Business Description

Overview We are a clinical-stage biotechnology company developing treatments for age-related brain disorders. Our lead drug candidate, neflamapimod, is an investigational, orally administered small-molecule drug that readily crosses the blood brain barrier and selectively inhibits the enzyme p38α, a key driver of neuroinflammation and synaptic dysfunction. By targeting the critical disease processes underlying degenerative disorders of the brain, neflamapimod has the potential to reverse synaptic dysfunction, improve neuron health, and slow or prevent disease progression. Neflamapimod is currently in clinical development for the treatment of DLB, our lead indication, as well as nfvPPA, RAS, and ALS. Our novel approach focuses on reducing the impact of neuroinflammation, which we believe is a key factor in the manifestation of degenerative diseases of the brain. Chronic activation of p38α in the brains of people with certain neurodegenerative diseases is believed to impair how neurons communicate through synapses. This synaptic dysfunction leads to deterioration of cognitive and motor abilities. Left untreated, synaptic dysfunction can result in irreversible neuronal loss that leads to devastating disabilities, significant reliance on a caretaker, long term care living, and, ultimately, death. However, before neuronal loss commences, disease progression in many major neurodegenerative disorders, including DLB, initially involves a protracted period of reversible functional loss, particularly with respect to the synapses. We believe that inhibiting p38α activity in the brain has the potential to reverse the clinical progression observed in the early stages of certain neurodegenerative diseases, as well as slow further progression by delaying permanent synaptic dysfunction and neuron death, by interfering with key pathogenic drivers of disease. We believe we are a leader in the industry in developing a treatment for DLB, a disease with no approved therapies in the US or European Union, despite being the second most common progressive dementia. Neflamapimod is the only clinical drug candidate that, to our knowledge, has shown statistically significant improvements on clinical endpoints and a biomarker of neurodegeneration in both a Phase 2a and Phase 2b clinical trial. Differentiating our approach from potential competitors, we believe we are also the only company specifically targeting the treatment of DLB patients without AD co-pathology. While DLB patients with AD co-pathology generally have significant, irreversible neuronal loss, DLB without AD co-pathology is primarily a disease of functional deficits of synapses that we believe is more treatable. We believe if neflamapimod is given in the early stages of certain degenerative diseases of the brain like DLB without AD co-pathology, it may reverse synaptic dysfunction, improve neuron health and function, and slow further progression by delaying synaptic dysfunction and neuronal death. We believe this approach enhances the alignment of our development path with neflamapimod’s mechanism of action, reduces the heterogeneity of our target patient population, and provides the opportunity to demonstrate heightened clinical effect in shorter duration trials. Our Pipeline Set forth below is a table presenting our clinical pipeline: Anticipated Milestones Set forth below are our anticipated clinical development milestones during the next 12 months, subject to, among other things, available funding: Anticipated Milestones 2Q26 Complete enrollment in Phase 2a clinical trial in nfvPPA Mid-2026 Complete enrollment in Phase 2a RESTORE Trial in RAS Mid-2026 Initial Phase 2a biomarker data in nfvPPA 2H26 Topline Phase 2a clinical data in RAS 2H26 Initial Phase 2a clinical data in nfvPPA 2H26 Initiation of planned Phase 3 trial in DLB YE26 First patient dosed with neflamapimod in EXPERTS-ALS trial in ALS 1H27 Topline Phase 2a clinical and biomarker data in nfvPPA Our Strategy Our mission is to develop and commercialize innovative medicines that change the course of the disease in patients who suffer from age-related brain disorders. 9 The key elements of our strategy are: ● Capitalize on neflamapimod ’ s unique properties and our differentiated approach to progress the development of neflamapimod for the treatment of age-related brain disorders with high unmet medical need.…