Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Q2 2022 Earnings Presentation
34 slides from Viridian Therapeutics's earnings presentation, Aug 15, 2022
Slide 1: VRDN-001 & VRDN-002 initial clinical data
Exhibit 99.3 VRDN-001 & VRDN-002 initial clinical data August 15, 2022
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: Agenda
INTRODUCTION 1 Jonathan Violin, Ph.D., President and Chief Executive Officer Agenda 2 VRDN-001 PROOF OF CONCEPT RESULTS Barrett Katz, M.D., Chief Medical Officer Jonathan Violin, Ph.D. President and Chief Executive Officer Raymond Douglas, M.D., Ph.D., Director, Thyroid Eye Disease Program, Cedars-S
Slide 4: VRDN-001 treated TED patients had rapid, significant response on a wide array of endpoints
VRDN-001 treated TED patients had rapid, significant response on a wide array of endpoints Two infusions of VRDN-001 10mg/kg three weeks apart resulted in rapid, significant improvements in proptosis, Clinical Activity Score (CAS), and diplopia at week 6 The initial dataset compares favorably to cli
Slide 5: All VRDN-001 efficacy measures compare favorably to Tepezza
All VRDN-001 efficacy measures compare favorably to Tepezza Signs Symptoms Improvement in Improvement in Clinical Activity Score (CAS) proptosis and diplopia score Overall response: Proptosis: Proptosis: CAS: CAS: Diplopia: Complete Signs + symptoms Responder rate Mean change Score of 0 or 1 Mean ch
Slide 6: VRDN-001 Phase 1/2 trial
VRDN-001 Phase 1/2 trial Population characteristics Healthy Data reported similar to Tepezza Phase 3: May 2022 volunteers • Active TED • CAS ≥4 TED 10mg/kg • Onset of active TED Q3W x2 Reporting today n=6 VRDN-001 symptoms within 12 months n=2 placebo Endpoints reported today: Planned for 4Q22 • Saf
Slide 7: VRDN-001 proof of concept results
VRDN-001 proof of concept results Raymond Douglas, M.D., Ph.D., Director, Thyroid Eye Disease Program, Cedars-Sinai Medical Center 7
Slide 8: Baseline patient characteristics similar to Tepezza trials
Baseline patient characteristics similar to Tepezza trials VRDN-001 Placebo Tepezza Ph2 Tepezza Ph3 n 42 41 6 2 23.4 22.6 Proptosis, mean (SEM) 24.8 (1.2) 22.5 (4.5) CAS, mean (SEM) 5.1 5.1 5.2 (0.3) 5.5 (1.5) 38 (90%) 28 (67%) Diplopia, n (%) 4 (67%) 1 (50%) Diplopia, mean (SEM) 1.8 Not reported 1.
Slide 9: Significant improvement in both signs and symptoms of TED
Significant improvement in both signs and symptoms of TED Overall response defined as reduction in proptosis of ≥2mm and reduction in CAS ≥2 points Overall response Overall response Overall response at week 6 at week 6 at week 6 (% responder) 83% 83% 3 5/6 0% 0% Tepezza Tepezza VRDN-001 Placebo VRDN
Slide 10: Rapid and robust proptosis reduction in VRDN-001 treated patients
Rapid and robust proptosis reduction in VRDN-001 treated patients Change in proptosis (mm) Reduction in proptosis Mean proptosis change Reduction in proptosis (from baseline to week 6) (from baseline to week 6) (from baseline to week 6) (from baseline to week 6) VRDN-001 Active Pla Plac cebo ebo 10m
Slide 11: Proptosis reduction compares favorably with Tepezza
Proptosis reduction compares favorably with Tepezza Mean proptosis change Proptosis responder rate at week 6 Proptosis response rate at week 6 (from baseline to week 6) (Proptosis Responder ≥2mm change from baseline proptosis) 83% 83% Tepezza 56% 56% 55% 55% Tepezza Phase 2 VRDN-001 Phase 3 10mg/kg
Slide 12: Significant improvement in symptoms as measured by CAS
Significant improvement in symptoms as measured by CAS Clinical Activity Score (CAS): a composite 0-7 scale scoring signs and symptoms of TED Change in CAS score Mean change in CAS Reduction in CAS CAS of 0 or 1 at week 6 (from baseline to week 6) Reduction in CAS score (from baseline to week 6) (fr
Slide 13: Symptoms improvement compares favorably with Tepezza
Symptoms improvement compares favorably with Tepezza Clinical Activity Score (CAS): a composite 0-7 scale scoring signs and symptoms of TED Mean CAS Change CAS of 0 or 1 at week 6 Mean CAS improvement at week 6 CAS of 0 or 1 at week 6 (to week 6) (Change from baseline score) Tepezza VRDN-001 Tepezza
Slide 14: 75% of VRDN-001 patients had complete diplopia resolution at week 6
75% of VRDN-001 patients had complete diplopia resolution at week 6 Diplopia resolution rate defined as % of patients with diplopia at baseline that improved to a score of 0 Diplopia resolution vs. Tepezza Complete diplopia resolution at week 6 Complete dip( l% op rie as r pe os no de lu rs t)ion at
Slide 15: Favorable safety profile
Favorable safety profile VRDN-001 10mg/kg TED cohort TEPEZZA label VRDN-001 Placebo TEPEZZA Placebo 10mg/kg (n=84), n (%) (n=86), n (%) (n=2), n (n=6), n ADVERSE REACTIONS: 21 (25%) 6 (7%) Muscle spasms 2 - No serious Nausea - - 14 (17%) 8 (9%) adverse events (SAEs) Alopecia - 1 11 (13%) 7 (8%) 1* -
Slide 16: Takeaways for VRDN-001 and VRDN-002 Data
Takeaways for VRDN-001 and VRDN-002 Data Jonathan Violin, Ph.D., President and Chief Executive Officer 16
Slide 17: All VRDN-001 efficacy measures compare favorably to Tepezza
All VRDN-001 efficacy measures compare favorably to Tepezza Signs Symptoms Improvement in Improvement in Clinical Activity Score (CAS) proptosis and diplopia score Overall response: Proptosis: Proptosis: CAS: CAS: Diplopia: Complete Signs + symptoms Responder rate Mean change Score of 0 or 1 Mean ch
Slide 18: VRDN-001 is a more complete antagonist of IGF-1R
VRDN-001 is a more complete antagonist of IGF-1R, potentially explaining favorable clinical activity Mutational scan reveals unique signatures of IGF-1R antibodies Maximal IGF-1R inhibition (%) Residual IGF-1R activity (% Inhibition) VRDN-001 Tepezza VRDN-002 lonigutumab 10% 00% 4% 26% 24% 39% 25 7%
Slide 19: Responses occur rapidly and compare favorably to Tepezza
Responses occur rapidly and compare favorably to Tepezza Mean time to response (weeks) Median time to proptosis response Mean time to overall response Mean time to response (weeks) VRDN-001 VRDN-001 VRDN-001 VRDN-001 3.0 4.8 3.0 4.8 10mg/kg 10mg/kg 10mg/kg 10mg/kg weeks weeks Tepez Tepezzz aa Tepezz
Slide 20: VRDN-001 may offer a shorter treatment course
VRDN-001 may offer a shorter treatment course Change in proptosis from baseline Phase 3 program: standard and shortened treatment course in active and chronic TED 2023 2024 • THRIVE trial: placebo- Topline controlled, active TED Active TED (n=120) • THRIVE-2 trial: placebo- Topline controlled, chron
Slide 21: VRDN-002 has extended half-life of 30-40 days
VRDN-002 has extended half-life of 30-40 days Non-human primate PK VRDN-001 HV VRDN-002 HV Time (days) Time (days) Time (days) • VRDN-001 and Tepezza PK very similar in non-human primates: ~6 day half-life 1-6 • VRDN-001 and Tepezza PK very similar in oncology patients: ~10-11 day half-life across 6
Slide 22: VRDN-002 mediates IGF-1 increases similar to teprotumumab
VRDN-002 mediates IGF-1 increases similar to teprotumumab PK/PD modeling indicate potential feasibility of 2mL 300mg Q2W to Q4W SC dosing % Change from Baseline of IGF-1 in normal healthy volunteers Teprotumumab VRDN-002 Planned SC POC trial in TED Presumed Placebo Removed Randomized, double-blind t
Slide 23: Key takeaways from today's data
VRDN-001 demonstrated rapid, significant Key improvement across all efficacy measures in takeaways TED at week 6, surpassing prior TED trials from today’s VRDN-001 produces more complete IGF-1R data inhibition via unique receptor interaction VRDN-001 was well tolerated with favorable safety profile
Slide 24: Phase 3 and SC Plans
Phase 3 and SC Plans Jonathan Violin, Ph.D., President and Chief Executive Officer 24
Slide 25: Next steps: large commercial opportunity guides our urgency
Next steps: large commercial opportunity guides our urgency Tepezza annualizing ~$2B in US 2 years after first product approval; substantial room for growth Active TED moderate to severe $3.5B+ 20-25K 35-40K 20-40% Tepezza 0% Tepezza share share today (not approved) ~$2B (2022E) Chronic TED 75K+ 150
Slide 26: Corporate objectives in TED: Build a market-leading franchise
Corporate objectives in TED: Build a market-leading franchise 2 1 Launch Rapidly follow potential best- IV launch with in-class IV TED potential best- therapy in US in-class SC TED and EU therapy 26
Slide 27: Highlights of global IV VRDN-001 Phase 3 program plan
Highlights of global IV VRDN-001 Phase 3 program plan • Designed to assess pivotal efficacy data Global registrational in active AND chronic disease 2023 2024 program for both active • Registrational program designed to enable approval in US, EU, and other and chronic TED Topline Active TED (n=120)
Slide 28: Viridian is poised to deliver a potential best-in-class SC TED therapy
Viridian is poised to deliver a potential best-in-class SC TED therapy Both VRDN-002 and VRDN-003 show potential for Q2W and Q4W dosing to be tested convenient, low-volume, SC product profile • VRDN-002 epitope and in vitro profile very similar • 2mL 300mg SC Q2W compares favorably with to teprotumu
Slide 29: Q2W to Q4W TED SC program plan
Q2W to Q4W TED SC program plan Clinical data allows us to select best program to start a planned Phase 3 trial in early 2024 Decision: Select one SC program to advance 2023 2024 VRDN-002 SC SC POC Q2W to Q4W Topline trial initiation SC patient data 2H23 SC Phase 3 Planned initiation Planning for act
Slide 30: Timeline and planned milestones
Timeline and planned milestones 4Q22 YE22 MY24 3mg/kg active TED data 20mg/kg Initiation data active TED data 1H23 YE24 upcoming medical congress Initiation data Chronic TED Chronic TED POC initiation POC data Q3 Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 2022 2023 2024 SC 003 IND SC phase 3 start SC 002 TED POC da
Slide 31: Second Quarter Financials
Second Quarter Financials Kristian Humer, Chief Financial and Business Officer 31
Slide 32: Q2 2022 Financial Results
Q2 2022 Financial Results CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS CONDENSED CONSOLIDATED BALANCE SHEET DATA (amounts in thousands, except share and per share data) (amounts in thousands) (unaudited) (unaudited) June 30, December 31, Three Months Ended June 30, Six Mont
Slide 33: We Viridian
WE VIRIDIAN
Slide 34: Thank you!
WE VIRIDIAN Thank you!
Viridian Therapeutics, Inc.\DE — Q2 2022 Earnings Presentation on 6ix