Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Investor Presentation (May 2022)
23 slides from Viridian Therapeutics's investor presentation, May 12, 2022
Slide 1: Viridian Therapeutics May 2022
Exhibit 99.2 Viridian Therapeutics May 2022
Slide 2: Cautionary note regarding forward-looking statements
Cautionary note regarding forward-looking statements This presentation contains forward-looking statements relating to Viridian Therapeutics, Inc., including statements about our plans to obtain funding, develop and commercialize our therapeutic candidates, our planned clinical trials, the timing of
Slide 3: Investment highlights
Investment highlights Viridian is engineering and developing best-in-class therapeutic antibodies for patients suffering from serious diseases that are underserved by current therapies Lead programs are novel and differentiated IGF-1R monoclonal antibodies being developed for Thyroid Eye Disease (“T
Slide 4: Viridian's discovery engine leverages core expertise in antibody discovery and engineering
Viridian’s discovery engine leverages core expertise in antibody discovery and engineering 3 5 2 4 1 First entrant Significant market First entrant Viridian Purpose-built validates a new opportunity; leaves room for engineering clinical team to mechanism of serious disease improvement creates improv
Slide 5: Pipeline
Pipeline Phase 2 Preclinical Phase 1 Discovery Phase 3 Proptosis reduction VRDN-001 – Intravenous (potentially subcutaneous) PoC data 3Q 2022 Thyroid Eye FIH data (IV, SAD, NHV) Disease VRDN-002 – Subcutaneous 3Q 2022 (IGF-1R) VRDN-003 Rare Disease VRDN-004 (Undisclosed) Undisclosed VRDN-005 5 5 Pip
Slide 6: Substantial market opportunity in thyroid eye disease
Substantial market opportunity in thyroid eye disease • Debilitating, progressive and sight-threatening orphan disease that significantly impacts quality of life 1 • 20-25,000 U.S. patients diagnosed with active disease each year 1 • Similar epidemiology ex-U.S. 2 • >$3.5B potential peak U.S. annual
Slide 7: Tepezza launch reset TED landscape, creating opportunities to evolve treatment paradigms
Tepezza® launch reset TED landscape, creating opportunities to evolve treatment paradigms PEAK SALES ESTIMATES >$3.5B; POTENTIAL TO IMPROVE VALUE NO COMPETITION PROPOSITION OF LEAD MOLECULE ® TEPEZZA Revenues • More compelling product profile by targeting 5 3 lower dose and treatment burden – ~65% >
Slide 8: History of IGF-1R antibodies in thyroid eye disease
History of IGF-1R antibodies in thyroid eye disease Teprotumumab (R1507), River Vision Development FDA grants Breakthrough Tepezza® approved IGF-1R mAb, enters clinic founded to develop designation for January 2020 for oncology teprotumumab for TED teprotumumab for TED 2016 2020 2006 2011 2004 2009
Slide 9: An IGF-1R antibody elicits rapid, robust response with dramatic change in proptosis after two doses
An IGF-1R antibody elicits rapid, robust response with dramatic change in proptosis after two doses Opportunity For Early And Clear Proptosis PoC TEPROTUMUMAB PH3 PROPTOSIS RESPONSE CHANGE FROM BASELINE IN PROPTOSIS Randomized, double-masked, placebo controlled 83 active TED patients: 41 active trea
Slide 10: VRDN-001: previous clinical data and successful pre-IND meeting confirms rapid path to clinical proof of concept
VRDN-001: previous clinical data and successful pre-IND meeting confirms rapid path to clinical proof of concept RAPID PROGRESS TO DATE NEAR TERM CLINICAL DATA • Identified AVE-1642 as a candidate for treating TED – Mechanism comparable to teprotumumab – Previously administered to >100 oncology pati
Slide 11: AVE-1642 and teprotumumab showed similar profiles in oncology trials
AVE-1642 and teprotumumab showed similar profiles in oncology trials AVE-1642 TEPROTUMUMAB Half-life (in oncology (1) (4,5) 10 days 10 days patients) (1) (6) IGF-1 increase 6X 3X (2) (7) Affinity (K ) 0.21 nM 5 nM D 5-52% 11-50% Hyperglycemia (3) (4,6,8,9) Severe in 5.6% of patients (n=106, 2 trials
Slide 12: IGF-1 levels generated in oncology patients suggest VRDN-001 target engagement at doses as low as 3 mg/kg
IGF-1 levels generated in oncology patients suggest VRDN-001 target engagement at doses as low as 3 mg/kg 3 mg/kg 6 mg/kg 12 mg/kg 18 mg/kg 24 mg/kg IGF1 IGF2 IGFBP3 12 Soria et al, EJC, 2013; n = 27; solid tumor; 3-24 mg/kg, q3w VRDN-001
Slide 13: VRDN-001 Phase 1/2 trial design: PoC data from two cohorts of TED patients expected in 3Q 2022
VRDN-001 Phase 1/2 trial design: PoC data from two cohorts of TED patients expected in 3Q 2022 POPULATION: • TED patients: active moderate to severe TED, same as teprotumumab Phase 3 • Normal Healthy Volunteers (NHVs): conducted in PROOF OF CONCEPT parallel to accelerate dose escalation decisions NH
Slide 14: Interim results from healthy volunteer portion of Phase 1/2 TED proof of concept trial
Interim results from healthy volunteer portion of Phase 1/2 TED proof of concept trial • Randomized double-blind placebo-controlled evaluation of safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers • Doses: two infusions of 3, 10, or 20 mg/kg q3w • Preliminary safety o
Slide 15: Interim safety and tolerability observations of VRDN-001 in healthy volunteers
Interim safety and tolerability observations of VRDN-001 in healthy volunteers (TEAE) ® TEPEZZA label Study VRDN-001-101, normal healthy volunteers VRDN-001 VRDN-001 Placebo Masked top dose cohort ® TEPEZZA Placebo Adverse Reactions 3 mg/kg 10 mg/kg (VRDN-001 20 mg/kg (n=84), n (%) (n=86), n (%) (n=
Slide 16: Plasma IGF-1 levels in healthy volunteers indicate VRDN-001 inhibits IGF1R at doses from 3-20 mg/kg
Plasma IGF-1 levels in healthy volunteers indicate VRDN-001 inhibits IGF1R at doses from 3-20 mg/kg Plasma levels of IGF-1 • Rapid and sustained increases in in healthy volunteers administered VRDN-001 plasma IGF-1, a biomarker for IGF- 1R inhibition nd 2 dose 1000 • Similar rate and extent of IGF-1
Slide 17: VRDN-002: a novel, next-generation IGF-1R mAb for TED
VRDN-002: a novel, next-generation IGF-1R mAb for TED • Treatment paradigm inconvenient: 8 IV infusions in 24 weeks (q3w) Current dosing • Dose is not conducive to low volume SC injection: 20mg/kg q3w paradigm suboptimal maintenance dose translates to ~500 mg/week • High affinity for IGF-1R offers p
Slide 18: VRDN-002 preclinical data supports potential best-in-class subcutaneous product profile
VRDN-002 preclinical data supports potential best-in-class subcutaneous product profile NHP PHARMACOKINETICS NHP PK • VRDN-002 binds IGF-1R with high affinity • IV and SC PK in non-human primates show 100 extended half-life and sustained exposure vs. teprotumumbab and VRDN-001 – VRDN-002 plasma conc
Slide 19: Leadership: Management team
Leadership Management team Jonathan Violin, Ph.D. President & CEO Vahe Bedian, Ph.D. Chief Scientist Kristian Humer Chief Financial & Business Officer Barrett Katz, M.D. Chief Medical Officer Deepa Rajagopalan, M.D. SVP, New Product and Portfolio Development Janielle Newland SVP, Human Resources Lar
Slide 20: Leadership: Board of Directors
Leadership Board of Directors Tomas Kiselak Chairman of the Board Managing Member, Fairmount Funds Management, LLC Peter Harwin Director Managing Member, Fairmount Funds Management, LLC Arlene Morris Director Chief Executive Officer, Willow Advisors, LLC Jennifer Moses, CPA Director, Audit Committee
Slide 21: 2022 milestones
2022 milestones IV VRDN-001 IV VRDN-001 IV VRDN-001 NHV data Proptosis PoC data 3mg/kg data VRDN-002 IV VRDN-002 IND clearance FIH data Q1 Q2 Q3 Q4 21 Milestones
Slide 22: Investment highlights
Investment highlights Viridian is engineering and developing best-in-class therapeutic antibodies for patients suffering from serious diseases that are underserved by current therapies Lead programs are novel and differentiated IGF-1R monoclonal antibodies being developed for Thyroid Eye Disease (“T
Slide 23: Viridian Therapeutics Contact us
Viridian Therapeutics Contact us: viridiantherapeutics.com IR@viridiantherapeutics.com
Viridian Therapeutics, Inc.\DE — Investor Presentation (May 2022) on 6ix