RedHill Biopharma has submitted its Investigational New Drug application for its drug opaganib, for the treatment of COVID-19, to the FDA.
RedHill Biopharma has announced it has submitted an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) to study its investigational drug, opaganib (Yeliva®, ABC294640), for the treatment of SARS-CoV-2, the virus causing the COVID-19 pandemic. The IND submission follows preliminary discussions with the FDA on the study design.
The proposed randomised, double-blind, placebo-controlled Phase IIa study is intended to evaluate the safety and efficacy of opaganib in up to 60 adult patients hospitalised with positive SARS-CoV-2 and pneumonia in the US.
Opaganib is a new chemical entity and is a proprietary, first-in-class, orally-administered, sphingosine kinase-2 (SK2) selective inhibitor with anticancer, anti-viral and anti-inflammatory activities, targeting multiple oncology, inflammatory and gastrointestinal indications. By inhibiting SK2, the drug blocks the synthesis of sphingosine 1-phosphate (S1P), a lipid-signalling molecule that promotes cancer growth and pathological inflammation.
Several pre-clinical studies support the potential role of SK2 in the replication-transcription complex of positive-strand single-stranded RNA viruses, similar to coronavirus. By inhibiting SK2, the drug potentially blocks viral replication and pathological inflammation.
“We are moving quickly to advance the clinical development of opaganib for COVID-19 and in parallel to expand access to opaganib for patients under compassionate use to help address the global pandemic caused by SARS-CoV-2,” said Dr Mark Levitt, Medical Director at RedHill. “We are encouraged by the preliminary findings from the first patients treated with opaganib under compassionate use indicating objective clinical improvement and look forward to collaborating closely with FDA to validate the effectiveness of opaganib in treating COVID-19 in a controlled clinical setting.”
According to the company, a compassionate use programme with opaganib is ongoing in Israel, with five patients treated to date in a leading hospital. Preliminary findings from all five patients have shown an improved C-Reactive Protein (CRP), an inflammatory biomarker, with four of the five patients also demonstrating measurable clinical improvement, including reduced supplemental oxygenation and higher lymphocyte counts within days following treatment initiation with the drug.
In order to facilitate access to opaganib, various programmes are currently under discussion in other countries, says the company. In line with the current global shift from compassionate use programmes with COVID-19 therapeutic candidates to more rigorously-controlled studies, the compassionate use programme in Italy is planned to be converted to a clinical study in order to generate more robust clinical data in a controlled setting for regulatory purposes.
A total of 131 subjects have been dosed with opaganib to date in ongoing and completed Phase I and Phase II clinical studies in oncology indications in the US.