SK biopharmaceuticals' Parkinson’s drug restores movement, clears toxic protein in early study

In preclinical models of GBA1-linked Parkinson’s disease, a genetic subset accounting for up to 15 percent of cases of the disease, SK biopharmaceuticals’ oral candidate SKPD restored motor function and maintained its effect for up to three months after treatment ended, the company reported last week.

The data, presented at the GBA1 Meeting 2025 in Montreal from Thursday to Saturday (local time, EDT), point to SKPD’s potential as a disease-modifying therapy, offering more than just symptom relief by directly targeting the biological drivers of the disease.

SKPD activates GCase, an enzyme that helps clear waste inside cells. In patients with GBA1 mutations, this enzyme is compromised, leading to the buildup of alpha-synuclein, a toxic protein that damages dopamine-producing neurons. 

SK biopharmaceuticals said SKPD reduced alpha-synuclein levels in multiple animal models and also activated lysosomal and autophagy pathways, the brain’s internal systems for clearing damaged proteins and cellular debris.

"SKPD not only reversed motor deficits in multiple animal models, but the effect persisted well beyond the treatment period," the company said in a Monday release. SK biopharmaceuticals positioned the drug as a potential disease-modifying therapy rather than a short-term symptomatic fix.

The drug is being developed as an oral therapy for Parkinson’s, with initial clinical trials planned for patients carrying GBA1 mutations. The company said it intends to broaden development to include the wider Parkinson’s population as more data become available.