- OPM will contribute its unique expertise in kinase inhibitor development and its Nanocyclix®-derived compound OPM-201 to the LITE program
- OPM will leverage the LITE consortium to accelerate the clinical development of OPM-201, strengthen the credibility of the drug candidate, and expand opportunities for fruitful collaborations
Regulatory News:
Oncodesign Precision Medicine (OPM) (ISIN: FR001400CM63; Mnemonic: ALOPM), a biopharmaceutical company specializing in precision medicine for the treatment of resistant and metastatic cancers, today announces it has joined The Michael J. Fox Foundation for Parkinson's Research's program the (MJFF) LRRK2 Investigative Therapeutics Exchange (LITE).
The participation of OPM to The Michael J. Fox Foundation for Parkinson's Research (MJFF) program the LRRK2 Investigative Therapeutics Exchange (LITE) follows the reacquisition of the rights to its OPM-201 program from Servier in December 2024. As a reminder, OPM and Servier have jointly developed OPM-201 a novel small molecule inhibitor of LRRK2 kinase (Leucine-Rich Repeat Kinase 2), a key therapeutic target in Parkinson's disease. This selective, potent, and orally active compound demonstrates the ability to block LRRK2 phosphorylation in the brain without observable side effects at effective doses. Initially developed in familial forms of Parkinson's linked to LRRK2 mutations, non-mutated LRRK2 inhibition also holds promise for a broader population with idiopathic Parkinson's disease. Given LRRK2's central role in disease progression, this candidate represents a compelling opportunity to advance disease-modifying treatments in Parkinson's disease.
The LITE initiative will be governed by a steering committee consisting of Foundation staff and key advisors with drug discovery and LRRK2 biology expertise. It will be implemented by the University of Dundee in the United Kingdom with strong collaborations from more than 20 leading academic institutions, more than 20 biotech and pharma key opinion leaders and more than 10 clinical advisors. The program will test multiple therapeutic strategies, generate critical tools and biomarkers, and build clinical cohorts to support future trials. With a three-year initial funding period, LITE aims to de-risk investment in LRRK2 therapies and create a scalable model to speed drug development for Parkinson's and other neurodegenerative diseases.
By leveraging LITE's open science platform and as an industrial partner, OPM aims to contribute its technological platforms to deepen understanding of LRRK2 biology and translate insights into transformative treatments. More specifically, OPM will contribute its unique expertise in kinase inhibitor development and its Nanocyclix®-derived molecule OPM-201 to the LITE program to advance the asset as a new therapeutic option for Parkinson's disease patients. Moreover, as OPM is actively seeking a strategic partner or investor with CNS expertise development capabilities to advance OPM-201 into Phase 2, being part of the LITE consortium will be an important catalyst to accelerate development, enhance credibility, and expand opportunities for meaningful collaborations.
"Joining the MJFF LITE consortium is an important milestone for OPM," said Dr. Jan Hoflack, CSO of OPM. "Our mission has always been to put patients at the center of our innovation. Through this collaboration, we will contribute to the development of breakthrough therapies targeting LRRK2, bringing hope to patients living with Parkinson's disease. 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 (POC in patients) by providing a networking to facilitate the search for a new suitable partner."
"The Michael J. Fox Foundation is unwavering in its commitment to drive the development of better treatments and to bring us closer than ever to a cure for Parkinson's disease," said Shalini Padmanabhan, PhD, senior vice president and head of translational research at MJFF. "Through LITE, we are advancing LRRK2 drug development while de-risking industry investment through open-science policies and expert collaboration. We welcome Oncodesign Precision Medicine to this global effort and look forward to advancing progress together on potential disease-modifying therapies for Parkinson's."
About OPM-201
This program began in 2011 in collaboration with Ipsen laboratories, it ended in 2017 following a change of strategy by our partner and all rights reverted in full to Oncodesign SA. This research collaboration enabled us to advance the program from "Hit stage" to "Advanced lead", without any investment of our own. We then pursued the Drug Discovery program within Oncodesign SA for 2 years, which led to the collaboration with Servier, starting in 2019. The collaboration led to the identification of a drug candidate in 2022, the date on which Servier exercised the option to license this molecule derived from Nanocyclix® technology. Until December 2024, the development of OPM-201 has remained entirely under Servier's management, with all preclinical and CMC development steps completed in a short timeframe and with convincing results. Servier initiated a Phase 1 trial in healthy volunteers, which demonstrated good tolerability (no serious side effects in any of the healthy volunteers), and interesting LRRK2 target engagement in the highest-dose healthy volunteers. OPM 201 thus naturally claims "Best in Class" status.
About LRRK2
Parkinson's disease is a progressive neurodegenerative disorder that affects 1% of the population over the age of 60. This disease, present in 8.5 million patients worldwide in 2019, is characterized by a progressive loss of dopaminergic neurons. LRRK2 is a major therapeutic target in Parkinson's disease. Activating mutations in the LRRK2 gene are associated with hereditary forms of Parkinson's disease. It is one of the only targets, along with alpha-synuclein, with the potential to modify the course of the disease. Current treatments are symptomatic, aiming to increase dopamine levels close to the remaining dopaminergic neurons. Although targeting LRRK2 is promising, there are challenges, including potential side effects of inhibitors on other organs such as the lungs and kidneys. However, recent advances in understanding the structure and function of LRRK2 are paving the way for more effective and specific therapies.
About the LRRK2 Investigative Therapeutics Exchange (LITE) Program
The Michael J. Fox Foundation for Parkinson's Research (MJFF) launched LITE in 2024 to pave the way for new therapeutic approaches for LRRK2, connect companies that are developing LRRK2-targeting therapies with pharma and biotech opinion leaders, and provide preclinical and clinical resources to establish best practices for advancing LRRK2 targeted therapeutics. Mutations in the LRRK2 gene linked to Parkinson's disease were first discovered in 2004 and are now understood to be the most common cause of inherited PD. Built on MJFF's dedication to open science, LITE fosters international collaboration across more than 30 academic and clinical centers and more than a dozen companies. The initiative is governed by an active steering committee consisting of MJFF staff and field leaders and is implemented by the University of Dundee in the United Kingdom. The LITE program also will benefit from collaboration with the Aligning Science Across Parkinson's (ASAP) initiative-supported programs including the Collaborative Research Network (CRN), the Parkinson's Progression Markers Initiative (PPMI) and the Global Parkinson's Genetics Program (GP2). Learn more here.
About Oncodesign Precision Medicine (OPM)
Oncodesign Precision Medicine (OPM), founded in 2022, is a biopharmaceutical company specializing in precision medicine, dedicated to discovering treatments for resistant and metastatic cancers.
OPM currently has two kinase inhibitors in clinical phase: OPM-101, intended for the treatment of chronic immuno-inflammatory digestive diseases and immuno-oncology, has demonstrated a significant therapeutic margin and absence of toxicity in its phase I healthy volunteers, with the start of phase 1b/2a in Oncology scheduled for September 2025. OPM-201, initially licensed to Servier and intended for the treatment of Parkinson's disease, completed its phase I trial in healthy volunteers at the end of 2024, and was reintegrated into OPM's portfolio.
Both molecules come from the Nanocyclix® technology platform, which enables the design and selection of small, highly effective and selective macrocyclic kinase inhibitors. We now have 12,000 molecules in our library and will be using AI to accelerate the discovery of drug candidates while reducing the cost of this phase.
OPM's other two technology platforms are:
OncoSNIPER, for the selection of therapeutic targets using artificial intelligence, on which we have a partnership with Servier for the search for targets in pancreatic cancer,
PROMETHE® for the design and selection of radiolabeled biological molecules for systemic radiotherapy, on which we are currently discussing partnerships with vectorization manufacturers.
OPM, co-founded by Philippe Genne, Jan Hoflack and Karine Lignel, is based in Dijon, at the heart of the university and hospital cluster, and employs 14 people.
More info at: oncodesign.com
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