Company Description

We are a molecular diagnostics company initially focused on the development and commercialization of innovative blood-based tests for detection and monitoring of Mild Cognitive Impairment (MCI), Alzheimer’s disease (AD), Parkinson’s disease (PD), and other neurodegenerative diseases (NDs). The proprietary technology we developed is based on quantitative analysis of “organ-enriched”, i.e. present at higher concentration in specific organs or tissues, microRNAs (miRNAs) in plasma. We believe that the technology can also be applied to disease areas beyond NDs. These projects include a test for a rare neurological and developmental disorder Rett syndrome which is currently being clinically validated, and cancer and inflammatory disorders, where the Company’s work is at an early stage. The tests being developed at the Company are currently not commercially available. While launching a lab-developed test (LDT) is our primary goal, we believe that providing testing services for pharma and biotech companies involved in the development of therapeutic treatments for MCI and AD will allow us to generate revenues prior to launching the LDT in the clinical setting, improve our test performance characteristics, and potentially allow us to offer our test as a companion diagnostic. However, since we do not currently have any definitive agreements to provide such testing services, such revenues cannot be guaranteed. Even if we are able to generate revenue from these testing services, we cannot be sure it will provide sufficient revenue to support our operation and R&D. miRNAs as biomarkers. miRNAs are a class of 21-23 base-pair, short non-coding functional RNA molecules that bind to messenger RNA (mRNA) and regulate protein synthesis and cellular processes, such as growth, inflammation, survival, and death. miRNAs may be enriched in certain organs (e.g., brain, liver, lung), tissues or organ regions (e.g. hippocampus, midbrain), cell types (e.g. neurons), and cellular compartments (e.g. synapses and neurites). miRNAs can cross the blood-brain barrier and appear in the bloodstream. These properties of brain-enriched miRNAs make them powerful and patient friendly biomarkers of NDs and other brain health conditions. miRNAs are quantified, from plasma, using established reverse transcription quantitative polymerase chain reaction, RT-qPCR, technology and their expression patterns can be used to develop testing algorithms to assign risk of progression to individual patients. In October 2024, the Nobel Assembly at Karolinska Institutet awarded the 2024 Nobel Prize in Physiology or Medicine jointly to Dr. Victor Ambros and Dr. Gary Ruvkun for the discovery of microRNA and its role in post-transcriptional gene regulation. The press release regarding this award stated that the recipients “groundbreaking discovery revealed a completely new principle of gene regulation that turned out to be essential for multicellular organisms, including humans. It is now known that the human genome codes for over one thousand microRNAs. Their surprising discovery revealed an entirely new dimension to gene regulation. MicroRNAs are proving to be fundamentally important for how organisms develop and function.” Press release: The Nobel Prize in Physiology or Medicine 2024 - NobelPrize.org High need for minimally invasive early detection of MCI and AD. According to Alzheimer’s Association Report: 2023 Alzheimer’s Disease Facts and Figures (https://www.alz.org/media/documents/alzheimers-facts-and-figures.pdf), 6.7M Americans over age 65 currently have AD; someone in the US develops AD every 67 seconds. In 2021, total cost of care for Alzheimer’s disease and other dementias was estimated at $321 billion (not including unpaid caregiving). AD dementia is typically preceded by 10-20 years of the disease development in the brain, initially without clinical symptoms (pre-symptomatic MCI due to AD), and then subsequently manifested as MCI, which itself is a precursor to AD-associated dementia. Detailed analyses of failed clinical trials of anti-AD therapies suggest that therapeutic benefit is mostly seen in the sub-groups of patients with MCI and/or moderate AD. Thus, there is a critical need for the development of methods for early AD detection. Because cognitive testing cannot reliably identify patients in pre-symptomatic stages of AD, effective biomarkers are now viewed as necessary targets for successful patient enrollment into clinical trials and treatment monitoring. Recent advances in detection techniques based on neuroimaging and cerebrospinal fluid (CSF) biomarkers have facilitated the shift in clinical research from the dementia phase to MCI preclinical AD. However, the high cost of over $3,000 with limited reimbursement, along with invasiveness of neuroimaging and cerebrospinal fluid needle draw for analysis, make their application for primary and monitoring purposes impractical. These techniques are also frequently not available in community centers, where blood-based tests can provide a viable solution as an initial diagnostic assessment tool. Over the past 15 years, significant progress has been made in the development of blood protein markers as valid diagnostic markers for MCI and AD and recently, the utility of such markers as surrogates for disease monitoring and therapeutic response measurements has been strongly considered (“Diagnostic Accuracy of a Plasma Phosphorylated Tau 217 Immunoassay for Alzheimer Disease Pathology”, by Ashton NJ et al. JAMA Neurol. 2024. doi:10.1001/jamaneurol.2023.5319; “Highly Accurate Blood Test for Alzheimer’s Disease Comparable or Superior to Clinical CSF Tests”, by Barthélemy NR et al., Nat Med. 2024 doi:10.1038/s41591-024-02869-z; “Discriminative Accuracy of Plasma Phospho-tau217 for Alzheimer Disease vs Other Neurodegenerative Disorders”, by Palmqvist S et al. JAMA. 2020, 324:772-781. doi:10.1001/jama.2020.12134; Palmqvist S, Tideman P, Cullen N, et al. “Prediction of future Alzheimer’s disease dementia using plasma phospho-tau combined with other accessible measures”, Palmqvist S et al. by Nat Med. 2021, 27:1034-1042. doi:10.1038/s41591-021-01348-z; “Differential roles of A?42/40, p-tau231 and p-tau217 for Alzheimer’s trial selection and disease monitoring”, by Ashton NJ et al., Nat Med. 2022, 28:2555-2562. doi:10.1038/s41591-022-02074-w; “Blood-based biomarkers for Alzheimer’s disease: towards clinical implementation”, by Teunissen CE et al. Lancet Neurol. 2022, 21:66-77. doi: 10.1016/S1474-4422(21)00361-6). Biomarker signatures for detection of NDs. We are working on developing a comprehensive portfolio of tests for early and specific detection of neurodegenerative diseases. Our primary testing platform targets brain-enriched and inflammation-associated miRNAs; however, we may add blood protein biomarkers and mutational markers to our test over time. We have completed several studies in independent cohorts of samples and in collaborations with academic centers. We believe that the data generated to date indicate that our approach to detecting and differentiating neurodegenerative disorders may provide clinically meaningful information to clinicians and patients. Clinically meaningful tests are tests that impact the practice of medicine by either changing the treatment course for a patient or making physician practice more efficient. Clinically meaningful tests can be LDTs or FDA-approved tests. Foundational articles in Alzheimer’s field have demonstrated that synaptic dysfunction/loss precede clinical manifestation of AD (“Hypothetical model of dynamic biomarkers of the Alzheimer’s pathological cascade” by Jack Jr CR et al. Lancet Neurol 2010 9:119, PMID: 20083042; “Toward defining the preclinical stages of Alzheimer’s disease: recommendations from the National Institute on Aging-Alzheimer’s Association workgroups on diagnostic guidelines for Alzheimer’s disease” by Sperling RA et al. Alzheimers Dement 2011 7:280, PMID: 21514248). We believe DiamiR’s miRNA biomarker approach captures synaptic dysfunction and loss, reflecting early stages of neurodegeneration, as reported in DiamiR publications listed elsewhere in the disclosure. Statements made by several experts within the field, in the report titled “Technology Niche Analysis for CogniMIR® titled “In Vitro Test for Early Detection and Monitoring of Alzheimer’s Disease” (Project #: NIH1052TN), in the publication Foresight Science & Technology, further support commercial and clinical potential of our approach. According to a comprehensive report published by the Alzheimer’s Association (“2019 Alzheimer’s Disease Facts and Figures”): “Finding a simple and inexpensive test, such as a blood test, to diagnose Alzheimer’s would be ideal for patients, physicians and scientists.” A more recent report from the Association (“2023 Alzheimer’s Disease Facts and Figures”) stated: “The relatively recent discovery that Alzheimer’s disease begins 20 years or more before the onset of symptoms suggests that there is a substantial window of time in which we may be able to intervene in the progression of the disease. Scientific advances are already helping the field to make progress in these presymptomatic years. … Biomarkers … enable earlier detection of Alzheimer’s, giving those affected the opportunity to address modifiable risk factors that may slow or delay cognitive decline. Biomarkers are already accelerating the development of new treatments by making it possible for clinical trials to specifically recruit individuals with the brain changes that experimental therapies target. In addition, biomarker, basic science and other research advances offer the potential to expand the field’s understanding of which therapies or combination of therapies may be most effective at which points in the Alzheimer’s disease continuum.” In summary, we believe CogniMIR® test may provide clinically meaningful information to clinicians as it may help identify individuals with pathological changes in the brain targeted by therapeutic agents in development. These individuals may then be recruited in appropriate clinical studies or selected for compassionate administration of experimental treatments. It is the Company’s belief that dynamic miRNA biomarkers of CogniMIR® test may also provide clinically meaningful information to patients, providing the affected individuals with “the opportunity to address modifiable risk factors that may slow or delay cognitive decline” (“2023 Alzheimer’s Disease Facts and Figures”). These assertions have not been evaluated by the FDA. DiamiR has not received FDA approval for CogniMIR® test, but intends to seek the launch of CogniMIR® test as an LDT in accordance with the FDA and CLIA guidelines. --- Our principal executive office is located at 11 Deer Park Drive, Suite 102G, Monmouth, NJ 08852. Our telephone number at our principal executive office is 732-823-1143. Our laboratory is located at 2 Church Street South, Suite B-5, New Haven, CT 06519. Our corporate website is www.diamirbio.com.