Oncodesign joins MJFF LRRK2 development collaboration
Program supports development of new therapies targeting the protein
Oncodesign Precision Medicine (OPM) joined a Michael J. Fox Foundation (MJFF) program to develop potential therapies that target the LRRK2 protein, a key contributor to some forms of Parkinson’s disease.
“Our mission has always been to put patients at the center of our innovation,” Jan Hoflack, PhD, OPM’s chief scientific officer, said in a company press release. “Through this collaboration, we will contribute to the development of breakthrough therapies targeting LRRK2, bringing Oncodesign hope to patients living with Parkinson’s disease.”
MJFF’s LRRK2 Investigative Therapeutics Exchange (LITE) program brings together academic and pharmaceutical researchers to support the development of novel therapies that target LRRK2 and advance scientific progress toward new LRRK2-relevant clinical biomarkers. OPM will bring its experimental LRRK2 inhibitor therapy, OPM-201, to the collaboration.
The progressive loss of certain nerve cells in the brain causes a variety of Parkinson’s symptoms. Although treatment can help manage these symptoms, no currently approved therapies can slow or stop disease progression. The underlying causes of Parkinson’s are often unclear, but for some people, the disease has genetic roots.
Mutations in LRRK2 — a gene that encodes the LRRK2 protein — are among the most common genetic contributors to Parkinson’s. These mutations usually increase LRRK2 activity, disrupting pathways involved in cellular signaling and waste clearance. Over time, this dysfunction can damage nerve cells and contribute to Parkinson’s development.
Targeting the underlying cause
Researchers believe that medications targeting LRRK2 or its associated gene may help people with these mutations. These drugs could potentially benefit a broader range of patients as well, OPM said.
OPM-201 is an oral therapy that aims to inhibit LRRK2 protein activity. It blocks phosphorylation, a regulatory process that might play a role in activating the protein. OPM believes this mechanism may address the underlying causes of Parkinson’s, making it a potential disease-modifying treatment.
OPM’s proprietary drug discovery platform, Nanocyclix, focuses on identifying molecules that inhibit kinases, a type of regulatory protein that includes LRRK2.
OPM and its collaborator Servier used Nanocyclix to identify OPM-201 as a therapeutic candidate in 2022. Servier licensed the molecule and took over further development. OPM reacquired rights to OPM-201 in late 2024. The company plans to continue to develop the therapy and is seeking collaborators to assist with future clinical trials.
In a Phase 1 clinical trial in France testing OPM-201 in healthy volunteers, the medication was found to be safe and well tolerated. There was also evidence that the highest tested dose of the therapy engaged the LRRK2 protein, according to OPM.
Hoflack said OPM, which primarily develops kinase inhibitors as cancer treatments, will work with other LITE collaborators to move OPM-201 toward trials in people with Parkinson’s. “In view of OPM’s focus in oncology the MJFF and its LITE program will play a critical role in advancing OPM-201 to the next stage,” he said.
OPM, in turn, will bring its experience with kinase inhibitors to the collaboration. The program might help OPM in its search for new partners, Hoflack said.
MJFF began the LITE program to facilitate LRRK2 research, including preclinical and clinical testing of therapies that target LRRK2 or its gene.
“Through LITE, we are advancing LRRK2 drug development while de-risking industry investment through open-science policies and expert collaboration,” said Shalini Padmanabhan, PhD, head of translational research at the foundation. “We welcome Oncodesign Precision Medicine to this global effort and look forward to advancing progress together on potential disease-modifying therapies for Parkinson’s.”
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