Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — miRagen Therapeutics — Investor Presentation (October 2020)
26 slides from Viridian Therapeutics's investor presentation, Oct 28, 2020
Slide 1: miRagen Therapeutics Corporate Overview
Exhibit 99.2 miRagen Therapeutics Corporate Overview NASDAQ: MGEN October 2020
Slide 2: Forward-looking statements
This presentation contains forward-looking statements relating to Miragen Therapeutics, Inc., including statements about our plans to obtain funding, develop and commercialize our therapeutic candidates, our planned clinical trials, the timing of and our ability to obtain and maintain regulatory app
Slide 3: Viridian Transaction Highlights
Viridian Transaction Highlights The acquisition of Viridian was structured as a stock-for-stock transaction whereby all of Viridian’s outstanding equity interests were exchanged for a combination of shares of miRagen common stock and shares of a newly designed non-voting Series A convertible preferr
Slide 4: Financial overview: Company Capitalization Structure
Financial overview Company Capitalization Structure As of October 28, 2020 Common Shares Series A Preferred Stock As Converted Common Shares1 Common stock outstanding 68,483,666 68,483,666 Series A Preferred Stock 398,498 398,498,499 68,483,666 398,498 466,982,165
Slide 5: Expanding the definition of innovation in drug discovery
Conventional approach to drug discovery prioritizes more differentiation than is necessary Focus on the “new” (targets, modalities, platform technologies) leaves many therapeutic spaces under competitive Still limited treatment options for patients, many left with only one option OCTOBER 2020 miRage
Slide 6: Disease areas to explore
Disease areas to explore Motivated patient populations with few options Few approved, high-priced therapies Limited market competition Compelling returns by new entrant Strategic gaps Price Access Delivery/Quality of Life Efficacy OCTOBER 2020 Focus for first indications: Thyroid Eye Disease Undiscl
Slide 7: Viridian Therapeutics Pipeline
Viridian Therapeutics Pipeline VRDN-001* (IV) VRDN-002 (SC) VRDN-003 (SC) VRDN-004 VRDN-005 and beyond New IND 3Q21 Ph2 pilot cohort data 1Q22 Ph2 data MY22 IND YE21 Ph1 data MY22 Ph2 data 2023 IND 2022 IND 2022 IND 2022 Thyroid Eye Disease (TED) Undisclosed Rare Orphan Disease *Previously developed
Slide 8: Program 1: IGF-1R & Thyroid Eye Disease (TED)
Program 1: IGF-1R & Thyroid Eye Disease (TED)
Slide 9: Thyroid Eye Disease (TED): patient snapshot
OCTOBER 2020 Debilitating orphan eye disease that significantly impacts quality of life 15,000-20,000 new patients per year1 Progresses from acute to fibrotic phase (chronic stage) after 6-24 months with potential for permanent scarring Historically poor treatment options (surgery, radiation, steroi
Slide 10: Teprotumumab: from failed oncology candidate to successful TED drug
OCTOBER 2020 Teprotumumab (IGF-1R mAb): from failed oncology candidate to successful TED drug Teprotumumab (R1507) enters clinic for oncology Ph2 oncology trials fail to meet efficacy criteria. Roche discontinues teprotumumab development. River Vision Development founded to develop teprotumumab for
Slide 11: Current U.S. TED market expanding towards $3B
Patient population*: “active/acute” TED: 15K-20K incidence/year “inactive/chronic” TED: 70K TEPEZZA wholesale acquisition cost: $343K/course (annual net realized price $200K/course)2 Market will remain branded until at least 2032 (BLA exclusivity for teprotumumab), potentially to 2039 (pending metho
Slide 12: Teprotumumab Ph3 results
Teprotumumab Ph3 Randomized, double-masked, placebo controlled 83 active TED patients 41 teprotumumab 42 placebo Dosing: q3w, first infusion 10 mg/kg, subsequent 7 infusions 20 mg/kg Primary endpoint: proptosis response OCTOBER 2020 An effective treatment: IGF-1R inhibition with anti-IGF-1R antibody
Slide 13: Our approach to TED
Convenient SC VRDN-002 Quick entrant, IV infusion VRDN-001 OCTOBER 2020 Our approach to TED Strategy in TED: Building our own momentum and growth Licensed a clinical IGF-1R antibody from ImmunoGen/Sanofi previously tested in >100 oncology patients Improve PK of existing mAb sequence with half-life e
Slide 14: IV Quick Entrant: VRDN-001
IV Quick Entrant: VRDN-001
Slide 15: VRDN-001: Leverage existing clinical stage mAb
OCTOBER 2020 Approach: Licensed AVE-1642 from ImmunoGen/Sanofi, a clinical-stage anti-IGF-1R mAb, to enable fastest possible clinical trial in TED Terms: worldwide rights, single digit million up-front, milestones, mid-single digit royalties Advantages: Existing GLP toxicology package Human PK/PD an
Slide 16: VRDN-001 and teprotumumab are comparable
OCTOBER 2020 VRDN-001 and teprotumumab are comparable VRDN-001 Teprotumumab Half-life (in oncology patients) 10 days(1) 10 days(4,5) IGF-1 increase 6X(1) 3X(3) Affinity (Kd) 0.21 nM(2) 5 nM(4) Hyperglycemia 5-52% Severe in 5.6% of patients (n=106, 2 trials) (3) 11-50% Severe in 3% of patients (n=335
Slide 17: Target engagement suggests receptor saturation at low doses
OCTOBER 2020 Target engagement suggests receptor saturation at doses as low as 3 mg/kg Soria et al, EJC, 2013; n = 27; solid tumor; 3-24 mg/kg, q3w VRDN-001
Slide 18: Explore accelerated adaptive trial design
OCTOBER 2020 Explore accelerated adaptive trial design 80% of the benefit with teprotumumab seen within 12 weeks à explore a 12-week endpoint (pending FDA feedback) Results from phase 2 will enable side-by-side comparison with teprotumumab 3 expansion cohorts: “Saturating” dose: Expected to saturate
Slide 19: Convenient SC: VRDN-002
Convenient SC: VRDN-002
Slide 20: Opportunity for VRDN-002
Opportunity for VRDN-002 Doses ≤ 300mg may enable SC injection Examples: Takhzyro (300 mg SC) and Dupixent (300 mg SC) An antibody with a longer half-life will require lower cumulative dose Teprotumumab half-life can be exceeded by use of FcRn-binding mutations Precedent: adding half-life extending
Slide 21: VRDN-002: Convenient SC timeline
Timeline IND: YE 2021 Ph1 healthy volunteer data: mid-year 2022 Ph2 efficacy in patients: 2023 OCTOBER 2020 Goal: Develop biosuperior SC injection Approach: Half-life extension technology VRDN-002: Convenient SC VRDN-002 Ph2 Data 2023 Timeline: YE 2021 MY 2022 YE 2022 MY 2023 IND YE 2021 Ph1 data MY
Slide 22: Our Pipeline, Team, and Financials
Our Pipeline, Team, and Financials
Slide 23: miRagen leadership, board of directors
Board of directors Jeffrey Hatfield Chairman of the Board of miRagen Peter Harwin Managing Member, Fairmount Funds Tomas Kiselak Managing Member, Fairmount Funds Lee Rauch CEO, miRagen Arlene M. Morris CEO, Willow Advisors, LLC Joe Turner Board Member and Audit Chair OCTOBER 2020 Management Lee Rauc
Slide 24: Cash and Milestones
Cash with completion of $91M PIPE is expected to be approximately $140M Expected cash runway: through 2023 OCTOBER 2020 Cash and Milestones VRDN-002 IND YE 2021 Q3 2021 VRDN-001 New IND 3Q 2021 VRDN-005 Program announcement 2021 Q4 2021 Q1 2022 Q2 2022 Q3 2022 Q4 2022 Q1 2023 Q2 2023 VRDN-001 Ph2 pi
Slide 25: miRagen takeaways
OCTOBER 2020 miRagen takeaways Attractive markets, high probability of success, multiple shots on goal Tepezza has been a highly successful launch, and there is currently no other competition in the market $3B+ addressable market opportunity in thyroid eye disease We have an antibody that Binds the
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miRagen Therapeutics — Investor Presentation (October 2020) on 6ix