In-Vitro Diagnostic test for NASH
NIS4 for the identification of patients with NASH
A key differentiator of our pipeline is our NASH biomarker-based2 diagnostic program, in which we are developing a new in vitro diagnostic test, or IVD, to identify patients with NASH who may be appropriate candidates for drug therapy. We believe this standalone diagnostic has the potential to benefit patients, improve overall clinical care and facilitate the identification of NASH patients to be treated, thereby decreasing the need for liver biopsy.
Since our inception, we have developed a recognized expertise in transcriptomics, which is the study of the RNA transcripts in cells. We initially focused this expertise on mRNA and have expanded to the study of specific microRNAs, or miRNA, short non-coding RNA molecules that are master regulators of many biological processes, and play important role in the regulation of human liver development and pathophysiology. MiRNAs are expressed at different levels in patients with NASH, and as they are released from cells in response to stress, they can be detected in most biological fluids, including blood.
Through our in-house program, we have found four unique biomarkers that we believe provide the best overall diagnostic performance: alpha-2-macroglobulin (A2M), chitinase-3-like protein 1 (CHI3L1), hemoglobin A1c (HbA1c), and microRNA-34a (miR-34a). Our diagnostic test combines the results from these four independent assays through a single proprietary algorithm, referred to as NIS4, to assist in identifying patients with both NASH and significant fibrosis who should be considered for therapeutic intervention with elafibranor or any other suitable therapeutic.
We use advanced technologies, such as next generation sequencing, or NGS, which allows us to perform sequencing of millions of small fragments of DNA in parallel. NGS represents a significant improvement over first-generation sequencing technology, increasing speed and accuracy.
In January 2019, we took a first step towards making this test available as we signed a licensing agreement with LabCorp, a leading drug development and NASH clinical trial company, to expand access to NIS4 in the global clinical research community. It is a first commercial opportunity and this test will provide more visibility to stakeholders in NASH and ensure a good adoption of the test when it is regulatory validated as IVD for clinical decisions.
We therefore anticipate marketing our IVD test first as a laboratory developed test, or LDT, in 2019, and then submitting our IVD test for FDA marketing authorization in 2020.
We believe that broad adoption of our non-invasive, accessible test, if validated and authorized for marketing, would provide physicians with a tool to identify patients who would benefit from treatment and consequently help solve the problem of NASH under-diagnosis.
1Noncirrhotic Nonalcoholic Steatohepatitis with Liver Fibrosis: Developing Drugs for Treatment; Guidance for Industry; FDA 2018
2Biomarkers are characteristics of the body that can be objectively measured and correlate to a biological state or condition. Circulating biomarkers are biological molecules, such as proteins, DNA or RNA, found in body fluids such as cerebrospinal fluid, blood or urine that modulate with disease. A single circulating biomarker or a panel of different markers can be used to not only identify but also monitor the evolution of disease.