Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Investor Presentation (June 2025)
56 slides from Viridian Therapeutics's investor presentation, June 2025
Slide 1: Corporate Presentation June 2025
Corporate Presentation June 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 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 moving towards the clinic DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 STATUS Reported positive Veligrotug topline data; BLA Thyroid Eye Intravenous planned 2H 2025 Disease (anti–IGF-1R) Portfolio VRDN-003
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: 2H 2025 • Strong durability of proptosis response in THRIVE Veligrotug EU MAA submission: 1H 2026
Slide 6: Near-term anticipated clinical and regulatory catalysts
Near-term anticipated clinical and regulatory catalysts offer potential to drive significant value Veligrotug BLA EU MAA Potential PDUFA Intravenous submission submission date & U.S. launch Thyroid Eye 2H 2025 1H 2026 2H 2026 Disease (anti–IGF-1R) Phase 3 topline data for Portfolio BLA VRDN-003 REVE
Slide 7: 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 8: Thyroid Eye Disease (TED) Portfolio
Thyroid Eye Disease (TED) Portfolio 8
Slide 9: 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 10: 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 Teprotumumab Subcutaneous and potential Steroids/ best-in-class therapy THRIVE/THRIVE-2 topline data show a Surgery Q8W or Q4W dosing; robust clinical prof
Slide 11: Positive THRIVE 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 12: Veligrotug
Veligrotug Intravenous anti–IGF-1R 12
Slide 13: Veligrotug met all endpoints in two phase 3 trials
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 14: Veligrotug well-positioned to become treatment-of-choice for 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 15: Robust clinical profile expected to drive rapid commercial adoption
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 16: THRIVE in Active TED
THRIVE in Active TED Global phase 3 clinical trial 16
Slide 17: THRIVE: robust and consistent clinical activity in active TED
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 18: THRIVE phase 3 trial design
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 19: THRIVE baseline characteristics well-balanced
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 20: THRIVE 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 21: Proptosis responder rate: 70% veligrotug vs 5% placebo
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 22: Complete resolution of diplopia and minimal 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 23: Consistency between Hertel and MRI / CT proptosis 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 24: Veligrotug generally well-tolerated, 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 25: Adverse events including hearing impairment in THRIVE
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 26: 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 27: THRIVE-2 in Chronic TED
THRIVE-2 in Chronic TED Global phase 3 clinical trial 27
Slide 28: THRIVE-2: robust clinical activity in 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 29: 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 30: THRIVE-2 baseline characteristics well-balanced
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 31: 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 32: 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 33: First phase 3 in chronic TED with 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 34: THRIVE-2 consistency between Hertel 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 35: 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 with
Slide 36: 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 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 37: VRDN-003
VRDN-003 Subcutaneous half-life extended anti–IGF-1R 37
Slide 38: Positive veligrotug phase 3 data supports VRDN-003 development
Positive phase 3 data for veligrotug in active and chronic TED 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 Curr
Slide 39: Later-entrant SC therapies expand market and take share from 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 40: VRDN-003 designed as 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 41: Phase 1 HV study: 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 42: 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 43: PK model shows Q4W and Q8W dosing achieves predicted exposure
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 44: Ongoing phase 3 trials for VRDN-003 and path to BLA
Ongoing phase 3 clinical trials for VRDN-003 and path to BLA 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 Design •
Slide 45: REVEAL-1 & REVEAL-2 evaluate Q4W and Q8W arms versus placebo
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 46: FcRn Inhibitor Portfolio
MEVIRIDIAN FcRn Inhibitor Portfolio
Slide 47: 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 48: 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 49: 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 close to $10B by 2030 Projected WW MG and CIDP FcRn Market1 ...with Potential in Additional Autoimmune Indications $9.1B Graves’ Myositis Disease $2.5B Lupus Sjogren’s Nephritis Syndrome 2024 2030 So
Slide 50: Potential best-in-class portfolio to capture autoimmune market share
Viridian’s potential best-in-class portfolio is designed 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 • Fc fragment is a clinically and commercially • Targeting more du
Slide 51: 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 52: VRDN-006 spares albumin and LDL in 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 53: Single dose VRDN-008 longer half-life and deeper IgG reduction
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 54: 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
Slide 55: Appendix
AV ax VIRIDIAN Appendix
Slide 56: Tepezza Prescribing Information durability of proptosis responders
Tepezza Prescribing Information shows 53% of proptosis responders maintained their response Tepezza Durability (Label) of Week 24 proptosis 53% responders maintained a proptosis response at (16/30 participants) Week 721 56 Source: TEPEZZA U.S. Prescribing Information. Cross-trial comparisons should
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