Abstract Details

Title Empasiprubart Versus Placebo in Chronic Inflammatory Demyelinating Polyradiculoneuropathy: EMNERGIZE Phase Three Study Design

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P1 - Poster Session 1 (8:00 AM-9:00 AM)

Poster/Presentation
Number 9-021

Objective To describe the study design of the EMNERGIZE trial, which compares empasiprubart versus placebo in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP).

Background CIDP is a rare, autoimmune neuropathy characterized by nerve damage, resulting in disability due to limb weakness, sensory disturbances, and fatigue. Nerve biopsies in CIDP feature complement deposition. Additionally, complement activation promotes macrophage-mediated demyelination. Empasiprubart binds complement C2, blocking activation of classical and lectin complement pathways.

Design/Methods

This Phase 3, randomized, double-blinded, placebo-controlled study will randomize ~160 adults in a 2:1 ratio to receive either empasiprubart IV or placebo on Days 1 and 8 then once every 4 weeks in a 24-week double-blind treatment period (Part A), followed by a 4-week double-blind rollover and a 23-month open-label period (Part B), and a 15-month safety follow-up after the last dose.

Results

The primary endpoint is a reduction of ≥1-point versus baseline in adjusted Inflammatory Neuropathy Cause and Treatment (aINCAT) score at Week 24. Key secondary endpoints include changes from baseline at Week 24 in Inflammatory Rasch-Built Overall Disability Scale score, Medical Research Council Sum score, dominant hand grip strength, and Timed Up and Go, and time to reduction of ≥1 point versus baseline in aINCAT score.

Conclusions

This Phase 3 study will compare the efficacy and safety of empasiprubart versus placebo in patients with CIDP, focusing on functional ability, muscle strength/function, clinical status, gait impairment, and patient-reported outcomes.

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.
Simon Rinaldi, PhD, MBChB	Dr. Rinaldi has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Aregnx. Dr. Rinaldi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hansa. Dr. Rinaldi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Annexon. Dr. Rinaldi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Rinaldi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for CSL Behring. The institution of Dr. Rinaldi has received research support from MRC. The institution of Dr. Rinaldi has received research support from BMA. The institution of Dr. Rinaldi has received research support from GBS/CIDP Foundation International.
Pietro E. Doneddu, MD	The institution of Dr. Doneddu has received research support from GBS CIDP foundation.
Karissa Gable, MD, FAAN	Dr. Gable has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Accordant. Dr. Gable has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Gable has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Takeda. Dr. Gable has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Gable has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Gable has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astrazenca. Dr. Gable has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Guidepoint. Dr. Gable has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for CSL Behring. Dr. Gable has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Grifols . Dr. Gable has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for annexon. Dr. Gable has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Gable 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. Gable has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx.
Mark Stettner, MD (University of Duesseldorf)	No disclosure on file
Luis Querol, MD, PhD (Hospital de la Santa Creu i Sant Pau)	Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CSL Behring. The institution of Dr. Querol has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Querol has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for ArgenX. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alnylam. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Annexon. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avilar. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biocryst. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lundbeck. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for KPL. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lycia. The institution of Dr. Querol has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Querol has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Querol has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis. Dr. Querol has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche. The institution of Dr. Querol has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for ArgenX. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Merck. Dr. Querol has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Grifols. The institution of Dr. Querol has received research support from GBS-CIDP Foundation International. The institution of Dr. Querol has received research support from Grifols. The institution of Dr. Querol has received research support from ISCIII. The institution of Dr. Querol has received research support from CIBERER. The institution of Dr. Querol has received research support from UCB. The institution of Dr. Querol has received research support from ArgenX.
Reena Patel	No disclosure on file
Hafedh Haddad (argenx)	No disclosure on file
Olivier Van de Steen (argenx)	No disclosure on file
Kevin Budding (argenx)	No disclosure on file
Inge Van de Walle	No disclosure on file
Susan V. Ellor, MD, PhD (UC Health)	No disclosure on file
Martin Markov (argenx)	No disclosure on file
Jeffrey A. Allen, MD (University of Minnesota)	Dr. Allen has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for csl behring. Dr. Allen has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Takeda. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Grifols. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alnylam. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astra Zeneca. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Johnson and Johnson. Dr. Allen has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Annexon. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amgen. Dr. Allen has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Dianthus. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL Behring. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Annexon. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Allen has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Allen has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dianthus. Dr. Allen has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for CSL behring. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Takeda. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alnylam. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion. Dr. Allen has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Johnson and Johnson.

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