Freeline reports Q1 results

Syncona Limited

Freeline Reports First Quarter Financial Results

18 May 2021

Syncona Ltd, a leading healthcare company focused on founding, building and funding a portfolio of global leaders in life science, notes the announcement that its portfolio company, Freeline Therapeutics Holdings plc (Nasdaq: FRLN), a clinical-stage, fully integrated, next generation, systemic, liver directed, AAV-based gene therapy company announced its financial results for the first quarter 2021 and provided an update on recent business highlights.

The announcement can be accessed on Freeline's website at: https://www.freeline.life/investors-media/newsroom/ and the full text of the announcement from Freeline is contained below.

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About Syncona

Syncona's purpose is to invest to extend and enhance human life. We do this by founding and building companies to deliver transformational treatments to patients in areas of high unmet need.

Our strategy is to found, build and fund companies around exceptional science to create a dynamic portfolio of 15-20 globally leading healthcare businesses for the benefit of all our stakeholders. We focus on developing treatments for patients by working in close partnership with world-class academic founders and management teams. Our strategic balance sheet underpins our strategy enabling us to take a long-term view as we look to improve the lives of patients with no or few treatment options, build sustainable life science companies and deliver strong risk-adjusted returns to shareholders.

Freeline Reports First Quarter 2021 Financial Results and Recent Business Highlights

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 - 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.

Contact

David S. Arrington
Vice President Investor Relations & Corporate Communications
Freeline Therapeutics
[email protected]
+1 (646) 668 6947