Corporate Overview. Dr Robert Scoffin CEO. http;// STAND NUMBER: 27

Transcription

1 Corporate Overview Dr Robert Scoffin CEO http;// STAND NUMBER: 27

2 Re-Pharm Summary > Focus on commercially valuable early stage assets. > Repositioned compounds > Using a combination of biological understanding and Cresset tools > Non Repositioned assets > Significant value added by appropriate pre-clinical development > Targeted development to advanced pre-clinical/early clinical > Combination of Cresset technology and a very experienced team > Deep re-profiling expertise > Proven track record (NVA237 licensed to Novartis) > Extensive computational chemistry and cheminformatics knowledge

3 Cresset Technology

4 Field Points Condensed representation of electrostatic, hydrophobic and shape properties ( protein s view ) > Molecular Field Extrema ( Field Points ) F F F N N H 2 N S O O Br 2D 3D Molecular Electrostatic Potential (MEP) Field Points = Positive = Negative = Shape = Hydrophobic

5 Biologically Relevant Molecular Comparisons Bioisosteres Bioisosteric groups

6 Cresset Software > Virtual screening platform > Massive cost reduction in wet screening campaigns > Library Design > HTS Rescue > Similarity matrices > Used to compare compounds for potential re-profiling/sideeffect prediction. > Applications in ADME/Tox as well as targeted re-profiling

7 Introduction

8 Reprofiling - Advantages > Greater knowledge of agent compared to classical NCE discovery > Existing toxicology, clinical safety, pharmacokinetics > Lower risk of failure > Faster development track > Create and maintain markets through new patents > Lowered R&D costs > Orphan indications > Better profitability > Commercially Valuable? > Examples of valuable re-profiled compounds > Nexium (single enantiomer Omeprazole) > Advair (combination of Salmeterol & Fluticasone) > Sildenafil: hypertension to ED > Raloxifene: Breast Cancer to Osteoporosis > Milnacipran: antidepressant to fibromyalgia > NVA 237 Inhaled Glycopyrronium Bromide for COPD developed by Arakis/Sosei and Vectura licensed to Novartis

9 How does Re-Pharm fit? Cost Target ID Pre-Clinical Development Early Clinical Phase 1 and POP Phase II Phase III Registrati on Marketing Re-Pharm Manageable costs Expertise in identifying reprofiling opportunities Smart identification of reduced risk assets Mid and Major Pharma Huge Resources Expertise in clinical development and approval

10 Management Team & Board > Rob Scoffin (CEO) Chemist by training with 23 years experience in developing businesses in the life sciences and drug discovery services market. > Alan Rothaul (CSO) Biologist by training. Over 30 years of drug company experience in major Pharma and biotech and founding VC based company, Serentis (raised 15M). Experienced in preclinical, clinical and regulatory areas. Has successfully been involved in drug reprofiling for~15 years and had a key involvement in the identification, development and licencing of NVA237 to Novartis. > Andy Vinter (Board) 40 years experience in pharmaceutical R&D, working with major companies and leading drug discovery groups, including Sir James Black at Smithkline & French. > David Bardsley (CBO) Microbiology background and 20 years of commercial experience in a variety of roles across the life sciences industry. An experienced management team and Board with an excellent external network

11 RP0217 NON-CONFIDENTIAL

12 RP0217 Discovery example of smart re-profiling > Identification of novel target > Virtual screening using known inhibitor as starting point > Start with database of known drugs (2,000 compounds) > Generation of long list of candidates for re-profiling (100 compounds) > Triage the long list by properties, drug-likeness, etc. (50 compounds) > Triage the long list by medicinal chemistry eyeballing (24 compounds) > Triage compounds by availability (6 compounds) > Screen using in vitro assay > Found RP0217 active at nm concentration 12

13 Demonstration that RP0217 synergises with Steroids Human Cord Blood Mast Cells Selection of minimally active Dexamethasone conc. for synergy study Selection of minimally active Dexamethasone concentration for synergy study Clear synergy with glucocorticoid ~100 fold left shift in D/R curve RP0217 alone RP nM Dex Clear anti-inflammatory activity at very low concentrations of RP0217 in combination with steroid (threshold ~5x10-11 RP x10-10 Dexamethasone) Indication of steroid sparing activity-attractive profile for ocular allergy/inflammation

14 RP0217 suitability as a reprofiled drug > Old drug (1960 s) > No S.O.M. patent complications > Unexpected and unreported activity for a clinically and commercially desirable target > Patentability, accepted target biology > Considerable clinical experience (>40 years) > Comprehensively understood and acceptable safety profile and pharmacokinetics for both adult and paediatric use by the oral route > A highly abridged development path - no need for systemic toxicology

15 IP and licensing > Two GB patents filed April 2013, two PCT applications field as follow-on April > RPN001/RPN003 > Narrow claims in ophthalmic inflammatory conditions RP0217 with or without steroids > RPN002/RPN004 > Broad claims in systemic and topical inflammatory indications RP0217 and related compounds, with or without steroids > Licensing > Option to licence RP0217 in separate indications > Topical eye (allergic conjuncto-rhinitis) > Topical lung (COPD, Asthma, IPF) > Topical to gut (IBS) > Would accept single licensee for all above > Could be enhanced by NCE possibilities as follow on > Adds systemic indications (Rheumatology, etc)

16 STAND NUMBER: 27 Dr Robert Scoffin Dr Alan Rothaul Chief Executive Officer Chief Scientific Officer Dr David Bardsley Chief Business Officer t: +44 (0)