Viridian Therapeutics, Inc. · NASDAQ:VRDN

Viridian Therapeutics — Technical Presentation (December 2024)

18 slides from Viridian Therapeutics's technical presentation, Dec 16, 2024

  1. Slide 1: Veligrotug THRIVE-2 Topline Results

    Veligrotug THRIVE-2 Topline Results December 16, 2024

  2. 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,” “become,” “continue,” “could,” “design,” “estimate,” “expect,” “intend,” “may,” “mi

  3. Slide 3: THRIVE-2 topline results of veligrotug in chronic TED build on robust & consistent clinical activity

    THRIVE-2 topline results of veligrotug in chronic TED build on robust & consistent clinical activity observed in THRIVE ACTIVE TED CHRONIC TED  Achieved all primary and secondary endpoints with high level of significance Topline data today  Rapid onset of treatment effect  Generally well-tolerate

  4. Slide 4: THRIVE-2: Demonstrated robust and consistent clinical activity in the largest and broadest TED phase 3 study

    THRIVE-2: Demonstrated robust and consistent clinical activity in the largest and broadest TED phase 3 study to date Achieved all primary and secondary endpoints with high level of statistical significance in largest IGF-1R antibody study in TED to date Rapid onset of treatment effect, with statisti

  5. Slide 5: THRIVE-2 is a phase 3 randomized, controlled, double-masked trial of veligrotug in chronic TED

    THRIVE-2 is a phase 3 randomized, controlled, double-masked trial of veligrotug in chronic 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 = 125 Primary efficacy

  6. Slide 6: THRIVE-2 baseline characteristics were well-balanced between active and placebo arms

    THRIVE-2 baseline characteristics were well-balanced between active and placebo arms Veligrotug Placebo (n = 125) (n = 63) Age in years, mean (SD) 50.5 (13.5) 50.7 (12.0) Participant Female sex, n (%) 95 (76%) 46 (73%) Demographics White race, n (%) 94 (75%) 48 (76%) Months since TED onset, mean (SD

  7. Slide 7: THRIVE-2 met all primary and secondary endpoints at 15 weeks with high level of statistical significance

    THRIVE-2 met all primary and secondary endpoints at 15 weeks with high level of statistical significance Veligrotug Placebo p-value (n=125) (n=63) Primary Endpoint: 56% 8% p < 0.0001 Proptosis responder rate (exophthalmometry)1 Proptosis Proptosis mean change from baseline (exophthalmometry) -2.34 m

  8. Slide 8: Statistically significant proptosis responder rate at all time points, including at 3 weeks

    Statistically significant proptosis responder rate at all time points, including at 3 weeks, after just one infusion of veligrotug Proptosis Responder Rate Proptosis Mean Change from Baseline Proptosis Mean Reduction (mm) Proptosis Responder Rate (%) 100 0 -0.46 -0.62 -0.72 -0.69 80 -0.75 56% -1 60

  9. Slide 9: THRIVE-2 is the first phase 3 study in chronic TED to demonstrate significant diplopia response & resolution

    THRIVE-2 is the first phase 3 study in patients with chronic TED to demonstrate statistically significant diplopia response & resolution Diplopia Responder Rate Diplopia Complete Resolution Diplopia Responder Rate (%) Diplopia Resolution Rate (%) 100 100 80 80 52% 55% 56% 60 47% 60 36% 33% 32% 40 29

  10. Slide 10: THRIVE-2 demonstrated consistency between Hertel exophthalmometry and MRI / CT as measurements of proptosis

    THRIVE-2 demonstrated consistency between Hertel exophthalmometry and MRI / CT as measurements of proptosis Hertel exophthalmometry MRI / CT Veligrotug Placebo Veligrotug Placebo (n=125) (n=63) (n=125) (n=63) Proptosis responder rate Proptosis responder rate 56% 8% 48% 3% at week 15 at week 15 Propt

  11. Slide 11: Veligrotug was generally well-tolerated and 94% of veligrotug-treated patients completed their treatment course

    Veligrotug was generally well-tolerated and 94% of veligrotug-treated patients completed their treatment course Veligrotug Placebo N=125 N=63 n (%) n (%) Participants with any treatment-emergent 106 (85%) 43 (68%) • Vast majority of TEAEs in adverse event (TEAE) both arms were mild Participants with

  12. Slide 12: Veligrotug was generally well-tolerated, with a 9.6% placebo-adjusted rate of hearing impairment AEs

    Veligrotug was generally well-tolerated, with a 9.6% placebo-adjusted rate of hearing impairment AEs Veligrotug Placebo AEs occurring at ≥10% frequency in N=125 N=63 either arm n (%) n (%) Muscle spasms 45 (36%) 4 (6%) Headache 18 (14%) 8 (13%) Hearing impairment1 16 (13%) 2 (3%) Fatigue1 15 (12%) 5

  13. Slide 13: THRIVE-2: Demonstrated robust and consistent clinical activity in the largest and broadest TED phase 3 study

    THRIVE-2: Demonstrated robust and consistent clinical activity in the largest and broadest TED phase 3 study to date Achieved all primary and secondary endpoints with high level of statistical significance in largest IGF-1R antibody study in TED to date Rapid onset of treatment effect, with statisti

  14. Slide 14: Veligrotug is well-positioned to become the treatment-of-choice for active & chronic TED

    Veligrotug is well-positioned to become the treatment-of-choice for active & chronic TED, with BLA submission expected in 2025 Active & chronic data Robust clinical responses Significant clinical activity on in BLA submission across all primary & secondary diplopia resolution & response endpoints Su

  15. Slide 15: Veligrotug's differentiated profile expected to drive rapid commercial adoption in TED, if approved

    Veligrotug’s differentiated profile expected to drive rapid commercial adoption in TED, if approved Large & Growing Market Focused Footprint • ~$2B single-product market in U.S.1 • Narrow and well-defined call point supports • Tepro launch as first entrant: $166M net sales in first small, efficient

  16. Slide 16: Viridian experience with veligrotug supports ongoing VRDN-003 development

    Viridian experience with veligrotug supports ongoing VRDN-003 development v Veligrotug VRDN-003 Veligrotug & VRDN-003 share the same binding domain Half life extension technology THRIVE & REVEAL clinical programs share key features and operations Robust and consistent Actively dosing clinical respon

  17. Slide 17: Multiple meaningful catalysts expected across Viridian's portfolio in 2025

    Multiple meaningful catalysts expected across Viridian’s portfolio in 2025, including BLA submission of veligrotug Phase 3 topline Phase 3 topline Veligrotug BLA Potential PDUFA data for THRIVE data for THRIVE-2 Intravenous submission date & launch in active TED in chronic TED Thyroid Eye September

  18. Slide 18: Viridian is building a leadership position in autoimmune disease

    Viridian is building a leadership position in autoimmune disease Revenue-Funded Growth Commercial Transition v v v 2027+ v v BLA Submission & Commercial VRDN-003 Launch** 2026 VRDN-003 BLA Active & chronic TED Readiness v Submission* Advance Potential Best-in-Class Veligrotug BLA Veligrotug Launch**

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