Slide 1: MOVE-FA Topline Results
MOVE-FA Topline Results Matthew B. Klein, MD CEO Patient Living with FA
Slide 2: Forward-Looking Statements
Forward-Looking Statements 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 statements wit
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 Primary Analysis Population: Placebo-Controlled Open-Label Extension 7-21 years old Overall Enrolled Population: Vatiquinone included patients over age 21 Vatiquinone R Placebo Primary Endpoint Change in mFA
Slide 4: mFARS Disease Rating Scale Measures Disease Progression Across Four Domains
mFARS Disease Rating Scale Measures Disease Progression Across Four Domains Bulbar Upright stability Lower limb Upper limb coordination coordination mFARS scores worsen (increase) on average by 2 to 3 points per year in pediatric patients based on natural history studies1 4 MOVE-FA Topline Results 1
Slide 5: Baseline Patient Characteristics: Primary Analysis & Overall Enrolled Populations
Baseline Patient Characteristics: Primary Analysis & Overall Enrolled Populations Primary Analysis Population Overall Enrolled Population Placebo Vatiquinone Total Placebo Vatiquinone Total n (%) n (%) n (%) n (%) n (%) n (%) Subject Number 62 61 123 73 70 143 Mean Age at Baseline [min,max] 14.3 [8,
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 Primary Analysis Population Overall Enrolled Population Change from Baseline to Week 72 Change from Baseline to Week 72 Analysis Placebo Vatiquinone Difference P-value Placebo Vatiqu
Slide 7: Vatiquinone Treatment Resulted in Meaningful Slowing of Disease Symptom Progression
Vatiquinone Treatment Resulted in Meaningful Slowing of Disease Symptom Progression Primary Analysis Population Overall Study Population p = 0.14 p = 0.098 p = 0.021 p = 0.025 p = 0.14 p = 0.082 p = 0.025 p = 0.0095 p = 0.21 p = 0.13 7 MOVE-FA Topline Results
Slide 8: Pre-Specified Completers Sensitivity Analysis
Pre-Specified Completers Sensitivity Analysis 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 study without treatment assignmen
Slide 9: Completer Sensitivity Analysis Population
Completer Sensitivity Analysis Population Primary Analysis Population Overall Enrolled Population Placebo Vatiquinone Total Placebo Vatiquinone Total n (%) n (%) n (%) n (%) n (%) n (%) Randomized 48 48 96 56 54 110 Mean Age at Baseline [min,max] 14.2 [8,20] 15.4 [9,21] 14.8 [8,21] 17.6 [8,68] 19.0
Slide 10: Vatiquinone Treatment Demonstrated Greater Magnitude of Effect on Disease Progression in Completers Sensitivity Analysis
Vatiquinone Treatment Demonstrated Greater Magnitude of Effect on Disease Progression in Completers Sensitivity Analysis Primary Analysis Population Completers Overall Enrolled Population Completers Change from Baseline to Week 72 Change from Baseline to Week 72 Analysis Placebo Vatiquinone Differen
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 Primary Analysis Population Completers -2 Placebo Change from Baseline in mFARS total score Vatiquinone -1 0 0.77 1 Treatment Difference 2 2.31 Points 3 3.08 at Week 72 4 Baseline Week 12 Week 24 We
Slide 12: Vatiquinone Demonstrated to Be Well Tolerated
Vatiquinone Demonstrated to Be Well Tolerated Similar Adverse Event Profile Between Vatiquinone and Placebo Subjects Most Common Treatment-Related Adverse Events Were GI Symptoms MOVE-FA Safety Profile Consistent with Other Vatiquinone Pediatric Studies 12 MOVE-FA Topline Results
Slide 13: Overview of Treatment-Emergent Adverse Events in Overall Study Population
Overview of Treatment-Emergent Adverse Events in Overall Study Population Category Placebo Vatiquinone (N=73) N (%) (N=73) N (%) Subjects with at least one TEAE 73 (100) 71 (97) Subjects with TEAEs by maximum severity Mild 31 (42.5) 26 (35.6) Moderate 32 (43.8) 37 (50.7) Severe 9 (12.3) 7 (9.6) Life
Slide 14: MOVE-FA Results Support Discussions With Regulatory Authorities
MOVE-FA Results Support Discussions With Regulatory Authorities Evidence of meaningful High unmet need Discuss potential path clinical benefit on key for pediatric to registration with aspects of FA disease FA patients regulatory authorities 14 MOVE-FA Topline Results
Slide 15: Strategic Portfolio Prioritization
Strategic Portfolio Prioritization • Discontinuation of preclinical and early research programs in gene therapy • Reduction in workforce related to deprioritized programs and SG&A • Estimated reductions in residual 2023 OPEX of approximately 15% • Updated 2023 full-year OPEX to be shared at Q2 earni