Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Corporate Update (January 2023)
23 slides from Viridian Therapeutics's corporate update, Jan 9, 2023
Slide 1: VRDN-001 3 mg/kg results in patients with TED
VRDN-001 3 mg/kg results in patients with TED January 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: Viridian programs rapidly advancing towards an estimated $4B+ TED market
Viridian programs are rapidly advancing towards an estimated $4B+ TED market Key takeaways Source: Horizon Therapeutics Q3 22 earnings: peak U.S. annual net sales of >$3B and ex-U.S. estimate of >$1B peak annual net sales, includes expected entrance to Europe; Viridian-sponsored market research All
Slide 4: Viridian rapidly advancing differentiated intravenous and subcutaneous anti-IGF-1R TED programs
Viridian is rapidly advancing differentiated intravenous and subcutaneous anti-IGF-1R TED programs VRDN-001, -002, and -003 are investigational therapies that are not approved for any use in any country. 1) Rodon J et al, J Clin Onc, 2518 Suppl), June 2007 3) Gore L et al, Mol Cancer Ther 8(12 Suppl
Slide 5: VRDN-001 proof-of-concept cohorts tested doses in active TED
VRDN-001 proof-of-concept cohorts tested 3 doses in active TED Active TED Clinical activity score (CAS) of ≥4 Onset of symptoms within past 12 months VRDN-001 10 mg/kg Q3W x 2 (n=6) Placebo (n=2) VRDN-001 20 mg/kg Q3W x 2 (n=6) Placebo (n=2) VRDN-001 3 mg/kg Q3W x 2 (n=9) Placebo (n=1)* *2 patients
Slide 6: Baseline patient characteristics similar to Tepezza trials
Baseline patient characteristics similar to Tepezza trials SEM = Standard error of the mean VRDN-001 (3, 10, and 20 mg/kg) VRDN-001 3 mg/kg VRDN-001 10 mg/kg VRDN-001 20 mg/kg Placebo Tepezza Ph2 Tepezza Ph3 n 21 9 6 6 5 42 41 Proptosis, mean (SEM) 23.7 (0.7) 23.1 (1.2) 24.8 (1.2) 23.6 (1.3) 22.8 (2
Slide 7: VRDN-001 efficacy measures compare favorably to Tepezza
VRDN-001 efficacy measures compare favorably to Tepezza Clinical Activity Score (CAS) = a composite 0-7 scale scoring signs and symptoms of TED *Diplopia was present at baseline in 13 out of 21 drug-treated patients; 4 in 10 mg/kg cohort, 4 in 20 mg/kg cohort and 5 in the 3 mg/kg cohort Overall resp
Slide 8: Efficacy at all doses consistent with full target engagement
Efficacy at all doses consistent with full target engagement IGF-1 data presented as mean ± SEM *Preliminary 3 mg/kg plasma IGF-1 levels include n=5 at week 6 and n=9 at week 3 TED IGF-1 Individual patient CAS Individual patient proptosis Measured by Hertel exophthalmometer Dose (mg/kg) Plasma IGF-1
Slide 9: Exophthalmometry and MRI together provide robust assessment of proptosis
Exophthalmometry and MRI together provide robust assessment of proptosis Hertel Exophthalmometer Most commonly used modality measurement of proptosis Successfully used as primary endpoint in prior clinical trials in TED Magnetic Resonance Imaging (MRI) More precise measurement Centrally read by 2 bl
Slide 10: VRDN-001 responses by exophthalmometer confirmed by centrally reviewed MRI
Adj. mean proptosis change (by exophthalmometer) Mean proptosis change (by MRI) VRDN-001 responses by exophthalmometer are confirmed by centrally reviewed MRI Time (weeks) Proptosis change by MRI (from baseline to week 6) Blinded, centrally-reviewed MRI data were available for 5/5 placebo patients a
Slide 11: Proptosis reduction compares favorably with Tepezza
Proptosis reduction compares favorably with Tepezza Proptosis change from baseline presented as mean ± SEM Mean proptosis change (from baseline to week 6) Proptosis responder rate at week 6 (Proptosis responder ≥2mm change from baseline proptosis) VRDN-001 Tepezza Phase 3 Tepezza Phase 2 n=42 n=41 n
Slide 12: Significant improvement in signs and symptoms as measured by CAS
Significant improvement in signs and symptoms as measured by CAS Clinical Activity Score (CAS): a composite 0-7 scale scoring signs and symptoms of TED Mean change in CAS (from baseline to week 6) Reduction in CAS (from baseline to week 6) Placebo VRDN-001 Placebo VRDN-001 n=21 n=5 3 mg/kg: 67% 10 m
Slide 13: CAS improvement compares favorably with Tepezza
CAS improvement compares favorably with Tepezza Clinical Activity Score (CAS): a composite 0-7 scale scoring signs and symptoms of TED Change in Clinical Activity Score (CAS) from baseline presented as mean ± SEM Tepezza Phase 3 Tepezza Phase 2 VRDN-001 CAS of 0 or 1 at week 6 n=21 n=42 n=41 3 mg/kg
Slide 14: Significant improvement in both signs and symptoms of TED
S Significant improvement in both signs and symptoms of TED Overall response defined as reduction in proptosis of ≥2mm and reduction in CAS ≥2 points Tepezza Phase 2 data: FDA clinical review of Tepezza (BLA 761143) Tepezza Phase 3 data: Douglas RS, et al, NEJM 382:4, Jan 2020 VRDN-001 Tepezza Phase
Slide 15: 54% of VRDN-001 patients had complete diplopia resolution at week 6
S 54% 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 For patients with diplopia at baseline, complete diplopia resolution defined as Gorman subjective diplopia score of zero FDA
Slide 16: Adverse reactions: favorable safety profile
ADVERSE REACTIONS: VRDN-001 3 mg/kg (n=9), n VRDN-001 10 mg/kg (n=6), n VRDN-001 20 mg/kg (n=6), n Placebo (n=5), n TEPEZZA (n=84), n (%) Placebo (n=86), n (%) Muscle spasms 2 2 2** - 21 (25%) 6 (7%) Nausea 2 - - - 14 (17%) 8 (9%) Alopecia - - - 1 11 (13%) 7 (8%) Diarrhea 1 2** 1* - 10 (12%) 7 (8%)
Slide 17: Patient case report from 3 mg/kg cohort
Patient case report from 3 mg/kg cohort Baseline patient characteristics Proptosis: 29 mm CAS: 7 Following VRDN-001 treatment, at week 6 Change in proptosis by HE*: -5 mm Change in proptosis by MRI: -5.2 mm Change in CAS: -6 points Case report patient information and photos taken by patient used wit
Slide 18: Global Phase 3 program includes standard and accelerated treatment courses
Global Phase 3 program includes standard and accelerated treatment courses to maximize differentiation Potential improvements on Tepezza Accelerated, 12-week course of therapy Lower dose – 10mg/kg Shorter infusion times (30 min for VRDN-001 vs. 60-90 min for Tepezza) Faster onset and improved effica
Slide 19: Potential best-in-class subcutaneous (SC) programs in TED
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. 1) Rodon J et al, J Clin Onc, 2518 Suppl), June 2007 3) Gore L et al, Mol Cancer Ther 8(12 Suppl), 2009 2) Kurzrock R et al, Clin Cancer
Slide 20: VRDN-001 data validate potential monthly low volume SC product profile
VRDN-001 data validate potential convenient, monthly low volume SC product profile for VRDN-002 and VRDN-003 PK model shows VRDN-002 and VRDN-003 dosed 2mL 300mg q2w and q4w, with a 600mg loading dose, exceed exposure of 3 mg/kg VRDN-001 IV, suggesting potential for robust efficacy via subcutaneous
Slide 21: Launch with self-administered pen
VRDN-002 SC Q2W or Q4W VRDN-003 SC Q2W or Q4W Launch with self-administered pen: Selection of 1 program based on clinical data in 2H23 Phase 3 trial initiation in early 2024 Viridian plans to deliver a potential best-in-class SC therapy for patients with TED
Slide 22: Corporate priorities
Corporate priorities VRDN-001 IV Phase 2 results in chronic TED expected in 1H23 THRIVE Phase 3 trial enrollment with topline results in active TED expected mid-2024 THRIVE-2 Phase 3 trial initiation in chronic TED planned for 1H23 THRIVE-2 Phase 3 trial topline results in chronic TED expected by YE
Slide 23: Our long-term vision: building a fully-integrated biopharmaceutical company
Launch potential best-in-class IV TED therapy in US and EU Rapidly 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 Potenti
Viridian Therapeutics, Inc.\DE — Corporate Update (January 2023) on 6ix