Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.
Julian D. Gillmore, National Amyloidosis Centre, University College London, London, UK

Main Body of the Abstract
Introduction: The V30M TTR variant can manifest in hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). This exploratory analysis evaluated the efficacy of eplontersen in patients from the NEURO-TTRansform Phase 3 trial (NCT04136184) with either early-onset (aged <50 years) or late-onset (aged ≥50 years) V30M ATTRv-PN.
Methods: Changes from baseline to Week 65/66 in modified Neuropathy Impairment Score +7 (mNIS+7) composite score (range, –22.3 to 346.3), Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) total score (range, –4 to 136), and modified body mass index (mBMI; nutritional status) were measured.
Results: Eighty-five patients with early-onset (n=54) or late-onset (n=31) V30M ATTRv-PN were randomized to receive eplontersen. A further 33 patients from NEURO-TTR (NCT01737398) with early-onset (n=16) or late-onset (n=17) V30M ATTRv-PN served as a historical placebo group. At baseline, in eplontersen versus placebo groups, mean age was 39.2 versus 40.1 years in patients with early-onset V30M ATTRv-PN and 68.2 versus 69.1 years in patients with late-onset V30M ATTRv-PN. In both subgroups (early- and late-onset), changes from baseline to Week 66 in mNIS+7 composite score and Norfolk QoL-DN total score were in favour of eplontersen compared with placebo. Changes in mBMI from baseline to Week 65 were modest with eplontersen compared with marked reduction (worsening) for placebo (Figure).
Conclusions: The effect of eplontersen (change from baseline to Week 65/66) was consistent in early- and late-onset V30M ATTRv-PN in reducing neuropathy impairment, improving quality of life, and in maintaining better nutritional status, versus historical placebo.

**Author Listing - Lead/ Submitting Author (we will be communicating with this individual): Name, Institution, City and State where the work was done. Co-authors will be added in a separate section.**
Julian D. Gillmore, National Amyloidosis Centre, University College London, London, UK

**Main Body of the Abstract**
**Introduction:** The V30M _TTR_ variant can manifest in hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). This exploratory analysis evaluated the efficacy of eplontersen in patients from the NEURO-TTRansform Phase 3 trial (NCT04136184) with either early-onset (aged <50 years) or late-onset (aged ≥50 years) V30M ATTRv-PN.
**Methods:** Changes from baseline to Week 65/66 in modified Neuropathy Impairment Score +7 (mNIS+7) composite score (range, –22.3 to 346.3), Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) total score (range, –4 to 136), and modified body mass index (mBMI; nutritional status) were measured.
**Results:** Eighty-five patients with early-onset (n=54) or late-onset (n=31) V30M ATTRv-PN were randomized to receive eplontersen. A further 33 patients from NEURO-TTR (NCT01737398) with early-onset (n=16) or late-onset (n=17) V30M ATTRv-PN served as a historical placebo group. At baseline, in eplontersen versus placebo groups, mean age was 39.2 versus 40.1 years in patients with early-onset V30M ATTRv-PN and 68.2 versus 69.1 years in patients with late-onset V30M ATTRv-PN. In both subgroups (early- and late-onset), changes from baseline to Week 66 in mNIS+7 composite score and Norfolk QoL-DN total score were in favour of eplontersen compared with placebo. Changes in mBMI from baseline to Week 65 were modest with eplontersen compared with marked reduction (worsening) for placebo (**Figure**).
**Conclusions:** The effect of eplontersen (change from baseline to Week 65/66) was consistent in early- and late-onset V30M ATTRv-PN in reducing neuropathy impairment, improving quality of life, and in maintaining better nutritional status, versus historical placebo.

Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy: An Exploratory Analysis in Patients With the V30M TTR Variant and Early-Onset or Late-Onset Disease PC118 (#230)

We invite you to attend the XIX International Symposium on Amyloidosis to be held in Rochester, MN.

This symposium is organized as a hybrid meeting (with the focus on in-person) held from Sunday, May 26 through Thursday, May 30, 2024.

Together with the ISA Board and members of Mayo Clinic, a very attractive scientific hybrid program is being planned. The program will cover a variety of types of systemic amyloidosis: ATTR (hereditary and wild-type), AL (light-chain), AA and less frequent forms. There will be key lectures, specific symposia including those company sponsored, oral presentations and poster sessions of accepted abstracts.

COURSE DESCRIPTION
This program will provide a comprehensive review of state-of-the-art education in systemic amyloidosis: basic science, pathophysiology, diagnosis, current and emerging treatments. Scientific presentations will include competitive oral and poster abstracts. The rapid evolution of scientific discovery and expanding treatment options has created tremendous interest in the field and this meeting will fill and important gap in educating scientists and clinicians.