NI004 / Neurimmune - LARVOL DELTA

A Study to Assess Safety and Tolerability and to Explore Efficacy of NSC001 in Mild to Moderate Alzheimer's Disease (clinicaltrials.gov) - P1/2 | N=90 | Not yet recruiting | Sponsor: NSC-Therapeutics

New P1/2 trial • Alzheimer's Disease • CNS Disorders

CNS muscarinic receptors and muscarinic receptor agonists in Alzheimer disease treatment. (PubMed, Handb Clin Neurol) - "Based on the acceptance criteria for a preferred M1 muscarinic agonist for the treatment of AD, aiming for efficacy, specificity, and safety in clinical use, few M1 muscarinic agonists fulfill these requirements, such as orthosteric M1 agonists-cevimeline (aka AF102B), the first FDA-approved M1 agonist, and NSC001 (aka AF267B). The pros and cons of various muscarinic agonists developed are critically discussed in comparison to these drugs. The review proposes new alternatives to cholinergic therapy, particularly selective M1 muscarinic drugs, that should be designed to amplify its clinical effect and supplement the disease-modifying effect of new treatments to slow down or arrest disease progression."

Journal • Review • Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Dementia • Lewy Body Disease • Movement Disorders • Parkinson's Disease • Psychiatry • Schizophrenia

PLEIOTROPIC EFFECTS OF THE CNS-SELECTIVE M1 MUSCARINIC AGONIST NSC001: FROM FAST SYMPTOMATIC ACTIVITY TO LONG-TERM DISEASE MODIFICATION IN AD (ADPD 2025) - "Conclusions : NSC001 has entered a Phase 2a proof-of-concept trial in patients with mild to moderate AD, with up-titration from 10 to 40 mg od. The trial will explore safety, PK, AD biomarkers, and cognitive effects, with Results informing a future Phase 3 program."

Alzheimer's Disease • CNS Disorders • Cognitive Disorders • CSF P-tau

MULTIMODAL ACTIVITY IN AD AND RELATED DISORDERS BY PRECISE MODULATION OF THE M1 MUSCARINIC RECEPTOR (ADPD 2025) - "Conclusions : Compared to other muscarinic agonists and M1 positive modulators, NSC001 appears uniquely positioned to deliver enhanced therapeutic effects in MCI, early and later stages of AD, when acetylcholine release is severely diminished. NSC001 represents a novel, multi-modal therapy for AD and related neurodegenerative conditions by delivering rapid cognitive-enhancing effects alongside long-term disease-modifying impact on AD pathology."

Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Psychiatry • Schizophrenia • ADAM17 • PRNP

NSC001 - A SELECTIVE M1 MUSCARINIC ACETYLCHOLINE RECEPTOR AGONIST FOR THE TREATMENT OF ALZHEIMER'S DISEASE AND RELATED DISORDERS (ADPD 2024) - "The mechanism of action of NSC001 via activation of postsynaptic M1mAChR provides a compelling rationale in support of its cognitive benefits and potential disease-modifying properties in AD and other related CNS diseases. Compared with several muscarinic agonists and M1 positive modulators, NSC001 may also have an improved therapeutic value in later neurodegeneration stages, when ACh release is mostly depleted. NSC001 may become a novel therapy in AD with fast cognitive-enhancing effects paralleled by long-term disease-modifying effects on AD pathology."

Alzheimer's Disease • CNS Disorders • Cognitive Disorders • Psychiatry • Schizophrenia • ADAM17 • PRNP

A 28-DAYS PROSPECTIVE, MULTI-CENTER, DOUBLE-BLIND, PLACEBO-CONTROLLED, RANDOMIZED, STUDY TO EVALUATE SAFETY, TOLERABILITY, PK, AND EFFECTS ON PATHOGENETICALLY IMPORTANT BIOMARKERS and qEEG OF EITHER PLACEBO OR FOUR DIFFERENT ORAL DOSES OF NSC001 IN HEALTHY ELDERLY VOLUNTEERS (ADPD 2024) - "Long-term exposure to NSC001 was well tolerated and safe in healthy elderly subjects. The PK profile was consistent with absent drug accumulation, and comparisons between plasma and CSF levels indicated high BBB penetration. Absent effects on CSF biomarkers may be related to low doses and short treatment durations."

Biomarker • Clinical • Alzheimer's Disease • CNS Disorders • Depression • Psychiatry • APOE • Aβ42

Small Molecule Anticancer Compound Modulates Cell Cycle DNA Damage Response Pathway and Inhibit Tumorigenesis in Triple Negative Breast Cancer. (PubMed, FASEB J) - "Overall, our data suggest that NSC001 may be a potent small molecule anticancer compound which should be further investigated for its potential to be used clinically in the treatment of TNBC patients."

Journal • Breast Cancer • HER2 Breast Cancer • Hormone Receptor Breast Cancer • Oncology • Solid Tumor • Triple Negative Breast Cancer • CDKN1A • ER • HER-2 • PGR