NEURODEX INC — Department of Health and Human Services SBIR Phase I: NIA

Description

Alzheimer s DiseaseADis a common and devastating disease affecting overmillion patients worldwideAD patients lose their independence and are in need of constant carewhich costs estimate above $B in the US aloneAD is considered a graveyard for therapeutic programs with success rate ofan order of magnitude lower than theindustry averageEven successful therapeutics offer only moderate symptomatic relief and do not halt diseases progressionWe believe that a main reason for the repeated failures is the lack of a diagnostic toolbox enabling early detectionbefore clinical symptoms appear and neurons already lostselection of patients with more homogeneous pathology and routine monitoring of treatment responseNeuroDex aims to develop a blood test that allows rapidaccurate and robust population screening for early detection and frequent monitoring of treatment responseThis test is based on immune purification of Neuronal Derived ExosomesNDEfrom plasma samplesExosomes are small vesicles secreted from all cells while containing a sample of their parent cell proteins and RNA signatureThuscapturing NDE provide a surrogate sample of brain neurons of a leaving individualA prototype of the test was already shown to distinguish between AD and controlsas well as Frontotemporal DementiaFTDcommon dementiawith overspecificity and sensitivityMoreoverusing samples fromperson visits in Baltimore Longitudinal Study on AgingBLSAshowed that the test predicted AD development up toyears prior to clinical diagnosis withsensitivity andspecificityThe aim of this study is to develop the tools necessary for further developing the assay into a diagnostic assay to be performed in our CLIA cap certified lab and further down the road developing an IVD diagnostic kitValidating this first in class methodology requires development of a controlled spike in system that allows evaluation of the assay efficiencyaccuracyspecificity and sensitivity to individual plasma matrixWe will determine the assay specified rangerequired volume and validate its linearity and precision between experiments daysWe will use mice model of AD to validate our hypothesis that NDE contents reflects that of brain neuronsWe believe that these steps are needed to confidently advance to clinical validation of the test in a large retrospective clinical studywhich is the goal of phase II of this grantAttaining these aims will provide a robust test with established analytic and quality controlsSuch an assay will put us in a strong position to engage co development agreement with pharmaceutical companies to develop diagnostic and companion diagnostic assays that will contribute to the development of diseases modifying treatment for AD We are developing a unique blood test for Alzheimer s diseaseADpreclinical detectionpatient selection and treatment response monitoringThe test is based on immunoaffinity isolation of neuronal derived exosomes from plasma samples and measurement of their content as surrogates to brain neuronsOur blood test will have the potential to transform the way AD clinical trials are performedfrom attempting to rescue severely injured neurons to earlier preventionpotentially improving the harshsuccess rate of AD therapeutic development to the industry average of close to