Regenxbio Inc. · NASDAQ:RGNX
Regenxbio — WORLDSymposium 2023
15 slides from Regenxbio's conference presentation, March 2023
Slide 1: RGX-121 Gene Therapy for Severe MPS II: CAMPSIITE Clinical Study Update
RGX-121 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type II (MPS II): CAMPSIITE™ Phase I/II/III: A Clinical Study Update Can Ficicioglu, MD, PhD Professor of Pediatrics at Perelman School of Medicine at the University of Pennsylvania Director of the Newborn Metabolic Screening Pro
Slide 2: AAV Gene Therapy Has the Potential to Address Unmet Need in MPS II
AAV Gene Therapy Has the Potential to Address Unmet Need in MPS II High Unmet Need in MPS II Potential of RGX-121 for MPS II Incidence of MPS II Prevalence AAV9 Vector + IDS Attenuated MPS II Severe Transgene 1 in 100,000 MPS II to ~25% ~75% 1 in 170,000 ▪ One-time administration RGX-121 May ▪ Image
Slide 3: RGX-121: CAMPSIITE Part 1, Phase I/II
RGX-121: CAMPSIITE Part 1, Phase I/II NCT03566043 on ClinicalTrials.gov Participants Cohorts (dose levels) Data Genome copies/g brain mass Enrollment up to 16 Primary Endpoint: Safety severe MPS II partcipants RGX-121 (≥ 4 months to ˂ 5 years of age) AAV9 + IDS Secondary & Exploratory May be on Stan
Slide 4: RGX-121 Phase I/II Cohorts
RGX-121 Phase I/II Cohorts ▪ 15 participants dosed as of January 3, 2023 ▪ Age at dosing ranged from 5 months to 59 months ▪ IDS Mutations among severe MPS II trial participants included deletion, frameshift, gene inversion, insertion, missense, splicing, and substitution ▪ Study duration 104 weeks.
Slide 5: CAMPSIITE Safety Summary
CAMPSIITE Safety Summary ▪ 12 serious adverse events (SAE; 8 in main study, 4 in LTFU) reported in 7 participants: None are considered related to RGX-121 ▪ SAEs reported in main study: HSV gingivostomatitis*, fever requiring hospitalization, SAE infection of VP shunt, viral meningitis*, hydrocephalu
Slide 6: Cerebrospinal Fluid GAGs: Heparan Sulfate
Cerebrospinal Fluid (CSF) GAGs: Heparan Sulfate (HS) Cohort 1 n = 3 Cohort 2 n = 7 Cohorts (median†) Individual Participants Cohort 3 n = 4 % Reduction 1600 Week Week Week Cohort 8 24 48 1400 -29.5% -20.6% -33.5% 1 n=3 n=2 n=3 -42.3% -36.4% -48.9% 1200 CSF HS (ng/mL) 2 n=6 n=7 n=6 -58.7% -74.9% -64.
Slide 7: CSF GAGs: HS D2S6, a Trisulfated Disaccharide
CSF GAGs: HS D2S6, a Trisulfated Disaccharide D2S6 is a Correlate of Neuropathology Phenotype in Severe MPS II1-3 Cohort 1 n = 3 Cohort 2 n = 7 Cohort 3 n = 4 CSF D2S6 (ng/mL) Maximum attenuated Time on Study (Weeks) • Week 48 CSF HS D2S6 measurements continued to show dose-dependent reductions acro
Slide 8: Neurodevelopment Assessments
Neurodevelopment Assessments Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) ▪ Participants were assessed using the BSID-III ▪ Age Equivalent (AEq) data is presented for the BSID-III Cognitive, Expressive Language and Fine Motor subtests ▪ BSID-III manual normative data were
Slide 9: Neurodevelopmental Function: Baseline Cognitive Function > -2SD
Neurodevelopmental Function Baseline BSID-III Cognitive Function > -2SD Cohort 1 Cohort 2 Cohort 3 Cognition Expressive Language Fine Motor +/- 1S D +/- 1S D +/- 1S D +/- 2S D +/- 2S D +/- 2S D Age Equivalence (months) Age (months) The majority of participants with baseline function >-2SD have devel
Slide 10: Neurodevelopmental Function: Baseline Cognitive Function Below -2 SD
Neurodevelopmental Function Baseline BSID-III Cognitive Function Below -2 SD Cognition Expressive Language Fine Motor Cohort 2 Cohort 3 Age Equivalence (months) Age (months) The majority of participants with baseline function below -2SD stabilized or had an increase of > 3 mo in AEq on cognitive, ex
Slide 11: Vineland Adaptive Behavior Scales Second Edition (VABS-II)
Vineland Adaptive Behavior Scales Second Edition (VABS-II) VABS measures adaptive behavior, defined as the personal and social skills for everyday living* Communication Daily Living Socialization The majority of participants demonstrated stabilization or ongoing skill acquisition on age-appropriate
Slide 12: VABS-II Maladaptive Behavior Index
VABS-II Maladaptive Behavior Index ▪ Maladaptive behaviors, a measure of undesirable behaviors that interfere with daily functions, are associated with neurodegeneration Cohort 1 ▪ Participants are classified according to level of maladaptive behavior at baseline and compared to average performance
Slide 13: RGX-121 CAMPSIITE Part 1, Phase I/II Summary of Results
RGX-121 CAMPSIITE Part 1, Phase I/II Summary of Results Safety: RGX-121 was well tolerated ▪ As of January 3, 2023, 15 participants have been dosed with RGX-121 ▪ RGX-121 has been well tolerated across 3 cohorts with no SAE related to study drug CNS: CSF GAGs and neurodevelopmental assessments conti
Slide 14: RGX-121: CAMPSIITE Part 2, Phase III
RGX-121: CAMPSIITE Part 2, Phase III NCT03566043 on ClinicalTrials.gov Participants Dose Data Enrollment up to 30 Primary Endpoint: CSF GAGs neuronopathic MPS II participants RGX-121 Co-primary Endpoint: (≥ 4 months to ˂ 5 years of age) AAV9 + IDS • Neurodevelopmental Assessments (Bayley, Mullen) Ma
Slide 15: Acknowledgements
Acknowledgements The RGX-121-101 Investigators REGENXBIO ▪ Can Ficicioglu, Children’s Hospital of Philadelphia ▪ Nidal Boulos ▪ Joe Hagood ▪ Roberto Giugliani, Hospital de Clinicas, Porto ▪ Yoonjin Cho ▪ Caroline Mulatya Alegre, Brazil ▪ Paulo Falabella ▪ Laura Pisani ▪ Paul Harmatz, UCSF Benioff Ch