharmaceutical…

    CAMBRIDGE, England, July 5, 2021 /PRNewswire/ -- Transine Therapeutics, a biotechnology company developing a novel class of therapeutic RNAs based on its pioneering SINEUP® platform technology, today announced the appointment of Jan Thirkettle, Ph.D. as its Chief Executive Officer. He joins Transine's team at a time of rapid growth following the Company's recent successful £9.1 million extended Seed financing, which was co-led by Takeda Ventures, Inc. and the Dementia Discovery Fund.

    Dr Thirkettle has close to 25 years' experience of building cross-functional teams to drive delivery of innovative technologies. Jan has built platforms and played a leadership role in the commercialisation of multiple therapeutics across small molecule, biopharmaceutical and gene therapy modalities.

    Dr Thirkettle joins Transine from Freeline Therapeutics Holdings (NASDAQ:FRLN) where he was start-up CEO and more recently Chief Development Officer. There he led the early establishment of the company's pipeline, overseeing the deployment of a novel AAV manufacturing platform and playing a core role in delivering Series B & C raises and a successful IPO in 2021.  

    Prior to Freeline Therapeutics Jan spent 18 years at GSK in multiple leadership roles spanning from Discovery to Manufacturing, including overseeing the establishment of GSK's Cell & Gene Therapy platform unit and playing a pivotal role in the commercialisation of Strimvelis, the first ex-vivo gene therapy to receive an EU Marketing Authorisation Application. 

    Jan holds an MA in Chemistry and a Ph.D. in Biological Chemistry from Oxford University.  

    "Jan is a highly experienced leader with a proven track record of establishing novel platforms and developing new therapeutics, making him a perfect fit for Transine," said Robbie Woodman, Chairman of Transine's Board of Directors. "His expertise in guiding biotechnology ventures across all stages of development and his deep scientific knowledge will be crucial in the development of Transine's pipeline of novel mRNA-targeted therapeutics, initially focused on central nervous system and ophthalmology indications. We are thrilled to have him join."

    "Transine has a world-class academic foundation based on 10 plus years of innovation from the Labs of Professors Stefano Gustincich and Piero Carninci who identified a new functional and naturally occurring class of long non-coding RNAs. Combined with an outstanding team the Company has the opportunity to build an entire new class of therapeutic RNAs that can exquisitely modulate protein expression by interacting with the natural cellular translation machinery," said Dr Thirkettle, CEO of Transine Therapeutics. "This novel approach has tremendous potential, in particular as it overcomes the limitations of current gene therapy and RNA technology approaches. I am delighted that the Board has asked me to lead Transine as we work to develop our pioneering SINEUP® platform technology."

    About Transine Therapeutics

    Transine Therapeutics is a private biotechnology company focused on the development of a novel class of therapeutic RNAs that can upregulate protein expression with an unprecedented level of control and specificity: the SINEUPs®. The company's pioneering technology is expected to offer an entirely unique mechanism of action for currently hard-to-treat diseases by massively extending the druggable proteome and allowing it to address diseases which have been beyond the reach of small molecules, conventional biologics or gene therapies.

    Transine, which was founded in 2020 and is headquartered in Cambridge UK, is backed by Takeda Ventures, Inc. and the Dementia Discovery Fund.

    For more information, please visit: www.transinetx.com

    For more information please contact:

    Transine Therapeutics

    Dr Jan Thirkettle, CEO

     

    MEDiSTRAVA Consulting

    Sylvie Berrebi, David Dible, Frazer Hall

     

    T: +44 (0)7714 306525

     

    Cision View original content:https://www.prnewswire.com/news-releases/transine-therapeutics-appoints-industry-veteran-jan-thirkettle-phd-as-ceo-301325424.html

    SOURCE Transine Therapeutics

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  2. Appointment of Chief Technical Operations Officer James Bircher continues evolution of leadership team to execute across near- and long-term priorities

    Amit Nathwani, MD, PhD to return to Chief Scientific Officer role on an interim basis to replace departing Chief Scientific Officer Romuald Corbau

    LONDON, June 30, 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 the expansion of its executive leadership team with the appointment of James "Jay" Bircher as Chief Technical Operations Officer effective July 1…

    Appointment of Chief Technical Operations Officer James Bircher continues evolution of leadership team to execute across near- and long-term priorities

    Amit Nathwani, MD, PhD to return to Chief Scientific Officer role on an interim basis to replace departing Chief Scientific Officer Romuald Corbau

    LONDON, June 30, 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 the expansion of its executive leadership team with the appointment of James "Jay" Bircher as Chief Technical Operations Officer effective July 1, 2021. Mr. Bircher brings nearly three decades of operational excellence in leadership roles across a wide range of pharmaceutical, biotechnology and medical device manufacturing platforms, including AAV gene therapy.

    Mr. Bircher will report to Chief Executive Officer Theresa Heggie as part of the executive leadership team. He will bring to Freeline his deep experience leading manufacturing site teams through successful regulatory inspections conducted by the US Food and Drug Administration ("FDA"), including the Center for Biologics Evaluation and Research ("CBER"), the Center for Drug Evaluation and Research ("CDER"), the European Medicines Agency ("EMA") and other regulatory agencies.

    "Jay's deep knowledge base across manufacturing platforms, compliance, validation, quality control and regulatory matters adds tremendous value to Freeline," said Theresa Heggie, Chief Executive Officer of Freeline. "We are building a world-class gene therapy company, with a leadership team that has the strength, experience and vision to realize the potential of our leading capsid and platform technology. Jay's expertise in AAV gene therapies and successful manufacturing track record will help enhance the Company's ability to advance our gene therapy candidates from the clinic to commercialization."

    Mr. Bircher said, "I am excited to join Freeline now as the Company gains momentum with plans for a third program in the clinic by the end of this year. Freeline has developed industry-leading AAV platform technology and manufacturing capabilities, which together offer the possibility of delivering potentially transformative medicines that could greatly improve patients' lives."

    The Company also announced that Chief Scientific Officer ("CSO"), Romuald Corbau, PhD, has decided to leave Freeline to pursue other opportunities. Dr. Corbau will continue in his current role until August 20, 2021, to facilitate the smooth transition of his responsibilities. Freeline has begun an international search for his replacement.

    Amit Nathwani, MD, PhD, who previously served as the CSO of Freeline, will assume the CSO role on an interim basis. Professor Nathwani, who is the Company's co-founder, Scientific Advisor and a member of the Board of Directors, has conducted pioneering research on gene therapy in Hemophilia B.

    Ms. Heggie added, "I thank Romu for his many contributions to Freeline, including building a stellar team of scientists whose protein engineering and other complementary expertise have been instrumental in the development and maturation of our differentiated platform technology. We are pleased that Amit will be stepping into an interim role as we undertake a comprehensive search for the next CSO."

    About James "Jay" Bircher

    Mr. Bircher joins Freeline from Abeona Therapeutics, where he held several roles of increasing responsibility, serving most recently as the company's Chief Technology Officer leading Process Development, Assay Development, Quality, Validation, Manufacturing, Facilities, CMC Program Management and Supply Chain. Before Abeona, he was Head of Quality at Becton, Dickinson and Company (BD), where he led the company from the groundbreaking of a 120,000-square-foot sterile manufacturing facility to an FDA pre-approval inspection and the eventual launch of several ready-to-administer, prefilled syringe products. Later in his tenure at BD, Mr. Bircher served as Head of Manufacturing, where he was responsible for all formulation, fill and packaging operations. He also held positions of increasing responsibility related to quality with GlaxoSmithKline, Amgen and Tyco Healthcare. Mr. Bircher has a Bachelor of Science degree from Marshall University and is Six Sigma certified.

    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 a missing 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, as well as a preclinical program in Hemophilia A. 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 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, discussion of the Company's strategies, statements regarding the initiation, timing, progress and results of the Company's preclinical studies and clinical trials, including the advancing of the dose-escalation for the Phase 1/2 clinical study of FLT190; statements that the Company will continue to progress its FLT190 clinical trial; business plans and prospects; capital allocation objectives; and manufacturing, research, pipeline, and clinical trial plans, including anticipated clinical development milestones for the Company's product candidates, as well as its management succession 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 uncertainties inherent in research and 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 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 Company's ability to design and implement successful clinical trials for its product candidates; whether the Company's cash resources will be sufficient to fund the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements for the Company's 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 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 enroll 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, regulatory approval or commercialization of its product candidates; the Company's ability to identify or discover additional product candidates, or failure to capitalize on programs or product candidates; as well as the ability of the Company to successfully recruit, hire and retain officers and other employees with required or desired skills, training and education on a timely basis to replace departing officers or otherwise, which could adversely impact the Company's development, regulatory approval or commercialization of its 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 our Annual Report on Form 20-F for the fiscal year ended December 31, 2020 and in our 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 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.

    Contact

    David S. Arrington

    Vice President, Investor Relations & Corporate Communications

    Freeline Therapeutics

     

    +1 (646) 668 6947



    Primary Logo

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  3. Lowest dose cohort complete and study positioned for dose escalation

    Company to present FLT190 data by year-end

    LONDON, June 16, 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 dosing of the second patient in the ongoing Phase 1/2 MARVEL-1 clinical trial of FLT190, its liver-directed AAV gene therapy candidate for Fabry disease.

    "Dosing the second patient in the MARVEL-1 study is an important milestone for Freeline and evidence of progress in our Fabry program," said Theresa Heggie, Chief Executive Officer…

    Lowest dose cohort complete and study positioned for dose escalation

    Company to present FLT190 data by year-end

    LONDON, June 16, 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 dosing of the second patient in the ongoing Phase 1/2 MARVEL-1 clinical trial of FLT190, its liver-directed AAV gene therapy candidate for Fabry disease.

    "Dosing the second patient in the MARVEL-1 study is an important milestone for Freeline and evidence of progress in our Fabry program," said Theresa Heggie, Chief Executive Officer of Freeline. "Easing of COVID-19 restrictions, together with geographic expansion of study sites, should enable continued enrollment as we work to make FLT190 available to the Fabry disease patient community. With dosing complete in our first cohort, we look forward to advancing through the next cohorts in our dose-finding study. We plan to present data later this year to demonstrate the potential for FLT190 to be a transformative treatment for patients with Fabry disease."

    MARVEL-1 is a multicenter, international, Phase 1/2 dose-finding trial in adult males with classic Fabry disease being conducted in two parts: in previously treated patients and in previously untreated patients. The study is evaluating the safety and efficacy of FLT190 across four dose levels in approximately 10 patients and monitoring patients for nine months after dosing. Patients will be eligible to participate in a long-term follow-up study for further evaluation of the safety and efficacy of FLT190. In addition to safety, efficacy endpoints include levels of αGLA production, clearance of globotriaosylceramide ("Gb3") and globotriaosylsphingosine ("LysoGb3") from the plasma, changes in renal and skin biopsies, renal and cardiac function, αGLA immune response, and quality of life.

    About FLT190

    FLT190 is an investigational AAV gene therapy in development as a potential treatment for patients with Fabry disease that is currently being studied in a Phase 1/2 clinical trial. FLT190 consists of our next generation potent AAVS3 engineered capsid, containing an expression cassette with a codon-optimized human GLA cDNA under the control of the liver-specific promoter FRE1. 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.

    About Fabry Disease

    Fabry disease is a genetic lysosomal storage disorder that leads to the deficiency of a key enzyme needed to break down a fatty substance called globotriaosylceramide ("Gb3"). Without the enzyme, this fatty substance builds up throughout the body, affecting tissues and organs. Fabry disease occurs in all ethnic groups and is estimated to affect one in every 40,000 people.

    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 a missing 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, as well as a preclinical program in Hemophilia A. 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 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, discussion of the Company's strategies, anticipated operating and financial performance and financial condition; the Company's expectations regarding its use of cash and cash runway; statements regarding the initiation, timing, progress and results of the Company's preclinical studies and clinical trials, including the advancing of the dose-escalation for the Phase 1/2 clinical study of FLT190; statements that the Company will continue to progress its FLT190 clinical trial; business plans and prospects; capital allocation objectives; and manufacturing, research, pipeline, and clinical trial plans, including anticipated clinical development milestones for the Company's 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 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 uncertainties inherent in research and 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 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 Company's ability to design and implement successful clinical trials for its product candidates; whether the Company's cash resources will be sufficient to fund the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements for the Company's 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 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 enroll 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, regulatory approval 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. 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 our Annual Report on Form 20-F for the fiscal year ended December 31, 2020 and in our 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 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.

    Contact

    David S. Arrington

    Vice President, Investor Relations & Corporate Communications

    Freeline Therapeutics



    +1 (646) 668 6947



    Primary Logo

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  4. FLT190 Phase 1/2 dose-finding study in Fabry disease on track to dose additional patients; Company to present data by year-end

    FLT201 Phase 1/2 dose-finding study in Gaucher disease Type 1 expected to be in the clinic by year-end

    FLT180a Phase 1/2 dose-confirmation study in Hemophilia B on track to initiate trial sites by year-end

    LONDON, May 17, 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 reported financial results for the first quarter of 2021 and provided an update on recent business highlights.

    "2021 is an…

    FLT190 Phase 1/2 dose-finding study in Fabry disease on track to dose additional patients; Company to present data by year-end

    FLT201 Phase 1/2 dose-finding study in Gaucher disease Type 1 expected to be in the clinic by year-end

    FLT180a Phase 1/2 dose-confirmation study in Hemophilia B on track to initiate trial sites by year-end

    LONDON, May 17, 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 reported financial results for the first quarter of 2021 and provided an update on recent business highlights.

    "2021 is an important year of execution for Freeline and we are encouraged by the progress we see in patient identification and screening in the FLT190 Phase 1/2 dose-finding trial for the treatment of Fabry disease," said Theresa Heggie, Chief Executive Officer of Freeline. "The easing of COVID restrictions and our strategic geographical diversification of trial sites, along with the ongoing identification of patients through our close working relationships with investigators and patient organizations, give us confidence with respect to recruitment into this important trial for people with Fabry disease. We look forward to sharing more information about study progress later this year."

    "We are advancing our FLT201 program for the treatment of Gaucher disease Type 1 into the clinic in a Phase 1/2 dose-finding study, which we plan to initiate by year-end," continued Ms. Heggie. "Our FLT180a program for the treatment of Hemophilia B also continues to progress and we will initiate the dose-confirmation trial later this year."

    "We also continue to leverage our differentiated AAVS3 technology platform, which underlies each of our programs and consists of a proprietary, rationally-designed capsid, optimized expression cassette design, novel protein engineering capabilities, rapid candidate screening and cutting-edge analytics," added Ms. Heggie. "We are committed to providing patients with safe and effective gene therapy and our potent platform, together with a long-standing focus on quality, provides us with the possibility of delivering functional cures to patients and the ability to address indications considered beyond the reach of first-generation vectors."

    Recent Business Highlights

    • Continued enrollment in the ECLIPSE run-in study for FLT180a for the treatment of Hemophilia B, supports enrollment in the Phase 1/2 dose-confirmation trial: Freeline continues to enroll patients into the ECLIPSE run-in study and anticipates that this will accelerate enrollment in the Phase 1/2 dose-confirmation trial, which will include only a small number of patients.
    • Presented FLT190 data that demonstrated GLA uptake and metabolic cross-correction in Fabry disease-relevant cell lines: In May 2021, the Company presented data at the American Society of Gene and Cell Therapy ("ASGCT") Annual Meeting 2021 demonstrating GLA uptake and metabolic cross-correction in Fabry disease relevant cell lines supportive of the Company's ongoing development of FLT190.
    • Advanced FLT201 for the treatment of Gaucher disease Type 1 in key markets: The Company previously announced that it filed a Clinical Trial Authorization ("CTA") with the UK Medicines and Healthcare products Regulatory Agency ("MHRA") and has also completed a similar filing with the Ministry of Health ("MOH") in Israel, a key market for Gaucher disease Type 1.
    • Presented data on foundational technology supporting the Company's robust gene therapy platform: In May 2021, Freeline presented four posters at ASGCT, which detailed advancements in its high-throughput rAAV manufacturing platform optimization and candidate selection, development scale-up of suspension cell-based AAV manufacturing and quantification assay strategy for AAV-based gene therapies, as well as assay development to measure transduction efficiency.
    • Strengthened the Freeline Board of Directors: In March 2021, the Company announced the appointment of Colin Love, PhD to the Freeline Board of Directors. Dr. Love has extensive experience in manufacturing complex biotechnology products and currently serves as the Chief Operating Officer of Replimune.
    • Strengthened the leadership team: Today, the Company announced the appointment of Eric Fink to the Freeline leadership team. Effective May 24, 2021, Mr. Fink will join Freeline in a newly-created position of Chief Human Resources Officer ("CHRO"). Mr. Fink joins from Global Blood Therapeutics, Inc., where he was the CHRO. Mr. Fink brings significant expertise in building organizations as they progress toward commercialization, and he will be based in the Company's New York office. The role builds on the recent appointment of Michael Parini, who joined the Company in March 2021 from Vertex, Inc. in the newly-created role of President and Chief Operating Officer. Mr. Parini is a global pharmaceutical executive and a recognized biotechnology leader in areas such as corporate strategy, operational effectiveness and building high-performing organizations. Mr. Parini is based in the Company's New York office.

    Selected Anticipated Milestones in 2021

    • Progress dose escalation for the FLT190 Phase 1/2 dose-finding trial in Fabry disease.
    • Commence clinical development of FLT201 for Gaucher disease Type 1 by initiating a Phase 1/2 dose-finding trial.
    • Report four-year durability data for FLT180a from the Company's Phase 1/2 dose-finding trial in Hemophilia B.
    • Initiate Phase 1/2 dose-confirmation trial for FLT180a to confirm both the dose and immune management regimen for the planned pivotal Phase 3 trial. The Company is targeting full enrollment of the Phase 1/2 dose-confirmation trial during the first half of 2022 with a six-month data readout by the end of 2022.
    • Complete IND/CTA enabling studies for FLT 210 for Hemophilia A.

    Q1 2021 Financial Highlights

    Cash Position: Cash and cash equivalents were $195.8 million as of March 31, 2021, as compared to $230.0 million as of December 31, 2020. Based on the Company's current operating plan, Freeline expects that its current level of cash and cash equivalents will enable the Company to fund its operating expenses into the third quarter of 2022.

    R&D Expenses: Research and development ("R&D") expenses for the three months ended March 31, 2021 were $23.9 million, as compared to $17.5 million for the same period in 2020. The increase of $6.4 million was driven by an increased investment in activities related to the current and proposed clinical trials for FLT201 and FLT210 and overall research and development, which includes earlier pipeline programs, discovery and further development of the Freeline platform.

    G&A Expenses: General and administrative ("G&A") expenses for the three months ended March 31, 2021 were $10.1 million, as compared to $3.7 million for the same period in 2020. The increase of $6.3 million was driven primarily by legal and professional fees related to expenses associated with the Company's obligations as a public company, including annual and periodic reporting, equity compensation programs, more extensive governance requirements and increased audit fees and expenses related to US GAAP requirements, as well as an increase in headcount and related personnel costs.

    As of March 31, 2021, the Company had 35,843,902 ordinary shares outstanding.

    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 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 and Fabry disease, as well as preclinical programs in Gaucher disease and Hemophilia A. 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 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, discussion of the Company's strategies, anticipated operating and financial performance and financial condition; the Company's expectations regarding its use of cash and cash runway; statements regarding the initiation, timing, progress and results of the Company's preclinical studies and clinical trials, including the initiation of the Phase 1/2 dose confirmation trial for FLT180a and data readouts from that trial, progress with respect to the dose-escalation for the Phase 1/2 clinical study of FLT190, commencement of clinical trials in our FLT201 program, and completion of proof-of-concept studies of FLT210; statements that the Company's updated clinical development plan for FLT180a will allow it to address CMC feedback from the FDA in parallel with the Phase 1/2 dose confirmation trial; business plans and prospects; capital allocation objectives; and manufacturing, research, pipeline, and clinical trial plans, including anticipated clinical development milestones for the Company's 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 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 uncertainties inherent in research and 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 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 Company's ability to design and implement successful clinical trials for its product candidates; whether the Company's cash resources will be sufficient to fund the Company's foreseeable and unforeseeable operating expenses and capital expenditure requirements for the Company's 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 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 enroll 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, regulatory approval 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. 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 our Annual Report on Form 20-F for the fiscal year ended December 31, 2020 and in our 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 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 (the "SEC"). You may review these documents by visiting EDGAR on the SEC website at www.sec.gov.

    Contact

    David S. Arrington

    Vice President Investor Relations & Corporate Communications

    Freeline Therapeutics

     

    +1 (646) 668 6947





    Freeline Therapeutics Holdings plc

    Unaudited Condensed Consolidated Statements of Operations Data

    (in thousands of U.S. dollars, except per share data)

     Three Months Ended March 31,
      2021   2020 
    OPERATING EXPENSES:   
    Research and development$        23,863  $        17,457 
    General and administrative 10,078   3,735 
    Total operating expenses 33,941   21,192 
    LOSS FROM OPERATIONS: (33,941)  (21,192)
    OTHER INCOME (EXPENSE) NET:   
    Other income (expense), net (1,733)  863 
    Interest income, net 140   53 
    Benefit from R&D tax credit 598   3,702 
    Total other income (expense), net (995)  4,618 
    Loss before income taxes (34,936)  (16,574)
    Income tax expense (9)  (131)
    Net loss (34,945)  (16,705)
    Net loss per share attributable to ordinary shareholders—basic and diluted$        (0.98) $        (1.85)
    Weighted average ordinary shares outstanding—basic and diluted 35,655,443   9,010,843 
            
            

    Freeline Therapeutics Holdings plc

    Unaudited Condensed Consolidated Balance Sheet Data

    (in thousands of U.S. dollars, except per share data)

     March 31, December 31,
      2021   2020 
    ASSETS   
    CURRENT ASSETS:   
    Cash and cash equivalents$        195,765  $        229,974 
    Account receivable    97 
    Prepaid expenses and other current assets 27,718   28,105 
    Total current assets 223,483   258,176 
    Property and equipment, net 10,093   8,608 
    Intangible assets, net 16   23 
    Other non-current assets 1,655   1,805 
    Total assets$        235,247  $        268,612 
    LIABILITIES, PREFERRED SHARES AND SHAREHOLDERS' EQUITY   
    CURRENT LIABILITIES:   
    Accounts payable$        5,528  $        8,093 
    Accrued expenses and other current liabilities 10,292   10,719 
    Total current liabilities 15,820   18,812 
    Total liabilities 15,820   18,812 
    Commitments and contingencies   
    SHAREHOLDERS' EQUITY:   
    Deferred shares 137   155 
    Additional paid-in capital 458,857   456,293 
    Accumulated other comprehensive loss 11,368   9,342 
    Accumulated deficit (250,935)  (215,990)
    Total shareholders' equity 219,427   249,800 
    TOTAL LIABILITIES AND SHAREHOLDERS' EQUITY$        235,247  $        268,612 

     



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  5. LONDON, May 12, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline"), a clinical-stage biotechnology company developing transformative AAV gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that senior management will participate in a Fireside Chat at the virtual 2021 RBC Capital Markets Global Healthcare Conference on Tuesday, May 18, 2021 at 4:50 pm EDT.

    A webcast of the event will be available on the Investors section of the Freeline website. Senior management will also participate in virtual one-on-one meetings with investors at the conference.

    About Freeline Therapeutics

    Freeline is a clinical-stage biotechnology company developing…

    LONDON, May 12, 2021 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (NASDAQ:FRLN) (the "Company" or "Freeline"), a clinical-stage biotechnology company developing transformative AAV gene therapies for patients suffering from inherited systemic debilitating diseases, today announced that senior management will participate in a Fireside Chat at the virtual 2021 RBC Capital Markets Global Healthcare Conference on Tuesday, May 18, 2021 at 4:50 pm EDT.

    A webcast of the event will be available on the Investors section of the Freeline website. Senior management will also participate in virtual one-on-one meetings with investors at the conference.

    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 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 and Fabry disease, as well as preclinical programs in Gaucher disease and Hemophilia A. Freeline is headquartered in the UK and has operations in Germany and the US.

    Contact

    David S. Arrington

    Vice President Investor Relations & Corporate Communications

    Freeline Therapeutics



    +1 (646) 668 6947 



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