Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Investor Presentation (January 2024)
31 slides from Viridian Therapeutics's investor presentation, Jan 16, 2024
Slide 1: Corporate Presentation
Corporate Presentation January 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 on 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 a unique market opportunity First-generation product • Determine potential key areas of establishes significant differentiation opportunity for next- generation
Slide 4: Viridian pipeline: late-stage TED programs and anti-FcRn therapies
Viridian pipeline: late-stage TED programs and differentiated portfolio of Anti-FcRn therapies Expected 2024 DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Milestones Upcoming Milestone Topline data Thyroid Eye VRDN-001 THRIVE: mid-2024 Disease Intravenous, five-dose regimen THRIVE-2: YE 2024 (anti-I
Slide 5: Recent progress and near-term catalysts
Recent progress and near-term catalysts 2H 2023 Accomplishments 2024 Anticipated Key Catalysts TED Portfolio • Selected VRDN-003 as subcutaneous (SC) program • Mid-2024: VRDN-001 IV Phase 3 topline results in with best-in-class potency and 40-50 day half-life THRIVE active TED • Two VRDN-001 IV Phas
Slide 6: Thyroid Eye Disease (TED) Franchise
Thyroid Eye Disease (TED) Franchise 6
Slide 7: Thyroid Eye Disease (TED) Overview
Thyroid Eye Disease (TED) Normal Eye Anatomy Overview • Autoantibodies trigger IGF-1R/TSHR pathway1 • Heterogeneous autoimmune disease with Optic Nerve clinical signs and symptoms that can vary or modulate following onset, in some cases for the rest of a patient’s life Thyroid Eye Disease (TED) • Ma
Slide 8: Thyroid eye disease represents a large market opportunity with potential for growth
Thyroid eye disease represents a large market opportunity with potential for growth Opportunities for growth Annualized Revenues ($) • Continued penetration into the active US TED market ~$1.8B1 • Growth into chronic TED market • Market expansion from subcutaneous • Ex-US opportunity remains untappe
Slide 9: Developing two anti-IGF-1Rs: VRDN-001 (IV) and VRDN-003 (SC)
Viridian is developing two anti-IGF-1Rs: VRDN-001 (IV) and VRDN-003 (SC) VRDN-001 VRDN-003 VRDN-003 has half-life engineering via clinically validated, 3 amino acid (YTE) modification in Fc region VRDN-001 & VRDN-003 are nearly identical Differ in YTE change in Fc domain to enable 4-5x extended half
Slide 10: VRDN-001
VRDN-001 Intravenous anti-IGF-1R 10
Slide 11: VRDN-001 IV in active TED compares favorably to teprotumumab
VRDN-001 IV in active TED provides a range of therapeutic exposures comparing favorably to teprotumumab 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 12: VRDN-001 IV well tolerated in Phase 2 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 Global Phase 3 Development in TED
VRDN-001 IV Global Phase 3 Development in TED ACTIVE TED CHRONIC TED • 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 • N = 90 • N = approx. 159 • Double-masked, randomized, placebo-controlled • Double-mask
Slide 14: VRDN-003
VRDN-003 Subcutaneous long-acting anti-IGF-1R 14
Slide 15: Later-entrant SC therapies could displace incumbent IV and grow market
Later-entrant SC therapies have the potential to significantly displace incumbent IV and grow market IV to SC with same molecule IV to SC with new SC entrant 85% of IV market converted in 2 years 1 30% of new prescriptions converted in 3 years 2 Target: CD38 Target: CD20 • IV Launch: Nov 2015 by J&J
Slide 16: VRDN-003 selected as lead SC program based on PK and PD profile
VRDN-003 selected in December 2023 as lead SC program based on PK and PD profile versus -001 SC and -002 SC Phase 1 HV Pharmacokinetics (PK) Phase 1 HV Pharmacodynamics (PD) VRDN-001 300 mg VRDN-001 300 mg SC 100 6 VRDN-002 300 mg VRDN-002 300 mg SC VRDN-003 300 mg VRDN-003 300 mg SC Fold Change fro
Slide 17: VRDN-003 was well tolerated in healthy volunteers
VRDN-003 was well tolerated in healthy volunteers VRDN-003 (Cohorts 1-4) VRDN-002 VRDN-001 Placebo IV (n=8) SC (n=12) IV (n=8) SC (n=8) IV (n=8) SC (n=8) (n=8) Treatment-Related AEs -- 3 (25%) -- 1 (12.5%) -- 3 (37.5%) ** 3 (37.5%) • No treatment Muscle Spasms -- -- -- -- -- 1 (12.5%) -- discontinua
Slide 18: VRDN-001 IV clinical experience informs VRDN-003 SC exposures
VRDN-001 IV clinical experience informs exposures of VRDN-003 SC expected to lead to clinical responses VRDN-001 IV Phase 2 Clinical Responses in Active TED 250 VRDN-001 IV Pharmacokinetics Proptosis: CAS*: Diplopia**: Responder Rate Score of 0 or 1 Complete resolution 200 % with ≥ 2mm reduction fro
Slide 19: VRDN-003 SC demonstrates exposures associated with VRDN-001 IV response
VRDN-003 SC demonstrates exposures associated with VRDN-001 IV clinical response VRDN-003 PK Modeling Q8W dosing of VRDN-003 is predicted to 250 achieve 3 mg/kg VRDN-001 IV Cmin and Cavg 003 Q8W 24 wks – Maximizes convenience (least frequent dosing) 003 Q4W 24 wks 200 003 Q2W 24 wks – Lowest exposur
Slide 20: VRDN-003 SC: potential pivotal development in TED via established path to BLA
VRDN-003 SC: potential pivotal development in TED via an established path to BLA* Two Expected Possible Potential Global Pivotal Active Arms Endpoints VRDN-003 Studies To include at least one of: Primary: Goals: Evaluating safety Q8W Proptosis and efficacy in responder rate • Preserve compelling IGF
Slide 21: FcRn Franchise: Expansion Beyond TED
FcRn Franchise: Expansion Beyond TED 21
Slide 22: Anti-FcRns are a large market opportunity in myasthenia gravis
Anti-FcRns are a large market opportunity with >$4B projected revenues in myasthenia gravis alone by 2028 Projected WW Myasthenia Gravis FcRn Market1,2 ... with potential in additional autoimmune indications $4.3B Vyvgart Key Other FcRns $1.5B • Myositis • Membranous CIDP Nephropathy In registration
Slide 23: Building a potentially best-in-class FcRn franchise
Building a potentially best-in-class FcRn franchise VRDN-006: highly selective Fc fragment VRDN-008: novel extended half-life anti-FcRn • The only other known Fc fragment in • Targeting deeper & more durable IgG development suppression • FcRn inhibition via Fc fragment has proven • Maintain safety p
Slide 24: VRDN-006 shows comparable potency and IgG-lowering to efgartigimod
VRDN-006 shows comparable potency in vitro and IgG- lowering in NHPs to best-in-class anti-FcRn efgartigimod VRDN-006 pharmacodynamic effect on IgG is VRDN-006 potency is comparable to efgartigimod comparable to efgartigimod in head-to-head NHP study Potency measured by competition ELISA (in triplic
Slide 25: VRDN-006 shows similar safety profile to efgartigimod in NHP study
VRDN-006 shows similar safety profile to efgartigimod in head- to-head NHP study Designed to selectively bind FcRn VRDN-006 does not lower albumin in VRDN-006 does not increase LDL in without impacting albumin recycling NHPs, comparable to efgartigimod NHPs, comparable to efgartigimod HSA VRDN-006 F
Slide 26: VRDN-008 designed as half-life extended anti-FcRn
VRDN-008 is designed to be a half-life extended anti-FcRn 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 NHP data expected in 2H 2024 Humanized mice as a model to demonstrate
Slide 27: Well funded through anticipated key catalysts into 2026
Viridian is well funded through anticipated key catalysts into 2026 Cash Position Selected VRDN- Initiate expected Phase 3 Phase 3 • Ended Q3 with $313M in cash and cash 003 as potential pivotal SC THRIVE data in THRIVE-2 data equivalents best-in-class program for active TED for in chronic TED TED S
Slide 28: Appendix
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Slide 29: TED Epidemiology in US and Europe
TED Epidemiology in US and Europe Active TED moderate to severe 45K+ 45K+ Chronic TED moderate to severe 145K+ 95K+ More than 200K patients in US and 140K patients in Europe with moderate to severe TED 29 = 1,000 estimated patients. Source: Viridian-sponsored market research
Slide 30: VRDN-001 IV in chronic TED showed robust clinical response
VRDN-001 IV in chronic TED showed robust clinical response at 3 and 10 mg/kg 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 by Score of 0 or 1** Mea
Slide 31: VRDN-001 IV well tolerated in Phase 2 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%
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