Slide 1: Corporate Presentation September 12, 2016
Corporate Presentation September 12, 2016 Exhibit 99.1
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, regarding, among other things, the timing and success of preclinical studies and clinical trials conducted by REGENXBIO and its development
Slide 3: NAV Gene Therapy – using a modified virus as a delivery vehicle for healthy genes
RNA Protein NAV Gene Therapy – using a genetically modified virus as a delivery vehicle for healthy genes Problem: Patient cannot make a protein the body needs because of a defective gene Healthy gene copy NAV Vector Cell Nucleus NAV Gene Therapy delivers a healthy gene copy to cells to address the
Slide 4: REGENXBIO: AAV gene therapy company developing life-altering treatments
REGENXBIO: adeno-associated virus (AAV) gene therapy company developing life-altering treatments for patients with severe diseases Proprietary NAV® Technology Platform includes exclusive worldwide rights to over 100 AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10 Lead program clinical trial init
Slide 5: REGENXBIO's NAV Technology Platform has been widely adopted
Nasdaq: ADVM REGENXBIO’s NAV Technology Platform has been widely adopted / / Nasdaq: AVXS Nasdaq: DMTX FRA: BAYN Nasdaq: VYGR NYSE: SNY Nasdaq: SHPG Nasdaq: BIIB Nasdaq: BOLD
Slide 6: REGENXBIO's lead programs
REGENXBIO’s lead programs Metabolic Diseases Neurodegenerative Diseases Retinal Diseases Indication Development Stage Regulatory / Clinical Research Preclinical Clinical Status Homozygous familial hypercholesterolemia (HoFH) IND active, Phase I/II trial initiation anticipated 2H 2016 Mucopolysacchar
Slide 7: Internal Development Programs
Internal Development Programs
Slide 8: RGX-501 for treatment of homozygous familial hypercholesterolemia (HoFH)
RGX–501 for treatment of homozygous familial hypercholesterolemia (HoFH) Defective LDLR gene leads to limited / no LDL cholesterol receptors, allowing LDL to build up in bloodstream Total cholesterol levels >500 mg/dL Coronary artery disease at young age Even with existing standard of care therapies
Slide 9: Preclinical NAV Gene Therapy improvement in familial hypercholesterolemia mice
Familial hypercholesterolemia mice injected with AAV8 Mouse LDLR gene or control Preclinical studies using NAV Gene Therapy have shown a significant improvement in familial hypercholesterolemia mice Stable correction of cholesterol levels seen for nearly one year 0 100 200 300 400 500 600 700 800 7
Slide 10: RGX-501 Phase I/II clinical trial in HoFH
RGX–501 Phase I/II clinical trial in HoFH Objectives Key inclusion criteria Male or female >= 18 years of age Untreated and/or treated LDL-C levels and clinical presentation consistent with the diagnosis of HoFH Molecularly defined LDLR mutations at both LDLR alleles NAbs titer <=1:5 Stable concurre
Slide 11: RGX-501 Phase I/II trial – administration and dose escalation
RGX–501 Phase I/II clinical trial – administration and dose escalation Expected dose escalation pathway Low Dose n = 3 Safety review High Dose n = 3 High Dose n = up to 6 2.5x1012 gc/kg Safety review 7.5x1012 gc/kg 7.5x1012 gc/kg Screening stage Follow up Treatment evaluation Administration 0 4 8 26
Slide 12: RGX-314 for treatment of wet age-related macular degeneration
RGX–314 for treatment of wet age-related macular degeneration (wet AMD) Blurring of central vision and progressive vision loss due to formation of leaky blood vessels in the eye VEGF inhibitors can prevent / reverse new blood vessel formation Frequency / uncomfortable administration of current anti-
Slide 13: RGX-314 – potential for long-term therapeutic anti-VEGF expression
RGX–314 – potential for long-term therapeutic anti-VEGF expression Improved AAV vector technology Potentially eliminate frequent, uncomfortable administration of current anti-VEGF therapies that affect compliance, and ultimately efficacy Current standard of care includes FDA-approved mAbs and mAb fr
Slide 14: RGX-314 expression in non-human primates vs earlier candidates
Sponsor Vector ROA Transgene Dose (GC/eye) Max. expression (ng/ml)2 AAV8 Subretinal anti-VEGF fab 1.0e11 4,992 AAV2 Subretinal sFlt 8.0e11 0.217 AAV2 Intravitreal sFLT01 2.4e10 528 RGX–314 has significant potential advantages over earlier generation candidates for wet AMD gene therapy RGX-314 expres
Slide 15: RGX-111 for treatment of mucopolysaccharidosis Type I (MPS I)
RGX–111 for treatment of mucopolysaccharidosis Type I (MPS I) Defective IDUA gene leads to reduced ability to process glycosaminoglycans (GAGs), a cellular waste product Severe GAG buildup causes neurodegeneration and early death Available treatment is inadequate to treat neurodegeneration; bone mar
Slide 16: Preclinical NAV Gene Therapy improvement in MPS I cats
Preclinical studies using NAV Gene Therapy have shown a significant improvement in MPS I cats IDUA Expression in Cats Following AAV9 Delivery CNS correction observed was substantially greater than that observed in a previous study of MPS I cats treated with BMT at similar ages 0 50 100 150 200 0.1 1
Slide 17: MPS I cats benefitted from cross-correction
In preclinical studies, MPS I cats benefitted from cross-correction Correction observed throughout the CNS Cerebrum Perivascular Meninges Source: Bell et al. 2014 Molecular Therapy
Slide 18: RGX-121 for treatment of mucopolysaccharidosis Type II (MPS II)
RGX–121 for treatment of mucopolysaccharidosis Type II (MPS II) Defective IDS gene leads to reduced ability to process GAGs Severe GAG buildup causes neurodegeneration and early death (similar phenotype to severe MPS I) X-linked recessive disease (predominantly occurs in males) Available treatment i
Slide 19: Preclinical NAV Gene Therapy improvement in MPS II mice
Preclinical studies using NAV Gene Therapy have shown a significant improvement in MPS II mice Reduction in Storage Lesions Following AAV9 Delivery Statistically significant reduction in storage lesions at all dose levels Source: Hinderer et al. 2016 Human Gene Therapy
Slide 20: A single transduced cell has potential to correct many other cells
A single transduced cell has potential to correct many other cells Cross–correction is a key treatment advantage in MPS diseases Protein secreted by transduced cells NAV Vector delivers healthy gene to cells RNA Gene NAV Vector Protein taken up by non-transduced cells 1 2 3 Protein
Slide 21: NAV Technology Platform
#6} REGENXBIO NAV® Technology Platform
Slide 22: The NAV Technology Platform is based on a broad and deep IP portfolio
Exclusive rights to more than 100 patents and patent applications worldwide The NAV Technology Platform is based on a broad and deep IP portfolio NAV Vectors … AAV7, AAV8, AAV9, AAVrh10 Over 100 other novel AAV sequences Sequences that are at least 95% identical to these capsids
Slide 23: Key features of REGENXBIO's NAV Technology Platform
Key features of REGENXBIO’s NAV Technology Platform Higher gene expression Longer-term gene expression Broad and novel tissue selectivity Lower immune response Improved manufacturability Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B Intravascular AAV9 Preferentially Targets
Slide 24: NAV Vectors – higher gene expression than early generation AAV vectors
NAV Vectors – higher gene expression than early generation AAV vectors AAV2 AAV8 NAV Vector AAV8: 10x–100x greater gene expression AAV2 AAV8 NAV Vector AAV8: More efficient gene delivery to sites of most retinal dystrophies1 Photoreceptors (PR) Retinal Pigment Epithelium (RPE) Photoreceptors (PR) Re
Slide 25: Team and Conclusion
#6} REGENXBIO Team and Conclusion
Slide 26: The REGENXBIO team
The REGENXBIO team Name Position Prior / Current Affiliations Ken Mills President, CEO & Co-Founder; Director James Wilson, M.D., Ph.D.* Chief Scientific Advisor & Co-Founder Vit Vasista Chief Financial Officer Stephen Yoo, M.D. Chief Medical Officer Faraz Ali Chief Business Officer Curran Simpson S
Slide 27: Financial results and guidance
Financial results and guidance 2015 financial highlights Program guidance Recruiting patients, Phase I/II trial initiation anticipated 2H 2016 Cash: R&D expense: G&A expense: Net loss: $199 $17 $12 $25 2016 financial guidance As of August 9, 2016, expect 2016 cash burn to be between $60 million and
Slide 28: REGENXBIO: AAV gene therapy company developing life-altering treatments
REGENXBIO: adeno-associated virus (AAV) gene therapy company developing life-altering treatments for patients with severe diseases Proprietary NAV® Technology Platform includes exclusive worldwide rights to over 100 AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10 Lead program clinical trial init
Slide 29: Leader in AAV gene therapy – Thank You
gos R=G=NXBIC" Leader in AAV gene therapy Thank You