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Silence Therapeutics Corporate Factsheet March 2012
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Silence Therapeutics Corporate Factsheet March 2012

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Silence Therapeutics Corporate Factsheet, March 2012, including: …

Silence Therapeutics Corporate Factsheet, March 2012, including:
> Pipeline of internal and partnered product candidates for RNAi therapeutics
> Partnerships and Collaborations
> Patented siRNA Delivery Technologies
> Structural Features of siRNA sequences
> Intellectual Property


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  • 1. Silence Therapeutics plc (AIM: SLN) is a leaderin the discovery, development and delivery ofRNA interference (RNAi) Therapeutics for thetreatment of serious diseases.Silence’s RNAi therapeutic platform comprises of proprietary delivery technologies, shortinterfering RNA (siRNA) sequences and innovative siRNA structural features that are critical Stock Information*in building, protecting and commercialising RNAi therapeutics -considered one of the most Ticker: AIM:SLNexciting and significant medial breakthroughs of recent years. Market Cap (GBP): £13.9million Market Cap (USD): $22.8millionAbout RNAi * as of 14 Feb, 2012RNAi is a natural cellular process that occurs in almost all organisms and results in thesilencing or shutting down of a target gene protein product. In an effort to combat awide range of diseases, therapeutics that exploit the RNAi process are being developed Corporate Highlightsto halt the production of disease-causing proteins. One of the sector’s most comprehensive RNAi platforms:Silence’s Pipeline I Unparalleled, proprietary delivery platformsSilence has developed a broad pipeline of internal and partnered I Potent siRNA sequencesproduct candidates. I Innovative siRNA structural featuresSilence’s technology serves as an important foundation in almost half of the siRNA programs inclinical development worldwide, demonstrating the company is a clear sector leader. Silence’s Robust clinical pipeline of siRNAimpressive pipeline of products not only validates the strength of the company’s technology but therapeutics:also the depth of knowledge that has guided the development path. I 5 clinical programs ongoing with partners and/or internallyProducts Partners Target Delivery Market Discovery Pre- PhaseI PhaseII PhaseIIb Tissue/ method size Clinical I Nearly half of the siRNA clinical programs Organ ($m) worldwide incorporate Silence’s technology RTP801PF-4523655 – Local Delivery to $1bn+ I Atu027 is one of most clinically advancedDiabetic Macular Naked sIRNA the Eye (potential) RNAi therapeutics in oncologyEdema I Indications include metabolic, pulmonaryPF-4523655 and vascular diseases, as well as cancer RTP801Age-related $3.1bn – Local Delivery Naked sIRNAMacular (2010) to the EyeDegeneration Validating partnershipsQPI-1002Prevention of P53 – Systematic $4.4bn with leading pharmaceutical Delivery to the Naked sIRNADelayed Graft Kidney (2010) companies:Function I Dainippon Sumitomo P53 – SystematicQPI-1002 Delivery to the $1bn+ I Pfizer/QuarkAcute Kidney Naked sIRNAInjury Kidney (potential) I Quark/Novartis I AstraZeneca PKN3 – Systematic $8.2bn+Atu027 Delivery to Tumor AtuPLEX (angiogenesisSolid Tumors Endothelium mkt 2010) Robust, global intellectual property portfolio providing a SystematicAtu111 $1bn+Acute Lung Injury Delivery to Lung DACC potential strong proprietary position: Endothelium I Issued US, EU and Japanese patents Systematic covering critical aspects of delivery,Liver diseases Delivery to Liver DBTC Undisclosed sequences and structures(DBTC programs) Hepatocytes and Endothelium P53 – SystematicLung diseases Delivery to the DACC Undisclosed(DACC system) Kidney 2012 GoalsSilence’s Partnerships I Complete Phase I clinical trial for Atu027 I Present final Phase I results on Atu027I Pfizer/Quark – Phase II products for diabetic macular edema and age-related macular I Advance preclinical programs for Liver & degeneration; $95M in milestones plus royalties Lung diseasesI Quark/Novartis (option) – Phase II study ongoing in kidney transplantation; Phase I study I Complete new corporate alliance complete in acute kidney injury; $82M in milestones plus royalties I Initiate Phase II trial of Atu027I Dainippon Sumitomo – siRNA delivery collaborationI AstraZeneca – $15m upfront; siRNA research and delivery collaboration (research phase now complete)
  • 2. RNAi Therapeutic Platform Silence’s siRNA Delivery Technologies Silences believes a single approach will not be sufficient to universally overcome the RNAi delivery Structural Features challenge. To achieve the goal of systemic delivery of RNAi therapeutics, Silence possesses a range of and IP complementary siRNA delivery technologies. AtuPLEX™ Delivery system  The AtuPLEX™ delivery system is Silence’s most advanced lipid based siRNA formulation. It is designed for a broad delivery to vascular endothelium and shows a high RNAi activity making it ideal for the development of anti-angiogenic/anti-vascular therapies.High-Value siRNA siRNA Delivery DACC Delivery systemSequences and IP Technologies and IP The DACC system represents a novel lipid-based formulation family which delivers siRNA efficiently to the vascular endothelium of the lung. Silence considers this delivery system as being exceptionally well suited to address lung-specific diseases e.g. acute lung injury/ARDS. The DACC system has demonstrated sustained gene silencing - a single dose is sufficient to inhibit target gene expression in Management Team lungs for up to a month. ANTONY SEDGWICK Chief Executive Officer DBTC Delivery system MAX HERRMANN Chief Financial Officer Delivery of siRNA to the liver has also been achieved by other delivery systems - but instead of merely targeting liver hepatocytes, Silence’s DBTC formulations target both liver hepatocytes and vascular KLAUS GIESE, Ph.D. endothelial cells of the liver. This unique feature opens up much broader therapeutic opportunities. Chief Scientific Officer GEORGE BUCHNER VP Business Development Successful siRNA delivery with Silence’s technology JÖRG KAUFFMAN VP Research & Development AtuRNAi™ Local and systemic delivery Phase II AtuPLEX™ Systemic delivery to endothelial cells in blood vessels Phase I RNAi Delivery Challenge DACC Systemic delivery to lung endothelial cells Preclinical The challenges of systemic RNAi DBTC Systemic delivery to hepatocytes Preclinical delivery are well documented and include numerous biological barriers that limit an RNAi therapeutic’s ability to reach its “target” safely and effectively. Silence’s Structural Features Silence structurally modifies its proprietary siRNA sequences to improve their efficacy and safety, while also offering an increased depth of patent protection. Two examples of Silence’s Delivery Solutions this work are: Silence’s sophisticated and flexible AtuRNAi™ delivery technologies provide Proprietary siRNA sequences containing the only naturally-occurring chemical modification the Company with unmatched (2’-O-methyl) to result in a stabilized molecule. potential to develop solutions for successfully delivering RNAi molecules to specific tissues thus Zamore “Design Rules” allowing specific therapeutic Technology in-licensed from UMass provides insight into methods for increasing siRNA benefits. This versatility positions potency and reducing off-target effects of its therapeutics. Silence to be a partner of choice for pharmaceutical and biotechnology companies currently working in, or seeking to enter, the RNAi field. Silence’s Intellectual Property Silence Therapeutics is executing a proactive strategy to continue to build and strengthen a global, diverse and competitive intellectual property portfolio that provides the Company and its partners with a strong proprietary position in the RNAi therapeutics space. The Company believes that it will continue to make significant progress in these efforts as it expects additional RNAi patents to be issued in Japan, the United States and Europe during 2012. At present, Silence’s global patent portfolio contains issued patents and pending applications covering strategic areas of RNAi therapeutic development including multiple proprietary siRNA delivery technologies, AtuRNAi (chemical modification, blunt ended), specific siRNA sequences and Zamore design rules. The Royal Institution of Great Britain, 21 Albemarle Street, London W1S 4BS, United Kingdom t: +44 (0) 20 7491 6520 www.silence-therapeutics.com e: london@silence-therapeutics.com