Opaganib by RedHill Biopharma submitted for approval of investigation for COVID-19 treatment

RedHill Biopharma Ltd., a biopharmaceutical company, has announced that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) to study its investigational drug, opaganib (Yeliva^®, ABC294640)1, for the treatment of SARS-CoV-2 infection (COVID-19). The IND submission follows preliminary discussions with the FDA on the study design.

The proposed randomized, double-blind, placebo-controlled Phase 2a study is intended to evaluate the safety and efficacy of opaganib in up to 60 adult patients hospitalized with positive SARS-CoV-2 and pneumonia in the U.S.

“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 Mark L. Levitt, MD, Ph.D., 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.”

A compassionate use program 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 opaganib.

In order to facilitate access to opaganib, various programs are currently under discussion in other countries. In line with the current global shift from compassionate use programs with COVID-19 therapeutic candidates to more rigorously-controlled studies, the compassionate use program 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.

RedHill is preparing for a potential ramp-up of manufacturing of opaganib.

A total of 131 subjects have been dosed with opaganib to date in ongoing and completed Phase 1 and Phase 2 clinical studies in oncology indications in the U.S., in pharmacokinetic studies in healthy volunteers in the U.S., and under the existing FDA-approved expanded access requests from physicians for individual oncology patients, establishing safety and tolerability in humans both in the U.S. and ex-U.S.

Pre-clinical data have demonstrated both anti-viral and anti-inflammatory activities of opaganib, with the potential to reduce lung inflammatory disorders, such as pneumonia, and mitigate pulmonary fibrotic damage. Several prior pre-clinical studies support the potential role of sphingosine kinase-2 (SK2) in the replication-transcription complex of positive-strand single-stranded RNA viruses, like coronavirus, and its inhibition may potentially inhibit viral replication. Pre-clinical in vivo studies2 have demonstrated that opaganib decreased fatality rates from influenza-virus infection and ameliorated Pseudomonas aeruginosa-induced lung injury.

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