Core Insights - NeuroSense Therapeutics is advancing its Alzheimer's disease program and has appointed Prof. Steven E. Arnold to its Scientific Advisory Board, enhancing its scientific leadership [1][2] - The company recently completed a proof-of-concept study for Alzheimer's disease, with results indicating a favorable safety and tolerability profile, and expects clinical and biomarker outcomes in Q1 2026 [2] Company Overview - NeuroSense Therapeutics is a clinical-stage biotechnology company focused on developing treatments for severe neurodegenerative diseases, including Alzheimer's disease and ALS, addressing significant unmet medical needs [5] - The company's strategy involves developing combined therapies that target multiple pathways associated with neurodegenerative diseases, leveraging strong scientific research on biomarkers [5] Alzheimer's Disease Context - Alzheimer's disease is a leading cause of dementia, affecting over 30 million people globally, characterized by memory loss and cognitive decline, with no current cure available [3] - There is a significant unmet need for disease-modifying treatments that can slow or halt the progression of Alzheimer's disease, making multi-target approaches like PrimeC potentially valuable [3] PrimeC Drug Candidate - PrimeC is NeuroSense's lead drug candidate, an extended-release oral formulation combining two FDA-approved drugs, ciprofloxacin and celecoxib, designed to target key mechanisms of neuron degeneration and inflammation [4] - The multi-target mechanism of PrimeC is relevant to both Alzheimer's disease and ALS, aiming to inhibit disease progression [4]

Core Insights - BioVie Inc. is a clinical-stage company focused on developing innovative drug therapies for neurological and neurodegenerative diseases, including Alzheimer's, Parkinson's, and Long COVID [4] - The company is hosting a webinar on December 9, 2025, featuring CEO Cuong Do, who will discuss the drug bezisterim (NE3107) and its potential benefits [2][3] - BioVie is advancing multiple late-stage clinical programs, including BIV201, an orphan drug candidate for refractory ascites, with significant market opportunities [2] Company Overview - BioVie Inc. is engaged in developing therapies targeting neuroinflammation and insulin resistance, which are critical factors in Alzheimer's and Parkinson's diseases [4] - Bezisterim is a first-in-class, orally available small molecule that has shown promising results in improving cognition and motor function in clinical studies [2][4] - BIV201, the company's late-stage candidate, is under FDA Fast Track status and aims to address complications of liver cirrhosis, with guidance from the FDA for Phase 3 clinical testing [4] Market Potential - The company is positioned to create significant value with its late-stage programs and strong safety data, targeting multi-billion-dollar market opportunities [2] - The ongoing clinical studies and potential partnerships are expected to enhance BioVie's market presence and financial performance [2]

MALVERN, Pa., Nov. 18, 2025 (GLOBE NEWSWIRE) -- Annovis Bio, Inc. (NYSE: ANVS) (“Annovis” or the “Company”), a late-stage clinical drug platform company pioneering transformative therapies for neurodegenerative diseases such as Alzheimer's disease (AD) and Parkinson's disease (PD), today announced that the U.S. Food and Drug Administration (FDA) has scheduled a Type C meeting in January 2026 to discuss the Company's pathway for Parkinson's disease dementia (PDD). Annovis also reaffirmed that its ongoing Pha ...

Core Insights - Buntanetap significantly improves cognition in Parkinson's patients, especially those with Alzheimer's co-pathology, showing a three-times greater response [1] - The treatment leads to reductions in plasma biomarkers pTau217, total tau, and brain-derived tau, indicating its potential to address cognitive decline and modify disease progression [1][4] - The findings support the need for therapies targeting multiple neurotoxic proteins, as neurodegenerative diseases often co-occur [3] Study Results - In a Phase 3 study, buntanetap halted cognitive decline in early Parkinson's patients, with the most significant improvements in those with mild dementia [2] - Approximately 25% of patients in the study exhibited amyloid co-pathology, which was associated with more pronounced cognitive decline that was counteracted by the treatment [2] Therapeutic Efficacy - Buntanetap treatment resulted in significant cognitive improvements in Parkinson's patients with amyloid co-pathology, supported by measurable reductions in established neurodegeneration biomarkers [4] - The data presented by the company is the first to explore treatment effects in Parkinson's patients with amyloid co-pathology, highlighting the drug's potential across multiple neurodegenerative diseases [5] Upcoming Presentation - Full biomarker data will be presented at the Clinical Trials on Alzheimer's Disease (CTAD) conference in San Diego from December 1-4, 2025, with further details to be announced [5] Company Overview - Annovis Bio, Inc. is focused on developing innovative therapies for neurodegenerative diseases, including Alzheimer's and Parkinson's, aiming to improve patient outcomes and quality of life [6]

Core Insights - Annovis Bio, Inc. is making significant progress in its pivotal Phase 3 Alzheimer's study, with all 84 clinical sites fully activated and participant recruitment ongoing [6][2] - The company has strengthened its intellectual property by transferring all patents to a new crystalline form of its drug, buntanetap, and published pharmacokinetic data supporting this transition [2][6] - Recent biomarker findings from a Phase 2/3 study indicate meaningful reductions in inflammation and neurodegeneration, suggesting the drug's potential as a disease-modifying therapy [2][6] Clinical Highlights - The pivotal Phase 3 study for early Alzheimer's disease is on track, with robust participation and a screen failure rate within expected projections [6] - The first patients have completed the 6-month treatment period, marking a key milestone for upcoming symptomatic readouts [6] - Biomarker results from the Phase 2/3 trial show significant reductions in neuroinflammation and neurodegeneration in patients treated with buntanetap compared to placebo [6] Business Highlights - The company appointed Mark Guerin as CFO, enhancing its leadership team during a critical phase [2][6] - Annovis presented four scientific posters at the AAIC 2025 conference, showcasing progress in its Alzheimer's trial and pharmacokinetics of buntanetap [6] Financial Results - As of September 30, 2025, Annovis reported cash and cash equivalents of $15.3 million, an increase from $10.6 million at the end of 2024 [6] - Research and development expenses for Q3 2025 were $6.3 million, up from $2.7 million in Q3 2024 [6] - The net loss per common share for Q3 2025 was $0.37, a decrease from $0.97 in Q3 2024 [7][15]

Core Insights - AC Immune SA has published preclinical data on its first-in-class TDP-43 PET tracer, ACI-19626, which shows potential for precision medicine in neurodegenerative diseases [2][5][6] - TDP-43 pathology is a significant factor in various neurodegenerative diseases, complicating diagnosis due to overlapping clinical features [3][6] - ACI-19626 demonstrates high specificity and selectivity for TDP-43 aggregates, with promising pharmacokinetic properties [4][7][8] Company Overview - AC Immune SA is a clinical-stage biopharmaceutical company focused on precision therapeutics for neurodegenerative diseases, including Alzheimer's and Parkinson's diseases [10] - The company utilizes two technology platforms, SupraAntigen® and Morphomer®, to develop a diversified pipeline of therapeutic and diagnostic programs [10] - AC Immune has established strategic partnerships with leading pharmaceutical companies, resulting in over $4.5 billion in potential milestone payments and royalties [10] Research and Development - The publication in Nature Communications details the characterization of ACI-19626, highlighting its potential to improve diagnosis and treatment of TDP-43 proteinopathies [5][6] - ACI-19626 has been advanced into Phase 1 clinical trials, with initial results expected in Q4 2025 [5][6][8] - The tracer shows excellent selectivity for TDP-43 over other common co-pathologies, indicating its potential as a diagnostic tool [7][8]

Summary of Conference Call on Gene Therapy for Neurodegenerative Diseases Industry Overview - The conference focused on gene therapy applications for neurodegenerative diseases, featuring speakers from three companies: Mira, uniQure, and Passage Bio [1][2][6]. Key Points by Company Mira - **Company Overview**: Mira specializes in genetic medicines, focusing on local delivery of small doses to treat severe indications, which enhances safety and reduces costs [2]. - **Recent Milestones**: Mira has two pivotal studies and two awaiting Biologics License Application (BLA) filings, including treatments for rare and common eye diseases and a Parkinson's treatment that has shown three positive studies [3][4]. - **Innovative Approach**: The company utilizes AI technology to analyze data from the largest neurohospital in Europe, demonstrating physiological changes in the brain related to their Parkinson's treatment [4][24]. - **Potential Applications**: Mira's gene therapy can convert glutamate to GABA, potentially treating various neurodegenerative diseases by calming hyperactive neurons [22][24]. uniQure - **Company Overview**: uniQure is a pioneer in genomic medicine, known for developing the first approved AAV gene therapies, including Glybera and Hemgenix [6][9]. - **Lead Program**: The company is focused on Huntington's disease, utilizing a platform called MyCure to suppress the aberrant Huntingtin protein [8]. - **Clinical Findings**: In a study involving 45 patients, uniQure reported a 75% statistically significant slowing of disease progression based on the UHDRS [9]. - **Regulatory Plans**: A pre-BLA meeting is expected in Q4, with a BLA submission planned for Q1 of the following year [9]. Passage Bio - **Company Overview**: Passage Bio is based on technology from the University of Pennsylvania, focusing on gene therapy for frontotemporal dementia and Huntington's disease [14]. - **Clinical Approach**: The company delivers AAV1 via a minimally invasive procedure, showing high levels of target engagement and stabilization of neurofilaments [14][15]. - **Future Directions**: Passage Bio is also developing a preclinical program targeting the DNA damage response pathway in Huntington's disease [15]. Challenges in Gene Therapy - **Delivery Issues**: The primary challenge remains delivering therapies to the correct brain regions, especially for diseases like Huntington's [18][19]. - **Slow Disease Progression**: Designing clinical studies for slowly progressing neurodegenerative diseases is difficult, as changes in function are hard to detect [19][20]. - **Heterogeneity**: Variability among patients complicates the assessment of treatment efficacy, necessitating innovative statistical methods to account for differences [31][32]. Regulatory Considerations - **FDA Flexibility**: The FDA is showing increased willingness to consider alternative trial designs and endpoints for rare diseases with high unmet needs [44][46]. - **Natural History Data**: Utilizing natural history datasets can help in understanding disease progression and support regulatory submissions [30][31]. Safety Concerns - **Patient Safety**: The panel acknowledged the importance of safety in gene therapy, especially following recent patient deaths in trials [35][37]. - **Local Delivery Advantages**: Localized delivery methods may reduce systemic exposure and associated risks, as demonstrated by lower doses used in certain therapies [38][41]. Access and Implementation - **Training and Infrastructure**: Successful implementation of gene therapies will depend on training healthcare providers and establishing treatment centers [52][54]. - **Data-Driven Adoption**: Strong clinical data will be crucial for gaining acceptance among medical professionals and ensuring broad access to therapies [55][56]. Conclusion - The conference highlighted the potential of gene therapy in treating neurodegenerative diseases, the challenges faced in delivery and study design, and the evolving regulatory landscape that may facilitate the development of these innovative treatments [1][17][42].

Summary of Conference Call on Gene Therapy for Neurodegenerative Diseases Companies Involved - **MeiraGTx Holdings** - **uniQure** - **Passage Bio** Key Points and Arguments MeiraGTx Holdings - Focuses on genetic medicines for inherited and larger indications, utilizing local delivery of small doses to enhance safety and reduce costs [2][3] - Owns in-house manufacturing facilities, which supports the rapid development of their programs [2] - Two pivotal studies and two awaiting BLA filings, including treatments for rare and common eye diseases and a Parkinson's treatment that has shown three positive studies [3][4] - Partnership with an AI company to analyze data from the largest neurohospital in Europe, demonstrating physiological changes in the brain with their Parkinson's treatment [4][24] uniQure - A pioneer in genomic medicine with two approved AAV gene therapies: Glybera for lipoprotein lipase deficiency and Hemgenix for hemophilia B [6] - Lead program focuses on Huntington's disease, utilizing a platform called MyCure to suppress the aberrant huntingtin protein [9] - Clinical testing of AMT-130 has shown a 75% statistically significant slowing of disease progression over three years [10] - Plans to hold a pre-BLA meeting in Q4 and submit a BLA in Q1 of the following year [10] Passage Bio - Focuses on gene therapy for frontotemporal dementia patients with GRN mutations, using AAV1 delivered via a minimally invasive procedure [15] - Demonstrated high levels of target engagement and stabilization of plasma neurofilaments [15] Challenges in Gene Therapy for Neurodegenerative Diseases - Delivery to the right location in the brain remains a significant challenge, especially for diseases like Huntington's [18][19] - Slow progression of neurodegenerative diseases complicates the design of clinical studies to detect meaningful changes [19][20] - Heterogeneity among patients complicates the assessment of treatment effects [32][34] Safety Considerations - Local delivery methods may reduce systemic exposure and associated risks compared to systemic therapies [39][40] - Smaller doses and targeted delivery are believed to enhance safety profiles [42] Regulatory Aspects - The FDA is showing increased flexibility in evaluating gene therapies for rare diseases, allowing for single-arm studies with supportive evidence [44][45] - Emphasis on using natural history data and biomarkers to assess treatment effectiveness [31][33] Access and Implementation Challenges - The complexity of procedures may limit access, but strong data can encourage training and adoption among healthcare providers [53][55] - Existing surgical techniques can facilitate the implementation of new therapies, particularly in diseases with significant unmet needs [55][56] Other Important Insights - AI technology is being utilized to analyze large datasets to identify physiological changes and improve the robustness of clinical trial endpoints [25][35] - The potential for gene therapy to address multiple neurodegenerative diseases by altering brain circuitry is highlighted [22][24]

Core Insights - Alterity Therapeutics announced positive results from the ATH434-201 Phase 2 clinical trial for Multiple System Atrophy (MSA), demonstrating clinically meaningful efficacy in modifying disease progression at both 50 mg and 75 mg doses [1][2][3] Company Overview - Alterity Therapeutics is a biotechnology company focused on developing disease-modifying treatments for neurodegenerative diseases, particularly MSA and Parkinson's disease [4][8] - The lead candidate, ATH434, is an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration, showing preclinical efficacy in reducing α-synuclein pathology and restoring normal iron balance in the brain [4][8] Clinical Trial Details - The ATH434-201 Phase 2 clinical trial was a randomized, double-blind, placebo-controlled study involving 77 adults, assessing the efficacy, safety, and pharmacokinetics of ATH434 over 12 months [5][6] - Results indicated that ATH434 produced clinically and statistically significant improvements on the modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I, with additional positive trends in motor performance and patient-reported outcomes [5][6] Efficacy and Safety - ATH434 demonstrated target engagement by reducing iron accumulation in MSA-affected brain regions, with both dose levels showing a favorable safety profile comparable to placebo [2][5] - The study reported no serious adverse events attributed to ATH434, reinforcing its tolerability [2][6] Regulatory Status - ATH434 has received Fast Track Designation and Orphan Drug Designation from the U.S. FDA and the European Commission for the treatment of MSA, highlighting its potential as a significant therapeutic option in a market with currently no approved disease-modifying treatments [4][8]

Health Risk - Research indicates that New Zealand's top male rugby players face a higher risk of developing neurodegenerative diseases like Alzheimer's compared to the general population [1]