Silence Therapeutics PLC · NASDAQ:SLN
Silence Therapeutics — H1 2018 Earnings Presentation
13 slides from Silence Therapeutics's earnings presentation, September 2018
Slide 1: Silence Therapeutics Plc June 2018 Interim Results
Silence Therapeutics Plc June 2018 Interim Results Dr David Horn Solomon, CEO David Ellam, CFO A new era in RNAi therapeutics © Silence Therapeutics 2018 1
Slide 2: Silence: Operational Highlights
Silence: Operational Highlights • New leadership in place with the recruitment of Dr David Horn Solomon as Chief Executive Officer, an experienced public company biotech CEO, board member and biotech investor. • The field is advancing: Gene-silencing as a therapeutic modality was granted its first d
Slide 3: Driving the Business Forward
Driving the Business Forward • Primary emphasis is on advancing our proprietary pipeline of valuable RNAi-derived assets to build significant shareholder value and to create innovative products for patients and their caregivers. • Building expertise around Hematology and Iron Metabolism Disorders. •
Slide 4: Evolution of our GalNAc-siRNA platform
Evolution of our GalNAc-siRNA platform • Patents filed • Patents filed • Clinical stage in 2019 (SLN124) and used • Further optimisation ongoing in late pre-clinical stage programmes • Plans to enter the pipeline in early 2019 (SLN226, CVD) • Advantages: improved potency, COG and • Robust performanc
Slide 5: Market opportunity for SLN124 (US and Europe only)
Market opportunity for SLN124 (US and Europe only) • β-Thalassemia intermedia & T. major (TDT) ‒ Combination with transfusions & chelators to reduce frequency & dose ‒ Improve erythropoiesis and reduce 40,000 20,000 secondary iron overload burden TDT NTDT • β-Thalassemia intermedia (NTDT) ‒ Monother
Slide 6: SLN124 mechanism of action: increasing Hepcidin by silencing its repressor TMPRSS6
SLN124 mechanism of action: increasing Hepcidin by silencing its repressor TMPRSS6 HJV BMPR Macrophages • TMPRSS6 (Transmembrane BMP Protease, Serine 6) is a negative regulator of the Ferroportin BMP/SMAD signaling TMPRSS6 P Fe pathway Iron recycling • Inhibition of TMPRSS6 in P SMAD hepatocytes ind
Slide 7: Therapeutic activity of SLN124 in a disease model of Hereditary Hemochromatosis type 1
Therapeutic activity of SLN124 in a disease model of Hereditary Hemochromatosis type 1 (iron overload) Study design Collaboration with d1 wk 3 Hfe-/- mice Prof. Dr. Martina Muckenthaler 1X sc, HFE-/- mice, females, 6 wks, n=6-7 Heidelberg University, Germany TMPRSS6 mRNA Hepcidin (Serum) Kidney Iron
Slide 8: SLN124 improves RBC maturation, anaemia and normalises spleen size in β-Thalassaemia model
SLN124 improves RBC maturation, anaemia and normalises spleen size in a murine model of β−Thalassaemia intermedia Collaboration with Dr. J. Vadolas & Dr. G. Grigoriadis. Monash Medical Centre/Melbourne, Australia Hbbth3/+ mice Study design d1 wk 2 wk 5 sc,Hbbth3/+ & wt mice, n=5-8 SLN124 treatment s
Slide 9: SLN124 Summary
SLN124 Summary • Preclinical package: Robust data generated in several disease models • Status: Non-clinical development work ongoing, with CTA filing expected in Q2 2019 • Clinical plans: Phase 1b planned in patients (β−Thalassaemia and MDS). Network of KOL’s established • Dosing regime: Patient-fr
Slide 10: H1 Financial Report 2018
Silence Therapeutics Plc David Ellam, CFO H1 Financial Report 2018 © Silence Therapeutics 2018 10
Slide 11: Increase in Operating Expenses to drive platform and indications
Increase in Operating Expenses to drive platform and indications 2018 2017 2017 Income statement (GBP '000) H1 H1 Full year Revenue 0 16 16 Research and development costs ¹ (5,212) (3,817) (7,943) General & Administration expenses² (4,681) (3,021) (6,464) Operating loss (9,893) (6,822) (14,391) Othe
Slide 12: Silence has >12 months cash to continue to advance the business
Silence has >12 months cash to continue to advance the business Cash, cash equivalents & R&D and G&A Expenses @ 30 term deposits of £34.3M at June each year 30 June 2018 ($45.1M) Cash @30 June each year £M 60 R&D and G&A Expenses £M 50 12.0 10.0 40 8.0 30 6.0 20 4.0 10 2.0 0.0 0 2014 2015 2016 2017
Slide 13: Expected newsflow outlook & milestones next 15 months
Expected newsflow outlook & milestones next 15 months Q4 2018 H1 2019 H2 2019 SLN124 CTA enabling toxicology File CTA First In Human dosing CV disease pPoC in NHP 2018 new Targets POM data available pPOC in mice Final candidate nomination Alcohol Use Disorder CTA enabling toxicology