A Targeted Approach

There is a high, unmet need for medicines which address the fundamental causes of Parkinson’s disease and GBA-AP.

Rather than merely providing symptomatic relief, therapies targeting the underlying biological causes could potentially slow disease progression for certain patients.

For GBA-AP patients with compromised GCase enzymatic activity, several lines of evidence suggest that pharmacological activation of the GCase enzyme could provide therapeutic benefits.

Compromised levels of GCase activity have been shown to increase Parkinson’s risk and accelerate the decline of Parkinson’s patients.
BIA 28-6156 / LTI-291is the first activator of the GCase enzyme to have been tested in clinical studies. It is designed to target the GCase enzyme to increase activity and improve glycosphingolipid metabolism in the lysosome. Preclinical studies have shown that BIA 28-6156 / LTI-291 easily crosses the blood-brain-barrier and accesses the GCase enzyme within the brain and central nervous system. BIA 28-6156 / LTI-291 is under development as a novel, first-in-class drug compound for the potential treatment of patients with GBA-AP.

Several studies in human cell-based systems and animal models with compromised GCase activity have shown that administration of BIA 28-6156 / LTI-291 restores the glycosphingolipid metabolism and lysosomal function. The effect of BIA 28-6156 / LTI-291 mediated GCase activation is more profound when the GCase enzyme activity is more impaired. BIA 28-6156 / LTI-291 normalized glycosphingolipid levels in a number of model systems.
BIAL Biotech clinical development program has progressed through the first stages of human clinical trials, including in healthy volunteers and GBA-AP patients. Based on the findings observed, the company is preparing the program for more advanced clinical development.