BioVie Inc (NASDAQ:BIVI, NASDAQ:BIVIW) said its drug candidate bezisterim met the prespecified endpoints of a Phase 2 trial in early-stage Parkinson's disease, with patients showing improvements in blood-based inflammatory markers and a range of clinical outcomes compared with placebo.
The SUNRISE-PD trial was a multicenter, randomized, double-blind, placebo-controlled study in patients with early-stage Parkinson's disease who had not previously been treated with carbidopa/levodopa.
The majority of patients treated with bezisterim showed improvement on EPNIC-15, a composite endpoint spanning 15 motor and non-motor measures of Parkinson's disease, while patients on placebo moved in the opposite direction.
Patients with higher baseline inflammation saw the greatest benefit. About half the trial population had baseline platelet counts above 230 x10³/µL, a marker linked to inflammation, and in this group bezisterim showed statistically significant advantages over placebo across every clinical measure, including EPNIC-15 and the MDS-UPDRS scores.
The company said benefits were seen across the full range of platelet levels, meaning the marker could help select patients for future trials rather than rule any out.
"Topline results of the SUNRISE-PD trial are encouraging and demonstrate the potential for bezisterim to address significant unmet clinical needs in Parkinson's disease through a differentiated therapeutic approach that targets the underlying disease biology, rather than focusing solely on symptomatic treatment provided by currently approved therapies," said Cuong Do, CEO of BioVie.
Dr Mark Stacy, William E Murray Professor of Neurology at the Medical University of South Carolina, said improvement across both motor and non-motor domains of the MDS-UPDRS is encouraging because these measures represent clinically meaningful outcomes recognized by physicians, patients, and the FDA. He added that the greater treatment effect in patients with higher platelet levels supports the rationale that reduction in neuronal inflammation remains an unmet need in neurodegenerative disease and may guide evaluation of a more targeted patient population in future studies.
James Beck, Chief Scientific Officer of the Parkinson's Foundation, said the trial results could represent a meaningful advancement in Parkinson's treatment and help address a significant unmet need in care.
BioVie said bezisterim was well tolerated, with a safety profile comparable to placebo. Adverse events occurred in 39.3% of patients on bezisterim compared with 51.7% on placebo, with no severe or serious adverse events and one treatment-related adverse event in each group.