Regenxbio Inc. · NASDAQ:RGNX
Regenxbio — ASGCT 2024
12 slides from Regenxbio's conference presentation, May 11, 2024
Slide 1: RGX-202, an Investigational Gene Therapy for Duchenne Muscular Dystrophy: Interim Clinical Data
RGX-202*, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data Jahannaz Dastgir, DO Sr Clinical Development Lead REGENXBIO ASGCT, May 11, 2024, Baltimore * RGX-202 is an investigational product that has not been approved by the FDA. No conclusions r
Slide 2: Forward-looking Statements
Forward-looking Statements This presentation includes “forward-looking statements,” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are gen
Slide 3: RGX-202 Transgene is Designed to Encode Key Elements of Naturally Occurring Dystrophin
RGX-202 Transgene is Designed to Encode Key Elements of Naturally Occurring Dystrophin DAPC DAPC DAPC RGX-202 expresses a new, differentiated microdystrophin with important biology that is the most similar to a natural shortened dystrophin that protects muscles from degenerating 1. England (1990) Na
Slide 4: RGX-202 is Novel Among Current Class of AAV-microdystrophins
RGX-202 is Novel Among Current Class of AAV- microdystrophins RGX-202 is the only gene therapy designed to deliver a transgene for a microdystrophin with the functional elements of the C-Terminal (CT) domain found in naturally occurring dystrophin AAV Serotype Promoter Transgene REGENXBIO1 8 Spc5-12
Slide 5: RGX-202 Study Overview
RGX-202 Study Overview Key Eligibility Criteria Study Plan • Boys aged 4 to 11 years at screening Dose Level 1 Dose Level 2 Dose Level 2 • Genetically confirmed DMD (mutations in exons 18 and above) Dose Evaluation Dose Evaluation Expanded Cohort 1x1014 GC/kg 2x1014 GC/kg 2x1014 GC/kg • 100-meter wa
Slide 6: Key Baseline Characteristics & Safety
Key Baseline Characteristics & Safety Post- Weight at Age at Dosing Administration Dose Cohort N Dosing (yrs) follow-up (kg) (months) 1 Dose 3 4.4-10.5 17.8 – 28.3 7.9-13.4 (1x1014 GC/kg) evaluation 2 Dose 2 8.1-12.1 24.3 – 31.2 3.5-5.9 (2x1014 GC/kg) evaluation 2 Expansion 2 5.8-8.5 17.3 – 24.3 1.2
Slide 7: Interim Data: Dose Level 1
Interim Data: Dose Level 1 RGX-202 Dose Level 1 Microdystrophin Western blot CK Levels, week 10 Age at Dosing (Jess method) (% reduction from Patient (years) (% Normal Control) baseline) • Robust RGX-202 microdystrophin expression observed Dose Level 1 1x1014 GC/kg • Serum CK levels markedly 1 4 yrs
Slide 8: Interim Data: Dose Level 2
Interim Data: Dose Level 2 RGX-202 Dose Level 2 Microdystrophin Western blot CK Levels, week 10 Age at Dosing (Jess method) (% reduction from • Robust RGX-202 microdystrophin Patient (years) (% Normal Control) baseline) expression observed Dose Level 2 • Serum CK levels markedly 2x1014 GC/kg decreas
Slide 9: RGX-202 Microdystrophin Expression at 3 months
RGX-202 Microdystrophin Expression at 3 months Robust RGX-202 microdystrophin expression was demonstrated at both dose levels Dose Level 1 Dose Level 2 % RGX-202 % RGX-202 Age range at microdystrophin microdystrophin screening (n = 3) (n = 2) 4 to 5 years 38.8 6 to 7 years 83.4 8 to 11 years 11.1 20
Slide 10: RGX-202 microdystrophin is localized to the sarcolemma
RGX-202 microdystrophin is localized to the sarcolemma Normal Baseline 3 months Muscle biopsies are collected from biceps at baseline and 3 months post RGX-202 administration 10 Representative IF images from healthy control, patient 1 at baseline, and participant 1 three months post RGX-202 administ
Slide 11: AFFINITY DUCHENNE: Summary
AFFINITY DUCHENNE: Summary As of May 3, 2024, RGX-202 has been well-tolerated at both dose levels with no SAEs Robust RGX-202 microdystrophin expression was observed at both dose levels in all ages Encouraging observations of early improvements in daily activities associated with strength and functi
Slide 12: Acknowledgements
Acknowledgements AFFINITY DUCHENNE Participants and their Families The AFFINITY DUCHENNE Investigators • Amy Harper, MD, Children’s Hospital of Richmond at Virginia commonwealth University REGENXBIO • Susan Iannacone, MD, The University of Texas Southwestern Medical Center Nidal Boulos John Hall Sta