Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Investor Presentation (October 2025)
56 slides from Viridian Therapeutics's investor presentation, October 2025
Slide 1: Corporate Presentation
Corporate Presentation October 2025
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
Slide 3: Building upon proven first market entrants to develop differentiated next-generation products
Viridian is building upon proven first market entrants to develop differentiated next-generation products Identify market opportunities with clear remaining unmet need Determine key areas of potential First-generation product product differentiation establishes significant opportunity for next-gener
Slide 4: Differentiated pipeline: TED portfolio and FcRn inhibitor portfolio
Differentiated pipeline: TED portfolio moving towards commercial and FcRn inhibitor portfolio continues to progress DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 STATUS BLA submission Veligrotug planned Thyroid Eye Intravenous imminently Disease (anti–IGF-1R) Portfolio VRDN-003 Pivotal trials fully
Slide 5: Viridian is well positioned to deliver significant catalysts
Viridian is well positioned to deliver significant catalysts Anticipated Catalysts • Positive THRIVE and THRIVE-2 topline data in active and chronic TED showed a robust clinical profile1 BLA submission: Imminently Veligrotug • Strong durability of proptosis response in THRIVE EU MAA submission: 1H 2
Slide 6: Building a leadership position in autoimmune disease
Viridian is building a leadership position in autoimmune disease Revenue-Funded Growth Commercial Transition BLA and MAA Submission & 2027+ Commercial Readiness 2026 VRDN-003 v BLA Launch** Submission* Active & Chronic TED Veligrotug v Advance Potential BLA EU MAA Launch** Best-in-Class Submission*
Slide 7: Thyroid Eye Disease (TED) Portfolio
Thyroid Eye Disease (TED) Portfolio 7
Slide 8: TED is an autoimmune condition affecting tissues around the eyes
TED is an autoimmune condition characterized by inflammation, growth, and damage to tissues around and behind the eyes Normal Eye Anatomy Thyroid Eye Disease (TED) Autoantibodies trigger IGF-1R/TSHR pathway1 Bulging Eyes Heterogeneous autoimmune disease with clinical signs and symptoms that can vary
Slide 9: Developing an IGF-1R antibody portfolio to transform TED treatment
Viridian is developing an IGF-1R antibody portfolio with the potential to transform the treatment for people living with TED VRDN-003 Veligrotug Subcutaneous and potential Teprotumumab best-in-class therapy Steroids/ Phase 3 clinical data shows a robust Q8W or Q4W dosing; clinical profile1 in a new
Slide 10: Positive THRIVE and THRIVE-2 results support veligrotug and VRDN-003 development
Positive THRIVE and THRIVE-2 results support the transformative potential of veligrotug and ongoing VRDN-003 development Current TED Market v Veligrotug VRDN-003 Primed for new Well-positioned to become the Subcutaneous and potential entrants and growth IV treatment-of-choice in TED best-in-class th
Slide 11: Veligrotug
Veligrotug Intravenous anti–IGF-1R 11
Slide 12: Veligrotug met all endpoints in two phase 3 trials, THRIVE and THRIVE-2
Veligrotug met all primary and secondary endpoints with statistical significance in two phase 3 trials, THRIVE and THRIVE-2 Topline results reported September 2024 Topline results reported December 2024 Met all primary & secondary endpoints Met all primary & secondary endpoints ACTIVE TED CHRONIC TE
Slide 13: THRIVE: robust clinical activity in active TED patients
THRIVE: Veligrotug showed robust and consistent clinical activity in active TED patients Achieved all primary and secondary endpoints with high level of statistical significance (p < 0.0001) Rapid onset of treatment effect in as few as 3 weeks Generally well-tolerated, with no treatment-related SAEs
Slide 14: THRIVE-2: robust clinical activity in the largest 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 statistical significance in largest IGF-1R antibody study in TED to date Rapid onset of treatment effect, with statistically signific
Slide 15: Veligrotug well-positioned to become treatment-of-choice for active and 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 & diplopia resolution & response secondary endpoints Su
Slide 16: Robust clinical profile expected to drive rapid commercial adoption in TED
Veligrotug’s robust clinical 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 small, efficient sales
Slide 17: VRDN-003
VRDN-003 Subcutaneous half-life extended anti–IGF-1R 17
Slide 18: Positive phase 3 veligrotug data support ongoing VRDN-003 development
Positive phase 3 data for veligrotug in active and chronic TED support 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 Fully
Slide 19: Later-entrant SC therapies expand the market and take share from incumbent IV
Later-entrant SC therapies have demonstrated ability to expand the market and take market share from incumbent IV IV to SC with same molecule IV to SC with new SC entrant IV Drug SC Drug IV Drug SC Drug CD38 CD20 IV Launch: Nov 2015 by SC Launch: May 2020 IV Launch: Mar 2017 SC Launch: Aug 2020 J&J
Slide 20: VRDN-003 designed to bring a potentially best-in-class therapy
VRDN-003 designed to bring a potentially best-in-class therapy for patients Teprotumumab IV 1 VRDN-003 Autoinjector Potential VRDN-003 Benefits2 8 INFUSIONS Phase 3 pivotal program is Easy self-administration transforms administered every 3 weeks evaluating two dosing regimens: patient convenience 3
Slide 21: PK model shows Q4W and Q8W VRDN-003 achieves predicted exposure levels
PK model shows Q4W and Q8W dosing of VRDN-003 SC achieves predicted exposure levels of veligrotug at 3-10 mg/kg Subcutaneous VRDN-003 Pharmacokinetic (PK) Modeling • Veligrotug exposures modeled from a phase 2 TED clinical trial inform the exposure ranges anticipated to produce clinical benefit – Tw
Slide 22: Enrollment complete in ongoing phase 3 trials for VRDN-003
Enrollment complete in ongoing phase 3 clinical trials for VRDN-003 ACTIVE TED CHRONIC TED Key Inclusion Criteria Key Inclusion Criteria • Proptosis of ≥3 mm • Proptosis of ≥3 mm • CAS ≥3 • Any CAS (0–7) • Onset of TED symptoms within 15 months • Onset of TED symptoms >15 months Trial Design Trial D
Slide 23: REVEAL-1 & REVEAL-2 trial designs
REVEAL-1 & REVEAL-2 will evaluate Q4W and Q8W active arms of VRDN-003 versus placebo control Treatment Phase (20 weeks treatment with primary endpoint at 24 weeks) Treatment Arms D11 W4 W8 W12 W16 W20 W24 Through W52 (1:1:1) Primary Endpoint VRDN-003 Q4W1 Analysis Primary efficacy endpoint: Addition
Slide 24: FcRn Inhibitor Portfolio
MEVIRIDIAN FcRn Inhibitor Portfolio
Slide 25: Pathogenic autoantibodies drive disease in autoimmune diseases
Pathogenic autoantibodies drive disease pathophysiology in a number of autoimmune diseases FcRn-Mediated Recycling of IgGs, Including Pathogenic Autoantibodies1 Pathogenic autoantibodies cause inflammation and damage to healthy 1 tissues and cells, driving the pathology of autoimmune diseases1 2 Ser
Slide 26: Viridian's FcRn inhibitors aim to reduce pathogenic autoantibodies
Viridian’s portfolio of FcRn inhibitors aims to reduce circulating levels of pathogenic autoantibodies by blocking FcRn Inhibition of FcRn Reduces IgGs, VRDN-006 Including Pathogenic Autoantibodies1 Fc fragment that blocks IgG from binding to FcRn 1 Fc fragment 2 3 4 VRDN-008 Binds to albumin and Fc
Slide 27: FcRn inhibitors are a large market opportunity
FcRn inhibitors are a large market opportunity; market size of MG and CIDP alone are projected to be over $11B by 2030 Projected WW MG and CIDP FcRn Market1 ...with Potential in Additional Autoimmune Indications $11.2B Graves’ Myositis Disease $2.5B Lupus Sjogren’s Nephritis Syndrome 2024 2030 Sourc
Slide 28: FcRn portfolio potential to capture market share in autoimmune indications
Viridian’s FcRn portfolio has the potential to capture significant market share in autoimmune indications v VRDN-006 v VRDN-008 Highly Selective Fc Fragment and FcRn Inhibitor Half-life Extended Bispecific FcRn Inhibitor IgG • IgG reduction data consistent with the FcRn • Deeper and more sustained r
Slide 29: Appendix
Appendix 1) THRIVE in Active TED Pivotal Data 2) THRIVE-2 in Chronic TED Pivotal Data 3) VRDN-003 Phase 1 Data 4) FcRn Non-Human Primate Data 29
Slide 30: THRIVE in Active TED
THRIVE in Active TED Global phase 3 clinical trial pivotal data 30
Slide 31: THRIVE phase 3 trial design 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 32: THRIVE baseline characteristics
THRIVE baseline characteristics were well-balanced between active and placebo arms 7.8 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 (S
Slide 33: THRIVE achieved statistical significance across all endpoints at 15 weeks
THRIVE achieved high level of statistical significance across all primary and 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.
Slide 34: Proptosis responder rate primary endpoint met
Primary endpoint of proptosis responder rate met at 15 weeks: 70% for patients receiving veligrotug compared with 5% on PBO 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% 64% -
Slide 35: Diplopia resolution and CAS at week 15
Majority of patients receiving veligrotug had complete resolution of diplopia and minimal disease activity (CAS) at week 15 Diplopia Complete Resolution CAS Score 0 or 1 Diplopia Resolution Rate (%) 100 100 CAS 0 or 1 Rate (%) 80 80 63% 64% 64% 60 Diplopia 54% Responder 60 53% 44% Rate at 46% Week 1
Slide 36: Consistency between Hertel and MRI / CT measurements
THRIVE demonstrated consistency between Hertel and MRI / CT and validates both as reliable tools for measurements of proptosis Hertel Exophthalmometry MRI / CT Veligrotug Placebo Veligrotug Placebo (n=75) (n=38) (n=75) (n=38) Proptosis responder rate Proptosis responder rate 70% 5% 69% 9% at week 15
Slide 37: Veligrotug well-tolerated at week 15, no treatment-related SAEs
Veligrotug was generally well-tolerated at week 15, 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 • Vast majority of TEAEs in 66 (88%) 24 (63%) adverse event (TEAE) both arm
Slide 38: Adverse events and hearing impairment rate at week 15
Veligrotug was generally well-tolerated at week 15, with a 5.5% placebo-adjusted rate of hearing impairment AEs Veligrotug Placebo AEs occurring at ≥10% frequency in either arm N=75 N=38 n (%) n (%) Muscle spasms 32 (43%) 2 (5%) Headache 16 (21%) 5 (13%) Infusion related reaction (IRR) 13 (17%) 1 (3
Slide 39: 70% of proptosis responders maintained response at Week 52
70% of proptosis responders in THRIVE maintained response at Week 52 in long-term follow up Proptosis Durability Safety Resolution of Week 15 proptosis • No changes to veli’s safety profile during the 70% responders maintained a proptosis response at follow-up period • Vast majority of adverse event
Slide 40: THRIVE-2 in Chronic TED
THRIVE-2 in Chronic TED Global phase 3 clinical trial pivotal data 40
Slide 41: THRIVE-2 phase 3 trial design 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
Slide 42: THRIVE-2 baseline characteristics
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
Slide 43: THRIVE-2 met all primary and secondary endpoints at 15 weeks
THRIVE-2 met all primary and secondary endpoints at 15 weeks 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 mm -0.46 mm p < 0.0001 Diplopia responder rat
Slide 44: Statistically significant proptosis responder rate at all time points
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 -0.75 80 56% -1 60
Slide 45: First phase 3 chronic TED study with significant diplopia response and 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
Slide 46: Consistency between Hertel exophthalmometry and MRI / CT
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
Slide 47: Veligrotug well-tolerated; 94% completed 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 • Vast majority of TEAEs in 106 (85%) 43 (68%) adverse event (TEAE) both arms were mild Participants wit
Slide 48: Adverse events and hearing impairment rate in THRIVE-2
Veligrotug was generally well-tolerated, with a 9.6% placebo-adjusted rate of hearing impairment AEs Veligrotug Placebo AEs occurring at ≥10% frequency in either arm N=125 N=63 n (%) n (%) Muscle spasms 45 (36%) 4 (6%) Headache 18 (14%) 8 (13%) Hearing impairment1 16 (13%) 2 (3%) Fatigue1 15 (12%) 5
Slide 49: VRDN-003 Phase 1 Data
MEVIRIDIAN VRDN-003 Phase 1 Data
Slide 50: Phase 1 HV study: VRDN-003 extended half-life of 40-50 days
Phase 1 HV Study: Subcutaneous VRDN-003 showed an extended half-life of 40–50 days and sustained IGF-1 levels after dosing Phase 1 HV Pharmacokinetics (PK) Phase 1 HV Pharmacodynamics (PD) VRDN-003 SC (300 mg single dose) VRDN-003 SC (300 mg single dose) VRDN-003 SC (600 mg single dose) VRDN-003 SC
Slide 51: Phase 1 HV study: subcutaneous VRDN-003 was well-tolerated
Phase 1 HV Study: Subcutaneous VRDN-003 was well-tolerated VRDN-003 Single Dose SC Two Doses SC Placebo (n = 12) (n = 4) (n = 6) All Observed AEs 9 (n = 3) 2 (n = 2) 2 (n = 2) AEs deemed to be related to VRDN-003 3 1 -- • No hearing-related AEs Injection Site Reactions (ISRs)1 1 (8%) -- -- • No trea
Slide 52: FcRn Non-Human Primate Data
FcRn Non-Human Primate Data 52
Slide 53: VRDN-006 in vitro and multi-dose NHP data vs efgartigimod
v VRDN-006 in vitro, multi-dose NHP PK and IgG reduction data compared to efgartigimod pH-Dependent Binding Multi-Dose NHP Pharmacokinetics Mean Flourescence Intensity 4×10 5 Dose VRDN-006 1000 Serum Concentration 3×10 5 VRDN-006 (20 mg/kg, IV) Efgartigimod pH 6 Efgartigimod (20 mg/kg, IV) WT Fc Con
Slide 54: VRDN-006 spares albumin and LDL in multi-dose NHP study
v VRDN-006 spares albumin and LDL in multi-dose NHP study VRDN-006 Spares Albumin VRDN-006 Spares LDL 150 200 Dose Dose CFB Concentrations (%) CFB Concentrations (%) 150 100 100 VRDN-006 (20 mg/kg, IV) 50 Efgartigimod (20 mg/kg, IV) VRDN-006 (20 mg/kg, IV) Vehicle 50 Efgartigimod (20 mg/kg, IV) Vehi
Slide 55: Single dose VRDN-008 longer half-life and deeper IgG reduction vs efgartigimod
v A single dose of VRDN-008 demonstrated a longer half-life, deeper and more sustained reduction of IgG vs. efgartigimod VRDN-008 Showed ~3x Longer Half-life VRDN-008 Showed ~20% Deeper and More Head-to-Head vs. Efgartigimod in NHPs Sustained IgG Reduction Head-to-Head vs. Efgartigimod in NHPs 10000
Slide 56: Single dose of VRDN-008 spares albumin and LDL in NHPs
v A single dose of VRDN-008 spares albumin and LDL in NHPs VRDN-008 Spares Albumin VRDN-008 Spares LDL 125 150 Mean % Change from Baseline Mean % Change from Baseline Albumin Concentration LDL Concentration 100 100 75 VRDN-008 (60 mg/kg, IV) VRDN-008 (60 mg/kg, IV) Efgartigimod (48.5 mg/kg, IV) Efga
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