Biognosys Partners with Michael J. Fox Foundation to Develop Critical LRRK2 Biomarker Assays for Parkinson's Disease
Key Insights
Biognosys Group (search) has announced a collaboration with The Michael J. Fox Foundation to develop biomarker assays for LRRK2 (search), the most common genetic risk factor for late-onset Parkinson's disease (search).
The partnership will support MJFF's LRRK2 (search) Investigative Therapeutics Exchange (LITE), a global initiative involving more than 50 academic, industry and clinical partners working to accelerate LRRK2-targeted therapy development.
The collaboration aims to address a significant barrier in Parkinson's drug development: the lack of sensitive and reliable biomarkers to measure LRRK2 (search) expression and activity as treatment indicators.
Proteomics specialist Biognosys Group (search) has announced a collaboration with The Michael J. Fox Foundation to develop biomarker assays for Parkinson's disease (search), specifically targeting leucine-rich repeat kinase 2 (search) (LRRK2 (search)), the most common genetic risk factor for late-onset Parkinson's disease.
The partnership will support MJFF's LRRK2 (search) Investigative Therapeutics Exchange (LITE), a global initiative designed to accelerate the development of therapies and biomarkers targeting LRRK2. LITE represents a collaborative effort involving more than 50 academic, industry and clinical partners who share resources, biosamples, tools and early findings in real time to quickly evaluate and advance promising therapeutic ideas.
Addressing Critical Development Barriers
LRRK2 (search) has emerged as one of the most compelling targets in Parkinson's research, with a growing number of therapeutic modalities under development to reduce excessive LRRK2 activity. These approaches could benefit individuals with LRRK2 mutations and a broader population of people with Parkinson's disease (search) who share the same dysregulated biological pathway.
However, a significant barrier to progress is the lack of sensitive and reliable biomarkers to measure LRRK2 (search) expression and activity as a treatment indicator. Developing these assays is challenging due to low signal abundance and biological complexity.
"Successful clinical development requires not only promising therapeutic approaches, but also reliable tools to measure whether those therapies are having their intended biological effect," said Dr Oliver Rinner, Senior Vice President at Biognosys Group (search). "By combining Biognosys' expertise in highly sensitive, quantitative proteomics with MJFF's collaborative LITE framework, we aim to accelerate the development of biomarkers that can meaningfully guide Parkinson's drug development and de-risk clinical trials."
Advanced Proteomics Technology
Biognosys will use the SISCAPA and TrueSignature ultra-sensitive proteomics platforms in this collaboration to tackle the technical challenges and support the use of LRRK2 (search) and related biomarkers in clinical trials. The quantitative biomarker assays being developed could be used both to guide the development of therapies for Parkinson's and, potentially, to identify patients that could be treated with LRRK2-directed therapies.
Current LRRK2 Therapeutic Landscape
The LRRK2 (search) inhibitor furthest along in clinical development is Denali Therapeutics' small-molecule candidate DNL151, which reached the middle stages of clinical development. The phase 2b LUMA trial of the Biogen-partnered therapy, also known as BIIB122, in early-stage Parkinson's is due to read out in the middle of this year, while a phase 2a study in LRRK2-mutated disease is ongoing.
Biogen bought into Denali's LRRK2 (search) programme in a deal potentially worth more than $2.1 billion in 2020, but three years later it abandoned a phase 3 trial of the drug, called LIGHTHOUSE, in LRRK2-positive patients and amended the protocol of LUMA to include patients without the mutations. At the time, Biogen said its decision was not taken due to any concerns about the safety or efficacy of BIIB122, but was the result of the long timeline for the LIGHTHOUSE study and "resource prioritisation."
Other companies working on LRRK2 (search) include Neuron23 (search), Oncodesign Precision Medicines (search), and Brenig Therapeutics (search), all of which have clinical-stage candidates in development. South San Francisco-based Neuron23 raised $96.5 million in funding for the ongoing phase 2 NEULARK trial of its NEU-411 candidate in early Parkinson's patients with elevated LRRK2 activity, with topline results due in 2027.
Dijon, France-based Oncodesign recently picked up nearly $7 million in funding from the MJFF for a phase 1b trial of OPM-201, which was partnered with Servier before it handed back rights to the project in 2024. Boston start-up Brenig recently presented interim data from an ongoing phase 1 clinical trial of BT-267 in healthy volunteers.
"Through LITE, the Michael J. Fox Foundation (search) supports collaborative efforts to develop reliable biomarkers that can measure target engagement and biological response in Parkinson's disease (search)," said Dr Shalini Padmanabhan, Senior Vice President of Discovery and Translational Research at MJFF.