BioVie (NASDAQ:BIVI) outlined upcoming clinical catalysts for its lead drug candidate bezisterim and provided an update on a separate liver-disease program during a presentation at the RedChip Biotech Investor Conference led by President and CEO Cuong Do.
Leadership background and pipeline overview
Do introduced himself as a biochemist who previously spent 17 years at McKinsey helping build its healthcare practice and later held strategy roles at pharmaceutical companies, adding that he has also founded biotech companies and invested in startups. He said he initially invested in BioVie and served on its board before stepping in to lead the company after helping orchestrate the acquisition of bezisterim.
- Bezisterim, described as a “novel modulator of TNF-alpha production” that is orally administered and crosses the blood-brain barrier.
- BIV201, a drug candidate aimed at ascites, an end-stage liver disease condition.
Bezisterim mechanism and the Parkinson’s program
Do said bezisterim works by blocking activation of ERK and NF-kappaB to prevent TNF-alpha production, which he connected to reduced inflammation and reversal of insulin resistance. He argued that insulin resistance is a key contributor across multiple disorders and linked Parkinson’s symptoms not only to low dopamine, but also to inflammation and insulin resistance.
In discussing existing Parkinson’s treatment, Do noted levodopa’s short half-life and the challenge of levodopa-induced dyskinesia as dosing increases over time. He described preclinical work in rodents and non-human primates in which bezisterim alone was “equally effective as levodopa” in restoring muscle control, and said the combination of bezisterim and levodopa showed a synergistic effect. He added that, at the end of the primate study, the company found animals treated with the combination retained “twice as many neurons” as those treated with levodopa alone, which he said suggests neuroprotective properties.
Do also described a 40-patient Phase IIa study in moderate Parkinson’s patients that tested bezisterim in combination with levodopa. He said the combination group showed better muscle control than levodopa alone, and that about a third of patients on the combination were “in on state first thing in the morning.”
BioVie is now running a Phase IIb trial evaluating bezisterim alone in earlier-stage Parkinson’s patients. Do said the study enrolled 60 patients who are starting therapy for symptoms for the first time, randomized between bezisterim and placebo. The trial is fully enrolled, and he said the last patient’s final visit is May 1, with top-line data expected by the end of June.
Do framed the Phase IIb objective as obtaining two key inputs to plan a Phase III trial: the magnitude of effect across endpoints and the standard deviation needed to power the next study. Based on what the company knows now, he estimated a Phase III trial could require roughly 300–500 patients.
Long COVID trial backed by a $13 million grant
Do said BioVie is also studying bezisterim in long COVID, pointing to an estimated 17 million Americans with lingering symptoms such as brain fog and fatigue, and about 3 million with severe impairment affecting employment. He described a hypothesis that persistent viral protein fragments may keep the immune system activated, driving TNF-alpha production and symptoms through pathways he said bezisterim affects.
BioVie received a $13 million grant to support an exploratory trial, which Do said makes the company “the only organization of any kind” to receive a grant to explore whether a therapeutic can address central nervous system symptoms of long COVID. The placebo-controlled trial plans to enroll 100 patients and evaluate cognitive and physical measures as well as biomarkers. Do said the trial is enrolling rapidly with participation from academic centers including Yale, Mount Sinai, Mayo Clinic, Stanford, and UCSF, and that it should be fully enrolled by the end of the month, positioning the company for a top-line readout by year-end.
Do said BioVie can see blinded data as it comes in and is observing “an encouraging pattern of some people getting better,” with some patients reporting they feel “back to normal,” while others are not improving or worsening. If the apparent improvements align with the treatment arm after unblinding, he said the company would seek discussions with the FDA in the fall about “Accelerated Approval or perhaps an Emergency Use Authorization.” He also said he believes positive data could drive partnering interest or acquisition interest.
BIV201 ascites program and potential spin-out
Do also discussed BIV201 for ascites, describing the condition as having no therapy beyond repeated fluid removal and citing “over 50% mortality rate within a year.” He said a Phase IIa trial showed the drug was safe with no drug-related serious adverse events, and that BioVie terminated a Phase IIb efficacy study midway after seeing data indicating patients on treatment had “greater than 50% reduction of ascites fluid buildup.” He said the company believed it was ethical to stop and engage the FDA on a registration pathway.
According to Do, BIV201 has Fast Track and Orphan Drug designations, and the company has received FDA feedback indicating it would need one Phase III trial for registration. He said BioVie needs to raise $25 million to proceed and has filed an S-1 to take a new company public—Option Therapeutics—with the intention to place BIV201 into the new entity to fund the Phase III effort when market conditions allow.
In Q&A, Do said bezisterim could be viewed as a molecule with applicability across central nervous system indications, with the company also evaluating “sister molecules” that do not cross the blood-brain barrier for other uses. He pointed to what he described as a consistent safety profile so far, saying the most reported side effect was a mild headache lasting about a day in roughly 8% of patients. He added that large pharmaceutical companies have expressed interest in the mechanism but want clinical data before partnering discussions advance.
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.
