Data from collaborative study between MiRXES and Janssen demonstrate how miRNA-based biomarker signatures could lead to potential in early identification and diagnosis of pulmonary hypertension.
Pulmonary hypertension is a progressive condition in which the blood pressure in the blood vessels of the lungs is abnormally high which could lead to heart failure and early death. Pulmonary arterial hypertension is a progressive form of pulmonary hypertension that takes, on average, two years to diagnose from the onset of symptoms. Many are diagnosed only at advance stages of the disease while there are others who remain unidentified. Currently, there is no simple, non-invasive test to identify and diagnose whether the patient has pulmonary hypertension. Delayed diagnosis has also proven to affect patient clinical outcomes and survival. Development of an early diagnosis method for treatment to kick in would thus have tremendous impact on the patients’ prognoses, clinical outcomes and survival.
In a collaboration between MiRXES, a microRNA (miRNA) diagnostic company headquartered in Singapore, and Actelion Pharmaceuticals Ltd, one of the Janssen Pharmaceutical Companies of Johnson & Johnson, preliminary data of the study revealed that miRNA biomarker signatures have the potential to support early identification and diagnosis of pulmonary hypertension.
The results were presented at the annual European Respiratory Society (ERS) International Congress held in September 2020.
“The positive results of this study show the potential of miRNA-based diagnostic signatures as a tool to help identify those in the early stages of pulmonary hypertension,” said Aaron Waxman, M.D., Ph.D., Director of the Pulmonary Vascular Disease Program at Brigham and Women’s Hospital, Boston, USA, and Associate Professor of Medicine at Harvard Medical School.
Dr Aaron Waxman also shared that a biomarker known as, N-terminal pro-brain natriuretic peptide (NTproBNP) is commonly used in certain centres for pulmonary hypertension. However, it is shown to not be specific to pulmonary hypertension and also found to be increased in patients with other comorbidities – limiting its utility in detecting early stage pulmonary hypertension.
This collaborative study aimed to assess miRNA-based biomarker signatures in patients in the early stage of the disease, as well as those with established pulmonary hypertension using MiRXES’ proprietary assay technology and biomarker discovery platform.
A representative sample of early stage pulmonary hypertension patients were drawn from patients with borderline and exercise pulmonary hypertension. Together with patients with established pulmonary hypertension and non-pulmonary hypertension symptomatic patients, a total of 245 plasma samples were collected for the study.
These samples underwent testing for the commonly used biomarker and 600 miRNAs for analysis. Results then showed that miRNAs were more superior in distinguishing non-pulmonary symptomatic patients from those who were early stage – suggesting the unique ability of miRNAs in identifying early stage pulmonary hypertension. The results also showed that combining miRNA with NTproBNP allows distinguishing of non-pulmonary hypertension symptomatic patients from established pulmonary hypertension patients.
Results from this present study added on to the evidence from an earlier phase of the global research collaboration conducted in the UK and presented in August 2020 at the American Thoracic Society International Conference.
Building on these data, Janssen has initiated the CIPHER trial, the design of which is also being presented at ERS. The CIPHER trial is an ongoing, prospective, multi-centre study that aims to identify miRNA biomarker signatures for early detection of pulmonary hypertension.
“We are very excited with the promising early PH miRNA biomarker data generated from the current studies. Having launched our first regulatory-approved blood-based miRNA oncology test in 2019, MiRXES is committed to leveraging our end-to-end In Vitro Diagnostic (IVD) test development and manufacturing capabilities to support the translation of the PH miRNA biomarker signatures from bench to bedside,” said Zhou Lihan, Ph.D., CEO of MiRXES Pte Ltd.
Further clinical studies are currently in being planned in Asia, starting with Singapore and Japan, to provide further evidence to support the development and validation of the miRNA-based diagnostic test for early identification and diagnosis of pulmonary hypertension.