Freeline Therapeutics Holdings Plc
Freeline Therapeutics — Investor Presentation (June 2021)
19 slides from Freeline Therapeutics's investor presentation, Jun 21, 2021
Slide 1: Corporate Presentation June 21, 2021
Corporate Presentation June 21, 2021
Slide 2: Legal disclaimer
Legal disclaimer This presentation 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, assum
Slide 3: Clinical-stage biotechnology company developing transformative AAV-mediated gene therapies
Clinical-stage biotechnology company developing transformative AAV-mediated gene therapies for patients suffering from inherited systemic debilitating diseases Proprietary capsid enables high protein expression at low doses in humans Freeline positioned to target diseases perceived as beyond the rea
Slide 4: In-house research and manufacturing platform allows us to rationally design & deliver novel AAV gene therapy programs
In-house research and manufacturing platform allows us to rationally design & deliver novel AAV gene therapy programs Vector design Protein Assay Rapid Manufacturing Cutting edge Optimise vector engineering development candidate technology & analytics design and Design and Develop assays to screenin
Slide 5: Hemophilia B data validates AAVS3 capsid and platform
Hemophilia B data validates AAVS3 capsid and platform Our rationally designed Potent liver High protein Lower dose levels & AAVS3 capsid enables: transduction expression improved safety margin 200 190% Factor IX (FIX) activity level (%) Mean: 171% *** 150 The normal range (50-150% FIX activity) 143%
Slide 6: Robust pipeline in place: Aim to have third program in the clinic by end of 2021
Robust pipeline in place: Aim to have third program in the clinic by end of 2021 Development IND enabling Patient No. & WW Program Research1 studies2 Phase 1/2 Phase 3 US, UK & EU43 Commercial rights4 Hemophilia B FLT180a ~ 9,000 (RMAT designation) 1. In the research stage, we conduct in vitro and i
Slide 7: Hemophilia B & A
The Freeline mission: To be life changers Hemophilia B & A
Slide 8: Verbrinacogene setparvovec (FLT180a): Potential to provide a functional cure by normalizing FIX activity
Verbrinacogene setparvovec (FLT180a): Potential to provide a functional cure by normalizing FIX activity Key learnings from the Phase 1/2 B-AMAZE dose-finding clinical trial Demonstrated that the dose with potential to achieve FIX activity in the normal range is expected to be between 6.4e11 vg/kg a
Slide 9: FLT180a clinical development plan to accelerated approval
FLT180a clinical development plan to accelerated approval • Six-month Phase 1/2 trial to confirm the dose and immune management regimen for use in a pivotal Phase 3 study • File accelerated approval using the surrogate endpoint of FIX activity levels combined with demonstration of a positive correla
Slide 10: Hemophilia A
The Freeline mission: To be life changers Hemophilia A
Slide 11: FLT210 contains the only Factor VIII construct that fits within the carrying capacity of an AAV capsid
FLT210 contains the only Factor VIII construct that fits within the carrying capacity of an AAV capsid Expression cassette size comparison Key attributes of FLT210 5.2 Smallest disclosed liver specific promoter in development 5.1 Shortened FVIII gene to reduce expression cassette size Note: Hem A ca
Slide 12: Fabry & Gaucher disease
The Freeline mission: Fabry & Gaucher To be life changers disease
Slide 13: FLT190 demonstrates increased GLA expression and reduction in pathologic substrate in Fabry mouse model
FLT190 demonstrates increased GLA expression and reduction in pathologic substrate in key tissues in Fabry mouse model Kidney GLA activity levels Heart GLA activity levels 300 400 300 200 GLA in Kidney (nmol/hr/mg) (nmol/hr/mg) GLA in Heart 200 100 WORLD symposia 2019: Jey Jeyakumar et 100 al. Liver
Slide 14: FLT190 phase 1/2 dose-finding study poised for dose escalation
FLT190 phase 1/2 dose-finding study poised for dose escalation Adaptive study design Highest dose Higher doses 1.5e13 vg/kg Part I: Part II: Patients previously treated Previously untreated patients; with ERT or chaperone therapy use dose selected from Part I Higher dose 4.5e12 vg/kg Mid dose Mid do
Slide 15: Low doses of FLT201 in Gaucher mice result in higher expression and increased uptake in tissues
Low doses of FLT201 in Gaucher mice result in higher expression and increased uptake in tissues affected by Gaucher Type 1 disease • Novel GCase variant produced by FLT201 is more stable in plasma than wild-type protein • This leads to a greater than 20-fold increase in potency vs. wild-type protein
Slide 16: Gba-deficient mice data demonstrates GCase tissue penetration by FLT201, enzymatic activity & substrate clearance
Gba-deficient mice data demonstrates GCase tissue penetration by FLT201, enzymatic activity & substrate clearance • Restoration of GCase activity after FLT201 injection was observed in Gba-deficient mice in difficult-to-reach tissues as shown by decreased levels of lyso-GB1 substrate* • Dose-depende
Slide 17: Milestones
The Freeline mission: To be life changers Milestones
Slide 18: Multiple potential near-term value-creating milestones targeted for 2021
Multiple potential near-term value-creating milestones targeted for 2021 2021 Objectives Hemophilia B – Initiate clinical trial sites for the Phase 1/2 dose confirmation trial Hemophilia B – Present durability data up to 4 years from Phase 1/2 B-AMAZE dose-finding clinical trial Fabry – Progress dos
Slide 19: Thank you
Thank you
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