Rapport's RAP-219 succeeds in Phase 2a trial for epilepsy

RAP-219’s Phase 2a win is exciting — a novel mGlu2/3 approach showing seizure reduction with a favourable safety profile. With many patients still drug-resistant and existing AEDs limited by side effects, this could be a real step forward in focal epilepsy care. #Epilepsy #FocalOnsetSeizures #Neurology #Neuroscience #RAP219 #Keppra #Lamictal #Briviact #UCB #Eisai #DrugDevelopment #ClinicalTrials #Innovation #PrecisionMedicine

September is World Alzheimer's Month, a time to unite with the millions of families worldwide impacted by this devastating disease. Our collective motivation is unwavering: to bring effective screening tools and treatments to patients as quickly as possible and provide better care. We are dedicated to addressing this challenge by advancing the NeuLogiq® Platform to enable better, faster decision-making in CNS clinical trials. By providing a new standard for measuring disease progression, we are empowering the biopharma industry to accelerate the development of much-needed therapies. Today we are excited to announce the publication of breakthrough research from the University of Bath and University of Bristol that demonstrates the real-world efficacy of a passive EEG test (Fastball EEG) in identifying memory decline years before a clinical diagnosis. Cumulus is developing a scalable version of Fastball EEG – AccelADx, which is currently deployed in two large grant-funded studies. This month, we stand in solidarity with everyone working tirelessly to end Alzheimer's. Let's continue to push the boundaries of what's possible in clinical research. Learn more about the study here: https://lnkd.in/eF3xJxTy #WorldAlzheimersMonth #WAM2025 #AccelerateResearch #Alzheimers #Neurology #ClinicalTrials #DigitalBiomarkers #Biopharma #CumulusNeuroscience

This World Alzheimer’s Month, we’re reminded of the global urgency to find earlier, more effective ways to diagnose and treat Alzheimer’s.   At Clarendon, we’re proud to back Cumulus Neuroscience, whose pioneering work is helping reshape how neurological diseases are detected and managed. Their recent collaboration with the University of Bath and University of Bristol highlights the potential for earlier intervention and better outcomes for patients worldwide.   Supporting innovators like Cumulus is central to our mission of driving impact through investment.   Learn more: https://lnkd.in/eF3xJxTy   Brian Cummings | Neil Simms | James Curran | Stuart Gaffikin | Claudine Owens | Sian Mclaughlin | Jill Wilson | Matthew Ward | Jack Langan | Shauna Leonard | Daniel Lartey | Christine Faloon   #WorldAlzheimersMonth #WAM2025 #AccelerateResearch #Alzheimers #CumulusNeuroscience

Excited to share our new publication in BMC Medicine! “Targeted limbic self-neuromodulation for alleviating central sensitization symptoms in fibromyalgia” Read the paper here - https://lnkd.in/dKCf3hVM Fibromyalgia is a complex brain-based syndrome involving pain, fatigue, cognitive, and affective symptoms. Current treatments remain limited and often focus narrowly on pain. In this double-blind, randomized, dual-controlled trial, we tested a novel fMRI-informed EEG neurofeedback approach targeting the amygdala. The results showed: - Significant, sustained improvements across multiple symptom domains (pain, fatigue, cognition, affect) - Benefits that persisted up to one year after training - Evidence that targeting limbic circuits and emotion regulation processes can relieve the broader disease burden, not just pain This work highlights the potential of mechanism-based, brain-guided therapies to address central sensitization syndromes such as fibromyalgia—moving beyond symptom relief toward long-term functional improvement. Excellent (and hard) work by our PhD student (now postdoctoral fellow) Ayelet Or-Borichev and co-mentor Yulia Lerner #ChronicPain #Fibromyalgia #Neuroscience #Neurofeedback #Neuromodulation #CentralSensitization

In the latest issue of Scientific American, Kejal Kantarci, MD, of the Mayo Clinic Alzheimer’s Disease Research Center and Daryl Rhys Jones, PhD of Eisai showcase and explore the concept of “Smoldering Alzheimer’s Disease,” which describes a constellation of pathologies that includes toxic soluble Aβ protofibrils, tau, inflammation, and neurodegeneration. These pathologies collectively fuel a slow-burning process that begins decades before symptoms appear, continues even in the absence of Aβ plaques, and ultimately leads to ongoing cognitive and clinical decline. This evolving model aligns AD with other chronic diseases and emphasizes the importance of a thoughtful, patient-focused approach to managing it. 🔗 US HCPs can access the Eisai-sponsored supplement here: https://ow.ly/ig9X50WY50f #AlzheimersDisease #neurology

We are very excited to announce the continuation of the first modifying, treatment for Dementia with Lewy Bodies (DLB) at the University of Miami, and continue last year's successful Phase 2 "SHIMMER" clinical trial. COG1202 EAP (Expanded Access Program) is a single group, open-label program for individuals between 50-85 years of age, diagnosed with mild to moderate DLB. My team and I at the University of Miami's, Comprehensive Center for Brain Health are testing CT1812 (Zervimesine) a modulating, small molecule that acts as a sigma-2 receptor antagonist, aiming to protect neurons from damage caused by protein buildup, specifically alpha-synuclein. Demonstrating significant improvements in behavioral, cognitive, and motor symptoms in patients with DLB, Zervimesine represents a potential new hope for millions of patients and their families affected by DLB as there is currently no FDA approved treatment for DLB. Cognition Therapeutics and the University of Miami aims to advance Zervimesine into late-stage clinical trials for both DLB and Alzheimer's disease as the molecule progresses in its testing phase. #lewybodydementia #dementiawithlewybodies #alzheimers #universityofmiami #universityofflorida #neurology #neurodegeneration #clinicaltrials #neurodegenerativedisease #phase2 #dementia

🚨 Interesting approval - Aqneursa (levacetylleucine) Aqneursa is indicated for the treatment of neurological manifestations in adults and children aged 6 years and older (≥20 kg) with Niemann-Pick type C (NPC), either in combination with miglustat or as monotherapy in patients for whom miglustat is not tolerated. Aqneursa is a modified form of the amino acid leucine, targeting neurological dysfunction. While its precise mechanism of action remains under investigation, non-clinical studies have demonstrated that Aqneursa improves energy metabolism and enhances adenosine triphosphate (ATP) production, the primary energy source for cerebellar tissues. Clinical efficacy was established in a randomised, double-blind, placebo-controlled, two-period crossover Phase 3 study involving 60 NPC patients. Following 12 weeks of treatment, Aqneursa demonstrated significant improvements in neurological signs and functional outcomes compared with placebo, as measured by the Scale for the Assessment and Rating of Ataxia (SARA). These findings represent an important advancement in the therapeutic management of NPC-related neurological dysfunction. #RareDisease #Neurology #ClinicalTrials #OrphanDrugs #RegulatoryAffairs

🚨 New Publication in Multiple Sclerosis Therapy  The results of the Phase 3 POINT study have just been published in Multiple Sclerosis and Related Disorders (July 2025), led by Prof. Ludwig Kappos and colleagues. The study evaluated ponesimod as an add-on to dimethyl fumarate (DMF) in patients with active relapsing MS despite DMF monotherapy. 🔑 Key takeaways: Premature termination of the study due to slow recruitment (136 of 600 planned patients enrolled) limits interpretation of the results. Adding Ponesimod to dimethyl fumarate (DMF) was not superior to continuing on DMF in Relapsing multiple sclerosis. Ponesimod+DMF treatment was associated with nominally lower MRI defined disease activity. During the limited exposure to combined treatment, no new safety concerns emerged. 🧠 These findings highlight the importance of MRI-based monitoring when evaluating treatment strategies and inform the continued exploration of combination therapies in MS. 🔗 Read full article here: https://lnkd.in/dGZMmWfN #MultipleSclerosis #MSResearch #Neurology #ClinicalTrials #Ponesimod #DimethylFumarate #RC2NB

"What other neurodegenerative diseases could benefit from this early detection approach? Share your thoughts below." A major breakthrough in neurology could change the timeline for diagnosing Amyotrophic Lateral Sclerosis (ALS). Researchers have developed a novel blood test capable of detecting biological signs of the disease years before the first clinical symptoms emerge. This is a paradigm shift. For a condition where early diagnosis is critical for intervention and management, this innovation offers a significant window of opportunity. The ability to identify at-risk individuals pre-symptomatically could revolutionize clinical trials for new therapies, allowing for earlier treatment and potentially slowing disease progression. This advancement isn't just about a single disease. It highlights a broader movement towards proactive and predictive diagnostics in medicine, moving from treating symptoms to identifying risk at its earliest molecular stages. The implications for patients and their families are profound, offering not just answers, but precious time. This research underscores the power of biomarker discovery and its potential to reshape the future of neurodegenerative disease care. Read more about this groundbreaking development here: https://lnkd.in/gjzS_9JH What other neurodegenerative diseases could benefit most from this early detection approach? Share your thoughts below. #ALS #Neurology #MedicalBreakthrough #Biomarkers #EarlyDetection #Neuroscience #HealthcareInnovation #ClinicalTrials #MedicalResearch

Over the summer, I had the privilege of shadowing leading epileptologists at NYU Langone Health, as part of Training in Clinical Epilepsy for Basic Scientists organized by the International League Against Epilepsy (ILAE) and Drs. Dan Friedman and Helen Scharfman. This experience deepened my understanding of the epilepsy in clinical settings and the pressing unmet needs in epilepsy care.  Epilepsy is one of the oldest known neurological diseases, affecting ~1/100 individuals in the US. Despite increasing number of antiseizure medications, the incidence of intractable epilepsy (where more than two antiseizure medications fail) has persistently remained around 30%. This highlights the urgent need for innovative, disease-modifying therapies and novel mechanisms of action.  I am optimistic about the future of this field, with emerging technologies on the horizon. Just to highlight a few, companies like UNEEG medical A/S and Epiminder are developing ultra-long-term EEG recordings to provide more accurate seizure pattern characterization, facilitating optimal treatment choices. Additionally, exciting developments in drug therapy are underway, including cell therapy innovations (Neurona Therapeutics), advances in antisense oligonucleotides (Stoke Therapeutics, Ultragenyx), and even psychedelic-based treatments (Bright Minds Biosciences). Moreover, we are discovering new genes associated with epilepsy every year. I envision a future where personalized patient treatments are guided by genetic and EEG biomarkers. I look forward to witnessing how these technological advancements will reshape epilepsy care. If you’re aware of or involved in groundbreaking technologies aimed at improving epilepsy treatments, please comment or DM me—I’d love to connect and learn more! #Epilepsy #InnovationInHealthcare #Neuroscience #PrecisionMedicine #EpilepsyResearch #FutureOfMedicine #HealthcareInnovation #PersonalizedTreatment