Slide 1: REGENXBIO Reports Second Quarter 2018 Financial and Operating Results
August 8, 2018 REGENXBIO Reports Second Quarter 2018 Financial and Operating Results and Interim Data from Ongoing Clinical Trials for Wet AMD and HoFH EXHIBIT 99.2
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: Second quarter 2018 earnings call agenda
REGENXBIO second quarter 2018 earnings call agenda Agenda Opening RGX–314 for the treatment of wet AMD – Phase I interim results RGX–501 for treatment of HoFH – Phase I/II interim results Other recent operational highlights Financial results and guidance Closing
Slide 4: REGENXBIO's lead programs
Research Preclinical Phase I / II Phase III Retinal Disease RGX–314 Wet age-related macular degeneration (wet AMD) Metabolic Disease RGX–501 Homozygous familial hypercholesterolemia (HoFH) Neurodegenerative Disease RGX–111 Mucopolysaccharidosis Type I (MPS I) RGX–121 Mucopolysaccharidosis Type II (M
Slide 5: Our Heroes
Our Heroes @#R=G=ENXBIO
Slide 6: RGX-314 Phase I clinical trial interim results summary
RGX–314 Phase I clinical trial interim results Summary Well–tolerated at all doses Dose–dependent protein expression levels + Reduction in anti–VEGF injections + maintained retinal thickness + maintained to improved vision Cohort 3 (6x1010 GC/eye dose) 50% free of anti–VEGF injections through six mo
Slide 7: RGX-314 for treatment of wet AMD
THE DISEASE Blurring of central vision and progressive vision loss due to formation of leaky blood vessels in the eye VEGF inhibitors are standard of care to treat fluid and associated vision loss Frequency / uncomfortable administration of current anti-VEGF therapies affects compliance and ultimate
Slide 8: RGX-314 Phase I interim results study design
RGX–314 Phase I clinical trial – interim results study design 1 Dose escalation safety review to occur four weeks after final subject in each cohort has been dosed Dosing complete in three cohorts Administration and follow-up timeline Expected dose escalation pathway Dose 1 n = 6 3x109 GC/eye Dose 2
Slide 9: RGX-314 Phase I study overview and objectives
Objectives Primary To determine the safety and tolerability of RGX-314 in subjects with wet AMD though six months Secondary Expression of RGX–314 protein in the eye Effect of RGX–314 on best corrected visual acuity (BCVA) and central retinal thickness (CRT) as measured by Spectral Domain Optical Coh
Slide 10: RGX-314 Phase I safety summary
RGX–314 Phase I clinical trial – safety summary RGX–314 was well–tolerated No drug-related AEs or drug-related SAEs Most AEs were assessed as mild (Grade 1 – 83%) No observed immune responses, drug-related ocular inflammation, or any post-surgical inflammation beyond what is expected following routi
Slide 11: RGX-314 protein levels at one month
RGX–314 protein levels at one month As measured from aqueous samples by ELISA 12.8 ng/ml 160.2 ng/ml 2.4 ng/ml 3 x 109 GC/eye N=6 1 x 1010 GC/eye N=6 6 x 1010 GC/eye N=6
Slide 12: Summary and comparison of mean anti-VEGF injections
* Number of anti-VEGF injections reported between a day before the first anti-VEGF injection preceding Day 1 visit and six months prior ** Number of anti-VEGF injections reported between RGX-314 administration date and six–month visit *** One subject in Cohort 1 discontinued from the study at four m
Slide 13: Change from baseline in central retinal thickness at six months
Cohort 1 3 x 109 GC/eye Cohort 2 1 x 1010 GC/eye Cohort 3 6 x 1010 GC/eye N 5* 6 6 Mean change from baseline in CRT (µm) -14 +26 -14 Median change from baseline in CRT (µm) +22 +23 -16 Range (low, high) (-181, +92) (-7, +62) (-27, +7) * One subject in Cohort 1 discontinued from the study at four mon
Slide 14: Change from baseline in best corrected visual acuity at six months
* One subject in Cohort 1 discontinued from the study at four months Change from baseline in mean best corrected visual acuity at six months Cohort 1 3 x 109 GC/eye Cohort 2 1 x 1010 GC/eye Cohort 3 6 x 1010 GC/eye N 5* 6 6 Mean change from baseline in BCVA (ETDRS Letters) -2 +7 +8 Median change fro
Slide 15: Mean CRT and BCVA following RGX-314 administration
Cohort 1 Cohort 2 Cohort 3 Summary of mean central retinal thickness and best corrected visual acuity following RGX–314 administration Average injections: 4.7 Average injections: 3.8 Average injections: 1.3 +8 letters -14 μm -14 μm -2 letters +7 letters +26 μm
Slide 16: Cohort 3: three subjects with no additional anti-VEGF injections
Cohort 3: Three subjects with no additional anti–VEGF injections through month six Previous therapy Study subjects received on average >35 injections since wet AMD diagnosis Post-RGX–314 anti–VEGF injections 0 injections through six months post-RGX–314 BCVA Mean gain in BCVA of +8 ETDRS letters from
Slide 17: RGX-314 clinical trial summary through six months
Aqueous RGX–314 protein one month post–treatment Mean # of anti-VEGF injections through six months Mean change in CRT through six months Mean change in BCVA through six months Cohort 1 3 x 109 GC/eye (N=6) 2.4 ng/ml 4.7 inj* -14 µm** ‑2 letters** Cohort 2 1 x 1010 GC/eye (N=6) 12.8 ng/ml 3.8 inj +26
Slide 18: Summary of Cohort 3 and Cohort 4 expansion
Summary of Cohort 3 and Cohort 4 expansion Cohort 3 Positive signals from clinical measures at six months indicate that this may be a clinically meaningful dose which we expect to take into a Phase II trial for wet AMD Mean RGX–314 protein levels >100 ng/ml were observed post–treatment Subjects exhi
Slide 19: RGX-314 Phase I updated study design
RGX–314 Phase I clinical trial – updated study design 1 Dose escalation safety review to occur four weeks after final subject in each cohort has been dosed Dosing completed in three cohorts and initiated in Cohort 4 Administration and follow-up timeline Expected dose escalation pathway Dose 1 n = 6
Slide 20: RGX-501 for treatment of HoFH
THE DISEASE 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, the mean age of death is 32 years Approx. 11,000 patients w
Slide 21: RGX-501 Phase I/II clinical trial study design
RGX–501 Phase I/II clinical trial – study design 1 Dose escalation safety review to occur four weeks after final subject in each cohort has been dosed Three subjects dosed in each of the first and second cohorts Dose 1 n = 3 Dose 2 n = 3 Dose 2 n = up to 6 7.5x1012 gc/kg 7.5x1012 gc/kg 2.5x1012 gc/k
Slide 22: RGX-501 Phase I/II interim results summary
RGX–501 Phase I/II clinical trial interim results Summary Transaminase elevations observed in Cohort 2 Administration of steroid appears to mitigate transaminase elevations and related effects Clinical trial protocol expected to be amended to enroll additional subjects using steroid prophylaxis Addi
Slide 23: REGENXBIO's lead programs and anticipated milestones
Research Preclinical Phase I / II Phase III Retinal Disease RGX–314 Wet age-related macular degeneration (wet AMD) Complete dosing of Cohort 4 Q4 2018 and initiate Phase II trial in 2019 Metabolic Disease RGX–501 Homozygous familial hypercholesterolemia (HoFH) Additional data Q4 2018 Neurodegenerati
Slide 24: NAV Technology Platform has been widely adopted
REGENXBIO’s NAV Technology Platform has been widely adopted Over 20 partnered product candidates being developed by NAV Technology Licensees Research Preclinical Phase I / II Phase III Indication Licensee Indication Licensee Indication Licensee Indication Licensee Liver / hematologic Citrullinemia T
Slide 25: Financial results and guidance
1 As of August 3, 2018 2 Cash includes cash, cash equivalents and marketable securities for purposes of this presentation Financial results and guidance 2018 financial guidance: Expect 2018 ending cash balance to be between $250 million and $260 million2 2018 1H financials (mm) R&D expense: $41 G&A
Slide 26: Closing and Q&A
Closing and Q&A @ REGEN: Bo