Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Technical Presentation (September 2024)
19 slides from Viridian Therapeutics's technical presentation, Sep 10, 2024
Slide 1: Veligrotug (VRDN-001) THRIVE Topline Results
Veligrotug (VRDN-001) THRIVE Topline Results September 10, 2024
Slide 2: Cautionary note regarding forward-looking statements
Cautionary note regarding forward-looking statements This presentation contains forward-looking statements. These statements may be identified by the use of words such as, but not limited to, “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “on track,” “p
Slide 3: Viridian is developing an IGF-1R antibody franchise with the potential to transform the treatment of patients with TED
Viridian is developing an IGF-1R antibody franchise with the potential to transform the treatment of patients with TED VRDN-003 Topline data today Veligrotug (VRDN-001) Teprotumumab Potential for best-in-class subcutaneous Steroids/Surgery therapy Potential for Q8W or Q4W 2nd-to-market self-administ
Slide 4: THRIVE: Veligrotug showed robust and consistent clinical activity in active TED patients
THRIVE: Veligrotug showed robust and consistent clinical activity in active TED patients, with a favorable dosing regimen Achieved all primary and secondary endpoints with high level of statistical significance (p < 0.0001) in largest IGF-1R antibody study in TED Rapid onset of treatment effect in a
Slide 5: THRIVE is a phase 3 randomized, controlled, double-masked trial of veligrotug in active TED
THRIVE is a phase 3 randomized, controlled, double-masked trial of veligrotug in active TED Treatment Phase (12-week treatment period with primary endpoint at 15 weeks) Treatment Arms D1 W3 W6 W9 W12 W15 Through W52 (2:1 randomization) Primary Endpoint Analysis Veligrotug n = 75 Primary efficacy end
Slide 6: Baseline characteristics were well-balanced between active and placebo arms
Baseline characteristics were well-balanced between active and placebo arms Veligrotug Placebo (n = 75) (n = 38) Age in years, mean (SD) 48.9 (12.4) 49.1 (12.5) Participant Female sex, n (%) 56 (75%) 31 (82%) Demographics White race, n (%) 51 (68%) 19 (50%) Months since TED onset, mean (SD) 7.9 (3.7
Slide 7: THRIVE achieved high level of statistical significance across all primary & secondary endpoints at 15 weeks
THRIVE achieved high level of statistical significance across all primary & secondary endpoints at 15 weeks Veligrotug Placebo p-value (n=75) (n=38) Primary Endpoint: 70% 5% p < 0.0001 Proptosis responder rate (exophthalmometry)1 Proptosis Proptosis mean change from baseline (exophthalmometry) -2.89
Slide 8: Primary endpoint of proptosis responder rate met: 70% veligrotug vs 5% placebo
Primary endpoint of proptosis responder rate met at 15 weeks: 70% for patients receiving veligrotug compared with 5% on placebo Proptosis Responder Rate Proptosis Mean Change from Baseline Proptosis Mean Reduction (mm) Proptosis Responder Rate (%) 100 0 -0.51 -0.48 -0.67 -0.71 -0.72 80 72% 70% 64% 6
Slide 9: Majority of patients receiving veligrotug had complete resolution of diplopia and minimal disease activity at week 15
Majority of patients receiving veligrotug had complete resolution of diplopia and minimal disease activity at week 15 Diplopia Complete Resolution CAS Score 0 or 1 Diplopia Resolution Rate (%) 100 100 CAS 0 or 1 Rate (%) 80 80 64% 64% 60 54% 60 53% 44% 46% 32% 35% 40 26% 40 30% 25% 24% 20% 18% 20 12
Slide 10: THRIVE data showed high consistency between Hertel exophthalmometry and MRI / CT measurements of proptosis
THRIVE data showed high consistency between Hertel exophthalmometry and MRI / CT measurements of proptosis Hertel exophthalmometry MRI / CT Veligrotug Placebo Veligrotug Placebo (n=75) (n=38) (n=75) (n=38) Proptosis responder rate 70% 5% Proptosis responder rate 69% 9% Proptosis mean change Proptosi
Slide 11: Veligrotug was generally well-tolerated, with no treatment-related SAEs
Veligrotug was generally well-tolerated, with no treatment-related SAEs, and 96% of veligrotug-treated patients completed all doses Veligrotug Placebo N=75 N=38 n (%) n (%) Participants with any treatment-emergent 66 (88%) 24 (63%) • Vast majority of TEAEs in adverse event (TEAE) both arms were mild
Slide 12: Veligrotug was generally well-tolerated, with a 5.5% placebo-adjusted rate of hearing impairment AEs
Veligrotug was generally well-tolerated, with a 5.5% placebo- adjusted rate of hearing impairment AEs Veligrotug Placebo AEs occurring at ≥10% N=75 N=38 frequency in either arm n (%) n (%) Muscle spasms 32 (43%) 2 (5%) Headache 16 (21%) 5 (13%) Infusion related reaction (IRR) 13 (17%) 1 (3%) Hearing
Slide 13: THRIVE: Veligrotug showed robust and consistent clinical activity in active TED patients
THRIVE: Veligrotug showed robust and consistent clinical activity in active TED patients, with a favorable dosing regimen Achieved all primary and secondary endpoints with high level of statistical significance (p < 0.0001) in largest IGF-1R antibody study in TED Rapid onset of treatment effect in a
Slide 14: THRIVE-2, the largest randomized, controlled study in chronic TED, is on track for topline data readout year-end 2024
THRIVE-2, the largest randomized, controlled study in chronic TED, is on track for topline data readout year-end 2024 CHRONIC TED Key Inclusion Criteria Trial Design • Proptosis of ≥3 mm • N = approx.159 (actual enrollment: 188 patients) • Any CAS (0-7) • 2:1 randomization veligrotug:placebo arm • O
Slide 15: Veligrotug experience strongly supports REVEAL pivotal program for subcutaneous VRDN-003
Viridian believes veligrotug experience strongly supports REVEAL pivotal program for subcutaneous VRDN-003 dosed Q4W and Q8W Veligrotug (IV) VRDN-003 (SC) Shared pharmacology: Veligrotug & VRDN-003 have the same binding domain VRDN-003 has the potential to have a best-in-class profile 15 Source: Vir
Slide 16: Positive THRIVE data validate the transformative potential for Viridian's TED franchise
Positive THRIVE data validate the transformative potential for Viridian’s TED franchise v Teprotumumab1 (IV) v Veligrotug 2 (IV) VRDN-0032 (SC) Mechanism Half-life extended IGF-1R partial antagonist IGF-1R full antagonist of Action IGF-1R full antagonist Treatment 8 infusions 5 infusions At-home aut
Slide 17: Viridian's TED franchise is designed to bring transformative therapies to patients
Viridian’s TED franchise is designed to bring transformative therapies to patients Current TED Market v Veligrotug VRDN-003 Primed for new Well-positioned as Potential best-in-class entrants and growth potential 2nd-to-market IV subcutaneous therapy • Lower IV burden compared with • Transformative c
Slide 18: Multiple meaningful catalysts expected across Viridian's TED and FcRn portfolios
Multiple meaningful catalysts expected across Viridian’s TED and FcRn portfolios Phase 3 topline Veligrotug BLA Potential PDUFA data for THRIVE-2 Intravenous submission date & launch in chronic TED Thyroid Eye Year-End 2024 2H 2025 2H 2026 Disease (anti-IGF-1R) Phase 3 topline data for Franchise BLA
Slide 19: Thank you to the TED community
Thank you to the TED community: patients, advocates, investigators, research staff, and partners who made this trial a success 19
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