An experimental Parkinson’s pill was linked to improvements in movement and non-motor symptoms in a subgroup of patients in a small Phase 2 trial.
The oral treatment, bezisterim, was associated with significantly greater improvements in motor and non-motor symptom scores than placebo among patients with higher levels of a potential inflammation marker.
The findings came from an exploratory subgroup analysis. Results from the wider trial also suggested improvements in symptoms and daily functioning.
Biovie, the company developing bezisterim, presented the findings from its Phase 2 SUNRISE-PD trial at the 151st Annual Meeting of the American Neurological Association in San Diego.
The company is planning a Phase 3 trial involving approximately 175 people with early-stage Parkinson’s disease and has requested a meeting with the US Food and Drug Administration to discuss the study.
Bezisterim is designed to enter the brain, reduce inflammation and improve insulin sensitivity, which describes how effectively cells respond to the hormone insulin.
Research suggests that inflammation in the brain, known as neuroinflammation, may contribute to the progressive loss of dopamine-producing nerve cells in Parkinson’s disease.
Dopamine is a chemical messenger involved in controlling movement. As levels decline, people with Parkinson’s can develop tremor, stiffness and slowed movement.
The condition also causes non-motor symptoms, including sleep disturbances, cognitive difficulties, depression and anxiety.
Researchers have also identified problems with insulin signalling in Parkinson’s, which may affect how nerve cells use energy and contribute to neurological damage.
By targeting inflammation and insulin resistance, bezisterim is intended to address processes thought to contribute to symptoms and disease progression.
The SUNRISE-PD trial enrolled 57 people with early-stage Parkinson’s who had not previously received levodopa treatment, apart from limited use during diagnostic testing.
Participants were randomly assigned to receive either 20mg of bezisterim or placebo twice daily for 12 weeks.
Researchers assessed inflammation markers, motor and non-motor symptoms, daily functioning, sleep and overall clinical benefit.
The latest analysis focused on 27 participants whose platelet counts were above 230bn per litre of blood at the start of the trial.
Platelets are small blood cell fragments involved in clotting. Higher platelet counts can indicate inflammation throughout the body.
This subgroup had been identified before the trial data were analysed.
Among these patients, those taking bezisterim showed statistically significant improvements in motor and non-motor symptom scores compared with placebo.
The researchers used the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale, which assesses symptoms and their impact on daily activities.
The motor assessment was modified to exclude tests of muscle stiffness and postural stability.
Bezisterim was associated with significantly greater improvements in motor scores, non-motor scores and the overall assessment.
The subgroup also showed greater improvement on the Composite Benefit Score, an exploratory measure combining several aspects of Parkinson’s symptoms and daily functioning.
In the wider efficacy analysis, 25 per cent of participants taking bezisterim experienced clinically meaningful improvements on this measure, compared with four per cent receiving placebo.
Meanwhile, 14 per cent of those taking bezisterim experienced clinically meaningful worsening, compared with 46 per cent in the placebo group.
Higher initial platelet counts also emerged as a potential marker for predicting treatment response, which could help researchers select participants for future trials.
Bezisterim was generally well tolerated, with no severe or serious adverse events reported in either treatment group.
Clarence Ahlem, Biovie’s senior vice president and programme lead for Parkinson’s disease, said the findings supported further investigation of anti-inflammatory treatments.
He said: “Because non-motor symptoms remain a significant unmet need, the breadth of activity observed with bezisterim is particularly encouraging for its development as a treatment for early Parkinson’s disease.”
The company believes the findings warrant further research into whether bezisterim can relieve symptoms and potentially affect the underlying disease process.
However, the evidence remains preliminary, with the strongest improvements found in a subgroup of just 27 participants and the wider findings based on an exploratory measure.
The planned Phase 3 trial will further assess the treatment’s safety and effectiveness in early-stage Parkinson’s disease, including whether the improvements can be reproduced in a larger population.
Further research will also be needed to determine whether bezisterim has any effect on disease progression.
Gene editing could transform Huntington's treatment - study
