Latest news with #neuroprotection
The Australian
11 hours ago
- Health
- The Australian
Neurizon's NUZ-001 shows promise in Huntington's disease
Neurizon's NUZ-001 and active metabolite NUZ-001 Sulfone show strong neuroprotective effects in a zebrafish Huntington's model Results show potential of drug to counteract early neurodegenerative damage caused by disease Neurizon plans to initiate additional validation studies in mammalian models of Huntington's Special Report: Clinical-stage biotech Neurizon Therapeutics has reached a milestone in development of its lead drug candidate NUZ-001 to treat Huntington's disease. Neurizon Therapeutics (ASX:NUZ), which is dedicated to advancing treatments for neurodegenerative diseases, has rolled out new preclinical data demonstrating significant neuroprotective effects of NUZ-001 and its active metabolite NUZ-001 Sulfone, in a zebrafish model of Huntington's disease. Huntington's disease is a rare, inherited neurodegenerative disorder that causes progressive degeneration of motor function, cognition and mental health. The disease affects between 2.7 and 4.8 per 100,000 people globally with no cure and no disease-modifying treatment. The treatments available only manage symptoms. In the Huntington's disease model, targeted mRNA knockdown of the Htt (huntingtin) protein triggered hallmark disease characteristics, including: Increased cell death Morphological malformations (smaller eyes and swollen hindbrains) Impaired haemoglobin production and Reduced expression of brain-derived neurotrophic factor (BDNF), a critical biomarker of neuronal function and survival. mRNA knockdown is a lab technique that reduces the activity of a specific gene, in this case the HTT gene, which produces the protein involved in Huntington's disease. Treatment with either NUZ-001 or NUZ-001 Sulfone after Htt knockdown: Prevented developmental and morphological abnormalities Attenuated neuronal cell death Restored the delayed production of haemoglobin; and Rescued BDNF expression. Neurizon said the results provided evidence of NUZ-001 and NUZ-001 Sulfone's potential to counteract early neurodegenerative damage. Study details For the preclinical study, wild-type zebrafish embryos were raised in standard conditions. Morpholino antisense oligonucleotides (MOs) targeting Htt mRNA were then injected into one-cell stage embryos to decrease Htt expression. NUZ-001 or NUZ-001 Sulfone at 1 and 10 μM concentrations were added to the embryonic media six hours post-fertilisation to evaluate the protective effects on Htt knockdown-induced deficits. At two days post-fertilisation changes in morphology (eye size and hindbrain swelling), neuronal cell death (apoptosis), haemoglobin levels, and the BDNF expression levels were analysed. Source: Neurizon Therapeutics Knockdown of Htt (Htt MO) resulted in smaller eyes and swollen hindbrain ventricles in zebrafish embryos. Neurizon said partial rescue of eye size and full reversal of hindbrain swelling were observed with 10 μM NUZ-001 and NUZ-001 Sulfone (Figure 2b). Source: Neurizon Therapeutics Other key findings of the study include: Neuronal cell death was significantly higher in the Htt knockdown group, while treatment with 1 μM and 10 μM NUZ-001, and 10 μM NUZ-001 Sulfone, significantly reduced apoptosis Haemoglobin levels were significantly decreased in the Htt knockdown group but partially restored by both concentrations of NUZ-001 and NUZ-001 Sulfone; and Expression of BDNF transcripts was significantly rescued with 10 μM NUZ-001 and 10 μM NUZ-001 Sulfone. Watch: Last patient completes treatment in OLE study NUZ-001 showing promise NUZ-001 is currently in clinical development for the most common form of motor neurone disease (MND) called amyotrophic lateral sclerosis (ALS), where it has shown: Preclinical efficacy in enhancing proteostasis Reducing pathological protein aggregation; and Preserving neuronal function. The company said new findings in the Huntington model further underscore NUZ-001's potential as a platform therapy targeting core cellular stress and clearance mechanisms common to multiple neurodegenerative diseases. Neurizon plans to advance additional preclinical studies in mammalian models of Huntington's disease as part of its broader strategy to expand NUZ-001's therapeutic applications to other progressive neurological disorders with high unmet need. 'These results mark another important milestone in the realisation of the potential for NUZ-001 to treat a range of neurodegenerative diseases,' CEO and managing director Dr Michael Thurn said. 'Huntington's disease is a devastating, rare genetic disorder that causes the progressive breakdown of nerve cells in the brain, leading to a range of symptoms including uncontrolled movements, cognitive decline, and emotional disturbances. 'These exciting results demonstrate NUZ-001 has consistent neuroprotective effects beyond amyotrophic lateral sclerosis (ALS), strengthening our conviction in NUZ-001's potential as a disease-modifying platform therapy across a range of neurodegenerative conditions.' This article was developed in collaboration with Neurizon Therapeutics, a Stockhead advertiser at the time of publishing. This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.
National Post
20-05-2025
- Health
- National Post
Enveric Biosciences Identifies Neuroplastogen Candidates with Potential to Address Neurodegenerative Disease
Article content CAMBRIDGE, Mass. — Enveric Biosciences (NASDAQ: ENVB) ('Enveric' or the 'Company'), a biotechnology company dedicated to the development of novel neuroplastogenic small-molecule therapeutics for the treatment of psychiatric and neurological disorders, today announced that it has filed a new provisional patent application with claims to methods and pharmaceutical formulations for use on a family of molecules discovered to offer potential to address neurodegenerative disease. If confirmed through further testing, the potential of this newly identified group of molecules expands the market potential for Enveric drug candidates in a new direction. Article content Neurodegenerative diseases are a heterogenous group of progressive neurological disorders characterized by the gradual loss of neurons, leading to a decline in cognitive, motor and other neurological functions. It has been estimated by the World Health Organization (WHO) that approximately 50 million people worldwide are affected by neurodegenerative diseases, with seven million in the United States. Article content Broad claims in the patent application describe a group of molecules that enhance brain derived neurotrophic factor ('BDNF') activity and address conditions where the BDNF/TrKB pathway plays a major role. BDNF and its receptor TrKB support neuroprotection and adult neurogenesis and enhance myelination by increasing the density of oligodendrocyte progenitor cells. While additional pre-clinical testing is needed to confirm the enhancement of BDNF activation, preliminary studies have revealed promotion of neuronal development in a cell-based assay. Article content 'Based on our team's research, future molecules that emerge from this family could represent therapeutic candidates to develop for Alzheimer's Disease and other neurodegenerative diseases,' said Joseph Tucker, Ph.D., CEO and Director of Enveric. 'In addition to the potential to promote BDNF signaling, an established therapeutic target in neurodegenerative diseases, a prototype molecule from the family has been tested in vivo and revealed encouraging pharmaceutical and pharmacokinetic (PK) characteristics.' Article content The preliminary PK profile suggests that the orally delivered compound effectively enters the bloodstream, penetrates the brain, and is subsequently and rapidly cleared from plasma. Further, this molecule did not display 5HT2B receptor activation, which would have been an indicator of cardiovascular risk potential. Article content 'Based on the discovery and characterization work done to date, we believe further investigation of this family of molecules is warranted,' said Dr. Tucker. Article content Enveric Biosciences (NASDAQ: ENVB) is a biotechnology company dedicated to the development of novel neuroplastogenic small-molecule therapeutics for the treatment of psychiatric and neurological disorders. Leveraging its unique discovery and development platform Psybrary™, which houses proprietary information on the use and development of existing and novel molecules for specific mental health indications, Enveric seeks to develop a robust intellectual property portfolio of novel drug candidates. Enveric's lead molecule, EB-003, is a potential first-in-class neuroplastogen designed to promote neuroplasticity, without inducing hallucinations, in patients suffering from difficult-to-address mental health disorders. Enveric is focused on advancing EB-003 towards clinical trials for the treatment of neuropsychiatric disorders while out-licensing other novel, patented Psybrary™ platform drug candidates to third-party licensees advancing non-competitive market strategies for patient care. Enveric is headquartered in Naples, FL with offices in Cambridge, MA and Calgary, AB Canada. For more information, please visit Article content Forward-Looking Statements Article content This press release contains forward-looking statements and forward-looking information within the meaning of applicable securities laws. These statements relate to future events or future performance. All statements other than statements of historical fact may be forward-looking statements or information. Generally, forward-looking statements and information may be identified by the use of forward-looking terminology such as 'plans,' 'expects' or 'does not expect,' 'proposes,' 'budgets,' 'explores,' 'schedules,' 'seeks,' 'estimates,' 'forecasts,' 'intends,' 'anticipates' or 'does not anticipate,' or 'believes,' or variations of such words and phrases, or by the use of words or phrases which state that certain actions, events or results may, could, should, would, or might occur or be achieved. Forward-looking statements may include statements regarding beliefs, plans, expectations, or intentions regarding the future and are based on the beliefs of management as well as assumptions made by and information currently available to management. Actual results could differ materially from those contemplated by the forward-looking statements as a result of certain factors, including, but not limited to, the ability of Enveric to: finalize and submit its IND filing to the U.S. Food and Drug Administration; carry out successful clinical programs; achieve the value creation contemplated by technical developments; avoid delays in planned clinical trials; establish that potential products are efficacious or safe in preclinical or clinical trials; establish or maintain collaborations for the development of therapeutic candidates; obtain appropriate or necessary governmental approvals to market potential products; obtain future funding for product development and working capital on commercially reasonable terms; scale-up manufacture of product candidates; respond to changes in the size and nature of competitors; hire and retain key executives and scientists; secure and enforce legal rights related to Enveric's products, including patent protection; identify and pursue alternative routes to capture value from its research and development pipeline assets; continue as a going concern; and manage its future growth effectively. Article content A discussion of these and other factors, including risks and uncertainties with respect to Enveric, is set forth in Enveric's filings with the Securities and Exchange Commission, including Enveric's Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q. Enveric disclaims any intention or obligation to revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Article content Article content Article content Article content Contacts Article content Investor Relations Tiberend Strategic Advisors, Inc. David Irish (231) 632-0002 dirish@ Article content Article content Article content
Yahoo
15-05-2025
- Health
- Yahoo
AB Science today reports a new publication in the scientific journal PLOS One, showing promising neuroprotective effects of masitinib
PRESS RELEASE MASITINIB SHOWS PROMISING NEUROPROTECTIVE EFFECTS IN NEUROIMMUNE-DRIVEN NEURODEGENERATIVE DISEASE MODEL A NEW PUBLICATION IN THE SCIENTIFIC JOURNAL SHOWS THAT MASITINIB CAN LOWER SERUM NEUROFILAMENT LIGHT CHAIN, AN IMPORTANT BIOMARKER FOR NEURODEGENERATIVE DISORDERS, INCLUDING MULTIPLE SCLEROSIS, AMYOTROPHIC LATERAL SCLEROSIS AND ALZHEIMER'S DISEASE Paris, May 15, 2025, 8am CET AB Science SA (Euronext - FR0010557264 - AB) today announced publication of research in the peer-reviewed scientific journal PLOS One [1], highlighting the neuroprotective potential of masitinib in a model of neuroimmune-driven neurodegenerative disease. This research demonstrates masitinib's ability to limit neuronal damage, as measured by serum neurofilament light chain (NfL) concentration, and reduce pro-inflammatory cytokine biomarkers, offering hope for its application in treating neurodegenerative diseases. NfL is a non-specific marker of axonal loss that can serve as a biomarker of a drug's ability to produce a neuroprotective effect. Importantly, neuronal damage, or prevention thereof, can be rapidly assessed by measuring serum NfL concentration in EAE-induced mice. Because EAE is a model of neuroimmune-driven neurodegenerative disease, it is highly relevant to masitinib's mechanism of action in diseases such as progressive multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), Alzheimer's disease and Parkinson's disease. The article, entitled 'Tyrosine kinase inhibitor, masitinib, limits neuronal damage, as measured by serum neurofilament light chain concentration in a model of neuroimmune-driven neurodegenerative disease', is freely accessible online from the PLOS One website. Professor Olivier Hermine, MD, President of the Scientific Committee of AB Science and co-author of this article commented: 'This study is the first to demonstrate that masitinib can lower serum NfL levels, a key biomarker of neuronal damage, while also reducing neuroinflammation and slowing functional decline in a neuroimmune-driven disease model. Masitinib has already shown clinical benefits in progressive MS, ALS, and mild-to-moderate AD in previous trials, and this study further strengthens its therapeutic promise. Overall, these findings support masitinib's potential as a disease-modifying therapy for neurodegenerative diseases.' Masitinib's mechanism of action targets the innate neuroimmune system, specifically mast cells and microglia, which are increasingly recognized as contributors to the pathophysiology of neurodegenerative diseases. These results provide additional evidence regarding the anti neuro-inflammatory properties of masitinib and add further credibility to the masitinib associated neuroprotection observed in late phase clinical trails of progressive multiple sclerosis, amyotrophic lateral sclerosis and Alzheimer's disease. Key findings include: Masitinib significantly reduced serum neurofilament light chain (NfL) levels, indicating its neuroprotective effects in a neuroimmune-driven neurodegenerative disease model. Reduction in relative NfL Levels: At Day 8, masitinib treatment reduced the relative increase in serum NfL levels as compared with the EAE control group. Specifically: Masitinib 50 mg/kg/day reduced NfL levels by 43%. Masitinib 100 mg/kg/day reduced NfL levels by 60%. This reduction was dose-dependent, with the higher dose showing greater efficacy. Reduction in absolute NfL Levels: At Day 8, absolute serum NfL concentrations were approximately 25% lower in both masitinib treatment groups as compared with the EAE control group. At Day 15, masitinib further reduced absolute serum NfL levels. Specifically: Masitinib 50 mg/kg/day reduced levels by 6%. Masitinib 100 mg/kg/day reduced levels by 26%. Masitinib treatment significantly reduced the levels of several pro-inflammatory cytokine biomarkers in the EAE mouse model, indicating its anti-inflammatory effects. Masitinib demonstrated beneficial effects on functional performance in the EAE mouse model, particularly in grip strength: Masitinib-treated mice initially showed deterioration in grip strength but recovered to their pretreatment (Day 1) levels by Day 15. Both masitinib groups (50 mg/kg/day and 100 mg/kg/day) showed significantly less relative deterioration in grip strength at Day 15 compared to the EAE control group (p < 0.001). These findings suggest that masitinib slows the deterioration of grip strength, indicating a protective effect on motor function under conditions of chronic neuroinflammation. Overall, these findings demonstrate that masitinib effectively limits neuronal damage, as reflected by lower serum NfL levels, and supports its potential as a neuroprotective agent in neurodegenerative diseases. [1] Hermine O, Gros L, Tran T-A, Loussaief L, Flosseau K, Moussy A, Mansfield CD, Vermersch P (2025) PLoS ONE 20(4):e0322199. About the neurofilament light chain (NfL) biomarkerThe measurement of neurofilament light chain (NfL) in biological fluids has been proposed for monitoring the therapeutic effect of drugs aimed at reducing axonal damage. NfL are cytoskeletal proteins that are highly specific for neurons in both the central nervous system (CNS) and the peripheral nervous system. NfL in cerebrospinal fluid or the bloodstream is therefore indicative of axonal lesions and/or degeneration and elevated NfL levels are associated with traumatic brain injuries or neurodegenerative diseases (NDD), including amyotrophic lateral sclerosis, multiple sclerosis, and Alzheimer's disease. A growing body of literature shows that because the level of free NfL in serum/plasma directly reflects neuronal damage within the CNS, it can be used as a reliable and easily accessible marker of disease intensity and/or activity across a variety of neurological disorders. About masitinibMasitinib is a orally administered tyrosine kinase inhibitor that targets mast cells and macrophages, important cells for immunity, through inhibiting a limited number of kinases. Based on its unique mechanism of action, masitinib can be developed in a large number of conditions in oncology, in inflammatory diseases, and in certain diseases of the central nervous system. In oncology due to its immunotherapy effect, masitinib can have an effect on survival, alone or in combination with chemotherapy. Through its activity on mast cells and microglia and consequently the inhibition of the activation of the inflammatory process, masitinib can have an effect on the symptoms associated with some inflammatory and central nervous system diseases and the degeneration of these diseases. About AB ScienceFounded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment. AB Science has developed a proprietary portfolio of molecules and the Company's lead compound, masitinib, has already been registered for veterinary medicine and is developed in human medicine in oncology, neurological diseases, inflammatory diseases and viral diseases. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB). Further information is available on AB Science's website: Forward-looking Statements - AB ScienceThis press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance. These forward-looking statements can often be identified by the words "expect", "anticipate", "believe", "intend", "estimate" or "plan" as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents published by AB Science. AB Science disclaims any obligation or undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. of the AMF General Regulations. For additional information, please contact: AB ScienceFinancial Communication & Media Relations investors@ Attachment Masitinib NfL PLOS One vENGError in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
