Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Investor Presentation (October 2023)
36 slides from Viridian Therapeutics's investor presentation, October 2023
Slide 1: Corporate Overview
® Corporate Overview October 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: 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 • ~$900 million1 market capitalization • Q2 2023 cash of ~$334.3 million2, cash runway into 2H:25 • Initial focus on thyroid eye di
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 First market entrant • Determine potential key areas of establishes significant differentiation 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 THRIVE-2 Phase 3 Chronic TED VRDN-001 SC Phase 1 VRDN-002 SC Phase 1 VRDN-003 SC Phase 1 Autoimmune & Rare Diseases VRDN-004 Rare Disease VRDN-005 Autoimmune VRDN-006 Auto
Slide 6: Thyroid Eye Disease (TED)
MEVIRIDIAN Thyroid Eye Disease (TED)
Slide 7: TED overview
TED overview Normal Eye Anatomy • Autoantibodies trigger IGF-1R/TSHR pathway1 • Heterogeneous autoimmune disease with clinical signs and symptoms that can vary or Optic Nerve modulate following onset in some cases for the rest of a patient’s life • Main signs include proptosis (eye bulging), Thyroid
Slide 8: Historically described as a biphasic disease course from active to chronic TED
Historically described as a biphasic disease course from active to chronic TED Inflammatory signs and symptoms, such as pain, redness, swelling, proptosis, diplopia, and eyelid retraction are at peak levels Inflammatory signs such as redness and swelling are reduced but the pain, proptosis, diplopia
Slide 9: Large commercial opportunity in US and ROW
Large commercial opportunity in US and ROW New start market provides rare opportunity for a differentiated entrant to rapidly capture market share Active TED Chronic TED >3 moderate to severe moderate to severe Billion ~$2 Billion Approved 2020 20-40% ~10% Estimated Tepezza market share >1 ~$851 Bil
Slide 10: VRDN-001 a potentially differentiated intravenous anti-IGF-1R antibody in TED
VRDN-001 a potentially differentiated intravenous anti-IGF-1R antibody in TED Tepezza VRDN-001 (teprotumumab) Mechanism of Action Full antagonist1 Partial antagonist Evaluating shortened,12-week / Treatment regimen 5 infusions FDA approved for 21-week / 8 infusions Starting dose of 10 mg/kg, remaini
Slide 11: Viridian is focused on 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 TED blockbuster prescriber base TED franchise US + ex-US Establish new treatment market access paradigms in TED a
Slide 12: VRDN-001 Phase 1/2 data in TED
VRDN-001 Phase 1/2 data in TED 12
Slide 13: VRDN-001 Phase 1/2 proof-of-concept trial in patients with TED
VRDN-001 Phase 1/2 proof-of-concept trial in patients with TED ACTIVE TED COHORTS CHRONIC TED COHORTS • Proptosis of ≥3 mm • Proptosis of ≥3 mm • Clinical activity score (CAS) of ≥4 • CAS of 0-7 • Onset of symptoms within past 12 months • Onset of symptoms >12 months prior Cohort 1 VRDN-001 10 mg/kg
Slide 14: VRDN-001 clinical activity observed in patients with TED
VRDN-001 clinical activity observed in patients with TED Signs Symptoms Improvement in Improvement in CAS and diplopia proptosis score Proptosis: Proptosis: Diplopia: CAS: CAS: Responder rate Mean change by Complete Score of 0 or 1 Mean change (% with ≥2mm exophthalmometry resolution** (% achieving
Slide 15: Proptosis change from baseline to week 6 by exophthalmometry
Proptosis change from baseline to week 6 by exophthalmometry ACTIVE TED CHRONIC TED Individual proptosis change Individual proptosis change 0 0 0 0 Change from baseline proptosis (mm) Change from baseline proptosis (mm) -1 -2 -2 -3 -4 VRDN 3 mg/kg -4 VRDN 10 mg/kg VRDN-001 3 mg/kg VRDN 20 mg/kg VRDN
Slide 16: Change in proptosis vs placebo in active TED
Reported change in proptosis in all VRDN-001 treated patients compared to placebo from baseline to week 6 in patients with active TED Individual proptosis change Mean proptosis change Adj. mean proptosis change (by MRI**) (by MRI**) (by exophthalmometer) 2 1 1 Change from baseline proptosis (mm) Cha
Slide 17: Proptosis reductions consistent with centrally reviewed MRI in chronic TED
VRDN-001 proptosis reductions by exophthalmometer are consistent with centrally reviewed MRI in patients with chronic TED Individual proptosis change Mean proptosis change Adjusted mean proptosis (by MRI**) (by MRI**) change (by exophthalmometer) 2 1 0 Change from baseline proptosis (mm) Change from
Slide 18: Improvement in signs and symptoms as measured by CAS in patients with TED at week 6
Improvement in signs and symptoms as measured by CAS in patients with TED at week 6 ACTIVE TED CHRONIC TED Individual CAS change Individual CAS change 0 CAS at baseline 1 0 0 1 6 1 2 5 5 6 6 6 0 0 0 0 Change from baseline score Change from baseline score -2 -1 -4 -2 -3 -6 VRDN 3 mg/kg -4 VRDN-001 3
Slide 19: Safety profile across all TED Phase 2 cohorts
Safety profile across all TED Phase 2 cohorts Reported adverse events occurring in ≥ 10% of patients ACTIVE TED COHORTS CHRONIC TED COHORTS VRDN-001 VRDN-001 Placebo 3 mg/kg 10 mg/kg 20 mg/kg Placebo (n=5), n 10 & 3 mg/kg Reported (n=9), n (n=6), n (n=6), n (n=6), n Reported Events: Events: (n=13),
Slide 20: THRIVE: VRDN-001 global Phase 3 trials in TED
THRIVE: VRDN-001 global Phase 3 trials 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 = up to 159 • Double-masked, randomized, placebo-controlled • Double-maske
Slide 21: Subcutaneous candidates in TED
Subcutaneous candidates in TED 21
Slide 22: Viridian is developing 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 YTE substitutions for extended half-life Mechanism of Action Full antagonist Full antagonist Partial antagonist Expected to match or exceed Half-life ~10 to 11
Slide 23: 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-001 Phase 1 trial fully enrolled • VRDN-002 Phase 1 trial fully enrolled • VRDN-003 Phase 1 trial with single IV
Slide 24: Ongoing Phase 1 trials in healthy volunteers
Ongoing Phase 1 trials in healthy volunteers Single SC dose of 300 mg VRDN-001 N=16 Single IV dose of 3.5 mg/kg Single SC dose of 300 mg VRDN-002 N=16 Single IV dose of 3.5 mg/kg Cohort 1 Cohort 3 Cohort 5 Single SC dose of 300 mg Single SC dose of 600 mg Repeat SC dose of VRDN-003 N=34 Cohort 2 Coh
Slide 25: Plan to launch with a potentially best-in-class IV followed by a self-administered SC pen
Plan to launch with a potentially best-in-class IV followed by a self-administered SC pen in TED 2 VRDN-001 IV Potential best-in-class Q3W IV treatment for TED VRDN SC Potential first/best-in-class self-administered SC pen 25 * Pictures used for illustrative purposes only
Slide 26: Corporate priorities
Corporate priorities VRDN-001 IV • 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 device supply partnership expected in 2H23 • VRDN-001, VRDN-002, & VRDN-0
Slide 27: Appendix
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Slide 28: TED is associated with significant vision-threatening impairments
TED is associated with significant vision- threatening impairments Dry Eye Diplopia Photophobia Decreased Visual Field Reduced Color Optic Neuropathy Disease (double vision) (light sensitivity) Visual Acuity Defects Vision Vision impairment in patients with TED 1. Bartley GB. Trans Am Ophthalmol Soc
Slide 29: 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 29 Source: Viridian-sponsored market research
Slide 30: 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 in prior clinical trials • Exploratory measure to potentially in TED confirm and
Slide 31: Evaluating symptoms of TED using clinical activity score (CAS)
Evaluating symptoms of TED using clinical activity score (CAS) 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 orbital pain redn
Slide 32: Baseline patient characteristics in active TED cohorts
Baseline patient characteristics in active TED cohorts VRDN-001 VRDN-001 VRDN-001 VRDN-001 (3, 10, and 20 mg/kg) 3 mg/kg 10 mg/kg 20 mg/kg Placebo n 21 9 6 6 5 Proptosis, mean (SEM) 23.7 (0.7) 23.1 (1.2) 24.8 (1.2) 23.6 (1.3) 22.8 (2) CAS, mean (SEM) 5.4 (0.2) 5.4 (0.4) 5.2 (0.3) 5.5 (0.4) 5.0 (0.5)
Slide 33: Baseline patient characteristics in chronic TED cohorts
Baseline patient characteristics in chronic TED cohorts VRDN-001 VRDN-001 VRDN-001 (10 and 3 mg/kg) 10 mg/kg 3 mg/kg Placebo n 12 6 6 5 Proptosis, mean (SEM) 22.2 (1.2) 21.1 (2.2) 23.4 (1.1) 25.0 (1.6) CAS, mean (SEM) 3.3 (0.8) 2.5 (1.1) 4.0 (1.0) 2.8 (0.8) Diplopia, n (%) 5 (41.7%) 3 (50.0%) 2 (33.
Slide 34: VRDN-001 clinical activity observed in patients with TED
VRDN-001 clinical activity observed in patients with TED Signs Symptoms Improvement in Improvement in CAS and diplopia proptosis score Proptosis: Proptosis: CAS: CAS: Diplopia: Responder rate Mean change by Score of 0 or 1 Mean change Complete (% with ≥2mm exophthalmometry (% achieving CAS of (chang
Slide 35: THRIVE Phase 3 trial design in patients with active TED
THRIVE Phase 3 trial design in patients with active TED Week 15 Double-Blind Treatment Period Primary Endpoint (15 weeks) Analysis Active TED • Proptosis of ≥3 mm VRDN-001 • CAS ≥3 10 mg/kg Q3W* • Onset of active TED (n=60) Screening symptoms within (up to 4 weeks) prior 15 months Randomization 2:1
Slide 36: THRIVE-2 Phase 3 trial design in patients with chronic TED
THRIVE-2 Phase 3 trial design in patients with chronic TED Week 15 Double-Blind Treatment Period Primary Endpoint (15 weeks) Analysis Chronic TED • Proptosis of ≥3 mm VRDN-001 • Any CAS (0-7) 10 mg/kg Q3W* • Onset of TED (n=106) Screening symptoms >15 (up to 4 weeks) months Randomization 2:1 Placebo
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