Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Q1 2024 Earnings Presentation
31 slides from Viridian Therapeutics's earnings presentation, May 8, 2024
Slide 1: Corporate Presentation
Corporate Presentation May 8, 2024
Slide 2: Cautionary note regarding forward-looking statements
Cautionary note regarding forward-looking statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as, but not limited to, “anticipate,” “believe,” “continu
Slide 3: Viridian is 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 that benefit patients Identify market opportunities with clear remaining unmet need Determine key areas of potential First-generation product product differentiation establishes significant oppo
Slide 4: Viridian's differentiated pipeline: late-stage TED programs and preclinical FcRn portfolio
Viridian’s differentiated pipeline: late-stage TED programs and preclinical FcRn portfolio DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 VRDN-001 Thyroid Eye Intravenous Disease (anti-IGF-1R) Portfolio VRDN-003 Pivotal program initiation on track for mid-year 2024 Subcutaneous VRDN-006 FcRn-targetin
Slide 5: Significant progress in Q1 2024 – All catalysts on track
Significant progress in Q1 2024 – All catalysts on track Anticipated Catalysts THRIVE topline: Sept. 2024 • THRIVE: completed and exceeded enrollment in March VRDN-001 THRIVE-2 topline: Year-end 2024 Intravenous • THRIVE-2: topline data on track for year-end 2024 VRDN-001 BLA: 2H 2025 VRDN-003 • Pos
Slide 6: Thyroid Eye Disease (TED) Portfolio
Thyroid Eye Disease (TED) Portfolio 6
Slide 7: TED is an autoimmune condition characterized by inflammation, growth, and damage to tissues around and behind 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 8: TED represents a large market opportunity with global growth potential
TED represents a large market opportunity with global growth potential Opportunity for New Differentiated Treatment Options Large Market with Primed for New Entrants Teprotumumab Limited Options and Growth Net Sales (US) ~$1.8 B2 • Large Market: ~190k people with moderate- • New-Start Oriented: Flar
Slide 9: Building upon a proven MOA, Viridian is developing two differentiated anti-IGF-1R mAbs
Building upon a proven MOA with demonstrated efficacy, Viridian is developing two differentiated anti-IGF-1R mAbs VRDN-001 (IV) VRDN-003 (SC) VRDN-001 and VRDN-003 have the same binding domain VRDN-003 is engineered for a longer half-life, shown to be 40-50 days in a HV study, 4-5x that of VRDN-001
Slide 10: VRDN-001
VRDN-001 Intravenous anti-IGF-1R 10
Slide 11: VRDN-001 in active TED showed robust clinical activity after two infusions in all dose cohorts
VRDN-001 in active TED showed robust clinical activity after two infusions in all dose cohorts Signs Symptoms Improvement in Improvement in Clinical Activity proptosis Score (CAS) and diplopia score Overall response: Proptosis: Proptosis: CAS*: CAS*: Diplopia: Signs + symptoms Responder rate Mean ch
Slide 12: VRDN-001 IV was well tolerated in a Phase 2 clinical study in active TED
VRDN-001 IV was well tolerated in a Phase 2 clinical study in active TED VRDN-001 3 mg/kg, 10 mg/kg, & 20 mg/kg TED cohorts VRDN-001 VRDN-001 VRDN-001 Placebo 3 mg/kg 10 mg/kg 20 mg/kg (n=5), n Adverse Reactions: (n=9), n (n=6), n (n=6), n Muscle spasms 2 2 2** - Nausea 2 - - - Alopecia - - - 1 No s
Slide 13: VRDN-001 IV in chronic TED showed robust clinical activity after two infusions in both dose cohorts
VRDN-001 IV in chronic TED showed robust clinical activity after two infusions in both dose cohorts Symptoms Signs Improvement in Clinical Activity Improvement in proptosis Score (CAS) and diplopia score Proptosis: Proptosis: Proptosis: CAS: CAS: Diplopia: Responder rate Mean change by Mean change b
Slide 14: VRDN-001 IV was well tolerated in a Phase 2 clinical study in chronic TED
VRDN-001 IV was well tolerated in a Phase 2 clinical study in chronic TED Reported adverse events occurring in ≥ 10% of patients VRDN-001 Placebo 10 & 3 mg/kg (n=5), n (n=13*), n Back pain 2 (15%) 0 (0%) Muscle spasms 2 (15%) 0 (0%) Headache 1 (8%) 2 (40%) Ear discomfort 0 (0%) 1 (20%) Fatigue 0 (0%
Slide 15: THRIVE (active) and THRIVE-2 (chronic) are on track to deliver topline results this year
THRIVE (active) and THRIVE-2 (chronic) are on track to deliver topline results this year ACTIVE TED CHRONIC TED Key Inclusion Criteria Enrollment Complete 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
Slide 16: VRDN-001 has the potential to improve patient experience with a differentiated dosing regimen
VRDN-001 has the potential to improve patient experience with a differentiated dosing regimen VRDN-001 Teprotumumab Viridian Differentiators Mechanism IGF-1R antagonist IGF-1R antagonist Same target, same MOA of Action Treatment 5 infusions given 8 infusions given ~2/3 less volume infused Regimen ev
Slide 17: VRDN-003
VRDN-003 Subcutaneous half-life extended anti-IGF-1R 17
Slide 18: Later-entrant SC therapies (VRDN-003) have the potential to expand the market and take share from incumbent IV
Later-entrant SC therapies (VRDN-003) have the potential to expand the market and take 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 by SC Launch: Aug 2020 J&J
Slide 19: Phase 1 HV Study: Subcutaneous VRDN-003 showed an 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 20: 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 21: VRDN-003 SC is predicted by modeling to achieve exposure levels associated with a VRDN-001 IV clinical response
VRDN-003 SC is predicted by modeling to achieve exposure levels associated with a VRDN-001 IV clinical response Subcutaneous VRDN-003 Pharmacokinetic (PK) Modeling Key Takeaways • VRDN-001 IV showed robust clinical activity 200 at all dose levels: 3, 10, and 20 mg/kg 003 Q8W 24 wks 003 Q4W 24 wks •
Slide 22: Positive VRDN-003 FDA Type C meeting completed; on track to start pivotal program mid-year
Positive VRDN-003 FDA Type C meeting completed; on track to start pivotal program mid-year Two Expected Possible Potential Global Pivotal Active Arms Endpoints VRDN-003 Studies To include at least one of: Primary: Goals: Evaluating safety Proptosis and efficacy in Q8W responder rate • Preserve compe
Slide 23: FcRn Inhibitor Portfolio: Expansion Beyond TED
FcRn Inhibitor Portfolio: Expansion Beyond TED 23
Slide 24: FcRns have multiple large market opportunities, including Myasthenia Gravis
FcRns have multiple large market opportunities, including Myasthenia Gravis with >$4B projected revenues by 2028 Projected WW Myasthenia Gravis FcRn Market1,2 ... with potential in additional autoimmune indications $4.3B Vyvgart Other Key FcRns Graves Disease $1.5B CIDP In registration Myositis Lupu
Slide 25: Potential best-in-class FcRn inhibitor portfolio could capture large market share in autoimmune indications
Potential best-in-class FcRn inhibitor portfolio could capture large market share in autoimmune indications VRDN-006 VRDN-008 Highly selective FcRn-targeting Fc fragment Half-life extended FcRn inhibitor target profile • The only other known Fc fragment in Aim to: development • Target deeper & more
Slide 26: Viridian anticipates multiple key catalysts across the TED and FcRn portfolios in 2024 & 2025
Viridian anticipates multiple key catalysts across the TED and FcRn portfolios in 2024 & 2025 Phase 3 THRIVE data Phase 3 THRIVE-2 data VRDN-001 BLA submission in active TED in chronic TED Intravenous September 2024 2H 2025 Thyroid Eye Year-End 2024 Disease (anti-IGF-1R) Selected VRDN-003 Portfolio
Slide 27: Significant progress in Q1 2024 – All catalysts on track
Significant progress in Q1 2024 – All catalysts on track Anticipated Catalysts THRIVE topline: Sept. 2024 • THRIVE: completed and exceeded enrollment in March VRDN-001 THRIVE-2 topline: Year-end 2024 Intravenous • THRIVE-2: topline data on track for year-end 2024 VRDN-001 BLA: 2H 2025 VRDN-003 • Pos
Slide 28: Appendix
Appendix 28
Slide 29: VRDN-006 shows comparable potency in vitro and IgG-lowering in NHPs to efgartigimod
VRDN-006 shows comparable potency in vitro and IgG-lowering in NHPs to best-in-class FcRn inhibitor efgartigimod VRDN-006 in vitro potency is comparable VRDN-006 pharmacodynamic effect on IgG is to efgartigimod comparable to efgartigimod in head-to-head NHP study Rat isotype Vehicle wt-hIgG1 Fc Cyno
Slide 30: VRDN-006 shows similar safety profile to efgartigimod in head-to-head NHP study
VRDN-006 shows similar safety profile to efgartigimod in head- to-head NHP study Designed to selectively bind VRDN-006 does not lower VRDN-006 does not increase FcRn without impacting albumin in NHPs, comparable to LDL in NHPs, comparable to albumin recycling efgartigimod efgartigimod Vehicle Efgart
Slide 31: VRDN-008 is designed to be a half-life extended FcRn inhibitor
VRDN-008 is designed to be a half-life extended FcRn inhibitor with potential for best-in-class efficacy and convenience VRDN-008 demonstrates extended half-life and deeper and more durable reduction of IVIG in a humanized mouse model compared head-to-head with efgartigimod 100 125 Efgartigimod Vehi
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