Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Corporate Update (December 2023)
23 slides from Viridian Therapeutics's corporate update, Dec 18, 2023
Slide 1: Subcutaneous IGF-1R Program Selection
Subcutaneous IGF-1R Program Selection December 18, 2023
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: Subcutaneous VRDN-003 selected and anticipated to advance to pivotal trials in TED in mid-2024
Subcutaneous VRDN-003 selected and anticipated to advance to pivotal trials in TED in mid-2024 VRDN-003 sets a potential new bar for best-in-class product in TED based on Phase 1 HV study • Extended half-life: VRDN-003 half-life exceeds expectations at 40-50 days – 4-5x increase compared to its pare
Slide 4: VRDN-003 is nearly identical to its parent VRDN-001 and has a 4-5x extended half-life
VRDN-003 is nearly identical to its parent VRDN-001 and has a 4-5x extended half-life VRDN-003 VRDN-001 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 exten
Slide 5: 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 Validated MoA P
Slide 6: Later-entrant SC therapies have the potential to significantly 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 7: Phase 1 clinical trials in healthy volunteers for VRDN-003, VRDN-002, and VRDN-001
Phase 1 clinical trials in healthy volunteers for VRDN-003, VRDN-002, and VRDN-001 VRDN-003 HV (N=34) VRDN-002 HV (N=16) VRDN-001 HV (N=16) COHORT: COHORT: COHORT: 1 Single dose of SC 300 mg 1 Single dose of SC 300 mg 1 Single dose of SC 300 mg VRDN-003 (n=6), placebo (n=2) VRDN-002 (n=8) VRDN-001 (
Slide 8: Phase 1 healthy volunteer study pharmacokinetics and pharmacodynamics
Phase 1 healthy volunteer study pharmacokinetics and pharmacodynamics VRDN-003 Phase 1 Pharmacokinetics (PK) VRDN-003 Phase 1 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 from Baseline IGF-1 VRDN-003
Slide 9: 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 10: VRDN-001 IV clinical experience informs exposures of VRDN-003 expected to lead to clinical responses
VRDN-001 IV clinical experience informs exposures of VRDN-003 expected to lead to clinical responses 250 VRDN-001 IV Pharmacokinetics 200 Concentration (μg/mL) 150 • 20 mg/kg IV exposure range (Cmin to Cavg) 20 mg/kg IV Cmin 100 (20 mg/kg IV) • 10 mg/kg IV exposure range (Cmin to Cavg) 10 mg/kg IV C
Slide 11: VRDN-001 IV clinical experience informs exposures of VRDN-003 expected to lead to clinical responses
VRDN-001 IV clinical experience informs exposures of VRDN-003 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 from %
Slide 12: VRDN-003 demonstrates exposures associated with VRDN-001 IV clinical response
VRDN-003 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 003 Q4W 24 wks – Maximizes convenience (least frequent dosing) 200 003 Q2W 24 wks – Lowest exposure,
Slide 13: VRDN-003: potential pivotal development in TED with an established path to BLA
VRDN-003: potential pivotal development in TED with an established path to BLA subject to alignment with regulators Two Global Possible Potential Pivotal Studies Active Arms Endpoints VRDN-003 To include at least one of: Primary: Goals: Evaluating safety Q8W Proptosis and efficacy in responder rate
Slide 14: Viridian pipeline: registrational TED programs and differentiated portfolio of Anti-FcRn therapies
Viridian pipeline: registrational TED programs and differentiated portfolio of Anti-FcRn therapies DISCOVERY PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Thyroid Eye VRDN-001 Disease Intravenous, five-dose regimen (anti-IGF-1R) VRDN-003 Selected Subcutaneous Program Pivotal program planned Potential best-in-
Slide 15: Viridian is well funded through key catalysts into 2026
Viridian is well funded through key catalysts into 2026 Select VRDN- Plan to initiate Phase 3 Phase 3 Cash Position 003 as potential pivotal SC THRIVE data in THRIVE-2 data • Ended Q3 with $313M in cash IGF-1R best-in-class program for active TED for in chronic TED SC program and cash equivalents VR
Slide 16: Appendix
Appendix: – VRDN-001 IV Phase 2 Clinical Data: Active and Chronic TED – VRDN-001 and VRDN-003 PK Parameters – IGF-1 Pharmacodynamics Following IV Dosing 16 IGF-1 = insulin-like growth factor 1, IV = intravenous, PK = pharmacokinetics, TED = thyroid eye disease.
Slide 17: VRDN-001 IV in active TED provides a range of therapeutic exposures comparing 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 18: VRDN-001 IV was well tolerated in active TED
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 Adverse Reactions: (n=9), n (n=6), n (n=6), n Muscle spasms 2 2 2** - Nausea 2 - - - Alopecia - - - 1 No serious adverse Diarrhea 1 2*
Slide 19: VRDN-001 IV in chronic TED showed robust clinical response at 3 and 10 mg/kg
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 20: VRDN-001 IV was well tolerated in chronic TED
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%) Muscle spasms 2 (15%) 0 (0%) Headache 1 (8%) 2 (40%) Ear discomfort 0 (0%) 1 (20%) Fatigue 0 (0%) 1 (20%) Flatulence 0 (0%)
Slide 21: VRDN-001 IV clinical experience to-date informs exposures of VRDN-003 expected to produce clinical benefit
VRDN-001 IV clinical experience to-date informs exposures of VRDN-003 expected to produce clinical benefit VRDN-001 IV Pharmacokinetics 250 Projected Projected 200 Projected Projected AUC AUC Dose Cmin Cavg (µg*day/mL) (µg*day/mL) (µg/mL) (µg/mL) Concentration (μg/mL) (5-doses) (8-doses) 150 20 mg/k
Slide 22: VRDN-003 demonstrates exposures associated with VRDN-001 IV clinical response
VRDN-003 demonstrates exposures associated with VRDN- 001 IV clinical response VRDN-003 PK Modeling 250 003 Q2W 24 wks Projected Projected Projected 003 Q4W 24 wks Dose AUC 200 003 Q8W 24 wks Cmin (µg/mL) Cavg (µg/mL) (µg*day/mL) Concentration (μg/mL) 150 Clinically active Q2W 136 116 19,500 Cmin 10
Slide 23: VRDN IGF-1 levels post-dosing
VRDN IGF-1 levels post-dosing VRDN-003 Phase 1 Healthy Volunteers VRDN-001, VRDN-002, VRDN-003 (IV) (SC & IV) VRDN-001 3 mg/kg IGF-1 (Fold change from baseline) VRDN-001 10 mg/kg VRDN-001 20 mg/kg 6 6 VRDN-003 SC 300 mg Placebo Fold Change IGF-1 VRDN-003 SC 600 mg VRDN-002 3 mg/kg 4 VRDN-003 5 mg/kg
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