Viridian Therapeutics, Inc. · NASDAQ:VRDN

Viridian Therapeutics — Technical Presentation (September 2023)

17 slides from Viridian Therapeutics's technical presentation, September 2023

  1. Slide 1: Preclinical Pharmacokinetics and Clinical Exposure Prediction for VRDN-003

    Preclinical Pharmacokinetics and Clinical Exposure Prediction for VRDN-003, a Next- Generation Half-life Extended Antibody to the IGF-1 Receptor for Thyroid Eye Disease (TED) Kelly Foster, Brent Dickinson, Vahe Bedian Viridian Therapeutics, Inc., Waltham, MA #OP-09-02

  2. Slide 2: Disclosures

    Disclosures • These studies were sponsored by Viridian Therapeutics, Inc. All data are proprietary. • VRDN-001 and VRDN-003 are investigational therapies not approved in any country. All authors met the ICMJE authorship criteria and had full access to relevant data. • Kelly Foster, Brent Dickinson,

  3. Slide 3: Background

    Background • Clinical and preclinical evidence suggest a central role for IGF-1 receptor antagonism in reducing the inflammation and proptosis that occur in TED1-4 • VRDN-001, a full antagonist antibody to IGF-1R with subnanomolar affinity, is in development for the treatment of TED − 2 infusions of

  4. Slide 4: Objectives

    to the meeting. Kelly Foster named the meeting Review Objectives ETA 003 presentation. Kelly Foster named the meeting Review • Investigate the pharmacokinetic (PK) parameters of VRDN-003 ETA 003with compared presentation. those of VRDN-001 in nonhuman primates (NHPs) Today • Investigate VRDN-003 SC

  5. Slide 5: PK of VRDN-003 vs VRDN-001 in nonhuman primates (NHPs)

    PK of VRDN-003 vs VRDN-001 in nonhuman primates (NHPs)

  6. Slide 6: Methods for NHP study

    Methods for NHP study • VRDN-001 or VRDN-003 was administered to a total of 16 cynomolgus ROA n monkeys as a single dose by either IV IV 4 injection or SC injection at 7.5 mg/kg VRDN-001 SC 4 • PK samples were collected at 9 time points through 14 days IV 4 VRDN-003 • Data were analyzed using WinNon

  7. Slide 7: Concentration over time: VRDN-003 vs VRDN-001 in NHPs

    Concentration over time: VRDN-003 vs VRDN-001 in NHPs Intravenous injection Subcutaneous injection 100 VRDN-003 IV VRDN-003 SC VRDN-001 IV VRDN-001 SC Concentration (μg/mL) Concentration (μg/mL) 100 10 10 0 5 10 15 0 5 10 15 Time (days) Time (days) Exposure was greater for VRDN-003 than for VRDN-001

  8. Slide 8: PK parameters in NHPs

    PK parameters in NHPs Vza CLa t1/2 AUCinf ROA (mL/kg) (mL/day/kg) (days) (day*µg/mL) %F IV 78 ± 6 8.5 ± 2.1 6.6 ± 1.3 915 ± 191 VRDN-001 70 SC 112 ± 23 13.1 ± 5.6 6.3 ± 1.4 636 ± 222 IV 86 ± 17 5.2 ± 0.8 11.9 ± 3.4 1480 ± 223 VRDN-003 71 SC 132 ± 2 7.2 ± 1.2 12.8 ± 2.0 1050 ± 182 ROA, route of admin

  9. Slide 9: PK parameters in NHPs

    PK parameters in NHPs Vza CLa t1/2 AUCinf ROA (mL/kg) (mL/day/kg) (days) (day*µg/mL) %F IV 78 ± 6 8.5 ± 2.1 6.6 ± 1.3 915 ± 191 VRDN-001 70 SC 112 ± 23 13.1 ± 5.6 6.3 ± 1.4 636 ± 222 IV 86 ± 17 5.2 ± 0.8 11.9 ± 3.4 1480 ± 223 VRDN-003 71 SC 132 ± 2 7.2 ± 1.2 12.8 ± 2.0 1050 ± 182 ROA, route of admin

  10. Slide 10: VRDN-003 SC dosing regimen simulations using PK modeling in humans

    VRDN-003 SC dosing regimen simulations using PK modeling in humans

  11. Slide 11: Methods for PK modeling in humans

    Methods for PK modeling in humans • Population PK models were developed based on data obtained from VRDN-001 dosed in healthy volunteers • VRDN-001 is described by a 2-compartment model with linear elimination from the central compartment • VRDN-003 is described by a model that uses identical parame

  12. Slide 12: VRDN-003 SC dosing simulations in humans

    VRDN-003 SC dosing simulations in humans Median (95%) VRDN-001, 3 mg/kg IV Q3W VRDN-003, 600 mg loading dose + 300 mg SC Q4W

  13. Slide 13: VRDN-003 SC dosing simulations in humans

    VRDN-003 SC dosing simulations in humans Median (95%) VRDN-001, 3 mg/kg IV Q3W VRDN-003, 600 mg loading dose + 300 mg SC Q4W Dosing regimen Cmin (μg/mL) 3 mg/kg Q3W 14 VRDN-001 IV VRDN-003 SC 300 mg Q4W 49 (with 600 mg loading dose) Q2W, Q3W, Q4W; once every 2, 3, or 4 weeks

  14. Slide 14: VRDN-003 SC dosing simulations in humans

    VRDN-003 SC dosing simulations in humans Median (95%) VRDN-001, 3 mg/kg IV Q3W VRDN-003, 600 mg loading dose + 300 mg SC Q4W Dosing regimen Cmin (μg/mL) 3 mg/kg Q3W 14 VRDN-001 IV 10 mg/kg Q3W 46 VRDN-003 SC 300 mg Q4W 49 (with 600 mg loading dose) Q2W, Q3W, Q4W; once every 2, 3, or 4 weeks

  15. Slide 15: VRDN-003 SC dosing simulations in humans

    VRDN-003 SC dosing simulations in humans Median (95%) VRDN-001, 3 mg/kg IV Q3W VRDN-003, 600 mg loading dose + 300 mg SC Q4W Dosing regimen Cmin (μg/mL) 3 mg/kg Q3W 14 VRDN-001 IV 10 mg/kg Q3W 46 VRDN-003 SC 300 mg Q4W 49 (with 600 mg loading dose) 300 mg Q2W 104 Q2W, Q3W, Q4W; once every 2, 3, or 4

  16. Slide 16: Conclusions

    Conclusions • VRDN-003, a next-generation half-life extended version of VRDN-001 – Demonstrated greater exposure and twice the half-life of VRDN-001 for both IV and SC administration in NHPs – Demonstrated exposures with SC administration that matched or exceeded IV administration of VRDN-001 in hum

  17. Slide 17: Thank you! Questions?

    Thank you! Questions? Preclinical Pharmacokinetics and Clinical Exposure Prediction for VRDN-003, a Next- Generation Half-life Extended Antibody to the IGF-1 Receptor for Thyroid Eye Disease (TED) Kelly Foster Viridian Therapeutics, Inc., Waltham, MA #OP-09-02

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