RedHill Biopharma Ltd (RDHL), a specialty biopharmaceutical company, reports that all six hospitalized patients with confirmed SARS-CoV-2 infection (the cause of COVID-19) that received their experimental drug opaganib (Yeliva®) demonstrated measurable clinical improvement within days following treatment. At the time of treatment initiation, patients accessing the drug via this compassionate use program were hospitalized and suffered moderate-to-severe COVID-19. Additionally all of them were hypoxic despite being treated with supplemental oxygen. Preliminary findings suggest all six patients have shown a decrease in required supplemental oxygenation, higher lymphocyte counts, a sign of improvement from virus-induced lymphopenia, and decreased C-reactive protein (CRP) levels, an important inflammatory biomarker correlated with lung lesions which could reflect disease severity. The investigational drug was well tolerated both with and without hydroxychloroquine.
The company reported in a press release that five of the six patients analyzed were weaned from oxygen, and three were discharged from the hospital within days of treatment initiation. The 6th patient, whose therapy was initiated more recently, is improving. To date, two patients have safely completed 14 days of Opaganib therapy, which has been well tolerated. The company reports a seventh patient on a hydroxychloroquine and azithromycin regimen suffered from side effects of diarrhea, which resolved quickly following cessation of all therapies. This patient received only 1 day of Opaganib dosing and hence wasn’t included in the analysis.
No Provider/Site Quotes
The company press release included a quote from Medical Director Mark L. Levitt, MD, PhD, noting the encouraging nature of these preliminary findings based on the compassionate use program. However, no quotes were included from any treating facility physician.
FDA Clinical Study
Dr. Levitt of Redhill Biopharma reports that the company will seek to conduct an Opaganib-based clinical trial in the United States. Additionally, the company will work to expand access to the drug via the compassionate use and clinical programs in more countries as well.
What is Opaganib?
TrialSite News profiled this investigational drug when it was approved by Italian regulators for SARS-CoV-2 expanded use for those patients experiencing acute respiratory distress syndrome (ARDS) in Italy. We noted that the investigational drug described by the company as a new, first-in-class orally administered, sphingosine kinase-2 (SK2) inhibitor with anti-viral, anti-inflammatory and anti-cancer characteristics. Originally developed by a U.S. company called Apogee Biotechnology crop, the drug targets oncology, inflammatory and gastrointestinal disorders. In 2015, Redhill Biopharma acquired the drug from Apogee.
By inhibiting SK2, opaganib blocks the synthesis of sphingosine 1-phosphate (S1P), a lipid-signaling molecule that promotes cancer growth and pathological inflammation. By inhibiting SK2, opaganib potentially blocks viral replication complex and pathological inflammation.
The company touts that preclinical data reveals “unique anti-viral” activity in a number of the viruses as well as anti-inflammatory activities and potential to reduce lung damage,” reported Dr. Levitt in a previous company press release. The drug has been through multiple successful pre-clinical studies in oncology, inflammation, GI and radioprotection models.
Clinical Trials to Date
The company has used the experimental drug on 131 subjects in the United States as part of Phase I and Phase II oncology trials. Thus far, the clinical data has acceptable safety and tolerability in both healthy volunteers and cancer patients. Phase 1 clinical study in cancer patients with advanced solid tumors. Opaganib received Orphan Drug designation from the U.S. FDA for the treatment of cholangiocarcinoma and is being evaluated in a Phase 1/2a in advanced cholangiocarcinoma and in a Phase 2 study in prostate cancer. Opaganib is also being evaluated for the treatment of coronavirus (COVID-19) in confirmed COVID-19 patients in Israel.
The development of opaganib has been supported by grants and contracts from U.S. federal and state government agencies awarded to Apogee Biotechnology Corp., including from the NCI, BARDA, the U.S. Department of Defense and the FDA Office of Orphan Products Development
With two approved gastrointestinal products, RedHill Biopharma is publicly traded on NASAQ (RDHL). The company has six drugs in the clinical pipeline. Founded in 2009 in Tel Aviv, they opened up a U.S. headquarters office in Raleigh, NC in 2017. Raleigh is in the famous life sciences cluster called Research Triangle Park or RTP. The company has about 160 employees in its U.S. operations, about 40 of who are based in the headquarters. The company inked a deal with Cosmo Pharmaceuticals to become its supplier in Europe.
Business & Finance
Like many biotech startups, its finances are somewhat precarious, although it’s impressive that they have actually secured market authorization for two products. At a share price of $8.60, as of this writing, their market capitalization equals $303.3 million. However, they forecast losses of $42 million on $6.29 revenue. With $47 million cash in hand, they report repeated losses. See financial disclosures. Its leadership can be viewed here.