Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Investor Presentation (November 2024)
42 slides from Viridian Therapeutics's investor presentation, Nov 12, 2024
Slide 1: Corporate Presentation November 2024
Corporate Presentation November 2024 Exhibit 99.2
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,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “might,” “plan,” “poten
Slide 3: First-generation product establishes significant opportunity for next-generation strategy
First-generation product establishes significant opportunity for next-generation strategy Identify market opportunities with clear remaining unmet need Determine key areas of potential product differentiation Engineer potential best-in-class antibodies and therapeutic proteins Rapidly advance progra
Slide 4: Differentiated pipeline: late-stage TED and preclinical FcRn portfolios
DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Thyroid Eye Disease (anti–IGF-1R) Portfolio Veligrotug Intravenous VRDN-003 Subcutaneous FcRn-Targeting Autoimmune Portfolio VRDN-006 FcRn-targeting Fc fragment VRDN-008 Bispecific, extended half-life FcRn inhibitor Differentiated pipeline: late-stage TE
Slide 5: Significant progress in Q3 2024
Significant progress in Q3 2024 Veligrotug Intravenous Reported THRIVE topline data in active TED in September: veligrotug achieved all primary & secondary endpoints with high levels of statistical significance (p < 0.0001) and was generally well-tolerated THRIVE-2 in chronic TED: completed and exce
Slide 6: Thyroid Eye Disease (TED) Portfolio
Thyroid Eye Disease (TED) Portfolio
Slide 7: TED is an autoimmune condition characterized by inflammation and tissue damage around the eyes
TED is an autoimmune condition characterized by inflammation, growth, and damage to tissues around and behind the eyes Normal Eye Anatomy Bulging Eyes Enlargement of extraocular muscles Optic Nerve Autoantibodies trigger IGF-1R/TSHR pathway1 Heterogeneous autoimmune disease with clinical signs and s
Slide 8: The only approved mAb treatment for TED targets IGF-1R
In teprotumumab2,3 clinical studies: Majority of AEs were mild AEs are generally transient & reversible Interviewed treating physicians cite comfort with managing AEs4 The only approved mAb treatment for TED targets IGF-1R Sources: 1 Teprotumumab Prescribing Information, 2 Douglas RS et al. NEJM. 20
Slide 9: Viridian is 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 of patients with TED BLA = Biologics License Application, IGF-1R = insulin-like growth factor-1 receptor, IV = intravenous, Q4W = every 4 weeks, Q8W = every 8 weeks, TED = thyroid eye disease. Steroids/
Slide 10: Transformative potential of Viridian's TED portfolio
Veligrotug2 (IV) Teprotumumab1 (IV) VRDN-0032 (SC) Mechanism of Action IGF-1R partial antagonist IGF-1R full antagonist Half-life extended IGF-1R full antagonist Treatment Regimen 8 infusions given every 3 weeks 5 infusions given every 3 weeks At-home autoinjector: dosed every 4 or 8 weeks Dose 20 m
Slide 11: Viridian's TED portfolio is designed to bring transformative therapies to patients
Current TED Market Transformative convenience of at-home autoinjector every 4 or 8 weeks2 Designed to replicate veligrotug clinical profile, including rapid onset of action2 BLA submission anticipated in the year following veligrotug BLA Potential to greatly expand TED market, if approved Lower IV b
Slide 12: Later-entrant therapies have demonstrated ability to take market share from incumbent IV
Later-entrant therapies have demonstrated ability to take market share from incumbent IV and expand the market Significant opportunity for 2nd to market IV and potential best-in-class SC therapies in TED Third party trademarks used are the property of their respective owners. Sources: 1 Azhar A et a
Slide 13: Veligrotug Intravenous anti–IGF-1R
Veligrotug Intravenous anti–IGF-1R
Slide 14: THRIVE: Veligrotug showed robust and consistent clinical activity in active TED
THRIVE: Veligrotug showed robust and consistent clinical activity in active TED patients, with a favorable dosing regimen AE = adverse event, IGF-1R = insulin-like growth factor-1 receptor, SAE = serious adverse event, TED = thyroid eye disease. Achieved all primary and secondary endpoints with high
Slide 15: THRIVE is a phase 3 randomized, controlled, double-masked trial in active TED
Treatment Phase (12-week treatment period with primary endpoint at 15 weeks) Veligrotug n = 75 THRIVE is a phase 3 randomized, controlled, double-masked trial of veligrotug in active TED D = day, TED = thyroid eye disease, W = week. D1 W3 W6 W9 W12 Placebo n = 38 Key: Veligrotug 10 mg/kg Placebo W15
Slide 16: Baseline characteristics were well-balanced between active and placebo arms
Baseline characteristics were well-balanced between active and placebo arms Veligrotug (n = 75) Placebo (n = 38) Participant Demographics Age in years, mean (SD) 48.9 (12.4) 49.1 (12.5) Female sex, n (%) 56 (75%) 31 (82%) White race, n (%) 51 (68%) 19 (50%) Disease Characteristics Months since TED o
Slide 17: THRIVE achieved high statistical significance across all endpoints at 15 weeks
THRIVE achieved high level of statistical significance across all primary & secondary endpoints at 15 weeks Source: Viridian THRIVE data on file. 1 Percentage of participants with ≥2 mm reduction in proptosis from baseline in the study eye, without deterioration in the fellow eye (≥2 mm increase), 2
Slide 18: Primary endpoint of proptosis responder rate met
Primary endpoint of proptosis responder rate met at 15 weeks: 70% for patients receiving veligrotug compared with 5% on placebo Proptosis Responder Rate Analysis Visits Proptosis Responder Rate (%) 53% 64% 64% 72% 70% Veligrotug Proptosis Mean Change from Baseline Analysis Visits Proptosis Mean Redu
Slide 19: Majority of patients had complete resolution of diplopia and minimal disease activity
Majority of patients receiving veligrotug had complete resolution of diplopia and minimal disease activity at week 15 Diplopia Complete Resolution Analysis Visits Diplopia Resolution Rate (%) 26% 32% 54% Veligrotug CAS Score 0 or 1 CAS 0 or 1 Rate (%) 7% 25% 46% 24% 20% 64% 18% 64% Analysis Visits V
Slide 20: High consistency between Hertel exophthalmometry and MRI / CT measurements
THRIVE data showed high consistency between Hertel exophthalmometry and MRI / CT measurements of proptosis Hertel exophthalmometry MRI / CT THRIVE represents the largest IGF-1R antibody study in TED to date and validates both exophthalmometry and MRI / CT as reliable tools for measurement of proptos
Slide 21: 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 N=75 n (%) Placebo N=38 n (%) Participants with any treatment-emergent adverse event (TEAE) 66 (88%) 24 (63%) Participants with any serious AE (SAE) 4 (5%)1
Slide 22: 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 Source: Viridian THRIVE data on file. 1 Terms aggregated utilizing methodology used by FDA for approved products for treatment of TED, 2 Reported as percentage of menstruating women. AE = adverse eve
Slide 23: THRIVE-2 in chronic TED on track for topline December 2024
CAS = clinical activity score, TED = thyroid eye disease. Enrollment completed in July; actual enrollment of 188 patients exceeded target by nearly 20% Key Inclusion Criteria Proptosis of ≥3 mm Any CAS (0–7) Onset of TED symptoms >15 months Trial Design N = approx.159 (actual enrollment: 188 patient
Slide 24: VRDN-003 Subcutaneous half-life extended anti–IGF-1R
VRDN-003 Subcutaneous half-life extended anti–IGF-1R
Slide 25: Veligrotug experience 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 Source: Viridi
Slide 26: VRDN-003 designed to bring a potentially best-in-class therapy
VRDN-003 designed to bring a potentially best-in-class therapy for patients Potential for reduced treatment burden to patients Source: 1 Teprotumumab Prescribing Information. IGF-1R = insulin-like growth factor-1 receptor, IV = intravenous, Q4W = every 4 weeks, Q8W = every 8 weeks, SC = subcutaneous
Slide 27: Phase 1 HV Study: VRDN-003 showed extended half-life of 40–50 days
Phase 1 HV Pharmacodynamics (PD) Phase 1 HV Pharmacokinetics (PK) Phase 1 HV Study: Subcutaneous VRDN-003 showed an extended half-life of 40–50 days and sustained IGF-1 levels after dosing Source: Preliminary Viridian clinical data on file as of April 12, 2024 data cut. Multi-dose cohort was a 600 m
Slide 28: Phase 1 HV Study: Subcutaneous VRDN-003 was well-tolerated
VRDN-003 Single Dose SC (n = 12) Two Doses SC (n = 4) Placebo (n = 6) All Observed AEs 9 (n = 3) 2 (n = 2) 2 (n = 2) AEs deemed to be related to VRDN-003 3 1 -- Injection Site Reactions (ISRs)1 1 (8%) -- -- Muscle Spasms -- -- -- Hyperglycemia -- 1 (25%) -- Hearing Impairment1 -- -- -- Insomnia 1 (8
Slide 29: Subcutaneous VRDN-003 Pharmacokinetic Modeling
Subcutaneous VRDN-003 Pharmacokinetic (PK) Modeling PK model shows Q4W and Q8W dosing of VRDN-003 SC achieves key exposure levels between 3–10 mg/kg of veligrotug IV VRDN-003 dosing regimens achieve veligrotug exposures already shown to be clinically active Veligrotug IV showed robust clinical activ
Slide 30: Ongoing phase 3 clinical trials for VRDN-003 and path to BLA
Ongoing phase 3 clinical trials for VRDN-003 and path to BLA REVEAL trials expected to deliver topline results in 1H 2026 to support BLA filing by year-end 2026 CHRONIC TED Key Inclusion Criteria Proptosis of ≥3 mm Any CAS (0–7) Onset of TED symptoms >15 months Trial Design N = 126 24-week primary e
Slide 31: REVEAL-1 & REVEAL-2 trial design
Treatment Phase (20 weeks treatment with primary endpoint at 24 weeks) VRDN-003 Q4W REVEAL-1 & REVEAL-2 will evaluate Q4W and Q8W active arms of VRDN-003 versus placebo control 1 600 mg loading dose given as two 300 mg injections. 2 Placebo injections administered at alternating study visits to main
Slide 32: FcRn Inhibitor Portfolio
FcRn Inhibitor Portfolio
Slide 33: Pathogenic autoantibodies drive disease pathophysiology in autoimmune diseases
Pathogenic autoantibodies drive disease pathophysiology in a number of autoimmune diseases Pathogenic autoantibodies cause inflammation and damage to healthy tissues and cells, driving the pathology of autoimmune diseases1 Serum levels of pathogenic autoantibodies are maintained, in part, by FcRn-me
Slide 34: Viridian's portfolio of FcRn inhibitors aims to reduce pathogenic autoantibodies
Viridian’s portfolio of FcRn inhibitors aims to reduce circulating levels of pathogenic autoantibodies by blocking FcRn Fc fragment that blocks IgG from binding to FcRn Binds to albumin and FcRn for a more sustained reduction of pathogenic autoantibodies Inhibition of FcRn Reduces IgGs, Including Pa
Slide 35: FcRn inhibitors are a large market opportunity
Projected WW MG and CIDP FcRn Market1 FcRn inhibitors are a large market opportunity; market size of MG and CIDP alone are projected to be close to $10B by 2030 ...with Potential in Additional Autoimmune Indications Sjogren’s Syndrome Lupus Nephritis Myositis Graves’ Disease Source: 1 Evaluate Pharm
Slide 36: Targeting more durable IgG suppression while maintaining Fc fragment safety profile
Targeting more durable IgG suppression while maintaining the Fc fragment safety profile Extended half-life for less frequent dosing Targeting a less frequent, self-administered, subcutaneous injection Potential to be best-in-class Viridian’s potential best-in-class portfolio is designed to capture s
Slide 37: VRDN-006 in vitro and multi-dose NHP data compared to efgartigimod
VRDN-006 in vitro, multi-dose NHP PK and IgG reduction data compared to efgartigimod Non-human primates (NHPs) were dosed with IV bolus of 20 mg/kg VRDN-006, 20 mg/kg efgartigimod (internally generated benchmark), or buffer vehicle every 4 days for 4 doses. Source: Viridian data on file. IgG = Immun
Slide 38: VRDN-006 spares albumin and LDL in multi-dose NHP study
VRDN-006 spares albumin and LDL in multi-dose NHP study Non-human primates (NHPs) were dosed with IV bolus of 20 mg/kg VRDN-006, 20 mg/kg efgartigimod (internally generated benchmark), or buffer vehicle every 4 days for 4 doses. Source: Viridian data on file. CFB = change from baseline, IV = intrave
Slide 39: Single dose of VRDN-008 showed longer half-life and deeper IgG reduction vs. efgartigimod
Non-human primates (NHPs) were given equimolar doses of 60 mg/kg VRDN-008, 48.5 mg/kg efgartigimod (internally generated benchmark), or buffer vehicle - all via IV bolus. Source: Viridian data on file. BLQ = below limit of quantification, IgG = Immunoglobulin G, IV = intravenous, NHP = non-human pri
Slide 40: Single dose of VRDN-008 spares albumin and LDL in NHPs
Non-human primates (NHPs) were given equimolar doses of 60 mg/kg VRDN-008, 48.5 mg/kg efgartigimod (internally generated benchmark), or buffer vehicle - all via IV bolus Source: Viridian data on file. IV = intravenous, LDL = low-density lipoprotein, NHPs = non-human primates. VRDN-008 Spares Albumin
Slide 41: Significant progress in Q3 2024
Significant progress in Q3 2024 Veligrotug Intravenous Reported THRIVE topline data in active TED in September: veligrotug achieved all primary & secondary endpoints with high levels of statistical significance (p < 0.0001) and was generally well-tolerated THRIVE-2 in chronic TED: completed and exce
Slide 42: Multiple meaningful catalysts expected across TED and FcRn portfolios
Thyroid Eye Disease (anti-IGF-1R) Portfolio Veligrotug Intravenous VRDN-003 Subcutaneous FcRn-Targeting Autoimmune Portfolio VRDN-006 FcRn-targeting Fc fragment VRDN-008 Bispecific, extended half-life FcRn inhibitor Multiple meaningful catalysts expected across Viridian’s TED and FcRn portfolios 202
Viridian Therapeutics, Inc.\DE — Investor Presentation (November 2024) on 6ix