Summit Therapeutics Inc. · NASDAQ:SMMT
Summit Therapeutics — Technical Presentation (January 2018)
17 slides from Summit Therapeutics's technical presentation, Jan 25, 2018
Slide 1: PhaseOut DMD: Review of Interim 24-Week Data
PhaseOut DMD: Review of Interim 24-Week Data January 25, 2018 Exhibit 99.2
Slide 2: Legal Disclaimer
Legal Disclaimer Statements in this presentation, other than statements of historical fact, constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include but are not limited to
Slide 3: Rare Diseases: Utrophin Modulation Program for DMD
Rare Diseases: Utrophin Modulation Program for DMD PhaseOut DMD Interim Data January 2018 Fatal Genetic disease caused by lack of dystrophin in muscle ~50,000 patients in US, EU and Japan (1) Ezutromid: a small molecule utrophin modulator in Phase 2 clinical trial in patients Collaboration with Oxfo
Slide 4: Utrophin Protein: Shares Structural Similarities with Dystrophin
Utrophin Protein: Shares Structural Similarities with Dystrophin PhaseOut DMD Interim Data January 2018 CRD CTD Actin Binding H1 H2 H4 H3 Rod domain Dystrophin protein Utrophin protein Source: Fairclough RJ, Wood MJ, Davies KE, Nat Rev Genet., 2013
Slide 5: Utrophin: Potential to Substitute for Dystrophin and Result in Less Severe Disease
Utrophin: Potential to Substitute for Dystrophin and Result in Less Severe Disease January 2018 PhaseOut DMD Interim Data
Slide 6: PhaseOut DMD: A Phase 2 Proof of Concept Trial of Ezutromid
PhaseOut DMD: A Phase 2 Proof of Concept Trial of Ezutromid PhaseOut DMD Interim Data January 2018 Design: Non-randomized, open label 48-week trial with extension phase Two formulations: F3 (suspension), F6 (powder) Subjects: 40 ambulatory DMD boys, fully enrolled 5-10 years old Able to walk >300m i
Slide 7: PhaseOut DMD: A Phase 2 Proof of Concept Trial of Ezutromid
Wk 48 PhaseOut DMD: A Phase 2 Proof of Concept Trial of Ezutromid PhaseOut DMD Interim Data January 2018 Cohort 1: 2.5 g BID F3 Formulation (n=30) Cohort 2: 1.0 g BID F6 Formulation (n=10) Optional Extension Phase Screening Optional Extension Phase Functional Tests D-28 Wk 12 MRI/MRS Wk 96+ Wk 72 Wk
Slide 8: Overview of the 24-Week Assessments
Overview of the 24-Week Assessments PhaseOut DMD Interim Data January 2018 Measure n Biopsy Assessments Developmental myosin (muscle damage) Utrophin expression Muscle fiber diameter (muscle maturity) 18-22 Magnetic resonance spectroscopy (MRS) 37 Functional Assessments: Six-minute walk distance Nor
Slide 9: Scientific Rigor Applied to Biopsy Analyses
Scientific Rigor Applied to Biopsy Analyses Automated muscle biopsy imaging removes human bias, ensures quality of measures Analytical validation followed strict international guidelines on validation of analytical procedures (ICH Q2(RI) 2005) Control, Becker muscular dystrophy and DMD biobank sampl
Slide 10: Significant Reduction in Muscle Damage after 24 Weeks of Ezutromid Treatment
Significant Reduction in Muscle Damage after 24 Weeks of Ezutromid Treatment PhaseOut DMD Interim Data January 2018 Developmental myosin marks areas of muscle damage/repair Number of developmental myosin positive fibers increased with disease severity in validation studies Baseline Week 24 0 = no re
Slide 11: 24 Weeks of Ezutromid Treatment Significantly Reduced Muscle Damage
24 Weeks of Ezutromid Treatment Significantly Reduced Muscle Damage PhaseOut DMD Interim Data January 2018 23% decrease in mean developmental myosin (MHCd) after 24 weeks 95% CI, -4.33, -0.90
Slide 12: 24 Weeks of Ezutromid Treatment Significantly Reduced Muscle Damage: Individual Patients
24 Weeks of Ezutromid Treatment Significantly Reduced Muscle Damage PhaseOut DMD Interim Data January 2018 Individual Patients
Slide 13: 24 Weeks of Ezutromid Treatment Increased Utrophin Intensity
24 Weeks of Ezutromid Treatment Increased Utrophin Intensity PhaseOut DMD Interim Data January 2018 Demonstrated 7% increase in mean utrophin intensity Baseline Week 24 Mean utrophin intensity 0.3 0.5 0.4 95% CI, -0.0052, 0.058
Slide 14: Additional 24-Week Findings in PhaseOut DMD
Additional 24-Week Findings in PhaseOut DMD Fat fraction was measured using MRS. Mean fat fraction in the vastus lateralis was 14.7% at baseline and 18.5% at 24 weeks (n=37). Longer term dosing of patients is expected to be required to detect changes. The mean six-minute walk distance was 404m at ba
Slide 15: Expect to See Positive Changes Across PhaseOut DMD Measurements Over Time
Expect to See Positive Changes Across PhaseOut DMD Measurements Over Time PhaseOut DMD Interim Data January 2018 Start of Dosing Utrophin engagement (biopsy) Muscle fiber damage (biopsy) Muscle health including fat fraction (MRI) Functional endpoints
Slide 16: Route to Market Strategy: Driven by Clinical Data
Route to Market Strategy: Driven by Clinical Data January 2018 PhaseOut DMD Interim Data Market PhaseOut DMD Proof of concept trial Pivotal Trial Data could support an accelerated or conditional approval pathway Market Positive 24-week data reported Top-line 48-week data expected to be reported Q3 2
Slide 17: Contact Details
Contact Details investors@summitplc.com Twitter: @summitplc 136a Eastern Avenue Milton Park Oxfordshire UK One Broadway Cambridge Massachusetts US January 2018 PhaseOut DMD Interim Data
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