Latest Conference Coverage

Efgartigimod Shows Consistent Safety Profile Across Immunoglobulin G-Mediated Autoimmune Diseases in Trials

The safety and tolerability of efgartigimod was consistent across different indications and doses, showing comparable rates of treatment-related adverse events with placebo in clinical trials for myasthenia gravis, primary immune thrombocytopenia, and pemphigus.

Zilucoplan Shows Reduction in MG Worsening in Phase 3 RAISE Study for Generalized Myasthenia Gravis

A post hoc analysis of the phase 3 RAISE study evaluating zilucoplan, a complement C5 inhibitor, showed significantly fewer patients experienced worsening and fewer requiring rescue therapy while on medication compared with placebo.

Results on Efgartigimod for Chronic Inflammatory Demyelinating Polyneuropathy Treatment: Richard Lewis, MD

The professor of neurology at Cedars-Sinai Medical Center talked about findings from the ADHERE trial assessing efgartigimod, a human IgG1 antibody Fc fragment, as a treatment for patients with chronic inflammatory demyelinating polyneuropathy. [WATCH TIME: 6 minutes] 

Social Determinants of Health Lead to Greater Suboptimal Treatment Response in Myasthenia Gravis

A retrospective study presented at the 2023 AANEM meeting revealed that social determinants of health are linked to poorer treatment outcomes in myasthenia gravis patients, emphasizing the need for identifying and supporting at-risk individuals.

Vutrisiran Shows Benefit Across Baseline Severities in Phase 3 HELIOS-A Study

In a recent post hoc analysis presented at the AANEM meeting, findings highlight the positive impact of vutrisiran on quality of life, disability, gait speed, and nutritional status across different neuropathy impairment score quartiles.

Creatine Kinase Levels Not Considered Risk Factor for COVID-19 Post-Acute Sequelae

Elevated creatine kinase levels were not found to be a prognostic factor for the development of PASC in encephalopathic COVID-19 patients, according to a retrospective chart review analysis.

Treatment Inequality Issues Identified for Patients With Generalized Myasthenia Gravis

In a recent online survey of 150 participating neurologists in the US, 74.7% reported that patients with generalized myasthenia gravis found it difficult to afford their prescribed therapies, and 60.0% noted an increased likelihood of exacerbation or hospitalization for these patients.

Understanding the ADAPT-SC Results of Subcutaneous Efgartigimod: Tuan Vu, MD

The division director of Neuromuscular Medicine & EMG at USF Health provided an overview of the open-label findings from ADAPT-SC, a phase 3 study assessing subcutaneous efgartigimod in patients with myasthenia gravis. [WATCH TIME: 3 minutes]

Decreased Neurofilament Light in ATTR Polyneuropathy Observed With Vutrisiran and Patisiran

Vutrisiran and patisiran treatment led to noticeable reductions in neurofilament light, indicating a potential biomarker of treatment response in hATTR amyloidosis patients.

Subcutaneous Efgartigimod PH20 Demonstrates Efficacy for Generalized Myasthenia Gravis in Open Label ADAPT-SC+ Trial

Presented at the 2023 AANEM meeting, new findings suggest that subcutaneous efgartigimod could be a valuable treatment option for patients with myasthenia gravis, with well-tolerated cycles and improved daily living activities.

Open-Label PROPEL Study Results Highlight Longterm Impact of Cipaglucosidase Alfa and Miglustat in Late-Onset Pompe Disease

The phase 3 PROPEL study demonstrated the long-term effectiveness of cipaglucosidase alfa/miglustat in late-onset Pompe disease, with improved 6-minute walk distance and lung function.

The Potential of Nipocalimab as a Treatment for Autoantibody Mediated Diseases: Hong Sun, MD, PhD

The senior director and global compound development team leader at Janssen discussed research on nipocalimab, an investigational agent for patients with myasthenia gravis that's shown promising data to this point. [WATCH TIME: 2 minutes]

Latozinemab Shows No Impact on Disease Progression in C9orf72 Frontotemporal Dementia Despite increasing Progranulin Expression

latozinemab treatment for C9orf72-associated frontotemporal dementia showed no significant impact on disease progression, although the treatment was generally safe and well-tolerated.

The Role of Biomarkers in Myasthenia Gravis Diagnosis and Treatment: Hong Sun, MD, PhD

The senior director and global compound development team leader at Janssen talked about the importance of biomarkers in detecting an earlier diagnosis of myasthenia gravis and the significant unmet medical need for better treatment options. [WATCH TIME: 3 minutes]

NeuroVoices: David Greeley, MD, on AR1001, an Oral PDE5 Inhibitor for Alzheimer Disease

David Greeley, MD, chief medical officer at AriBio and study author, provided commentary on the mechanism of action of AR1001, its promising effect on ptau181, and the next steps in its development.

Designing More Effective Proof-of-Concept Studies for Progressive MS Therapies: Robert J. Fox, MD

The vice-chair for research at Cleveland Clinic’s Neurological Institute discussed the way therapies are currently assessed for progressive MS and the need for disease-specific biomarkers. [WATCH TIME: 3 minutes]

Takeda’s TAK-861 Shows Promise in Improving Wakefulness in Phase 1 Trial Among Healthy Men

Lowering of Amyloid Plaque Through Monoclonal Antibodies Correlated With Better Clinical Benefit, Meta-Analysis Suggests

Results showed that the relative reduction (RR) in CDR-SB at end-of-study was moderately correlated with difference between treated and placebo in Centiloid change.

Reviewing the Phase 2 OVERTURE Results of Gamma Sensory Stimulation Device: Brent Vaughan

The chief executive officer of Cognito Therapeutics detailed clinical findings from the open-label extension of the phase 2 OVERTURE study presented at the recent Clinical Trials on Alzheimer’s Disease conference. [WATCH TIME: 4 minutes]

Alzheimer Agent Simufilam Not Associated With ARIA-E, Phase 3 Reports Show

At 40 weeks, there were no observed cases of ARIA-edema, and new microhemorrhages occurred predominantly in patients with pre-existing conditions.

Clinical Potential of Cognito’s Gamma Sensory Stimulation Device in Alzheimer Disease: Brent Vaughan

The chief executive officer of Cognito Therapeutics provided an in-depth overview of the company’s gamma sensory stimulation device, its mechanism of action, and why it serves as a promising therapy for patients with Alzheimer disease. [WATCH TIME: 5 minutes]

Anti-Amyloid-ß Oligomer Antibody ACU193 Reduces Amyloid Plaques in Dose-Dependent Manner

ACU193 treatment reduced amyloid plaques in early Alzheimer disease patients, suggesting AßOs as a viable target, with safety data and ARIA analysis presented at CTAD 2023.

Combination of Aducanumab Infusion and Focused Ultrasound Accelerates Reduction of Amyloid-ß Plaques

The use of focused ultrasound-mediated blood-brain barrier opening has the potential to advance neurotherapeutics in the treatment of Alzheimer disease and other progreesive neurological disorder impacted by that barrier.

Biogen’s BIIB080 Exhibits Favorable Outcomes in Phase 1b Trial for Early Alzheimer Disease

Patients with mild Alzheimer disease who received high doses of BIIB080 demonstrated improvement in cognitive and functional outcomes compared with placebo, supporting further investigation.

Mechanistic Potential of Pepinemab, a SEMA4D-Blocking Antibody: Terrence L. Fisher, PhD

The senior vice president of clinical development at Vaccinex provided commentary on a presentation from CTAD 2023 highlighting the therapeutic potential of peptinemab, an agent that’s shown success in Huntington disease, in patients with Alzheimer disease. [WATCH TIME: 3 minutes]

Eisai Plans to Submit BLA for Subcutaneous Lecanemab After Findings Show Promising Results

New subcutaneous lecanemab considered more effective than IV for amyloid plaque removal in Alzheimer disease; FDA application planned by March 2024.

The Complexities of Alzheimer Disease Treatment and Diagnosis in Clinical Trials: Sharon L. Rogers, PhD

The chief executive officer at AmyriAD Therapeutics discussed the limitations of clinical trials assessing potential disease-modifying therapies for Alzhiemer disease including the lack of a diverse patient population and nonthorough clinical assessments for diagnosis. [WATCH TIME: 6 minutes]

Phase 2b RewinD-LB Study Aims to Confirm Neflamapimod’s Potential in Dementia With Lewy Bodies

Findings from the phase 2a AscenD-LB, a study of neflamapimod in patients with mild-to-moderate Alzheimer disease, served as supportive data to guide the phase 2b study.

AXS-05 Shows Reduction in Relapse Risk in Phase 3 ACCORD Trial for Alzheimer Disease Agitation

Findings from the trial demonstrated rapid and sustained clinical response in patients with Alzhiemer disease agitation during the open-label treatment phase and did not show any new safety signals.

Therapeutic Advantages to Using EEG to Assess Alzheimer Agents: Williem de Haan, MD, PhD

The neurologist and senior research at the Amsterdam University Medical Centers’ Alzheimer Center discussed the reasons behind the use of quantitative EEG in assessing Cognition Therapeutics' Alzheimer agent CT1812. [WATCH TIME: 3 minutes]