Silence Therapeutics PLC · NASDAQ:SLN
Silence Therapeutics — 37th Annual Canaccord Genuity Growth Conference 2017
17 slides from Silence Therapeutics's conference presentation, August 2017
Slide 1: Silencing the gene, Curing the patient
37th Annual Canaccord Genuity Growth Conference Silencing the gene, Curing the patient AUGUST 2017
Slide 2: Forward looking statements
Forward looking statements The information contained in this presentation is being supplied and communicated to you on a Securities in the Company have not been, and will not be, registered under the United States confidential basis solely for your information and may not be reproduced, further dist
Slide 3: About Silence Therapeutics
About Silence Therapeutics Only quoted European RNA Our interference player facilities Key Performance Indicators: HQ in > Net cash position of £34M as of 6th of London January 2017 > Operating cash burn of £10M in 2016 > Market cap: £96M > EV: £67M (including £11M Arrowhead) R&D in Berlin Headcount
Slide 4: Our business model
Our business model Monetising our Drug development Innovation intellectual property • Develop enabling technology • Generate and maintain • Develop new technology patent protection for our to improve the • Identify suitable target genes inventions and performance of current technology pipeline candi
Slide 5: How do our drugs work?
How do our drugs work? RNA interference is… > A Nobel prize-winning discovery > A natural pathway that can be harnessed to inhibit the expression of disease-causing genes without altering DNA We can specifically target any gene in the genome with our short interfering RNA (siRNA) molecules © Silence
Slide 6: siRNA delivery: a look back
siRNA delivery: a look back Our previous delivery platform: Lipid nanoparticles (LNP) • Suitable for larger cargos (mRNA, CRISPR) • Our LNP systems are able to mediate RNAi outside the liver, in endothelial cells • Intravenous administration (infusion) required • Dose and clinical applications limit
Slide 7: Our new platform technology: the delivery issue is now solved in the liver
Our new platform technology: the delivery issue is now solved in the liver + = siRNA: Targeting ligand: Our medicines: Inhibition of a specific Specific delivery of GalNAc-siRNA target gene siRNA to a target cell conjugates type • GalNAc can deliver therapeutic siRNA specifically to hepatocytes • Ga
Slide 8: Competitive GalNAc-siRNA performance observed in higher species
Competitive GalNAc-siRNA performance observed in higher species Day -7 0 4 7 14 ..weekly.. 91 GalNAc-siRNA Quantification of serum protein Target Y at indicated time points s.c., 1 and 0.3 mg/kg (n=4) 1 mg/kg Control 1 mg/kg Target Y 0.3 mg/kg Target Y > No altered pro- inflammatory relative to pred
Slide 9: What are the key properties of our RNAi medicines?
What are the key properties of our RNAi medicines? Rapid drug discovery process: Speed and no Known target mRNA sequence. Algorithmic bioinformatic design and fast screen for lead identification druggability issues Highly specific: Specificity Single target gene, single cell type affected Tailored f
Slide 10: How do we select target genes and indications for our pipeline?
How do we select target genes and indications for our pipeline? Key criteria Target nomination Not limited to hepatic applications > In-depth analysis and Hepatocyte KOL survey expression > Defined TPP criteria Validated role in disease Attractive clinical path Commercially We are also able to treat
Slide 11: Our pipeline
Our pipeline Our programmes Out-licensed programmes (AtuRNAiTM) © Silence Therapeutics 2017 11
Slide 12: Our Intellectual Property on chemically modified siRNA structures
Our Intellectual Property on chemically modified siRNA structures © Silence Therapeutics 2017 12
Slide 13: What are chemical modifications?
What are chemical modifications? • Chemical modifications are Unmodified siRNA required to increase stability and potency, and to reduce immune stimulation of RNAi drugs • AtuRNAiTM: our proprietary siRNA modification patterns. Key IP: issued patents in EU, US, Japan, Australia, Korea, etc • Out-lic
Slide 14: Field leading IP position in modified siRNA
Field leading IP position in modified siRNA Chemical modifications are vital to prevent the degradation of therapeutic siRNA molecules and to enable potent RNA interference. Silence was a pioneer in developing a robust chemical modification technology. > Our patent portfolio protecting our modificat
Slide 15: Recent patent filings
Recent patent filings In addition to our patent estate protecting our chemical modification technology, we are strengthening our GalNAc-siRNA IP portfolio in 2017 by filing: • Several lead sequence patent applications • 2 linker chemistry patent applications • 4 RNAi construct patent applications •
Slide 16: Why Silence?
Why Silence? © Silence Therapeutics 2017 16
Slide 17: Value inflexion points and upside risk
Value inflexion points and upside risk ü R&D progress: Internal GalNAc-siRNA pipeline progressed rapidly, aiming to reach clinical development in late 2018. R&D day planned in Q4 2017 ü Licensee clinical progress: Quark Pharmaceuticals has reported a positive AKI Phase 2 readout and Phase 3 DGF re
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