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Riliprubart Shows Promise in CIDP Treatment with Significant Neurofilament Light Reduction

NeurologyMonoclonal Antibody
  • Phase 2 study results reveal that Sanofi's investigational riliprubart reduced plasma neurofilament light levels by 31% in patients with chronic inflammatory demyelinating polyneuropathy.
  • Greater reductions in neurofilament light levels correlated with higher treatment response rates, with up to 69% of patients showing improvement in disability scores.
  • Riliprubart, a selective inhibitor of the classical complement pathway, is now being evaluated in two global Phase 3 trials (MOBILIZE and VITALIZE) across 28 countries.

A Study to Test the Efficacy and Safety of Riliprubart Against the Usual Treatment of Intravenous Immunoglobulin (IVIg) in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

NCT06290141RecruitingPhase 3
Sanofi
Posted 8/21/2024

A Study to Assess the Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves)

NCT04281472CompletedPhase 2
argenx
Posted 4/15/2020

A Study to Test the Effects and Safety of Riliprubart in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) for Which the Usual Treatments do Not Work

NCT06290128RecruitingPhase 3
Sanofi
Posted 7/12/2024

Study to Evaluate Safety and Efficacy of Three Different Dosages of NewGam in Patients With Chronic Inflammatory Demyelinating Poly (Radiculo) Neuropathy

NCT02638207CompletedPhase 3
Octapharma
Posted 9/27/2017

Novel AAV Capsid Shows Unprecedented Brain Transduction Potential for Gene Therapy

NeurologyRare Disease
  • Latus Bio has developed AAV-Ep+, a novel AAV capsid variant that demonstrates exceptional ability to transduce ependymal cells and cerebral neurons in both mice and non-human primates.
  • The breakthrough capsid enables efficient protein secretion into cerebrospinal fluid, potentially allowing single-administration gene therapies for lysosomal storage disorders and other neurological diseases.
  • In preclinical studies, low-dose administration of AAV-Ep+ expressing human TPP1 achieved protein levels significantly exceeding those of natural AAV capsids, reaching potentially therapeutic levels for CLN2 disease patients.

Merck KGaA Joins Peregrine Ventures' Incentive Incubator as Strategic Partner

NeurologyOncology
  • Merck KGaA has become a strategic partner in Peregrine Ventures' Incentive Incubator, gaining early access to startups in bioconvergence, pharma, and biotechnology fields.
  • The collaboration will focus on ventures with significant market potential aligned with Merck's three global divisions: Healthcare, Life Science, and Electronics, with priority given to bioconvergence ventures.
  • Through this partnership, eligible startups will receive support including investments, regulatory guidance, business development assistance, and potentially early-phase trial planning.

Bright Minds' BMB-101 Completely Eliminates Drop Attacks in Preclinical SUDEP Model

Neurology
  • Bright Minds Biosciences announced that BMB-101 demonstrated complete elimination of drop attacks in the DBA/2 mouse model, a highly predictive model for sudden unexpected death in epilepsy (SUDEP).
  • The novel 5-HT2C receptor agonist achieved 100% survival in the preclinical model by reversing brainstem serotonin deficits and preventing seizure-induced respiratory arrest.
  • These findings highlight BMB-101's potential to address critical gaps in SUDEP prevention, particularly for drug-resistant epilepsy patients where SUDEP represents the leading cause of seizure-related mortality.
  • The results build on previous Phase 1 clinical data showing BMB-101 was safe and well-tolerated in 64 healthy volunteers with robust central target engagement confirmed.

Neurofibromatosis Type 1 with Plexiform Neurofibromas: Diagnosis, Epidemiology and Clinical Manifestations

Rare DiseaseNeurology
  • Neurofibromatosis Type 1 (NF1) affects approximately 1 in 3000 individuals globally, with plexiform neurofibromas (PNs) occurring in up to 50% of NF1 patients.
  • NF1 is typically diagnosed in early childhood between ages 4-6 using NIH clinical criteria, including café-au-lait macules, neurofibromas, and other characteristic manifestations.
  • Plexiform neurofibromas are usually congenital but become clinically apparent in early childhood, with symptoms including disfiguring masses, pain, neurological deficits, and compression of adjacent structures.

Rett Syndrome Pipeline Analysis Reveals 20+ Companies Developing Novel Therapeutics with Gene Therapy Leading Innovation

Rare DiseaseNeurology
  • A comprehensive 2025 pipeline analysis identifies over 20 companies developing more than 20 therapeutic candidates for Rett syndrome, highlighting significant industry investment in this rare neurological disorder.
  • Anavex Life Sciences leads with blarcamesine in Phase III trials, representing the most advanced treatment targeting SIGMA1 and muscarinic receptors to restore cellular homeostasis.
  • Gene therapy approaches dominate the pipeline with TSHA-102 and NGN-401 delivering MECP2 gene replacement using AAV9 vectors in Phase I/II trials.
  • The therapeutic landscape spans multiple modalities including small molecules, gene therapies, and biologics, addressing the critical unmet medical need in Rett syndrome treatment.

FDA-Approved Lenire Device Shows 91.5% Success Rate in Real-World Tinnitus Treatment Study

FDA ApprovalReal World EvidenceNeurology
• A new study published in Nature Communications Medicine reveals that 91.5% of tinnitus patients experienced clinically meaningful improvement after 12 weeks of treatment with the Lenire bimodal neuromodulation device.
• The retrospective analysis of 220 patients represents one of the largest real-world studies of tinnitus treatment, confirming results from previous clinical trials that led to FDA approval in March 2023.
• Lenire works by simultaneously delivering audio tones through headphones and mild electrical pulses to the tongue, offering a promising treatment option for the estimated 25 million Americans suffering from tinnitus.

Neurovation Labs Secures Patent for Novel PTSD Biomarker Detection Technology

AINeurologyPrecision Medicine
• Neurovation Labs has been granted U.S. Patent No. 12,274,761 for technology that detects GluA1-containing AMPA receptors in the brain, enabling objective PTSD diagnosis.
• The patented radiological imaging device represents a significant advancement in mental health diagnostics by providing a physiological biomarker for a condition traditionally diagnosed through subjective assessments.
• Beyond PTSD, Neurovation Labs is exploring applications of this technology for other neurological conditions including traumatic brain injury, Alzheimer's disease, and epilepsy.

Roche Adjusts Tominersen Dosing in GENERATION HD2 Trial for Huntington's Disease

Neurology
  • Roche has announced modifications to its GENERATION HD2 trial for tominersen, a huntingtin-lowering therapy for Huntington's disease, following a scheduled review by an independent data monitoring committee.
  • The trial will continue with the higher 100mg dose only, discontinuing the 60mg dose arm, as the higher dose was deemed more likely to demonstrate clinical benefit while maintaining a favorable safety profile.
  • This development represents a positive step forward for tominersen, which previously faced setbacks when its GENERATION HD1 trial was halted in 2021 due to safety concerns.

GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease

NCT05686551Active, Not RecruitingPhase 2
Hoffmann-La Roche
Posted 2/3/2023

A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease

NCT03761849CompletedPhase 3
Hoffmann-La Roche
Posted 1/23/2019

Study Reveals Significant Diagnostic Delays for Minority Patients with Generalized Myasthenia Gravis

Rare DiseaseNeurology
  • Project ASPIRE research reveals racial and ethnic minority patients with generalized myasthenia gravis (gMG) face nearly four-month longer diagnostic delays compared to white patients, despite seeking care at similar timeframes.
  • Key barriers to timely gMG diagnosis include poor symptom recognition by both patients and physicians, limited access to specialists, and challenges with medical record transfers between healthcare systems.
  • Minority patients reported significantly higher stress levels during their diagnostic journey, with nearly double the percentage rating their experience as "very stressful" compared to white patients.

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