Company to host a webcast today at 8:30 a.m. Eastern Time NEW YORK, Nov. 25, 2025 (GLOBE NEWSWIRE) -- Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL), a clinical-stage biopharmaceutical company focused on developing innovative treatments for Alzheimer's disease, Parkinson's disease, schizophrenia, neurodevelopmental, neurodegenerative, and rare diseases, including Rett syndrome, and other central nervous system (CNS) disorders, today reported financial results for its fourth quarter of ...

Core Insights - Klotho Neurosciences, Inc. is advancing the manufacturing and process development for KLTO-202, a gene therapy aimed at treating amyotrophic lateral sclerosis (ALS) [1] - The company has licensed a unique RNA splice variant of the alpha-Klotho gene from the Autonomous University of Barcelona, which is crucial for developing gene therapies [2] - Animal studies have shown that amplifying the levels of secreted alpha-Klotho (s-KL) through gene therapy leads to favorable therapeutic outcomes in models of ALS and other neurodegenerative diseases [2] - The company anticipates an eight-month timeline to complete process development and manufacturing, followed by four to six months for FDA-related activities, aiming to start clinical trials in ALS patients by Q3 of the following year [3] - The CEO highlighted a more efficient method for producing the AAV vector to deliver the s-KL gene directly to motor neurons, which are significantly affected by ALS [4] Company Overview - Klotho Neurosciences, Inc. focuses on innovative cell and gene therapies derived from the human Klotho gene, targeting neurodegenerative and age-related disorders such as ALS, Alzheimer's, and Parkinson's disease [5] - The company's portfolio includes proprietary cell and gene therapy programs utilizing DNA and RNA therapeutics, along with genomics-based diagnostic assays [5] - The management team consists of experienced individuals in biopharmaceutical product development and commercialization [5]

Core Insights - Cognition Therapeutics, Inc. reported promising preclinical data on zervimesine (CT1812) at the ARVO conference, indicating its potential to protect retinal pigment epithelial (RPE) cells from damage in dry age-related macular degeneration (dry AMD) [1][2][3] Company Overview - Cognition Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative small molecule therapeutics for age-related degenerative disorders of the central nervous system [9] - The company is currently investigating zervimesine in clinical programs for dementia with Lewy bodies and Alzheimer's disease, including the ongoing START study in early Alzheimer's disease [9] Product Details - Zervimesine is an investigational oral drug candidate that has shown the ability to reach therapeutic concentrations in the eye and is being studied for its effects on retinal cell health [2][4] - The drug binds to the sigma-2 receptor (TMEM97), which is crucial for retinal cell lipid uptake, a process impaired in dry AMD [2][3] - In a Phase 2 clinical trial, zervimesine slowed the rate of geographic atrophy lesion growth by 28.6% compared to placebo, resulting in smaller lesions for treated patients [4] Disease Context - Dry AMD accounts for up to 90% of age-related macular degeneration cases and leads to irreversible vision loss due to the death of retinal cells [6] - The disease is characterized by the accumulation of oxidized lipids and drusen, which damage RPE cells and contribute to vision loss [3][6] Research Findings - Preclinical research supports zervimesine's potential to protect retinal cells from oxidized lipids and enhance cellular function in degenerative diseases [2][3][4] - The drug has also shown robust clinical results in studies involving Alzheimer's disease and dementia with Lewy bodies, indicating its broader therapeutic potential [4][7]

Financial Data and Key Metrics Changes - As of December 31, 2024, the company's cash, cash equivalents, and short-term investments totaled $413.4 million [36] - Collaboration revenue for Q4 2024 was $54.2 million, up from $15.2 million in Q4 2023, while total collaboration revenue for the year was $100.6 million compared to $97.1 million in 2023 [37] - Total research and development expenses for Q4 2024 were $46.5 million, down from $47.7 million in Q4 2023, and for the year, they were $185.9 million compared to $192.1 million in 2023 [38] - Total general and administrative expenses for Q4 2024 were $15 million, slightly up from $14.9 million in Q4 2023, and for the year, they were $59.6 million compared to $56.7 million in 2023 [38] Business Line Data and Key Metrics Changes - The company is advancing two first-in-class late-stage clinical programs developed in collaboration with GSK, focusing on neurodegenerative disorders [9] - The pivotal Phase 3 trial in frontotemporal dementia with progranulin gene mutation is expected to read out later this year [10] - The ongoing PROGRESS-AD Phase 2 trial of AL101 in early Alzheimer's disease is expected to complete patient recruitment by early 2025 [10] Market Data and Key Metrics Changes - The company is targeting high unmet medical needs in neurodegenerative disorders such as frontotemporal dementia, Alzheimer's disease, and Parkinson's disease [7] - The company anticipates realizing a significant portion of its potential in 2025, with a focus on data-driven decisions to create sustainable value [10] Company Strategy and Development Direction - The company aims to discover and develop first or best-in-class disease-modifying therapies for neurodegenerative disorders [7] - The proprietary Alector Brain Carrier (ABC) platform is central to the company's strategy, enhancing the delivery of therapeutics to the brain [9] - The company is committed to advancing its preclinical pipeline, including programs targeting amyloid beta and tau pathology [33] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential for Latozinemab to provide a path to full approval based on the totality of evidence, including primary clinical endpoints and biomarkers [20][99] - The company remains optimistic about the upcoming readout of the Phase 3 trial and the potential for significant clinical benefits [10][124] Other Important Information - The company received a $1.7 million grant from the Michael J. Fox Foundation for Parkinson's Research to support research on GPNMB [34] - A virtual educational event is planned for Q2 2025 to share additional preclinical data on the ABC platform [35] Q&A Session Summary Question: Regarding the INFRONT study and patient enrollment - Management confirmed that they intentionally targeted early symptomatic populations and capped enrollment for more progressed patients to enhance efficacy [44][45] Question: On AL101 and its relation to INFRONT-3 - Management indicated that there is no significant read-through from the TREM2 trial to AL101 due to differing mechanisms [57][66] Question: About the ABC platform and its differentiation - Management highlighted the versatility and tunability of the ABC platform compared to competitors, emphasizing its potential for optimized efficacy and safety [75][78] Question: On siRNA versus ASOs - Management noted that siRNA may offer better on-target activity and fewer side effects compared to ASOs, with ongoing testing to determine efficacy [82] Question: Regarding the INFRONT-3 trial design and patient enrichment - Management explained the challenges in patient enrichment and the decision to focus on symptomatic patients based on observed progression rates [120][121]