Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Investor Presentation (July 2024)
33 slides from Viridian Therapeutics's investor presentation, Jul 15, 2024
Slide 1: Corporate Presentation July 2024
Exhibit 99.1 Corporate Presentation July 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: 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 product differentiation First-generation product establishes significant oppo
Slide 4: Differentiated pipeline: late-stage TED programs and preclinical FcRn portfolio
Differentiated pipeline: late-stage Thyroid Eye Disease programs and preclinical FcRn portfolio DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 VRDN-001 Thyroid Eye Intravenous Disease (anti–IGF-1R) On track to Portfolio VRDN-003 initiate Phase 3 Subcutaneous in August 2024 VRDN-006 FcRn-targeting Fc
Slide 5: Significant progress in 2024 to date – All catalysts on track
Significant progress in 2024 to date – 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 • THRIVE-2: enrollment target reached; topline data on track for year- Intravenous end 2024 VRD
Slide 6: Thyroid Eye Disease (TED) Portfolio
Thyroid Eye Disease (TED) Portfolio 6
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 Thyroid Eye Disease (TED) 1 Autoantibodies trigger IGF-1R/TSHR pathway Bulging Eyes Heterogeneous autoimmune disease with clinical signs and symptoms that can var
Slide 8: Anti-IGF-1R is the only approved targeted mAb treatment for TED
Anti-IGF-1R is the only approved targeted mAb treatment for TED 1 Anti–IGF-1R is the only targeted mechanism approved for TED VALIDATED TARGET WITH PROVEN EFFICACY Both active and chronic TED patients demonstrate 2,3 substantial benefit regardless of disease duration 2,3 In teprotumumab clinical stu
Slide 9: TED represents a large market opportunity for novel differentiated treatments
TED represents a large market opportunity for novel differentiated treatments and global growth potential Teprotumumab Large Market with Limited Options Primed for New Entrants and Growth Net Sales (US) 1 ~$1.8 B • ~190k people with moderate-to-severe • New-Start Market – every patient is a new 2 TE
Slide 10: Two differentiated anti–IGF-1R mAbs building on a proven MOA
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 11: VRDN-001 Intravenous anti–IGF-1R
VRDN-001 Intravenous anti–IGF-1R 11
Slide 12: Phase 2: VRDN-001 in active TED showed robust clinical activity
Phase 2: 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 rat
Slide 13: Phase 2: VRDN-001 IV was well tolerated in active TED
Phase 2: VRDN-001 IV was well tolerated 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 (n=9), n (n=6), n (n=6), n Adverse Reactions: Muscle spasms 2 2 2** - 2 - - - Nausea - - - 1 Alopecia No serious adverse Diar
Slide 14: Phase 2: VRDN-001 IV in chronic TED showed robust clinical activity
Phase 2: 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
Slide 15: Phase 2: VRDN-001 IV was well tolerated in chronic TED
Phase 2: VRDN-001 IV was well tolerated 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%) 2 (15%) 0 (0%) Muscle spasms 1 (8%) 2 (40%) Headache Ear discomfort 0 (0%) 1 (20%) 0 (0%) 1 (20%) Fatigue 0 (0%) 1 (
Slide 16: THRIVE and THRIVE-2 on track to deliver topline results this year
Phase 3 THRIVE (active) and THRIVE-2 (chronic) are on track to deliver topline results this year ACTIVE TED Enrollment Complete CHRONIC TED Enrollment Target Reached & Completion Expected in July Key Inclusion Criteria Key Inclusion Criteria >40% enrollment from the US • Proptosis of ≥3 mm • Proptos
Slide 17: VRDN-001 potential to improve patient experience with a differentiated dosing regimen
VRDN-001 has the potential to improve patient experience with a differentiated dosing regimen 1 VRDN-001 Teprotumumab Potential Viridian 2 Differentiators Mechanism Same target & MOA; more complete IGF-1R full antagonist IGF-1R partial antagonist of Action receptor inhibition with VRDN-001 Treatment
Slide 18: VRDN-003 Subcutaneous half-life extended anti–IGF-1R
VRDN-003 Subcutaneous half-life extended anti–IGF-1R 18
Slide 19: Planned Phase 3 trials for VRDN-003 and path to BLA
Planned phase 3 clinical trials for VRDN-003 and path to BLA following positive Type C meeting with FDA Core clinical package to support registration ACTIVE TED CHRONIC TED Randomized, double-masked, placebo- Randomized, double-masked, placebo- controlled phase 3 study to demonstrate controlled phas
Slide 20: Later-entrant SC therapies have expanded market and taken 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 by SC Launch: Aug 2020 J
Slide 21: VRDN-003 designed to bring a potentially best-in-class therapy for patients
VRDN-003 designed to bring a potentially best-in-class therapy for patients Potential VRDN-003 Benefits 1 Teprotumumab IV VRDN-003 Autoinjector Easy self-administration Ph3 pivotal program is evaluating transforms patient convenience 8 INFUSIONS two dosing regimens: administered every 3 weeks Infreq
Slide 22: HV Study: VRDN-003 showed 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 23: 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) 3 1 -- AEs deemed to be related to VRDN-003 • No hearing-related AEs 1 1 (8%) -- -- Injection Site Reactions (ISRs) • No tre
Slide 24: PK model shows Q4W and Q8W dosing achieves key exposure levels
PK model shows Q4W and Q8W dosing of VRDN-003 SC achieves key exposure levels between 3–10 mg/kg of VRDN-001 IV Subcutaneous VRDN-003 Pharmacokinetic (PK) Modeling • VRDN-003 dosing regimens achieve VRDN-001 exposures shown to be clinically active – VRDN-001 IV showed robust clinical activity 100 at
Slide 25: REVEAL-1 & REVEAL-2 on track to initiate in August 2024
VRDN-003 registrational trials – REVEAL-1 (active) & REVEAL-2 (chronic) – on track to initiate in August 2024 ACTIVE TED CHRONIC TED Key Inclusion Criteria Key Inclusion Criteria • Proptosis of ≥3 mm • Proptosis of ≥3 mm • Any CAS (0–7) • CAS ≥3 • Onset of TED symptoms within 15 months • Onset of TE
Slide 26: REVEAL-1 & REVEAL-2 will 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) 1 D1 W4 W8 W12 W16 W20 W24 Through W52 Treatment Arms (1:1:1) Primary Endpoint VRDN-003 Q4W Analysis Primary efficacy endpoint: Addition
Slide 27: VRDN-003 has the potential to transform TED treatment
VRDN-003 has the potential to transform TED treatment Leverages validated mechanism of IGF-1R inhibition in TED and same binding domain as VRDN-001 VRDN-003 (SC) Engineered for a longer half-life, shown to be 40–50 days in a HV study, 4–5x that of VRDN-001 Modeling of VRDN-003 clinical data in HV in
Slide 28: FcRn Inhibitor Portfolio: Expansion Beyond TED
FcRn Inhibitor Portfolio: Expansion Beyond TED 28
Slide 29: FcRns have multiple large market opportunities, including Myasthenia Gravis
FcRns have multiple large market opportunities, including Myasthenia Gravis with >$4B projected revenues by 2028 1,2 Projected WW Myasthenia Gravis FcRn Market ... with potential in additional autoimmune indications $4.3B Vyvgart Graves Other Key FcRns Disease $1.5B CIDP Approved Lupus Myositis Neph
Slide 30: VRDN-006 in vitro, NHP PK and IgG reduction data compared to efgartigimod
VRDN-006 in vitro, multi-dose non-human primate PK, and IgG reduction data compared to efgartigimod pH-Dependent Binding Multi-Dose NHP Pharmacokinetics 5 4×10 VRDN-006 Dose 1000 Efgartigimod 5 3×10 pH 6 Efgartigimod 20 mg/kg IV WT Fc Control VRDN-006 20 mg/kg IV 5 100 2×10 5 1×10 10 pH 7.4 0 1 0 5
Slide 31: VRDN-006 shows no effect on albumin or LDL in NHP study
VRDN-006 shows no effect on albumin or LDL in multi-dose non- human primate study VRDN-006 effect on albumin VRDN-006 effect on LDL in NHPs, compared to efgartigimod in NHPs, compared to efgartigimod 200 150 Dose Dose 150 100 100 VRDN-006 20 mg/kg IV 50 Efgartigimod 20 mg/kg IV VRDN-006 20 mg/kg IV
Slide 32: VRDN-008 half-life extended FcRn inhibitor with potential for best-in-class
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 deep, durable reduction of IVIg in a humanized mouse model 125 100 Vehicle Efgartigimod VRDN-008 Efgartigimod 100 VRDN-008 10 75 50 1
Slide 33: Expected catalysts across the TED and FcRn portfolios through 2026
Expected catalysts across the TED and FcRn portfolios through 2026 Phase 3 topline Phase 3 topline BLA VRDN-001 data for THRIVE data for THRIVE-2 submission Intravenous in active TED in chronic TED 2H 2025 Thyroid Eye September 2024 Year-End 2024 Disease (anti–IGF-1R) Phase 3 topline data for Initia
Viridian Therapeutics, Inc.\DE — Investor Presentation (July 2024) on 6ix