Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Investor Presentation (July 2023)
37 slides from Viridian Therapeutics's investor presentation, Jul 17, 2023
Slide 1: Corporate Overview July 2023
Exhibit 99.1 ® Corporate Overview July 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, continue, co
Slide 3: Who we are
Who we are Our mission: Developing best-in-class medicines for patients with serious and rare diseases • Founded in 2020 • Headquartered in Waltham, MA • 100+ employees 1 • ~$1.4 billion market capitalization 2 • Q1 2023 cash of ~$373.9 million , cash runway into 2H:25 • Initial focus on thyroid eye
Slide 4: Our approach is to leverage core expertise in antibody discovery and engineering
Our approach is to leverage core expertise in antibody discovery and engineering • Identify a unique market opportunity • Determine potential key areas of First market entrant differentiation establishes significant opportunity for second • Discover and engineer potential best-in- entrant strategy c
Slide 5: Viridian development pipeline
Viridian development pipeline PRECLINCAL PHASE 1 PHASE 2 PHASE 3 Thyroid Eye Disease (TED) VRDN-001 IV THRIVE Phase 3 Active TED Ongoing Phase 1/2 Active + Chronic TED THRIVE-2 Phase 3 planned Chronic TED Phase 1 ongoing VRDN-001 SC Phase 1 ongoing VRDN-002 SC Phase 1 planned VRDN-003 SC Autoimmune
Slide 6: Thyroid Eye Disease (TED)
ME VIRIDIAN Thyroid Eye Disease (TED)
Slide 7: TED overview
Normal Eye Anatomy TED overview • Autoantibodies trigger IGF-1R/TSHR 1 pathway • Progressive disease consisting of an initial active phase, followed by a transition to a Optic Nerve 2 secondary chronic phase • Main signs include proptosis (eye bulging), Thyroid Eye Disease (TED) redness, swelling, d
Slide 8: TED Epidemiology in US and Europe
TED Epidemiology in US and Europe Active TED moderate to severe 20K+ 35K+ Chronic TED moderate to severe 75K+ 150K+ More than 95K patients in US and 185K patients in Europe with moderate to severe TED = 1,000 estimated patients 8 Source: Viridian-sponsored market research
Slide 9: Large commercial opportunity in US and Europe
Large commercial opportunity in US and Europe Active TED Chronic TED moderate to severe moderate to severe $4B+ Approved 2020 20-40% ~10% ~$2B (2022) 0% 0% Not approved Estimated Tepezza market share Annual US sales 2 years Estimated annual peak following first product approval sales opportunity Sou
Slide 10: Key factors to build a successful blockbuster product franchise in TED
Viridian is focused on key factors to build a successful blockbuster product franchise in TED Best-in-class IV First / best-in-class product SC product Building a Active + Chronic Expand blockbuster TED prescriber base TED franchise US + ex-US Establish new treatment market access paradigms in TED a
Slide 11: Differentiated intravenous anti-IGF-1R antibody in TED
Viridian is advancing a differentiated intravenous anti-IGF-1R antibody in TED Tepezza VRDN-001 (teprotumumab) 1 Full antagonist Partial antagonist Mechanism of Action Evaluating shortened,12-week / FDA approved for 21-week / 8 infusions Treatment regimen 5 infusions Starting dose of 10 mg/kg, remai
Slide 12: VRDN-001 Phase 1/2 data in active TED
VRDN-001 Phase 1/2 data in active TED 12
Slide 13: VRDN-001 Phase 1/2 proof-of-concept cohorts in active TED
VRDN-001 Phase 1/2 proof-of-concept cohorts in patients with active TED Randomized, double-blind trial vs placebo Cohort 1 Active TED VRDN-001 10 mg/kg Q3W x 2 (n=6) 10 mg/kg results reported in • Clinical activity August 2022, presented at ATA 2022 Placebo (n=2) score (CAS) of ≥4 • Proptosis of ≥3
Slide 14: Baseline patient characteristics in active TED cohorts
Baseline patient characteristics in active TED cohorts VRDN-001 VRDN-001 VRDN-001 VRDN-001 Placebo (3, 10, and 20 mg/kg) 3 mg/kg 10 mg/kg 20 mg/kg n 9 21 6 6 5 Proptosis, mean (SEM) 23.1 (1.2) 23.7 (0.7) 24.8 (1.2) 23.6 (1.3) 22.8 (2) CAS, mean (SEM) 5.4 (0.4) 5.4 (0.2) 5.2 (0.3) 5.5 (0.4) 5.0 (0.5)
Slide 15: VRDN-001 clinical activity observed in patients with active TED
VRDN-001 clinical activity observed in patients with active TED Signs Symptoms Improvement in Improvement in CAS and diplopia proptosis score Overall response: Proptosis: Proptosis: CAS: CAS: Diplopia: Signs + symptoms Responder rate Mean change by Score of 0 or 1 Mean change Complete exophthalmomet
Slide 16: Clinical activity at all doses consistent with full target engagement
Clinical activity observed at week 6 at all doses consistent with full target engagement in patients with active TED Individual proptosis change Individual CAS change TED IGF-1 (by exophthalmometer) (All Patients) 0 0 VRDN-001 3 mg/kg* n=5 0 VRDN-001 10 mg/kg n=6 8 VRDN-001 20 mg/kg n=6 Placebo n=5
Slide 17: Reported change in proptosis versus placebo to week 6
Reported change in proptosis in all VRDN-001 treated compared to placebo from baseline to week 6 in patients with active TED Adj. mean proptosis change Individual proptosis change Mean proptosis change (by exophthalmometer) (by MRI**) (by MRI**) 2 1 1 Placebo * n=5 Placebo * 0 0 0 +0.03 mm n=3 * * -
Slide 18: Improvement in signs and symptoms as measured by CAS
Improvement in signs and symptoms as measured by CAS in patients with active TED Mean change in CAS Reduction in CAS CAS of 0 or 1 at week 6 (from baseline to week 6) (from baseline to week 6) 0 VRDN-001 Placebo -1 9/21 ≥5 43% 62% 62% Placebo -2 n=5 -1.75 16/21 76% ≥4 -3 19/21 2/5 -4≥3 90% 40% VRDN-
Slide 19: Safety profile across all active TED Phase 1/2 cohorts
Safety profile across all active TED Phase 1/2 cohorts VRDN-001 VRDN-001 VRDN-001 Placebo 3 mg/kg 10 mg/kg 20 mg/kg ADVERSE (n=6), n (n=9), n (n=6), n (n=6), n REACTIONS: Muscle spasms 2 2 2** - Nausea 2 - - - No serious adverse Alopecia - - - 1 events (SAEs), no Diarrhea 1 2** 1* - infusion reactio
Slide 20: THRIVE Phase 3 trial design in active TED
THRIVE Phase 3 trial design in patients with active TED Week 15 Double-Blind Treatment Period Primary Endpoint Analysis (15 weeks) Active TED VRDN-001 • CAS ≥3 10 mg/kg Q3W* • Proptosis of ≥3 mm (n=60) • Onset of active TED symptoms within Screening prior 15 months (up to 4 weeks) Randomization Plac
Slide 21: VRDN-001 Phase 1/2 data in chronic TED
VRDN-001 Phase 1/2 data in chronic TED 21
Slide 22: VRDN-001 Phase 1/2 trial design in chronic TED
VRDN-001 Phase 1/2 trial design in patients with chronic TED Randomized, double-blind trial vs placebo Cohort 1 Chronic TED VRDN-001 10 mg/kg Q3W x 2 (n=6) • Proptosis of ≥3 mm Placebo (n = 2) • CAS 0-7 Cohort 2 • Onset of TED symptoms >12 months prior VRDN-001 3 mg/kg Q3W x 2 (n=6*) Placebo (n=3) *
Slide 23: Baseline patient characteristics in chronic TED cohorts
Baseline patient characteristics in chronic TED cohorts VRDN-001 VRDN-001 VRDN-001 Placebo (10 and 3 mg/kg) 10 mg/kg 3 mg/kg n 6 12 6 5 Proptosis, mean (SEM) 23.4 (1.1) 22.2 (1.2) 21.1 (2.2) 25.0 (1.6) CAS, mean (SEM) 4.0 (1.0) 3.3 (0.8) 2.5 (1.1) 2.8 (0.8) Diplopia, n (%) 2 (33.3%) 5 (41.7%) 3 (50.
Slide 24: VRDN-001 clinical activity observed in chronic TED
VRDN-001 clinical activity observed in patients with chronic TED 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** Mean change** C
Slide 25: Clinical activity at both doses consistent with full target engagement
Clinical activity observed at week 6 at both doses consistent with full target engagement in patients with chronic TED Individual CAS change Mean change in CAS* Individual proptosis change (All Patients) (Patients CAS > 0 at baseline) (by exophthalmometer) CAS at baseline 1 0 0 5 6 1 6 1 2 5 6 6 0 0
Slide 26: Proptosis reductions by exophthalmometer consistent with MRI
VRDN-001 proptosis reductions by exophthalmometer are consistent with centrally reviewed MRI Adjusted mean proptosis Individual proptosis change Mean proptosis change change (by MRI**) (by MRI**) (by exophthalmometer) 1 2 0.0 * * 0 0 -0.5 Placebo 0 n=5 Placebo * -0.2 mm n=3 -0.7 mm -1 -2 -1.0 -2 -4
Slide 27: Safety profile across all chronic TED cohorts
Safety profile across all chronic TED cohorts 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 28: Preliminary THRIVE-2 Phase 3 trial design in chronic TED
Preliminary THRIVE-2 Phase 3 trial design in patients with chronic TED Week 15 Double-Blind Treatment Period Primary Endpoint Analysis (15 weeks) Chronic TED • Any CAS (0-7) VRDN-001 • Proptosis of ≥3 mm 10 mg/kg Q3W* • Onset of TED symptoms ≥15 Screening months (up to 4 weeks) Placebo Proptosis Res
Slide 29: Subcutaneous TED Candidates
Subcutaneous TED Candidates 29
Slide 30: Multiple potential best-in-class subcutaneous programs in TED
Viridian is developing multiple potential best-in- class subcutaneous (SC) programs in TED VRDN-001 SC VRDN-003 VRDN-002 Same binding domain Same extended half-life mutations Full antagonist Full antagonist Partial antagonist Mechanism of Action Expected to match or exceed ~10 to 11 days up to 43 da
Slide 31: VRDN-001, VRDN-002, & VRDN-003 SC program progress
VRDN-001, VRDN-002, & VRDN-003 SC program progress • SC formulation work completed for all three SC programs at 150 mg/mL concentration allowing for administration of 300 mg / 2 mL • VRDN-003 IND filing submitted to FDA • VRDN-002 Phase 1 trial with single IV and single SC dose cohorts enrolled • VR
Slide 32: Corporate priorities
Corporate priorities VRDN-001 IV • THRIVE-2 Phase 3 trial initiation in chronic TED planned for 3Q23 • THRIVE Phase 3 trial topline results in active TED expected mid-2024 • THRIVE-2 Phase 3 trial topline results in chronic TED expected by YE:24 VRDN-001, VRDN-002, & VRDN-003 SC programs • Pen devic
Slide 33: Plan to launch with IV followed by self-administered SC pen
Plan to launch with a potentially best-in-class IV followed by a self-administered SC pen in TED VRDN-001 IV Potential best-in-class Q3W IV treatment for TED VRDN SC Potential first/best-in-class self-administered SC pen 2 33 33 * Pictures used for illustrative purposes only
Slide 34: Our long-term vision: building a fully-integrated biopharmaceutical
Our long-term vision: building a fully- integrated biopharmaceutical Replicate drug development and company launch excellence in additional autoimmune and rare diseases Follow with potential first / best-in-class SC TED therapy Launch potential best-in-class IV TED therapy in US and ex-US Growth Pot
Slide 35: Appendix
av ax VIRIDIAN Appendix
Slide 36: Measuring proptosis change in TED
Measuring proptosis change in TED Hertel Exophthalmometer Magnetic Resonance Imaging (MRI) • Most commonly used measure of • More precise measurement proptosis • Centrally read by 2 masked reviewers • Successfully used to in prior clinical • Exploratory measure to potentially trials in TED confirm a
Slide 37: Evaluating symptoms of TED using clinical activity and diplopia scoring scales
Evaluating symptoms of TED using clinical activity and diplopia scoring scales Clinical Activity Score (0 to 7 points) 0 1 2 3 4 5 6 7 No pain, redness, severity or swelling +1 point for Spontaneous Gaze evoked Eyelid swelling Eyelid erythema Conjunctival Chemosis Inflammation of each orbital pain o
Viridian Therapeutics, Inc.\DE — Investor Presentation (July 2023) on 6ix