CytoDyn Inc announced positive preliminary results from the 20-patient-cohort who have completed the open-label portion of the Phase 2 trial for nonalcoholic steatohepatitis (NASH).
The Vancouver-based company’s current Phase 2 NASH trial is designed to test in 90 patients whether leronlimab may inhibit the devastating liver fibrosis associated with NASH. This trial consists of two parts: Part 1 is a double-blind placebo-controlled trial using 700 mg leronlimab versus a placebo in a 1:1 ratio. Part 2 is open-label, with all subjects receiving 350 mg leronlimab for 14 weeks.
The primary and secondary endpoints are 14-week changes from baseline in proton density fat fraction (PDFF) and cT1, an iron-corrected T1 mapping representative of liver inflammation and fibrosis.
READ: CytoDyn files for expanded access use of leronlimab for multi-drug resistance HIV patients
“We believe we have a very strong case to make for Breakthrough Therapy designation (BTD) for leronlimab treatment in NASH patients. We are eager to see the results of the blinded portion in the next couple of weeks,” CytoDyn CEO Nader Pourhassan said in a statement.
Nonalcoholic fatty liver disease (NAFLD) has become the most common cause of chronic liver disease in adults worldwide. NAFLD patients could also develop NASH due to problems with liver fibrosis (the thickening and scarring of liver tissue). There are currently no US Food and Drug Administration (FDA) approved treatments for NASH, and it was expected to be the number one cause of liver transplants in 2020. About 30-to-40% of adults in the US are living with NAFLD, and 3 to 12% of adults in the US are living with NASH.
CytoDyn previously demonstrated strong positive data in its pre-clinical study, highlighting the potential of leronlimab in treating nonalcoholic fatty liver disease (NAFLD), a common precursor to NASH leronlimab in treating nonalcoholic fatty liver disease.
Inhibition of CCR5 has been shown to reduce fibrosis in animal models of NASH liver fibrosis, and current data suggests the same trend in humans. Proton density fat fraction is an MRI-derived biomarker for fatty deposition, while cT1 is an iron-corrected T1 mapping representative of liver inflammation and fibrosis. They are both used to evaluate the risk of NASH.
“The cT1 and PDFF changes we are seeing in patients who have completed the open-label portion are showing that leronlimab in reducing markers of NASH. These results will guide us in developing our phase 3 NASH trial,” said Christopher P. Recknor, CytoDyn’s senior executive vice president of clinical operations.
Pourhassan concluded: “Upon unblinding, if the results are supportive, we intend to file for BTD and to seek accelerated approval for use of leronlimab in NASH and NAFLD patients in the US and abroad. We will also aggressively seek partnerships, especially with big pharmaceuticals who have experienced recent failures in their NASH trials.”
CytoDyn successfully completed a Phase 3 pivotal trial using leronlimab combined with standard antiretroviral therapies in HIV-infected patients who were heavily treatment-experienced individuals with limited treatment options.
The company is also conducting a Phase 2 clinical trial with leronlimab in mTNBC, a Phase 2 basket trial in solid tumor cancers (22 different cancer indications), Phase 2 investigative trial for post-acute sequelae of SARS COV-2, also known as coronavirus (COVID-19) long haulers.
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