Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Investor Presentation (January 2026)
56 slides from Viridian Therapeutics's investor presentation, January 2026
Slide 1: Corporate Presentation
Corporate Presentation January 2026
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: Viridian aspires to be a leading autoimmune company, starting with Best in TED
Viridian aspires to be a leading autoimmune company… … starting with Best in TED In thyroid eye disease (TED), we aim to bring new treatment options to patients that address unmet needs and expand the number of treated patients 3
Slide 4: Building towards a leadership position in TED enabling expansion to other autoimmune diseases
Viridian is building towards a leadership position in TED enabling our expansion to other autoimmune diseases Revenue Funded Growth Autoimmune Thyroid 5 Yrs Commercial Company TED 2 Yrs New Pipeline INDs and POCs* FcRn Expansion* TSHR POC* IGF-1R FcRn POC* 003 Launch** TSHR IND* 003 Topline* Veli La
Slide 5: Building a portfolio to address patient needs in TED
Viridian is building a portfolio to address patient needs in TED with veligrotug, elegrobart (VRDN-003), and targeted pipeline expansion Current TED Elegrobart TSHR Inhibitor Market v Veligrotug (VRDN-003) & Pipeline Primed for new Compelling profile with Subcutaneous and Innovate for the entrants a
Slide 6: Viridian has a proven track record of execution
Viridian has a proven track record of execution Granted 2 >1200 TED veligrotug regulatory BLA submitted patients enrolled in designations in 1200 Viridian clinical trials since January 1 during the US government 2 2025: Priority Review, shutdown 2024 Breakthrough Therapy Secured access to 8 phase 3
Slide 7: Strong and expanded TED portfolio and continued progress across FcRn inhibitors
Strong and expanded TED portfolio and continued progress across FcRn inhibitors Anticipated DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Milestones PDUFA target Veligrotug date June 30, Anti-IGF-1R; Intravenous 2026 Thyroid Eye Elegrobart Disease Topline data in Q1 (VRDN-003) Portfolio and Q2 2026
Slide 8: Veligrotug PDUFA date of June 30th and multiple additional catalysts in 2026
Veligrotug PDUFA date of June 30th and multiple additional anticipated value-creating catalysts portfolio-wide in 2026 2025 2026 BLA Accepted Veligrotug Dec 2025 ✓ PDUFA target Intravenous MAA Submission June 30, 2026 Q1 2026 Elegrobart REVEAL-1 Topline REVEAL-2 Topline (VRDN-003) Q1 2026 Q2 2026 Su
Slide 9: Veligrotug
Veligrotug Intravenous anti–IGF-1R 9
Slide 10: TED is an autoimmune condition characterized by inflammation, growth, and damage to tissues
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 11: Veligrotug granted Priority Review by the US FDA, PDUFA target date June 30, 2026
Veligrotug granted Priority Review by the US FDA, with PDUFA target date of June 30, 2026 Topline results reported September 2024 Topline results reported December 2024 Met all primary & secondary endpoints Met all primary & secondary endpoints ACTIVE TED CHRONIC TED Key Inclusion Criteria Key Inclu
Slide 12: THRIVE: robust and consistent clinical activity in active TED
THRIVE: Veligrotug showed robust and consistent clinical activity in active TED patients Achieved the primary and all 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
Slide 13: THRIVE-2: robust 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 completed to date Achieved the primary and all secondary endpoints with statistical significance in largest IV IGF-1R antibody study in TED to date Rapid onset of treatment effect, with stati
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 Active & chronic data Robust clinical responses Significant clinical activity on in BLA submission across all primary & diplopia resolution & response secondary endpoints Supported by one of the largest First pi
Slide 15: Veligrotug's 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 16: Launch preparation prioritizes stakeholder relationships and focus on patients
Viridian’s launch preparation prioritizes strong external stakeholder relationships and focus on patients Launch preparations are well underway with experienced leadership teams in place Field Medical Field Sales Patient Services Market Access Leadership in place, Leadership in place, Leadership in
Slide 17: Elegrobart (VRDN-003)
Elegrobart (VRDN-003) Subcutaneous half-life extended anti–IGF-1R 17
Slide 18: Positive phase 3 veligrotug data support ongoing elegrobart development
Positive phase 3 data for veligrotug in active and chronic TED support ongoing elegrobart development v Veligrotug Elegrobart Veligrotug & elegrobart share the same binding domain Half-life extension technology THRIVE & REVEAL clinical programs share key features and operations Robust and consistent
Slide 19: Later-entrant SC therapies have demonstrated ability to expand the market
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: Elegrobart designed to bring a potentially best-in-class therapy for patients
Elegrobart designed to bring a potentially best-in-class therapy for patients Teprotumumab IV 1 Elegrobart Autoinjector Potential Elegrobart Benefits2 8 INFUSIONS Phase 3 pivotal program is Infrequent administration administered every 3 weeks evaluating two dosing regimens: & low volume 3 SC Treatme
Slide 21: PK model shows Q4W and Q8W dosing achieves predicted exposure levels
PK model shows Q4W and Q8W dosing of elegrobart SC achieves predicted exposure levels of veligrotug at 3-10 mg/kg Subcutaneous Elegrobart Pharmacokinetic (PK) • Veligrotug exposures modeled from a phase 2 Modeling TED clinical trial inform the exposure ranges anticipated to produce clinical benefit
Slide 22: Enrollment complete in ongoing phase 3 clinical trials for elegrobart
Enrollment complete in ongoing phase 3 clinical trials for elegrobart 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
Slide 23: REVEAL-1 & REVEAL-2 will evaluate Q4W and Q8W elegrobart versus placebo
REVEAL-1 & REVEAL-2 will evaluate Q4W and Q8W active arms of elegrobart versus placebo 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 Elegrobart Analysis (Every 4 Weeks)1 Primary efficacy endpoint:
Slide 24: FcRn Inhibitor Portfolio
MEVIRIDIAN FcRn Inhibitor Portfolio
Slide 25: Pathogenic autoantibodies drive disease pathophysiology 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: Portfolio of FcRn inhibitors aims to reduce circulating 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; MG and CIDP projected over $11B by 2030
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 has potential to capture significant market share
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) Elegrobart (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 is a phase 3 randomized, controlled, double-masked trial 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 were well-balanced between arms
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 high 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: Primary endpoint of proptosis responder rate met: 70% 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 35: Majority of veligrotug patients had complete diplopia resolution and minimal CAS
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% Diplopia 60 54% Responder 60 53% 44% Rate at 46% Week 1
Slide 36: THRIVE demonstrated consistency between Hertel and MRI / CT
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 generally 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: Veligrotug well-tolerated at week 15, 5.5% placebo-adjusted hearing impairment AEs
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 is a phase 3 randomized, controlled, double-masked trial 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 were well-balanced between 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
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 study in chronic TED to show 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
Slide 46: THRIVE-2 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 generally 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: 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 49: Elegrobart (VRDN-003) Phase 1 Data
Elegrobart (VRDN-003) Phase 1 Data 49
Slide 50: Phase 1 HV Study: elegrobart extended half-life of 40–50 days
Phase 1 HV Study: Subcutaneous elegrobart 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 S
Slide 51: Phase 1 HV Study: subcutaneous elegrobart was well-tolerated
Phase 1 HV Study: Subcutaneous elegrobart was well-tolerated Elegrobart 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 elegrobart 3 1 -- • No hearing-related AEs Injection Site Reactions (ISRs)1 1 (8%) -- -- • N
Slide 52: FcRn Non-Human Primate Data
FcRn Non-Human Primate Data 52
Slide 53: VRDN-006 in vitro, multi-dose NHP PK and IgG reduction data compared to 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 of VRDN-008 showed 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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