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Annexon's Tanruprubart Shows Rapid and Durable Improvements in Guillain-Barré Syndrome Phase III Trial

NeurologyMonoclonal Antibody
  • Tanruprubart demonstrated significant functional improvements in Guillain-Barré syndrome patients as early as one week after treatment, with patients 14 times more likely to show improved mobility versus placebo.
  • The monoclonal antibody targeting C1q protein maintained durable benefits through 26 weeks, with twice as many treated patients achieving full recovery compared to placebo.
  • If approved, tanruprubart could address a significant unmet need for the estimated 7,000 U.S. and 15,000 EU patients diagnosed with GBS annually, potentially representing a $1 billion market opportunity.

Efficacy and Safety of ANX005 in Subjects With Guillain-Barré Syndrome

NCT04701164CompletedPhase 3
Annexon, Inc.
Posted 12/17/2020

Alzinova Secures Funding for Phase II Alzheimer's Drug Trial Preparations

Neurology
• Alzinova has successfully raised capital to advance preparations for Phase II clinical trials of its Alzheimer's disease therapeutic candidate, following promising early-stage results.
• The company is now fully focused on Phase II trial preparations, as highlighted in Carnegie's commissioned research reports and confirmed in Alzinova's Q1 review.
• This development represents a significant milestone in Alzinova's clinical pipeline, potentially offering a new approach to addressing the unmet needs in Alzheimer's disease treatment.

Specialty Pharmacists Drive Significant Improvements in Non-MS Neurology Care, Study Shows

Neurology
  • A single-center study at Vanderbilt University Medical Center found specialty pharmacists performed over 2,000 interventions across 741 neurology patients in just three months, with 98% of recommendations accepted by providers.
  • The most common interventions included medication changes (35%), patient counseling (10%), and care coordination (10%), demonstrating pharmacists' critical role in preventing treatment delays.
  • Researchers utilized a standardized 4-point impact scale, with most interventions (76%) rated as moderately impactful, suggesting significant improvements to patient quality of life and medication adherence.

Tofersen Shows Promise in Slowing Progression of SOD1 Motor Neuron Disease

Rare DiseaseNeurology
  • Tofersen, developed by Biogen, has demonstrated significant efficacy in slowing the progression of MND in patients with the SOD1 genetic mutation, offering a breakthrough in treatment for this previously untreatable condition.
  • Despite being available through Biogen's Extended Access Program at no cost, some patients face barriers to access as the NHS struggles with administration costs and capacity for the monthly lumbar puncture procedure.
  • Clinical trials, including the ATLAS study, show particularly promising results when treatment begins before symptom onset, with some patients remaining symptom-free despite elevated neurofilament levels indicating disease activation.

Neurogen Biomarking Partners with NeuroX to Revolutionize Early Alzheimer's Detection

Neurology
  • Neurogen Biomarking and NeuroX have formed a strategic partnership to accelerate early detection of Alzheimer's disease through an innovative patient-initiated platform combining blood biomarker testing and AI-enhanced cognitive assessments.
  • The collaboration addresses critical gaps in neurological care, with NeuroX's board-certified neurologists providing telehealth support that reduces typical specialist wait times from months to weeks for the 92% of Americans with MCI who remain undiagnosed.
  • Launching commercially in 2025, the integrated platform offers a comprehensive patient journey including digital cognitive assessment, at-home blood biomarker testing for ptau217, and personalized brain health action plans developed by specialists.

Regeneron Leads $60 Million Consortium to Accelerate UK Biobank Genome Sequencing for Alzheimer's and Parkinson's Drug Discovery

Drug DiscoveryNeurology
• Regeneron has formed a consortium with AbbVie, Alnylam, AstraZeneca, Biogen, and Pfizer, with each company committing $10 million to accelerate sequencing of 500,000 UK Biobank genomes by end of 2019. • The project aims to identify new biological targets for drug development, particularly for neurological diseases like Alzheimer's and Parkinson's, where current failure rates exceed 90%. • Regeneron's automated sequencing capabilities through its Genetics Center have dramatically reduced processing time, potentially revolutionizing drug discovery by linking genetic data with health records from 500,000 volunteers. • After an exclusive access period for consortium members, all sequencing data and research findings will be made publicly available to the broader scientific community.

Riliprubart Shows Promise as First-in-Class Treatment for Chronic Inflammatory Demyelinating Polyneuropathy

Rare DiseaseNeurologyMonoclonal Antibody
  • Sanofi's riliprubart demonstrated significant disease-controlling benefits across all patient cohorts in a Phase 2 study for chronic inflammatory demyelinating polyneuropathy (CIDP), including those who failed standard treatments.
  • The complement C1s inhibitor showed sustained efficacy for up to 48 weeks, with 87-92% of participants experiencing improvement or disease stabilization after 24 weeks of treatment.
  • Riliprubart reduced neurofilament light chain levels by 35% across all cohorts, suggesting potential reduction in nerve damage, while also improving patient-reported fatigue and quality of life outcomes.

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

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

Leqembi's Slow Adoption: Challenges and Progress in Alzheimer's Treatment

Monoclonal AntibodyDrug ApprovalNeurology
  • Leqembi, the first FDA-approved drug shown to slow Alzheimer's progression, has experienced slower-than-expected adoption despite its groundbreaking status in treating the disease.
  • Healthcare systems face significant implementation challenges, including establishing diagnostic protocols, monitoring systems for side effects, and navigating complex insurance coverage issues for the $26,000-per-year treatment.
  • Despite modest clinical benefits and potential side effects like brain swelling and bleeding, some patients report improvements in short-term memory, offering hope as researchers continue developing easier administration methods and complementary treatments.

FDA Clears Path for Cingulate's CTx-1301 ADHD Treatment NDA Submission

Neurology
  • The FDA has confirmed that Cingulate can proceed with its New Drug Application for CTx-1301, a novel ADHD treatment, without completing additional Phase 3 studies.
  • CTx-1301 utilizes Cingulate's proprietary Precision Timed Release (PTR) technology to deliver three precisely timed doses of dexmethylphenidate in a single daily tablet, potentially benefiting over 60% of ADHD patients currently using booster doses.
  • Cingulate plans to submit the NDA in the first half of 2025 and is advancing discussions with potential commercialization partners while preparing to engage with payers for market access.

FDA to Convene Advisory Committee for Eli Lilly's Alzheimer's Drug Donanemab

Monoclonal AntibodyNeurologyPrecision Medicine
• The FDA has announced plans to convene a Peripheral and Central Nervous System Drugs Advisory Committee to review donanemab's efficacy and safety profile before making a final approval decision.
• Donanemab's TRAILBLAZER-ALZ 2 trial employed innovative biomarker strategies, using both Amyvid® and Tauvid™ PET scans to identify early-stage patients with confirmed amyloid plaques most likely to benefit from treatment.
• Experts view anti-amyloid therapies like donanemab as just the first line of defense, with approximately 75% of Alzheimer's drugs in development now exploring novel targets related to aging pathways including inflammation and vascular dysfunction.

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