Ionis Pharmaceuticals, Inc. · NASDAQ:IONS

Ionis Pharmaceuticals — Technical Presentation (July 2023)

8 slides from Ionis Pharmaceuticals's technical presentation, Jul 10, 2023

  1. Slide 1: Eplontersen: NEURO-TTRansform Week 85 Topline Results

    Eplontersen: NEURO-TTRansform Week 85 Topline Results July 10, 2023 Nasdaq: IONS

  2. Slide 2: Forward-Looking Statements

    Forward-Looking Statements This presentation includes forward-looking statements regarding our business and the therapeutic and commercial potential of eplontersen, and Ionis' technologies. Any statement describing Ionis’ goals, expectations, financial or other projections, intentions or beliefs is

  3. Slide 3: NEURO-TTRansform Study Designed to Demonstrate Benefit in Patients with ATTRv-PN

    NEURO-TTRansform Study Designed to Demonstrate Benefit in Patients with ATTRv-PN A multicenter, open-label study in 168 patients with hereditary TTR amyloid polyneuropathy (ATTRv-PN) 1Benson et al, N Engl J Med (2018) 379:22-3 1. Figure adapted from Coelho et al, Neurol Ther (2021) 10:375-89. Random

  4. Slide 4: Treatment Resulted in Substantial and Sustained Reductions in Serum TTR Concentration Compared to Baseline

    Eplontersen Treatment Resulted in Substantial and Sustained Reductions in Serum TTR Concentration Compared to Baseline LSM: Least Square Mean; LSMD: Least Square Mean Difference Week 35:Mean = -11.1% (LSM = -14.5%) Week 81Last dose Mean (SE) % Change From Baseline in Serum TTR Concentration Week 35:

  5. Slide 5: Halt Neuropathy Progression at Week 85

    Eplontersen Continued to Halt Neuropathy Progression at Week 85 Substantial number of patients showed improvement in neuropathy impairment through 19 months of treatment mNIS+7 Composite Score Worse Better Mean (SE) Change From Baseline in mNIS+7 Composite Score Week 35:Mean = 9.8 (LSM = 10.0) Week

  6. Slide 6: Improve Quality of Life at Week 85

    Eplontersen Continued to Improve Quality of Life at Week 85 Norfolk QoL-DN Total Score Worse Better Mean (SE) Change From Baseline in Norfolk QoL-DN Total Score Week 35:Mean = 5.5 (LSM = 8.2) Week 35:Mean = -4.8 (LSM = -3.6) Week 66:Mean = 10.8 (LSM = 14.2) Week 66:Mean = -7.2 (LSM = -5.5) Week 85:M

  7. Slide 7: Demonstrate a Favorable Safety and Tolerability Profile at 85 Weeks

    Eplontersen Continued to Demonstrate a Favorable Safety and Tolerability Profile at 85 Weeks1 At 85 weeks, TEAEs incidence remains consistent with week 66 No TEAEs of special interest led to study drug discontinuation No imbalance of ocular events excluding vitamin A decrease or deficiency No SAEs w

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