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Sleep Disturbances in ICU Patients: New Research Reveals Impact on Recovery and Outcomes

Neurology
  • New research presented at ATS 2025 reveals that sleep patterns in ICU patients may serve as important biomarkers for predicting patient outcomes, with atypical N3 sleep linked to longer ICU stays.
  • A study of ICU patients found that hypersomnia (sleeping more than 11 hours in 24 hours) was associated with higher mortality rates, longer hospital stays, and lower rates of discharge to home.
  • Over 60% of ICU survivors with acute respiratory failure experience insomnia, with pre-existing mental health conditions and neurologic causes of respiratory failure significantly increasing this risk.

SP-102 Shows Clinically Meaningful Results for Sciatica Treatment in CLEAR Trial Analysis

Neurology
  • Scilex Holding Company presented post-hoc analysis of the C.L.E.A.R. trial demonstrating clinically meaningful safety and efficacy of SP-102 (SEMDEXA™) for lumbosacral radicular pain at the ASIPP Annual Meeting.
  • The analysis showed clear separation between SP-102 and placebo in multiple endpoints, with enhanced results in the modified intent-to-treat population that received confirmed study drug.
  • SP-102's viscous gel formulation was specifically designed as a safer alternative to current off-label products that carry warnings about potentially dangerous and life-threatening adverse events.

Acadia Pharmaceuticals Wins Patent Infringement Case Against Aurobindo for Parkinson's Drug Nuplazid

Neurology
  • A U.S. District Court ruled that Aurobindo Pharma infringes on two key patent claims held by Acadia Pharmaceuticals for its Parkinson's disease drug Nuplazid.
  • The court rejected Aurobindo's arguments that Acadia's patent claims were invalid, providing significant intellectual property protection for Acadia's flagship product.
  • Following the favorable ruling, Acadia's stock soared by over 34% to a one-year high of $23.61, reflecting investor confidence in the company's market position.

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 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

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

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.

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