Disease Modifying Therapy for DMD: Utrophin Modulation Programme

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1 Disease Modifying Therapy for DMD: Utrophin Modulation Programme PPMD Connect, June 2014

2 Legal Disclaimer FORWARD-LOOKING STATEMENTS This Document contains forward-looking statements. These statements relate to, among other things, analysis and other information that are based on forecasts of future results and estimates of amounts not yet determinable. These statements also relate to the Company s future prospects, developments and business strategies. Forward-looking statements are identified by their use of terms and phrases such as believe, could, envisage, estimate, expect, intend, may, plan, will or the negative of those, variations or comparable expressions, including references to assumptions. The forwardlooking statements in this Document are based on current expectations and are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by those statements. Given the risks and uncertainties associated with a company of this nature, potential investors should not place reliance on forward-looking statements. These forward-looking statements speak only as at the date of this Document. The Company does not undertake any obligation to update forward-looking statements or risk factors other than as required by any relevant regulations, whether as a result of new information, future events or otherwise. 2 PPMD Connect June 2014

3 Dystrophin or Utrophin Protein: Maintaining Integrity of Muscle Fibres Muscle fiber membrane Extracellular matrix Actin cytoskeleton Dystrophin or Utrophin Dystrophin or utrophin protein 3 PPMD Connect June 2014

4 Utrophin Modulation: A Disease-Modifying Approach for DMD Utrophin is functionally equivalent to dystrophin in both fetal and repairing muscle Utrophin is continually expressed at specialized sites in normal mature muscle fibers Normal Fiber Fetal (repairining) Time to Develop Fetal/ Immature Mature Utrophin modulation is a diseasemodifying strategy of potential utility for all DMD patients (independent of dystrophin mutation) DMD Fiber Degeneration Only normal utrophin levels required for DMD muscle recovery SMT C1100 designed to increase and maintain utrophin transcription DMD + SMT C1100 Fiber utrophin dystrophin 4 PPMD Connect June 2014

5 SMT C1100 Increased Utrophin Levels in Muscle Fibers Improves Disease Biomarkers In Vivo Reduced Fiber Regeneration (%CNFs) extensor digitorum longus tibialis anterior Reduced Muscle Leakiness (blood CK levels) mdx mdx + SMT C1100 Increased utrophin at the sarcolemma significantly reduces secondary disease effects 28 days of oral dosing (50mk/kg, QD) in mdx mice (n=5) Source: PLoS ONE, Vol 6, Issue 4, May 2011 mdx vehicle mdx+smt C PPMD Connect June 2014

6 UTROPHIN MODULATION PROGRAMME Clinical Data 6 PPMD Connect June 2014

7 Summary of Phase 1 Healthy Volunteer Trial Repeat dose Phase 1 healthy volunteer trial conducted in 2012 showed SMT C1100 was safe and well tolerated at all doses tested Achieved levels expected to increase utrophin expression for at least 14 hours/day Higher plasma levels achieved when SMT C1100 taken with food SMT C1100 taken with food v SMT C1100 taken after 12h fasting (same individuals after washout) 200mg/kg 7 PPMD Connect June 2014

8 Phase 1b Trial: Overview UK-based study 4 sites: London, Liverpool, Birmingham, Manchester 12 ambulatory DMD patients aged 5 to 11 years old 3 escalating dose cohorts, 4 patients per cohort: 50mg/kg BID, 100mg/kg BID, 100mg/kg TID 10 days of oral dosing Dosing to occur ideally within 10 minutes of consuming food Primary endpoint: Tolerability & Safety Secondary endpoint: PK levels of SMT C1100 and metabolites 8 PPMD Connect June 2014

9 Phase 1b Trial: Safety and PK First ever utrophin modulator trial in patients Safety: Primary endpoint of trial achieved SMT C1100 safe and well-tolerated in patients at all doses tested No apparent issues with patient compliance Variable Plasma levels of drug 2/12 boys had good SMT C1100 plasma levels appropriate for activating utrophin modulation 10/12 boys plasma levels were similar to fasted healthy adult volunteers 9 PPMD Connect June 2014

10 Phase 1b Trial: CK Observation Reduction in enzyme related to muscle damage Serum creatine kinase (CK) is an enzyme associated with muscle fibre damage and serum levels are elevated in DMD patients Reduced serum CK levels seen in non-clinical efficacy studies in mdx model after 15 days treatment with SMT C1100 Phase 1b data CK levels were reduced during dosing in the majority of boys Result is consistent with mdx data Consistent with reduction in muscle membrane damage 10 PPMD Connect June 2014

11 Next Steps: Targeting all Patients with DMD Future clinical trials of SMT C1100 to be modified to reflect our greater understanding of the importance of diet and other potential disease related factors Will seek to determine the optimal way to address drug exposure variability Dietary means and/or Drug formulation change Formulations already identified that increase exposure in animal studies Next patient study now expected to start in Q PPMD Connect June 2014

12 12 PPMD Connect June 2014

13 13 PPMD Connect June 2014

14 Alliance represents a multi-year strategic collaboration that combines extensive utrophin biology, chemistry and drug discovery expertise Commitment to build on the results obtained with SMT C1100 Aim to deliver first in-class and best in-class utrophin modulators for the therapy of DMD 14 PPMD Connect June 2014

15 Utrophin Modulation Programme Lead: SMT C1100 SMT C1100 related Next Generation DISCOVERY OPTIMISATION PRECLINICAL PHASE 1 PHASE 2 15 PPMD Connect June 2014

16 UTROPHIN MODULATION PROGRAMME Next Generation and Biomarkers 16 PPMD Connect June 2014

17 Next Generation Utrophin Modulators 1. Structurally Related to SMT C1100 Similar in vitro potency to SMT C1100, enhanced PK properties Clean safety/off target profile Rat In vivo PK Study 2. New structures, new mechanism New screening technology identifying differentiated utrophin modulators Potential new mechanism of action Increased in vitro activity compared to SMT C1100 Utrophin Levels in DMD patient Myoblasts 17 PPMD Connect June 2014

18 Biomarker Development Programme Utrophin / Mechanism Related UTRN localisation by quantitative IF UTRN mrna by QPCR UTRN protein by MS/MS Muscle Health From Muscle: Regeneration markers Inflammatory markers From Sera: Membrane damage (muscle enzymes, MicroRNAs) Active fibrosis (collagen fragments) Non-invasive Markers Muscle MRI 18 PPMD Connect June 2014

19 Thank You to the Community 19 PPMD Connect June 2014

20 Contact If you have further questions, do get in contact: Phone: +44 (0) Web: 20 PPMD Connect June 2014

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