BioVie (NASDAQ:BIVI) outlined Phase II results for its investigational drug bezisterim in Long COVID and Parkinson’s disease, while detailing plans to seek FDA feedback on potential Phase III programs.
President and CEO Cuong Do said the company’s two principal assets are bezisterim, an oral small-molecule treatment candidate intended to modulate inflammation, and BIV201, a candidate for ascites associated with end-stage liver disease. BioVie’s recent updates focused on the ADDRESS-LC Phase II Long COVID study and the SUNRISE-PD Phase II Parkinson’s study.
Long COVID Phase II Results
BioVie attributed the overall-population result to the heterogeneity of Long COVID symptoms. Do noted that patients without a particular symptom at baseline have limited ability to demonstrate improvement on measures related to that symptom.
The company had prespecified analyses of patient subgroups based on symptom severity before unblinding the study. In patients with high baseline fatigue, Do said bezisterim produced statistically significant improvements on five measures, including three fatigue-related endpoints. Patients with high post-exertional malaise showed statistically significant improvement on a post-exertional malaise measure as well as cognitive-related measures, according to the presentation. BioVie also reported statistically significant improvements across four cognitive impairment endpoints among patients with high baseline brain fog.
Do said the trial reported no serious adverse events and described the safety profile as favorable. The company is awaiting biomarker results from the study, which it expects to share before year-end. BioVie plans to request an end-of-Phase II meeting with the FDA to discuss a Phase III Long COVID program after reviewing the complete data set.
BioVie is also preparing an application for FDA Breakthrough Therapy designation, Do said. He added that the company is cautiously optimistic that one additional Phase III trial could potentially be sufficient for Long COVID, though FDA feedback will be needed.
Parkinson’s Data and Phase III Planning
In the SUNRISE-PD Parkinson’s trial, BioVie studied 57 patients and assessed clinical outcomes, inflammation, proteomic biomarkers and markers of neuronal injury. Do said the overall population showed slower worsening among bezisterim-treated patients relative to placebo, but the difference did not reach statistical significance.
In a prespecified subgroup of patients with higher baseline inflammation, identified by platelet levels, BioVie reported statistically significant results on components of the Unified Parkinson’s Disease Rating Scale, including motor symptoms, non-motor symptoms and activities of daily living. The company also reported results on its EPNIC-15 composite measure, stating that 25% of bezisterim-treated patients meaningfully improved compared with 4% of placebo patients, while 14% of treated patients meaningfully worsened compared with 46% of placebo patients.
BioVie said its biomarker analysis evaluated 380 markers. Do said all seven Parkinson’s-specific biomarkers moved in a favorable direction, more than 90% of neuronal-injury biomarkers moved favorably, and more than 75% of inflammation biomarkers did so. He highlighted reductions in neurofilament light chain, or NfL, and GFAP, which are biomarkers associated with neurodegeneration.
The company has requested an end-of-Phase II meeting with the FDA on Parkinson’s and expects formal feedback on its proposed Phase III design by the end of the year. BioVie currently expects to conduct two Phase III trials of approximately 160 patients each, one in the United States and one outside the U.S. Do said the primary endpoint would be assessed after six months, with a potential six- to 12-month extension intended to evaluate longer-term disease progression.
Funding and Other Programs
Do said BioVie will need to raise capital to fund future trials but does not intend to use its at-the-market equity program as its primary financing source at current share prices. He said potential investors have indicated that formal FDA feedback on the Phase III design could improve confidence in the company’s development plans.
BioVie’s BIV201 ascites program remains on hold, according to Do. He said the company has agreement with the FDA on requirements for a Phase III registration program but needs approximately $25 million in funding. BioVie had planned to place its ascites assets in a new company, Option Therapeutics, and fund the program through an initial public offering, but Do said market conditions have delayed that effort.
The company’s Alzheimer’s disease program is also progressing slowly because of funding constraints, Do said, as BioVie prioritizes the shorter and potentially less costly Long COVID and Parkinson’s studies.
About BioVie (NASDAQ:BIVI)
BioVie Inc (NASDAQ: BIVI) is a clinical-stage biopharmaceutical company developing therapies for neurological and liver diseases. The company focuses on treatments designed to address underlying disease mechanisms, including neuroinflammation, insulin resistance and fluid accumulation associated with advanced liver disease.
BioVie’s pipeline has included NE3107, an orally administered, blood-brain barrier-penetrant small molecule being investigated for neurodegenerative conditions such as Alzheimer’s disease and Parkinson’s disease.
