BioVie (NASDAQ:BIVI) President and CEO Cuong Do said during a RedChip Companies webinar that the company expects two near-term clinical readouts for its lead drug candidate, bezisterim, with Parkinson’s disease data anticipated in late July or early August and Long COVID data expected around late August to early September.
Do said BioVie is focused on inflammation and insulin resistance, with bezisterim designed to modulate production of TNF-alpha. He said the company believes the drug may have applications across Parkinson’s disease, Long COVID and Alzheimer’s disease, while a second asset, BIV201, is being developed for ascites, an end-stage liver disease condition.
Parkinson’s Trial Readout Expected This Summer
“If everything goes according to plan, we hope to announce the results of this data within the next month or two,” Do said, adding that the company is targeting “late July, early August” for the Parkinson’s trial results.
Do said BioVie’s approach to Parkinson’s differs from the traditional focus on low dopamine levels and motor symptoms. He said the company believes inflammation and insulin resistance are important metabolic drivers of the disease, and that future therapies should address both motor and non-motor symptoms.
Do discussed an earlier 40-patient trial in moderate to severe Parkinson’s disease in which bezisterim was tested in combination with levodopa. He said the combination showed greater muscle control than levodopa alone and that about one-third of patients receiving the combination were in an “on” state first thing in the morning before taking their morning medication. He also said some patients reported improvements in sleep, anxiety and depression measures.
Responding to a question about placebo effects in Parkinson’s studies, Do said BioVie used home-based nurse assessments, video recording and centralized rating to reduce variability. He also said the trial included a placebo run-in period to exclude patients whose assessments changed significantly before randomization.
Long COVID Study Fully Enrolled
Do said BioVie’s Long COVID trial is fully enrolled with 200 patients, split between placebo and bezisterim. He said the trial is evaluating endpoints related to cognition, malaise, fatigue and biomarkers, and described the study as exploratory, with about 20 endpoints.
According to Do, the trial is supported by a $13 million grant, and enrollment included participation from Long COVID centers including Yale, Mount Sinai, Mayo Clinic, UCSF and Stanford. He said Yale enrolled the last patient several weeks ago, putting the study on track for the final patient visit in early to mid-August.
If unblinded results are promising, Do said BioVie would seek discussions with the U.S. Food and Drug Administration in the fall about accelerated approval or Emergency Use Authorization, and said the company believes it could seek Breakthrough Therapy designation.
Alzheimer’s Program and Comparisons to Approved Drugs
In response to a question comparing BioVie’s Alzheimer’s approach to LEQEMBI and Kisunla, Do said bezisterim is different because it targets TNF-alpha production rather than amyloid plaques. He said BioVie believes amyloid and tau are inflammatory in nature and that blocking TNF-alpha may address multiple sources of inflammation.
Do said BioVie previously conducted a Phase 3 Alzheimer’s trial in which bezisterim showed a 68% slowing of cognitive decline versus placebo after six months on the CDR-SB endpoint. He contrasted that with anti-amyloid drugs, which he said showed roughly a 30% slowing of cognitive decline versus placebo after 18 months as a class.
Do also said BioVie’s Alzheimer’s program is currently on hold because of funding needs. He estimated that each Alzheimer’s trial could cost $25 million to $30 million and said two trials would be needed, for a total estimated cost of $50 million to $60 million to bring bezisterim to market for Alzheimer’s disease.
Ascites Asset May Be Moved Into Option Therapeutics
BioVie’s second asset, BIV201, is being developed for ascites. Do said the company has received the FDA feedback it needs to proceed to the next step and noted that BIV201 has orphan and Fast Track designation.
Do said BioVie has filed an S-1 for a company called Option Therapeutics and intends, when market conditions are appropriate, to place the ascites drug into that company. He said BioVie would aim to raise about $25 million to $30 million for Option Therapeutics to fund a registrational trial, which he estimated would take about two and a half years to conduct and support registration.
Cash Runway, Partnerships and Market Opportunities
Do said BioVie has “sufficient cash to last towards the tail end of the year” and described the company’s market capitalization as about $10 million, which he said was below its current cash balance. He also said the company has held conversations with larger pharmaceutical companies, but that potential partners have generally asked BioVie to return when it has additional clinical data.
Do provided company estimates for potential U.S. annual sales opportunities, saying BioVie believes ascites could represent $1.5 billion to $2 billion, Parkinson’s could represent $3 billion to $5 billion, Long COVID could represent more than $10 billion and Alzheimer’s could represent more than $30 billion. These figures were presented as BioVie’s internal views of potential market opportunities.
During the webinar, Do said safety monitoring continues in ongoing bezisterim trials and that, based on blinded data reviewed so far, the company has not seen drug-related severe adverse events and has observed “very few adverse events.” He said BioVie is not releasing blinded Parkinson’s analyses to avoid influencing patients or clinicians before the trial is unblinded.
About BioVie (NASDAQ:BIVI)
BioVie Inc is a clinical?stage biopharmaceutical company focused on developing novel therapies for chronic liver diseases and associated neurological complications. The company’s research and development efforts center on candidates designed to address serious unmet medical needs in hepatic encephalopathy and other liver?related disorders. BioVie advances its pipeline through controlled clinical trials and regulatory interactions in North America.
The company’s lead product candidate, BIV201, is undergoing Phase 2 clinical evaluation for the treatment of hepatic encephalopathy, a life?threatening condition marked by elevated neurotoxins in patients with advanced liver disease.
