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Abstract Details

Efficacy of Eplontersen According to Baseline Polyneuropathy Severity in Patients with Hereditary Transthyretin Amyloidosis: A Subgroup Analysis of the NEURO-TTRansform Index Study
General Neurology
S32 - General Neurology 1 (5:06 PM-5:18 PM)
009
To assess the impact of baseline polyneuropathy severity on eplontersen response, in patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN).
Patients with ATTRv amyloidosis experience motor, sensory, and autonomic neuropathies that impact quality of life (QoL). In NEURO-TTRansform (NCT04136184), eplontersen demonstrated benefits in QoL and neuropathy to Week 65/66 versus historical placebo in patients with ATTRv-PN.
NEURO-TTRansform participants (N=141) and participants who received historical placebo (N=59) were divided post hoc into quartiles based on baseline Neuropathy Impairment Score (NIS; 3.5–<21.5 [Q1; least impairment at baseline]; 21.5–<39.5 [Q2]; 39.5–<62.5 [Q3]; 62.5–<127.8 [Q4; most impairment at baseline]). Neuropathic impairment (modified NIS+7 [mNIS+7]) and QoL (Norfolk Quality of Life-Diabetic Neuropathy [Norfolk QoL-DN] questionnaire) were assessed.
Mean (SD) age was lowest in Q1 and highest in Q4 (placebo 52.1 [14.9]–64.6 [13.3] years; eplontersen 48.2 [15.8]–58.8 [14.1] years). Mean (SD) baseline mNIS+7 scores ranged from 28.8 (13.1)–123.2 (16.7) (placebo) and 31.7 (11.5)–132.7 (30.2) (eplontersen). Across quartiles, mean (SE) mNIS+7 scores increased numerically (worsened) from baseline to Week 66 for placebo (change from baseline: Q1, 13.8 [5.7]; Q2, 17.0 [7.1]; Q3, 41.9 [7.5]; Q4, 25.2 [4.7]), but remained stable for eplontersen (Q1, 0.4 [2.1]; Q2, −4.7 [3.6]; Q3, 2.2 [3.5]; Q4, 0.9 [3.2]). Mean (SD) baseline Norfolk QoL-DN scores ranged from 31.5 (25.6)–64.9 (20.9) (placebo) and 21.6 (19.9)–61.9 (24.7) (eplontersen). Mean (SE) scores increased numerically (worsened) to Week 66 for placebo (change from baseline: Q1, 7.2 [4.9]; Q2, 1.0 [6.7]; Q3, 27.0 [4.9]; Q4, 10.8 [4.6]), but improved for eplontersen (Q1, −5.9 [2.2]; Q2, −9.0 [3.7]; Q3, −9.0 [4.1]; Q4, −5.0 [2.8]). 
Consistent benefits of eplontersen treatment in the degree of neuropathy impairment and in QoL were observed to Week 66, regardless of baseline PN severity.
Authors/Disclosures
Thomas Brannagan III, MD, FAAN (Columbia University)
PRESENTER 		Dr. Brannagan has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Brannagan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Intellia. Dr. Brannagan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Astra Zenica. Dr. Brannagan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Annexon. The institution of Dr. Brannagan has received research support from Alnylam. The institution of Dr. Brannagan has received research support from Abcuro. The institution of Dr. Brannagan has received research support from Ionis. The institution of Dr. Brannagan has received research support from Vertex. The institution of Dr. Brannagan has received research support from NMD Pharma.
John L. Berk John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam Pharmaceuticals. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis Pharmaceuticals. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca/IONIS. John L. Berk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eidos/BridgBio. John L. Berk has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Intellia Therapeutics. John L. Berk has received research support from Alnylam . John L. Berk has received research support from Ionis. John L. Berk has received research support from Eidos/Bridgbio.
Morie Gertz, MD Dr. Gertz has received personal compensation for serving as an employee of AstraZeneca. Dr. Gertz has received personal compensation for serving as an employee of Al nylym. Dr. Gertz has received personal compensation for serving as an employee of Johnson Johnson. Dr. Gertz has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for beigene. Dr. Gertz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regeneron. Dr. Gertz has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for American board of Internal Medicine.
Sami L. Khella, MD, FAAN (Presbyterian Med Ctr/Dept of Neuro) Dr. Khella has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Ionis. Dr. Khella has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ionis. Dr. Khella has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Khella has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Khella has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Eidos.
Ahmad Masri, MD Dr. Masri has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Cytokinetics, BMS, BridgeBio, Pfizer, Ionis, Lexicon, Attralus, Alnylam, Haya, Alexion, Akros, Edgewise, Rocket, Lexeo, Prothena, BioMarin, AstraZeneca, Avidity, Neurimmune, and Tenaya.. The institution of Dr. Masri has received research support from Pfizer, Ionis, Attralus, and Cytokinetics.
Mathew a. Maurer, MD Dr. Maurer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Maurer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Maurer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Maurer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Intellia. Dr. Maurer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NovoNordisk. Dr. Maurer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bayer. Dr. Maurer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BridgeBIo.
Marcia Waddington Cruz, MD (Hospital Universitario) Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ionis. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sobi. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ionis. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC. Dr. Waddington Cruz has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NovoNordisk.
Theodore J. Kwoh, PhD Mr. Kwoh has received personal compensation for serving as an employee of Ionis Pharmaceuticals Inc. Mr. Kwoh has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Ionis Pharmaceuticals Inc. Mr. Kwoh has stock in Ionis Pharmaceuticals Inc.
Maksym Pola, MD, PhD Dr. Pola has received personal compensation for serving as an employee of Astrazeneca. Dr. Pola has stock in Astrazeneca.
Jonatan Nåtman, MSc Statistics Mr. Nåtman has received personal compensation for serving as an employee of AstraZeneca.
Laura Piera Obici (Fondazione IRCCS Policlinico San Matteo) Laura Piera Obici has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Laura Piera Obici has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AstraZeneca. Laura Piera Obici has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novo Nordisk. Laura Piera Obici has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for BridgeBio. Laura Piera Obici has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for AstraZeneca. Laura Piera Obici has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alnylam.