Slide 1: MOVE-FA Topline Results
Patient Living with FA Matthew B. Klein, MD CEO MOVE - FA Topline Results
Slide 2: Forward-Looking Statements
Forward - Looking Statements 2 This presentation contains forward - looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 . All statements contained in this presentation, other than statements of historic fact, are forward - looking statements, including state
Slide 3: MOVE-FA Is a Global Registration-Directed Trial of Vatiquinone in Friedreich Ataxia Patients
MOVE - FA Is a Global Registration - Directed Trial of Vatiquinone in Friedreich Ataxia Patients MOVE - FA Topline Results 3 Primary Endpoint Change in mFARS Score Open - Label Extension Placebo - Controlled Vatiquinone Placebo 72 weeks R 24 weeks Vatiquinone Primary Endpoint Primary Analysis Popula
Slide 4: mFARS Disease Rating Scale Measures Disease Progression Across Four Domains
mFARS Disease Rating Scale Measures Disease Progression Across Four Domains MOVE - FA Topline Results 4 mFARS scores worsen (increase) on average by 2 to 3 points per year in pediatric patients based on natural history studies 1 Bulbar Upright stability Lower limb coordination Upper limb coordinatio
Slide 5: Baseline Patient Characteristics: Primary Analysis & Overall Enrolled Populations
Baseline Patient Characteristics: Primary Analysis & Overall Enrolled Populations 5 Primary Analysis Population Overall Enrolled Population Placebo n (%) Vatiquinone n (%) Total n (%) Placebo n (%) Vatiquinone n (%) Total n (%) Subject Number 62 61 123 73 70 143 Mean Age at Baseline [min,max] 14.3 [
Slide 6: Vatiquinone Treatment Demonstrated Slowing of Disease Progression on mFARS with Nominal Significance in Key Subscales
Vatiquinone Treatment Demonstrated Slowing of Disease Progression on mFARS with Nominal Significance in Key Subscales MOVE - FA Topline Results 6 Primary Analysis Population Change from Baseline to Week 72 Overall Enrolled Population Change from Baseline to Week 72 Analysis Placebo Vatiquinone Diffe
Slide 7: Vatiquinone Treatment Resulted in Meaningful Slowing of Disease Symptom Progression
Overall Study Population p = 0.098 p = 0.025 p = 0.082 p = 0.0095 p = 0.13 Vatiquinone Treatment Resulted in Meaningful Slowing of Disease Symptom Progression MOVE - FA Topline Results 7 Primary Analysis Population p = 0.14 p = 0.021 p = 0.14 p = 0.025 p = 0.21
Slide 8: Pre-Specified Completers Sensitivity Analysis
Pre - Specified Completers Sensitivity Analysis MOVE - FA Topline Results 8 As the study was conducted during the COVID - 19 pandemic, a pre - specified sensitivity analysis was included for subjects that completed the study protocol on assigned treatment The total number of subjects completing the
Slide 9: Completer Sensitivity Analysis Population
Completer Sensitivity Analysis Population MOVE - FA Topline Results 9 Primary Analysis Population Overall Enrolled Population Placebo n (%) Vatiquinone n (%) Total n (%) Placebo n (%) Vatiquinone n (%) Total n (%) Randomized 48 48 96 56 54 110 Mean Age at Baseline [min,max] 14.2 [8,20] 15.4 [9,21] 1
Slide 10: Vatiquinone Treatment Demonstrated Greater Magnitude of Effect on Disease Progression in Completers Sensitivity Analysis
Vatiquinone Treatment Demonstrated Greater Magnitudeof Effect on Disease Progression in Completers Sensitivity Analysis MOVE - FA Topline Results 10 Primary Analysis Population Completers Change from Baseline to Week 72 Overall Enrolled Population Completers Change from Baseline to Week 72 Analysis
Slide 11: Vatiquinone Treatment Slowed Disease Progression by 75% in Completers Sensitivity Analysis at Week 72
Vatiquinone Treatment Slowed Disease Progression by 75% in Completers Sensitivity Analysis at Week 72 MOVE - FA Topline Results 11 Change from Baseline in mFARS total score Placebo Vatiquinone -2 -1 0 1 2 3 4 Week 60 Week 24 Week 36 Baseline Week 72 Week 48 Week 12 Treatment Difference 2.31 Points a
Slide 12: Vatiquinone Demonstrated to Be Well Tolerated
Vatiquinone Demonstrated to Be Well Tolerated MOVE - FA Topline Results 12 MOVE - FA Safety Profile Consistent with Other Vatiquinone Pediatric Studies Similar Adverse Event Profile Between Vatiquinone and Placebo Subjects Most Common Treatment - Related Adverse Events Were GI Symptoms
Slide 13: Overview of Treatment-Emergent Adverse Events in Overall Study Population
Overview of Treatment - Emergent Adverse Events in Overall Study Population MOVE - FA Topline Results 13 Category Placebo (N=73) N (%) Vatiquinone (N=73) N (%) Subjects with at least one TEAE 73 (100) 71 (97) Subjects with TEAEs by maximum severity Mild Moderate Severe Life - Threatening/Fatal 31 (4
Slide 14: MOVE-FA Results Support Discussions With Regulatory Authorities
MOVE - FA Topline Results 14 MOVE - FA Results Support Discussions With Regulatory Authorities Evidence of meaningful clinical benefit on key aspects of FA disease High unmet need for pediatric FA patients Discuss potential path to registration with regulatory authorities