Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Technical Presentation (July 2023)
41 slides from Viridian Therapeutics's technical presentation, Jul 10, 2023
Slide 1: VRDN-001 Phase 1/2 results in patients with chronic TED
VRDN-001 Phase 1/2 results in patients with chronic TED July 2023 Exhibit 99.2
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: Opening Remarks
Opening Remarks Scott Myers, MBA, President and Chief Executive Officer
Slide 4: Positive VRDN-001 preliminary data establish proof-of-concept in chronic TED
Positive VRDN-001 preliminary data establish proof-of-concept in chronic TED Key highlights VRDN-001 is an investigational therapy that is not approved for any use in any country. VRDN-001 data demonstrated clinically meaningful and rapid improvement in signs and symptoms of chronic TED at week 6 af
Slide 5: Viridian is focused on key factors to build a blockbuster TED franchise
Viridian is focused on key factors to build a successful blockbuster product franchise in TED Best-in-class IV product Active + Chronic TED Expand prescriber base US + ex-US market access and sales First / best-in-class SC product Establish new treatment paradigms in TED Building a blockbuster TED f
Slide 6: Building the Viridian team for execution and growth
Building the Viridian team for execution and growth Pre-commercial Product Development Medical Affairs Clinical Development Corporate Affairs
Slide 7: Today's Presenters
Today’s Presenters Kimberly Cockerham, MD, FACS Barrett Katz, MD, MBA CHIEF MEDICAL OFFICER Thomas Ciulla, MD, MBA CHIEF DEVELOPMENT OFFICER
Slide 8: TED Overview and Unmet Medical Need
TED Overview and Unmet Medical Need Kimberly Cockerham, MD, FACS
Slide 9: My background
My background George Washington Medical School: awarded a health professional scholarship from the US ARMY Fellowship-trained: Cosmetic, laser and reconstructive surgery of the face, eyelids, and eye muscles. Neuro-ophthalmology and orbital disease Former: Chief of Oculoplastics and Orbital Disease
Slide 10: TED overview
TED overview Autoantibodies trigger IGF-1R/TSHR pathway1 Progressive disease consisting of an initial active phase, followed by a transition to a secondary chronic phase2 Main signs include proptosis (eye bulging), redness, swelling, diplopia (double vision), and lid retraction3 Severe cases can cau
Slide 11: Vision impairment in patients with TED
Dry Eye Disease Diplopia (double vision) Photophobia (light sensitivity) Decreased Visual Acuity Visual Field Defects Reduced Color Vision Optic Neuropathy TED is associated with significant vision-threatening impairments 1. Bartley GB. Trans Am Ophthalmol Soc (1994) 92:477–588. 2. Chin et al. Clini
Slide 12: Historically described as a biphasic disease course from active to chronic TED
Historically described as a biphasic disease course from active to chronic TED Modified from Wang Y, Patel A, Douglas RS. Thyroid Eye Disease: How A Novel Therapy May Change The Treatment Paradigm. Ther Clin Risk Manag. 2019;15:1305-1318 Active Phase ~18 to 24 months Chronic Phase ~5 to 7+ years Inf
Slide 13: Current treatment options for patients with TED
Current treatment options for patients with TED 1. Stan MN, et al. . Randomized controlled trial of rituximab in patients with Graves’ orbitopathy. J Clin Endocrinol Metab. 2015;100(2):432–441. doi: 10.1210/jc.2014-2572 2. Perez-Moreiras JV, et al. Efficacy of tocilizumab in patients with moderate-t
Slide 14: Unmet medical needs
Unmet medical needs More convenient IV treatment options Availability of subcutaneous treatment option New treatment regimens to drive longer-term benefit and value for patients Achieving a similar benefit as IV with a convenient home- based subcutaneous treatment Chronic maintenance and/or retreatm
Slide 15: VRDN-001 Background and POC trials in TED
VRDN-001 Background and POC trials in TED Barrett Katz, MD, MBA, Chief Medical Officer
Slide 16: VRDN-001 IV is a differentiated full antagonist anti-IGF-1R humanized antibody in TED
VRDN-001 IV is a differentiated full antagonist anti-IGF-1R humanized antibody in TED *Zhao Y, et al. VRDN-001, a Full Antagonist Antibody to the Insulin-Like Growth Factor Receptor-1 (IGF-1R) in Development for Thyroid Eye Disease (TED), Binds to a Distinct Epitope from Teprotumumab, 91st ATA, Octo
Slide 17: Establish proof-of-concept in active and chronic TED
Proptosis of ≥3 mm Clinical activity score (CAS) of ≥4 Onset of symptoms within past 12 mo. Active TED Establish proof-of-concept in active and chronic TED 1st Proptosis of ≥3 mm Any CAS (0-7) Onset of symptoms >12 months prior Chronic TED 2nd
Slide 18: Presentations of VRDN-001 POC data in active TED at medical congresses
Presentations of VRDN-001 POC data in active TED at medical congresses in 1H:23 Presidential Poster Competition Winner
Slide 19: VRDN-001 Phase 1/2 trial design in patients with chronic TED
VRDN-001 Phase 1/2 trial design in patients with chronic TED Randomized, double-blind trial vs placebo Chronic TED Proptosis of ≥3 mm CAS 0-7 Onset of TED symptoms >12 months prior VRDN-001 10 mg/kg Q3W x 2 (n=6) Placebo (n = 2) VRDN-001 3 mg/kg Q3W x 2 (n=6*) Placebo (n=3) *In the 3 mg/kg dose coho
Slide 20: VRDN-001 POC data in Chronic TED
VRDN-001 POC data in Chronic TED Kimberly Cockerham, MD, FACS
Slide 21: Exophthalmometry and MRI together provide robust assessment of proptosis
Exophthalmometry and MRI together provide robust assessment of proptosis Hertel Exophthalmometer Most commonly used modality for measurement of proptosis Successfully used as primary endpoint in prior clinical trials in TED Magnetic Resonance Imaging (MRI) More precise measurement Centrally read by
Slide 22: Evaluating symptoms of TED using clinical activity scoring scales
Evaluating symptoms of TED using clinical activity scoring scales Clinical Activity Score (0 to 7 points) Source: Bartalena L, et al. Eur Thyroid J 2016;5:926. GO: Graves’ Orbitopathy, also known as thyroid eye disease, or TED.; Chemosis: Swelling of conjunctiva, which is the mucous membrane that co
Slide 23: Baseline patient characteristics in chronic TED
Baseline patient characteristics in chronic TED SEM = Standard error of the mean VRDN-001 (10 and 3 mg/kg) VRDN-001 10 mg/kg VRDN-001 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
Slide 24: VRDN-001 clinical activity observed in patients with chronic TED
VRDN-001 clinical activity observed in patients with chronic TED Preliminary data - *MRI available for 4 of 6 VRDN-001 10 mg/kg treated patients, 4 of 6 VRDN-001 3 mg/kg treated patients **2 patients with CAS of 0 at baseline excluded from calculation ***Includes only participants who had diplopia p
Slide 25: Reported change in proptosis from baseline to week 6
Reported change in proptosis from baseline to week 6 MRI data is preliminary. **Blinded, centrally-reviewed MRI data were available for 4 of 6 VRDN-001 10 mg/kg treated patients, 4 of 6 VRDN-001 3 mg/kg treated patients, and 5 of 5 placebo-treated patients. All MRI images were reviewed centrally by
Slide 26: Signs and symptoms as measured by CAS at week 6
Signs and symptoms as measured by CAS at week 6 *Includes patients with CAS > 0 at baseline only; 2 patients with CAS of 0 at baseline excluded from calculation, Baseline mean CAS for patients > 0: Placebo = 2.8 and VRDN-001 treated patients = 4.0 Mean change in CAS* (Patients CAS > 0 at baseline) C
Slide 27: Safety profile across all chronic TED cohorts
VRDN-001 10 & 3 mg/kg (n=13*), n Placebo (n=5), 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%) 1 (20%) Pruritus 0 (0%) 1 (20%) Safety profile across all chronic TED cohorts No serious adverse even
Slide 28: Summary of VRDN-001 Phase 1/2 data in patients with chronic TED
Summary of VRDN-001 Phase 1/2 data in patients with chronic TED With one approved medicine in TED, clinicians and patients are in need of new differentiated treatment options VRDN-001 showed improved clinical activity after 2 doses at week 6 VRDN-001 was generally well tolerated in TED patients with
Slide 29: THRIVE Phase program and SC progress
THRIVE Phase 3 program and SC progress Thomas Ciulla, MD, MBA, Chief Development Officer
Slide 30: THRIVE Phase 3 trial design in patients with active TED
THRIVE Phase 3 trial design in patients with active TED Topline results expected in the middle of 2024 *Q3W = once every three-week dosing Screening (up to 4 weeks) Double-Blind Treatment Period (15 weeks) Placebo (n=30) Active TED CAS ≥3 Proptosis of ≥3 mm Onset of active TED symptoms within prior
Slide 31: THRIVE Phase 3 amendment reflects confidence in 5-dose regimen
THRIVE Phase 3 amendment reflects our confidence in 5-dose regimen and key stakeholder feedback VRDN-001 IV Q3W x 5 (12 weeks) potentially improves benefit-risk for patients VRDN-001 data demonstrated clinically meaningful and rapid improvement in signs and symptoms of TED at week 6 after 2 infusion
Slide 32: THRIVE-2 Phase 3 trial design in patients with chronic TED
THRIVE-2 Phase 3 trial design in patients with chronic TED Trial initiation planned for 3Q 2023 with topline expected by YE:2024 *Q3W = once every three-week dosing Screening (up to 4 weeks) Double-Blind Treatment Period (15 weeks) Placebo Chronic TED Any CAS (0-7) Proptosis of ≥3 mm Onset of TED sy
Slide 33: 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, -002, and -003 are investigational therapies that are not approved for any use in any country. Mechanism Full antagonist Full antagonist Partial antagonist Half-life ~10 to 11 days Expected to match o
Slide 34: VRDN-001, VRDN-002, & VRDN-003 SC program progress and priorities
VRDN-001, VRDN-002, & VRDN-003 SC program progress and priorities SC formulation work completed for all three SC programs at 150 mg/mL concentration allowing for administration of 300 mg / 2 mL VRDN-001 SC IND amendment submitted to FDA VRDN-003 IND filing submitted to FDA VRDN-002 Phase 1 trial wit
Slide 35: Closing Remarks
Closing Remarks Scott Myers, MBA, President and Chief Executive Officer
Slide 36: VRDN SC: Potential first/best-in-class self-administered SC pen
VRDN SC Potential first/best-in-class self-administered SC pen Plan to launch with a potentially best-in-class IV followed by a self-administered SC pen in TED * Pictures used for illustrative purposes only VRDN-001 IV Potential best-in-class Q3W IV treatment for TED 2 1
Slide 37: Viridian is focused on key factors to build a blockbuster TED franchise
Viridian is focused on key factors to build a successful blockbuster product franchise in TED Best-in-class IV product Active + Chronic TED Expand prescriber base US + ex-US market access and sales First / best-in-class SC product Establish new treatment paradigms in TED Building a blockbuster TED f
Slide 38: Our long-term vision: building a fully-integrated biopharmaceutical company
Launch potential best-in-class IV TED therapy in US and ex-US Follow with potential best-in-class SC TED therapy Replicate drug development and launch excellence in additional autoimmune and rare diseases Our long-term vision: building a fully-integrated biopharmaceutical company Growth Potential
Slide 39: Q&A Session
Q&A Session
Slide 40: Q&A Session Participants
Q&A Session Participants Scott Myers, MBA PRESIDENT AND CEO Thomas Ciulla, MD, MBA CHIEF DEVELOPMENT OFFICER Todd James SVP, CORPORATE AFFAIRS & IR Kimberly Cockerham, MD, FACS Barrett Katz, MD, MBA CHIEF MEDICAL OFFICER Kristian Humer CHIEF FINANCIAL & BUSINESS OFFICER
Slide 41: Thank you!
WE VIRIDIAN Thank you!
Viridian Therapeutics, Inc.\DE — Technical Presentation (July 2023) on 6ix