Regenxbio Inc. · NASDAQ:RGNX
Regenxbio — WORLDSymposium 2023
14 slides from Regenxbio's conference presentation, Feb 24, 2023
Slide 1: RGX-111 Gene Therapy for Severe MPS I: Interim Analysis
RGX-111 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type I (MPS I): Interim Analysis of the First in Human Study and a Single Patient IND Presented by: Raymond Y. Wang, MD Division of Metabolic Disorders, CHOC Children’s Hospital / Department of Pediatrics, University of Californi
Slide 2: MPS I is a Systemic Disease Representing a Wide Spectrum of Severity
MPS I is a Systemic Disease Representing a Wide Spectrum of Severity Severity of Disease Manifestations Correlates with Degree of alpha-l-iduronidase (IDUA) Deficiency Hurler (60%) Hurler-Scheie (23%) Scheie (13%) Symptom Onset 0.5 y 3.0 y 7.8 y Age of diagnosis 0.8 y 3.9 y 9.3 y 35% IQ < 85 Cogniti
Slide 3: RGX-111: MPS I Phase I/II Clinical Study Summary
RGX-111: MPS I Phase I/II Clinical Study Summary NCT03580083 on ClinicalTrials.gov Participants Cohorts (dose levels) Data Enrollment of 8 MPS I Genome copies/g brain mass participants with CNS Primary Endpoint is Safety involvement or severe MPS I RGX-111 Secondary & Exploratory (≥ 4 months of age)
Slide 4: RGX-111 Phase I/II Trial and Single Patient Investigator-Initiated IND
RGX-111 Phase I/II Trial and Single Patient Investigator-Initiated IND ▪ As of January 17, 2023, 8 participants were dosed in the Phase I/II trial and 1 in the single patient IND protocol ▪ Age at dosing from 4 months to 13 years in Phase I/II trial and 20 months in single patient IND ▪ IDUA Mutatio
Slide 5: RGX-111 Safety Summary
RGX-111 Safety Summary Phase I/II Trial and Single Patient Investigator-Initiated IND ▪ 9 serious adverse events (SAE) reported in 4 participants: None are considered related to RGX-111 ▪ SAEs reported: Bronchiolitis, bronchopneumonia, sinusitis, 2 central line infections*, SAE COVID-19, RSV, sepsis
Slide 6: Cerebrospinal Fluid (CSF) GAGs Heparan Sulfate
Cerebrospinal Fluid (CSF) GAGs Heparan Sulfate (HS) Individual Participants Concentration (ng/mL) Cohort 1 – Phase 1/2 Cohort 2 – Phase 1/2 Single Patient IND Time on Study (Weeks) ▪ Decreased CSF heparan sulfate in majority of participants through last time point available ▪ Measurable CSF IDUA enz
Slide 7: Neurodevelopmental Assessments
Neurodevelopmental Assessments Age and developmentally appropriate validated instruments for neurodevelopmental testing were used to evaluate all participants n=6* Bayley Scale of Infant and Toddler Wechsler Abbreviated Scale of Development, Third Edition (BSID-III) Intelligence (WASI-II) for chrono
Slide 8: Neurodevelopmental Function BSID-III
Neurodevelopmental Function BSID-III Cohort 1 – Phase I/II Cohort 2 – Phase I/II Single Patient IND Cognition Expressive Language Fine Motor +/- 1S D +/- 2S D Age Equivalence (months) Age (months) ▪ All participants show continued developmental skill acquisition on all subtests ▪ At last assessment,
Slide 9: Neurodevelopmental Assessments and Function: BSID Cognitive Subtest
Neurodevelopmental Assessments and Function BSID* Cognitive Subtest Age Equivalence (months) BSID cognitive function in 2 participants** demonstrates higher AEq than available natural history data Cohort 1 – Phase I/II; n = 1 Cohort 2 – Phase I/II; n = 3 Single Patient IND; n = 1 Age (months) * Natu
Slide 10: Neurodevelopmental Assessments and Adaptive Function for 13 Year Old Participant
Neurodevelopmental Assessments and Adaptive Function for 13 Year Old Phase I/II Participant WASI-II and VABS-III VABS-III Age Equivalent Scores (year : month) BL Week of Assessment Week 52 Week 78 WASI- II Composite Scores Age 13y 14y 2m 14y 8m 2m Week of BL Week 52 Week 78 Assessment 13y 2m 14y 2m
Slide 11: Systemic Effects: Plasma I0S6
Systemic Effects: Plasma I0S6 I0S6 is a non-reducing end (NRE) disaccharide of glycosaminoglycans shown to be elevated in plasma, urine and CSF of MPS I patients1,2,3,4,5 Plasma I0S6 concentration (ng/ml) Cohort 1 – Phase I/II Cohort 2 – Phase I/II Single Patient IND Time on Study Not on ERT / ERT n
Slide 12: Systemic Effects: Urine Total GAGs
Systemic Effects Urine Total GAGs Total GAGs, urine (g/mol CK) ERT naïve at dosing Post ERT Withdrawal ERT Single Patient IND Time on Study Majority of patients maintained low levels of total urine GAGs Phase I/II Trial data cut: January 17, 2023 Single Patient IND data cut: December 12, 2021 12
Slide 13: RGX-111 Phase I/II Trial and Single Patient IND Summary of Results
RGX-111 Phase I/II Trial and Single Patient IND Summary of Results Safety: RGX-111 was well tolerated ▪ 8 participants were dosed in the Phase I/II trial and 1 in the single patient IND protocol ▪ RGX-111 has been well tolerated with no SAEs related to study drug CNS: Biomarkers and neurodevelopment
Slide 14: Acknowledgements
Acknowledgements The RGX-111-002 Investigators REGENXBIO ▪ Nidal Boulos ▪ Michelle Gilmor ▪ Can Ficicioglu, Children’s Hospital of Philadelphia ▪ Jenna Burke ▪ Caroline Mulatya ▪ Roberto Giugliani, HCPA, Porto Alegre, Brazil ▪ Yoonjin Cho ▪ Laura Pisani ▪ Yair Anikster, Sheba Medical Center, Israel