Roche Holding Ltd · SIX:RO
Roche — Investor Day (April 2025)
73 slides from Roche's investor day presentation, Apr 4, 2025
Slide 1: Neurology/ADPD 2025 Virtual IR Event
Neurology/ADPD 2025 Virtual IR Event April 4th 2025
Slide 2: Forward-looking statements
This presentation contains certain forward-looking statements. These forward-looking statements may be identified by words such as ‘believes’, ‘expects’, ‘anticipates’, ‘projects’, ‘intends’, ‘should’, ‘seeks’, ‘estimates’, ‘future’ or similar expressions or by discussion of, among other things, str
Slide 3: Welcome
Welcome Bruno Eschli Head of Investor Relations
Slide 4: Agenda
Agenda Welcome Bruno Eschli, Head of Investor Relations Neurology Update Azad Bonni, SVP and Global Head of Neuroscience & Rare Diseases, Roche Pharma Research & Early Development Prasinezumab in Parkinson’s disease: PhIIb PADOVA data Azad Bonni, SVP and Global Head of Neuroscience & Rare Diseases,
Slide 5: Roche is #1 in Neurology
Neurology Roche is #1 in Neurology Neurology products account for ~20% of 2024 Pharma sales ▪ Market leader in US/EU5 ▪ Ocrevus: #1 new to brand in US Ocrevus: ▪ Zunovo with >2,500 patients ww and >50% US starts are naive to Ocrevus FY 2024 sales 6.7 CHF bn ▪ HD Ocrevus: Ph3 RMS study (MUSETTE) did
Slide 6: Neurology diagnostics: Integrated solutions along the patient journey
Core Lab Neurology diagnostics: Integrated solutions along the patient journey Novel tests drive access to therapies and support disease management Screening and triage Diagnosis Treatment Disease monitoring Elecsys® pTau181 (rule out)* NPV>90% Elecsys® pTau181 / Aβ42 Development ongoing, filing exp
Slide 7: Identifying amyloid pathology is critical for early AD diagnosis
Core Lab Identifying amyloid pathology is critical for early AD diagnosis New study results presented at ADPD 2025 Strong rule-out performance (NPV>90%) in a prospective multicenter study Estimate for combined plasma Performance measure, % (95% CI) Estimate for plasma p-Tau181 p-Tau181 and ApoE4 Per
Slide 8: 2025: Significant key newsflow ahead
Neurology 2025: Significant key newsflow ahead* Compound Indication Milestone Itovebi + palbociclib + fulvestrant 1L PIK3CA-mut HR+ BC EU approval Columvi + GemOx 2L+ DLBCL US/EU approval Lunsumio SC 3L+ FL US approval/EU filing (EU filing) Elevidys DMD EU approval Regulatory Gazyva Lupus nephritis
Slide 9: IR events currently planned for 2025
Neurology IR events currently planned for 2025 Additional events driven by readouts Immunology/WCN Update on NGS Neurology Update Diagnostics Day Hematology Update Pharma Day 7 Feb 20 Feb 4 Apr 27 May 23 June 22 Sep 16:30 – 17:30 CET 20:30 - 21:30 CET 16:00 – 17:30 CEST 14:00 - 16:30 CEST 19:00-20:1
Slide 10: Neurology Update
Neurology Update Azad Bonni SVP and Global Head of Neuroscience & Rare Diseases, Roche Pharma Research & Early Development
Slide 11: Neurology R&D focus areas
Neurology Neurology R&D focus areas Preserving what makes people who they are Multiple sclerosis Neuromuscular disorders Neurodegenerative diseases Stop, reverse and prevent MS and Create a future of strength and Slow progression of Parkinson’s and preserve quality of life independence in NMD Alzhei
Slide 12: Neurology R&D focus areas
Neurology Neurology R&D focus areas Preserving what makes people who they are Multiple sclerosis Neuromuscular disorders Neurodegenerative diseases Stop, reverse and prevent MS and Create a future of strength and Slow progression of Parkinson’s and preserve quality of life independence in NMD Alzhei
Slide 13: Neurology pipeline
Neurology Neurology pipeline Industry leading portfolio differentiated on targets and platform technologies Ph I Ph II Ph III Registration Brainshuttle CD20 prasinezumab Enspryng Elevidys1 RG6035 RG7935 RG6168 RG6356 Multiple Sclerosis Parkinson’s MOG-AD RD DMD ** RD MAGLi trontinemab Enspryng RG618
Slide 14: Fenebrutinib: Rapid and sustained suppression of MS disease activity
Neurology Fenebrutinib: Rapid and sustained suppression of MS disease activity Compelling RMS data at weeks 12 and 48; 96-week data to be presented in 2025 Ph II (FENopta): New T1 Gd+ lesions1 and ARR reduction up to week 482 Total new T1 Gd+ lesions* CSF concentration ARR from Double-blind period a
Slide 15: RG6289, a FIC oral γ-secretase modulator in Alzheimer's disease
Neurology RG6289, a FIC oral γ-secretase modulator in Alzheimer’s disease Targeting amyloid precursor protein processing to prevent Aβ-aggregation GSMs reduce Aβ aggregation1 Ph I dose escalation results for RG62892 Ph II (GABriella) study design3 Mean change from baseline (%) Mean change from basel
Slide 16: Selnoflast (NLRP3i) is a potent inhibitor of the NLRP3 inflammasome
Neurology Selnoflast (NLRP3i) is a potent inhibitor of the NLRP3 inflammasome Potential to reduce pro-inflammatory responses in Parkinson’s and other diseases Selnoflast (NLRP3 inflammasome inhibitor) Development program Parkinson’s TA Indication Ph I Ph II Ph III Status Disease Inflammatory markers
Slide 17: Prasinezumab Ph2b PADOVA data in Parkinson's disease
Prasinezumab Ph2b PADOVA data in Parkinson’s disease Azad Bonni SVP and Global Head of Neuroscience & Rare Diseases, Roche Pharma Research & Early Development
Slide 18: Parkinson's: Progressive neurodegenerative disease
Neurology Parkinson’s: Progressive neurodegenerative disease No disease-modifying treatment available today Loss of dopaminergic neurons starts many years before Prevalence increases with age, significant economic onset of symptoms1 burden to people and HC systems Neurodegeneration ▪ PD affects ~1%
Slide 19: Prasinezumab is a mAb selectively binding aggregated α-synuclein
Neurology Prasinezumab is a mAb selectively binding aggregated α-synuclein Current symptomatic treatments only address motor symptoms, but do not affect PD progression Prasinezumab: First potential disease Ph II (PASADENA): Prasinezumab-treated individuals progress less modifying therapy in PD1, 2 t
Slide 20: PADOVA: Topline results from a Phase IIb study of prasinezumab
PADOVA: Topline results from a Phase IIb study of prasinezumab in early-stage Parkinson’s disease participants on stable symptomatic treatment Tania Nikolcheva,1 Gennaro Pagano,2,3 Judith Anzures-Cabrera,4 Tanya Simuni,5 Kenneth Marek,6 Nicola Pavese,7 Klaus Seppi,8 Fabrizio Stocchi,9 Ronald B. Post
Slide 21: Global Phase IIb PADOVA study design
Global Phase IIb PADOVA study design Novel time-to-event approach allows measuring treatment effect on top of standard-of-care symptomatic therapies 110 sites in Austria, Canada, France, Italy, Luxembourg, Poland, Spain, UK and US Key inclusion criteria Stratification Idiopathic PD L-DOPA or MAO-Bi
Slide 22: Baseline characteristics well balanced across arms
Baseline characteristics well balanced across arms Population consistent with early-stage PD; 74% of participants on L-DOPA Key baseline demographic and disease Placebo Prasinezumab All characteristics n=293 n=293 N=586 Age (yr) mean (SD) 64.4 (7.5) ` 64.2 (7.3) Sex, male n (%) 189 (64.5%) 183 (62.5
Slide 23: Primary endpoint: overall population
Primary endpoint Overall Prasinezumab showed a trend towards delaying motor progression Primary endpoint: Study missed statistical significance Time to ≥5 points on MDS-UPDRS Part III OFF Median n n events (weeks) 95% CI Placebo Prasinezumab 293 200 61.1 52.3, 71.9 100 Prasinezumab 90 Placebo 293 22
Slide 24: Primary endpoint: L-DOPA subgroup
Primary endpoint L-DOPA Prasinezumab showed a trend towards delaying motor progression Primary endpoint: Trend more pronounced in prespecified L-DOPA subgroup Time to ≥5 points on MDS-UPDRS Part III OFF Median n n events (weeks) 95% CI Placebo Prasinezumab 218 145 64.4 55.3, 81.1 100 Prasinezumab 90
Slide 25: Secondary endpoint: overall population
Secondary endpoint Overall Prasinezumab showed a trend towards delaying the decline of function Time to ≥3 points on MDS-UPDRS Part II in the presence of Median n n events (weeks) 95% CI a confirmed motor event Prasinezumab 293 142 112.1 81.1, NE Placebo 100 Prasinezumab 90 Placebo 293 163 88.3 62.7
Slide 26: Consistent trends in favour of prasinezumab across TTE endpoints
Primary and secondary endpoints Overall Consistent trends in favour of prasinezumab across TTE endpoints Forest plot of primary and secondary TTE endpoints Time to ≥5 point in MDS UPDRS Part III OFF Primary endpoint (confirmed) ≥3 points in MDS-UPDRS Part II in the presence of confirmed motor event
Slide 27: No effect on motor progression at 76 weeks
Secondary endpoint Overall Prasinezumab showed no effect on motor progression at 76 weeks Change from baseline to Week 76 on MDS-UPDRS Part III OFF 6 5 Difference in adjusted means (SE) change from baseline 4 –0.39 (0.74); 95% CI (–1.84 to 1.05) Adjusted mean 3 No difference vs placebo p=0.5994* 2 1
Slide 28: Trend towards reduction of motor progression at 104 weeks
Exploratory endpoint Overall Prasinezumab showed a trend towards reduction of motor progression at 104 weeks Treatment duration longer than 18 months may be needed to measure treatment effect Change from baseline to Week 104 on MDS-UPDRS Part III OFF 7 n=326 (56%) with double-blind data at 104 weeks
Slide 29: Motor progression at 104 weeks in L-DOPA-treated population
Exploratory endpoint L-DOPA Prasinezumab showed a trend towards reduction of motor progression at 104 weeks in the L-DOPA-treated population Treatment duration longer than 18 months may be needed to measure treatment effect Change from baseline to Week 104 on MDS-UPDRS Part III OFF 7 n=242 (56%) wit
Slide 30: Trend towards reduction in neuromelanin loss
Exploratory endpoint Prasinezumab showed a trend towards reduction in neuromelanin loss in substantia nigra Change from baseline to Week 76 in Neuromelanin MRI* Substantia nigra pars compacta intensity Substantia nigra pars compacta volume 0.4 p=0.079 p=0.1566 0.18 1.27 2.5 (0.185) (3.144) change fr
Slide 31: Trend towards reduction in iron accumulation
Exploratory endpoint Prasinezumab showed a trend towards reduction in iron accumulation in putamen and substantia nigra Change from baseline to Week 76 in Iron R2* MRI# Iron (R2*) putamen (Average) Iron (R2*) substantia nigra pars compacta (Average) 0.43 p=0.0025 0.50 p=0.1903 (0.197) 0.47 change fr
Slide 32: PADOVA: potential clinical benefit of prasinezumab
PADOVA: potential clinical benefit of prasinezumab in early-stage Parkinson’s disease on top of symptomatic treatment While primary endpoint missed, TTE analyses showed trends towards delaying motor progression ▪ More pronounced in L-DOPA treated population ▪ Consistent trends across secondary endpo
Slide 33: Acknowledgements
Acknowledgements We thank all the study participants and their families, and investigators and site staff, for their time and commitment to the prasinezumab clinical development programme 33
Slide 34: Trontinemab ADPD 2025 data
Trontinemab ADPD 2025 data Luka Kulic Vice President and Global Head of Early Development, Neuroscience and Rare Diseases, Roche Pharma Research & Early Development
Slide 35: Importance of substantial amyloid removal with anti-Aß therapies
Neurology Importance of substantial amyloid removal with anti-Aß therapies Improving efficacy, safety and brain penetrance are key considerations for new AD therapies Amyloid reduction in AD clinical trials1 Unmet needs for next gen Tx Faster/deeper amyloid clearance Prevention of new amyloid accumu
Slide 36: Trontinemab - a novel Brainshuttle bispecific 2+1 mAb targeting Aβ
Neurology Trontinemab - a novel BrainshuttleTM bispecific 2+1 mAb targeting Aβ Active transport across the BBB significantly increases brain penetration and target engagement Trontinemab Endothelial cell Microglia (brain capillary) Anti-Aβ binder IgG1 Fc domain Transferrin Receptor 1 (TfR1) Brainshu
Slide 37: Trontinemab - from preclinical groundwork to clinical validation of Brainshuttle
TRONTINEMAB - FROM EARLY PRECLINICAL GROUNDWORK TO THE CLINICAL VALIDATION OF THE BRAINSHUTTLETM PLATFORM Luka Kulic1, Fabien Alcaraz1, Gregory Klein1, Carsten Hofmann2, Stella Yilmaz2, João A. Abrantes2, https://ter.li/x3m6mw Denise Sickert2, Maddalena Marchesi3, Jakub Wojtowicz3, Ruth Croney4, Dav
Slide 38: Brainshuttle AD study design
BrainshuttleTM AD study design Phase Ib/IIa study assessing the safety, tolerability, PK and PD of trontinemab in participants with AD1 Dose Escalation (Part 1): Dose Expansion (Part 2) PK/PD (Part 3) n=14-16 per cohort n≈60 per cohort n≈40 (total) Cohort 1 0.2 mg/kg or Pbo (4:1) IV 7X Q4W ⮚ Assess
Slide 39: Brainshuttle AD study design
BrainshuttleTM AD study design Phase Ib/IIa study assessing the safety, tolerability, PK and PD of trontinemab in participants with AD1 Dose Escalation (Part 1): Dose Expansion (Part 2) PK/PD (Part 3) n=14-16 per cohort n≈60 per cohort n≈40 (total) Cohort 1 0.2 mg/kg or Pbo (4:1) IV 7X Q4W ⮚ Assess
Slide 40: Baseline characteristics at latest interim analysis
Baseline characteristics at latest interim analysis1 Population: 114 participants in cohort 3+4 who had the opportunity to complete treatment period PART 1 PART 2 PART 1 + 2 (COMBINED) (n = 32) (n = 82) ( n = 114) Cohort 4 Cohort 3 Cohort 4 Cohort 3 Cohort 3 Cohort 4 Baseline demographic and disease
Slide 41: Amyloid PET results in Part 1 (previously reported)
Amyloid PET results in Part 1 (previously reported) Rapid and robust amyloid PET reduction at 1.8 mg/kg and 3.6 mg/kg Cut-off date: 2 September 2024. Mean values ±SE (standard errors) of available PET results at the different visit time points are plotted. CL, Centiloid units. Florbetapir or florbet
Slide 42: Amyloid PET schedule in Part 2
Amyloid PET schedule in Part 2 Randomization of participants to three different intermediate PET time points Amyloid PET Scan Dose 1 Dose 2 Dose 3 Dose 4 Dose 5 Dose 6 Dose 7 Screening Part 4 (OLE) 0 4 8 12 16 20 24 28 weeks BL D50 D196 Randomization 1:1:1 BL D78 D196 BL D106 D196 BL, baseline; D, d
Slide 43: Amyloid lowering in the different PET randomization groups in Part 2
Amyloid lowering in the different PET randomization groups in Part 2 Preliminary results suggest earlier and deeper amyloid depletion at 3.6 mg/kg compared to 1.8 mg/kg Cut-off date: 14 November 2024. Mean values ±SE (standard errors) of available PET results at the different visit time points are p
Slide 44: Adjusted mean change from baseline in Part 1 + 2 (combined)
Adjusted mean change from baseline in Part 1 + 2 (combined)1 Rapid amyloid depletion at 3.6 mg/kg trontinemab (81% amyloid PET negative at 28 weeks) Participants 1.8 mg/kg 3.6 mg/kg ≤24 CL (%) (Part 1+2) (Part 1+2) BL 0/61 (0%) 0/31 (0%) D50 1/12 (8%) 1/6 (17%) D78 12/29 (41%) 11/19 (58%) D106 4/15
Slide 45: Deep amyloid depletion at 3.6 mg/kg trontinemab at 28 weeks
Deep amyloid depletion at 3.6 mg/kg trontinemab at 28 weeks Nearly all participants near or below amyloid positivity threshold (Part 1 + 2 combined) Amyloid PET Minimum -47 CL change from baseline 3.6 mg/kg trontinemab for 3.6 mg/kg @ D196 (Week 28) No amyloid reduction “non-responders” 24 CL n=31 n
Slide 46: Preliminary CSF biomarker results at 25 weeks
Preliminary CSF biomarker results at 25 weeks (Part 1 + 2 combined) Early and pronounced effects of trontinemab on key downstream biomarkers of disease in CSF n = 17 n = 46 n = 23 n = 18 n = 45 n = 26 Cut-off date: 14 November 2024. Annotated percentages show the median percent change from baseline
Slide 47: Preliminary CSF biomarker results at 25 weeks
Preliminary CSF biomarker results at 25 weeks (Part 1 + 2 combined) Early and pronounced effects of trontinemab on key downstream biomarkers of disease in CSF n = 17 n = 47 n = 24 n = 18 n = 46 n = 25 Cut-off date: 14 November 2024. Annotated percentages show the median percent change from baseline
Slide 48: Preliminary plasma biomarker results (Part 1 + 2 combined)
Preliminary plasma biomarker results (Part 1 + 2 combined) >50% reduction in plasma pTau217 at 3.6 mg/kg after 6 months of treatment ↓ 36% ↓ 51% n at 13 16 13 19 39 54 43 48 16 27 16 25 n at 12 16 11 18 38 54 39 47 16 27 16 28 visit visit Cut-off date: 14 November 2024. Plasma samples were measured
Slide 49: Blinded safety profile
Blinded safety profile1 Number of participants with safety events or study treatment discontinuations due to AE PART 1 PART 2 PART 1 + 2 (COMBINED) (n = 32) (n = 82) (n = 114) Cohort 3 Cohort 4 Cohort 3 Cohort 4 Cohort 3 Cohort 4 Total number of participants 1.8 mg/kg or Pbo 3.6 mg/kg or Pbo 1.8 mg/
Slide 50: Sustained low ARIA incidence with trontinemab
Sustained low ARIA incidence with trontinemab 3 ARIA-E cases in cohort 3 (1.8 mg/kg or Pbo), none in cohort 4 (3.6 mg/kg or Pbo) as of cut-off date PART 1 PART 2 PART 1 + 2 (COMBINED) (n = 32) (n = 82) (n = 114) Cohort 3 Cohort 4 Cohort 3 Cohort 4 Cohort 3 Cohort 4 Total number of participants 1.8 m
Slide 51: Infusion related reactions (IRRs) and transient anemia
Infusion related reactions (IRRs) and transient anemia Mild to moderate IRRs are common, but manageable AEs; limited anemia, not dose-dependent PART 1 PART 2 PART 1 + 2 (COMBINED) (n = 32) (n = 82) (n = 114) Cohort 3 Cohort 4 Cohort 3 Cohort 4 Cohort 3 Cohort 4 Total number of participants 1.8 mg/kg
Slide 52: Summary
Summary Recent interim data confirm rapid and deep amyloid lowering at higher trontinemab doses o Very rapid amyloid depletion at 3.6 mg/kg: 81% amyloid PET negative at 28 weeks o Nearly all participants near or below amyloid positivity threshold, no “amyloid non-responders” Early and pronounced eff
Slide 53: Acknowledgements
Acknowledgements We thank all the study participants and their families, the investigators, and site staff for their time and commitment to the BrainshuttleTM AD study https://ter.li/x3m6mw https://ter.li/irpp3b 53
Slide 54: Elevidys in Duchenne Muscular Dystrophy (EMBARK) – MDA data presentation
Elevidys in Duchenne Muscular Dystrophy (EMBARK) – MDA data presentation (March 12) Alex Murphy Senior Clinical Director
Slide 55: Duchenne muscular dystrophy: Unmet need remains critical
Neurology Duchenne muscular dystrophy: Unmet need remains critical DMD prevalence DMD is progressive, irreversible, and leading to early death Life expectancy in DMD Diagnosed population 202417 Pooled survival from systematic literature review on mortality in DMD up to 202018 27.810 10.994 8.769 US
Slide 56: Elevidys - first approved GT that provides functional benefits in DMD
Neurology Elevidys - first approved GT that provides functional benefits in DMD Elevidys Mode of Action Elevidys Clinical development program One time application First approved gene therapy with functional benefit Broader patient population than mutation-specific therapies ▪ Gene therapy for the tr
Slide 57: Elevidys providing clinically meaningful benefits in DMD
Neurology Elevidys providing clinically meaningful benefits in DMD Ph III (EMBARK): Results in DMD at 1 year1 Ph I (101): NSAA 5 years post Elevidys 2;3 Favors Elevidys LSMean difference (95% CI) p-value NSAA trajectories of 101 study (4pts) vs Natural History Centile NSAA Total - 90th - 75th - 50th
Slide 58: Long-Term Functional Outcomes and Safety of Delandistrogene Moxeparvovec in DMD
Long-Term Functional Outcomes and Safety of Delandistrogene Moxeparvovec in DMD: EMBARK 2-Year and Pooled 3-Year Analyses Crystal Proud, MD Children’s Hospital of The King’s Daughters, Norfolk, VA, USA
Slide 59: EMBARK: Study design
EMBARK: Study design PART 1: 52 weeks PART 2: 52 weeks Up to 5 years SINGLE IV INFUSION SINGLE IV INFUSION Key inclusion criteria:1 Delandistrogene Placebo • Ambulatory males aged ≥4 to <8 years at moxeparvovec OPEN-LABEL randomization Randomization EXTENSION • Confirmed DMD diagnosis (DMD mutation
Slide 60: EMBARK: External control (EC) cohort
EMBARK: External control (EC) cohort In the absence of a placebo arm, due to the crossover study design, 2-year data of EMBARK Part 1-treated patients were compared with an EC cohort of patients with DMD using propensity-score weighting1* EC cohort entry criteria: Patients receiving only ✔ Aged ≥4 a
Slide 61: EMBARK: Demographics and baseline clinical characteristics
EMBARK: Demographics and baseline clinical characteristics Baseline characteristics were well matched between patients receiving delandistrogene moxeparvovec in EMBARK Part 1 and EC patients after propensity-score weighting* EMBARK Part 1 Standardized mean Characteristic, Delandistrogene EC cohort d
Slide 62: EMBARK Part 1: Functional outcomes at 2 years
EMBARK Part 1: Functional outcomes at 2 years At 2 years, Part 1-treated patients demonstrated statistically significant and clinically meaningful functional benefit versus a propensity-score-weighted EC cohort LSMs (of change from baseline) and CIs were standardized by dividing by the SE. Negative
Slide 63: EMBARK Part 1: Functional outcomes at 2 years
EMBARK Part 1: Functional outcomes at 2 years At 2 years, Part 1-treated patients demonstrated statistically significant and clinically meaningful functional benefit versus a propensity-score-weighted EC cohort NSAA ∆ 2.88 points; P=0.0001 TTR ∆ –2.06 s; P=0.0033 10MWR ∆ –1.36 s; P=0.0028 Improvemen
Slide 64: EMBARK Part 1: Micro-dystrophin expression up to Week 64
EMBARK Part 1: Delandistrogene moxeparvovec micro-dystrophin expression and sarcolemmal localization up to Week 64 Delandistrogene moxeparvovec micro-dystrophin expression and sarcolemmal localization were sustained from Week 12 to Week 64 in a subset of patients Delandistrogene moxeparvovec Mean (S
Slide 65: Elevidys providing clinically meaningful benefits in DMD
Neurology Elevidys providing clinically meaningful benefits in DMD MRI continues to show minimal progression in underlying muscle pathology in 2 year follow up Musculoskeletal MRI FF outcomes from EMBARK Part 1 treated vs placebo up to 2 years Change from baseline to Week 52 and 104 in MRI FF (%) 1
Slide 66: EMBARK Part 1: Overview of 2-year safety results
EMBARK Part 1: Overview of 2-year safety results Between Weeks 52 and 104, Baseline to Week 52 Weeks 52–104* 15 (23.8%) patients Overview of AEs, n (%) N=63 N=63 experienced 34 TR-TEAEs Patients with any TEAEs 62 (98.4) 53 (84.1) One patient experienced two TR-SAEs of Patients with any SAEs 14 (22.2
Slide 67: EMBARK Part 1: Timeline of TR-TEAEs
EMBARK Part 1: Timeline of TR-TEAEs following treatment with delandistrogene moxeparvovec Evidence to date suggests that most TR-TEAEs were reported within the first 90 days of infusion Number of patients with TR-TEAEs, listed by frequency at 0–2 weeks, n (%)* Vomiting 31 (49.2) 3 (4.8) 0 (0) 0 (0)
Slide 68: 3-Year Functional Outcomes of Patients With DMD: Pooled Clinical Trial Data
3-Year Functional Outcomes of Patients With Duchenne Muscular Dystrophy: Pooled Delandistrogene Moxeparvovec Clinical Trial Data Vs External Controls (P167) Jerry R. Mendell,1 Anne M. Connolly,2 John Day,3 Craig M. McDonald,4 Crystal Proud,5 Perry Shieh,6 Craig M. Zaidman,7 Matthew Furgerson,1 Kai D
Slide 69: Pooled analyses: Study methodology and analysis
Pooled analyses: Study methodology and analysis Study 101 (NCT03375164) Analysis N=4 Delandistrogene moxeparvovec entry criteria: • Received target dose of 1.33×1014 vg/kg • ≥4 to <9 years old at baseline Study 102 (NCT03769116) Analysis n=26 • Had both baseline and Year 3 assessment values Total: N
Slide 70: Pooled analyses: Demographics and baseline clinical characteristics
Pooled analyses: Demographics and baseline clinical characteristics Baseline characteristics were well matched between the pooled delandistrogene moxeparvovec-treated patients and EC patients after propensity-score weighting* Standardized mean Pooled delandistrogene Characteristic, EC cohort differe
Slide 71: Pooled analyses: Functional outcomes at 3 years
Pooled analyses: Functional outcomes at 3 years At 3 years, patients treated with delandistrogene moxeparvovec demonstrated functional benefit versus the EC cohort NSAA ∆ 3.00 points; P=0.0003 TTR ∆ –1.80 s; P<0.0001 10MWR ∆ –0.40 s; P=0.0592 Improvement Improvement Improvement All P-values reported
Slide 72: Conclusions
Conclusions Results from 2-year EMBARK and 3-year pooled Healthy children usually reach the peak NSAA score of 34 after age 4 1 analyses indicate stabilization or slowing of disease progression compared with well Increased score/ higher peak2,3 matched ECs assessed by functional outcomes prognostic
Slide 73: QR codes
QR codes • QR code for EMBARK 2-Year poster (P169) • QR code for Pooled Analyses 3-Year poster (P167) The QR code is intended to provide scientific information for individual reference, and the information should not be altered or reproduced in any way.