Research Unit.
BIAL announces progress in a new Parkinson's treatment
BIAL, a hundred-year-old innovation-driven biopharmaceutical company focused on neurosciences and rare diseases, announced that the first patient has completed the full dose regimen in the ACTIVATE Phase 2 study.
BIA 28-6156 is a first-in-class, small molecule for once-daily oral administration, allosteric activator of beta-glucocerebrosidase (GCase), in development for the treatment of patients with Parkinson’s disease (PD) who have a mutation in the glucocerebrosidase 1 (GBA1) gene (GBA-PD). By increasing the activity of GCase, BIA 28-6156 may be the first drug to directly modify the underlying cause of the disease by re-establishing the sphingolipid recycling.
Joerg Holenz, BIAL´s Chief Scientific Officer, comments: “The first patient out in the ACTIVATE study marks a pivotal milestone in the development of BIA 28-6156, as well as for our ambition to create transformative value for people living with neurodegenerative diseases. We are confident that our medicine has the potential to become a groundbreaking, novel treatment for patients with a confirmed diagnosis of GBA-PD. BIA 28-6156 offers a specific, potentially disease-modifying mechanism of action, with the potential to delay clinical motor progression.”
The ACTIVATE study (clinicaltrials.gov: NCT05819359) is a Phase 2, multicenter, randomised, double-blind, placebo-controlled study evaluating the efficacy, safety, tolerability, pharmacodynamics, and pharmacokinetics of two fixed-dose levels (10mg/day and 60mg/day) of BIA 28-6156. Topline data from this Phase 2 study is expected to be released in mid-2026.
Joaquim Ferreira, Professor of Neurology and Clinical Pharmacology at Lisbon School of Medicine, ACTIVATE study investigator, and member of the ACTIVATE Steering Committee, comments: “The completion by the first patient in the ACTIVATE study marks a significant milestone in this journey. It reflects another step forward in this exceptional effort to advance treatment options for PD, particularly for patients with GBA1 mutations. This step follows the remarkable success of the recruitment phase, which has enrolled over 230 genetically confirmed GBA-PD patients across 85 sites in Europe and North America. There is immense anticipation surrounding the potential of BIA 28-6156, not only for the GBA-PD patient community but also for the broader Parkinson’s community.”
PD is the second most common neurodegenerative disorder affecting more than 10 million people globally, and between 5-15% of PD patients have mutations in the GBA gene, making it numerically the most important genetic risk factor for PD.
GBA-PD patients tend to have, on average, an earlier onset of symptoms compared to those with idiopathic PD(5). They also have more severe clinical symptoms, that progress significantly faster, leading to a worse overall prognosis, thus emphasizing the importance of developing new solutions that can impact the progression of the disease.