CytoDyn Inc announced positive results from the 350 mg weekly dose of its Phase 2 Nonalcoholic steatohepatitis (NASH) clinical trial.
The Vancouver, British Columbia-based company said that the trial was conducted in two parts. Part one compared a 700 mg weekly dose and placebo in a double-blind randomized manner and Part two evaluated a 350 mg weekly dose as an open-label study compared to the same placebo blinded arm.
“We are in the process of analyzing biomarker data including CCR5 haplotype information to better understand responder rates and mechanism of action,” said Christopher P. Recknor, CytoDyn’s senior executive vice president of clinical operations, in a statement. “Given 5% of the world’s population is estimated to have NASH with 20% progressing to cirrhosis, this signal gives hope for therapeutic intervention for this disease.”
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The primary endpoint, PDFF (proton density fat fraction), is an MRI-derived biomarker for fatty deposition, while the secondary endpoint, cT1, is an iron-corrected T1 mapping representative of liver inflammation and fibrosis. These two values are used to evaluate the risk of NASH.
CytoDyn’s Phase 2 clinical trial compared the changes from baselines in these endpoints. The leronlimab 350 mg dose versus placebo comparison for the primary endpoint PDFF was statistically significant. Leronlimab compared to placebo also reached near significance for the secondary endpoint cT1.
The company said that there were no significant differences in treatment-emergent adverse events between leronlimab and placebo groups.
“The main focus after seeking approval in the US will be the UK, Canada, Brazil, and the Philippines. Hitting our primary endpoint in both ITT and per protocol (PP) and secondary endpoint in PP with such short trial (14-week trial as compared to usually 24 to 32-week NASH trials) is a very strong statement of leronlimab’s potential,” said Nader Pourhassan, CytoDyn’s CEO in a statement.
Nonalcoholic steatohepatitis is a chronic liver disease characterized histologically by the presence of hepatic inflammation and cell injury (hepatocellular ballooning) due to hepatic fat accumulation (steatosis) equal or superior to 5% of hepatocytes. Unhealthy eating habits and lack of physical activity in the absence of excessive alcohol consumption contribute to the development of NASH.
CytoDyn is also conducting a Phase 2 clinical trial for NASH to evaluate the effect of leronlimab on liver steatosis and fibrosis. Pre-clinical studies revealed a significant reduction in Nonalcoholic fatty liver disease (NAFLD) and a reduction in liver fibrosis using leronlimab. There are currently no FDA-approved treatments for NASH, which is a leading cause of liver transplants.
About 30-40% of adults in the US live with NAFLD, and 3 to 12 percent of adults in the US live with NASH. There have been no strong safety signals identified in patients administered leronlimab in multiple disease spectrums, including patients with HIV, COVID-19, and oncology.
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