A US$6.92m grant from the Michael J. Fox Foundation will help prepare OPM-201 for clinical studies in people with early Parkinson’s disease.
Oncodesign Precision Medicine has been awarded the research grant to prepare its LRRK2 inhibitor. A Phase 1b trial, along with a Phase 2 study, is expected to start in 2027.
The funding follows OPM’s integration into the foundation’s LRRK2 Investigative Therapeutics Exchange programme and the evaluation of OPM-201 within its standardised testing pipelines.
It also builds on positive results from a Phase 1 trial in healthy volunteers, which the company said showed the treatment was safe and well tolerated. Evidence also showed that the highest OPM-201 dose tested interacted with the LRRK2 protein.
Jan Hoflack, OPM’s co-founder and chief scientific officer, said: “This grant shows the high interest of the best experts in the field for OPM-201 as a potential disease-modifying agent for people with Parkinson’s.
“The LITE advisory team has evaluated our available data in high levels of detail and validated the important properties of the compound: potency and selectivity, both in protein binding and cellular assays.”
Parkinson’s is marked by the progressive loss of dopaminergic neurons, the nerve cells that produce dopamine, a signalling molecule used by nerve cells to communicate.
Mutations in the LRRK2 gene are among the most common genetic causes of Parkinson’s, often leading to increased LRRK2 protein activity and disrupting cellular signalling and waste-clearance pathways.
OPM-201 is an oral small-molecule treatment designed to inhibit LRRK2 activity by blocking its phosphorylation, a regulatory process thought to contribute to the protein’s activation.
OPM said the molecule may have disease-modifying potential for Parkinson’s.
Preclinical studies have shown that OPM-201 has complete specificity for LRRK2 compared with a closely related protein, LRRK1.
The grant will support the development and validation of manufacturing processes to produce tablets for the trials. It will also support preparation for a long-term toxicology study required for Phase 2 and Phase 3 clinical development.
“We look forward to driving our program in this partnership at high speed to bring a new disease modifying treatment toward people with Parkinson’s disease who are in urgent need.”
The foundation’s LITE programme brings together researchers, clinicians and industry representatives to advance the development of novel LRRK2-targeting therapies.
By taking part, partners share data, samples, tools and early findings to help move promising ideas towards treatments faster.
Shalini Padmanabhan, the foundation’s senior vice president of discovery and translational research, said: “Advancing the LRRK2 therapeutic pipeline is an important part of our broader goal of fostering robust treatment development efforts for people with Parkinson’s disease.”
