MJFF grants $6.9M to prepare OPM’s Parkinson’s therapy for clinical trial
OPM-201 now part of foundation's LITE program for testing potential treatments
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- OPM-201, an LRRK2 inhibitor, is being developed by Oncodesign Precision Medicine as a potential disease-modifying therapy for Parkinson's.
- The Michael J. Fox Foundation for Parkinson's Research has granted OPM $6.9 million to prepare the therapy for clinical trials.
- The oral treatment candidate targets LRRK2 protein activity, a genetic cause of Parkinson's.
Oncodesign Precision Medicine (OPM) has been awarded a $6.92 million research grant from The Michael J. Fox Foundation (MJFF) to prepare its LRRK2 inhibitor, OPM-201, for a clinical study in people with early Parkinson’s disease.
The funding follows OPM’s integration into MJFF’s LRRK2 Investigative Therapeutics Exchange (LITE) program and the evaluation of OPM-201 within LITE’s standardized testing pipelines. The Phase 1b trial, along with a Phase 2 study, is expected to start in 2027, according to a company press release announcing the grant.
The MJFF award builds on positive results from a Phase 1 trial in healthy volunteers, which showed the medication was safe and well-tolerated. Evidence also demonstrated that the highest OPM-201 dose tested interacted with the LRRK2 protein, per the company.
“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,” said Jan Hoflack, PhD, OPM’s cofounder and chief scientific officer. “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 signaling molecule that neurons use 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 signaling and waste-clearance pathways.
MJFF grant will help produce drug tablets for trial
OPM-201 is an oral small-molecule therapy that aims to inhibit LRRK2 activity by blocking its phosphorylation, a regulatory process thought to contribute to the protein’s activation. OPM believes the molecule may be a potential disease-modifying treatment for Parkinson’s.
Preclinical studies have shown that OPM-201 has complete specificity for LRRK2, versus a closely related protein, LRRK1.
This new grant supports the development and validation of manufacturing processes to produce drug tablets for the trials, according to OPM. It also will support preparation of the therapy for a long-term toxicology study required for Phase 2 and Phase 3 clinical development, the company noted.
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.
“Following completion of the … work supported by this grant, OPM intends to advance OPM-201 into Phase 1b and Phase 2 clinical studies,” Hoflack said. “OPM will remain engaged with the LITE consortium for collaborative scientific exchange as development progresses.”
According to Hoflack, the foundation’s support will be helpful.
“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,” Hoflack said.
MJFF’s LITE program joins researchers, clinicians, and industry representatives to advance the development of novel LRRK2-targeting therapies. By taking part in the program, partners share data, samples, tools, and early findings, working together to move promising ideas to therapies faster.
“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,” said Shalini Padmanabhan, PhD, MJFF’s senior vice president of discovery and translational research.
