Oncodesign Precision Medicine Receives Support from The Michael J. Fox Foundation to Advance Development of LRRK2 Inhibitor OPM-201 for Parkinson’s Disease

• The grant will support the preparation of clinical Drug Product for Phase 1b and start of Phase 2 in people with Parkinson’s disease and the execution of long-term toxicology study including study product preparation

• The funding follows OPM’s integration into MJFF’s LRRK2 Investigative Therapeutics Exchange (LITE) program and evaluation of OPM-201 within LITE’s standardized testing pipelines

DIJON, France--(BUSINESS WIRE)--Regulatory News:

Oncodesign Precision Medicine (OPM) (ISIN: FR001400CM63; Mnemonic: ALOPM), a biopharmaceutical company specializing in precision medicine for the treatment of advanced cancers, today announces it has been awarded US $6.92 M (about €5.84 M) grant from the Michael J. Fox Foundation for Parkinson’s Research (MJFF) Therapeutic Pipeline Program (TPP). The grant aims to prepare OPM’s OPM-201 LRRK2 inhibitor to start its Phase 1b clinical study in 2027 in early people with Parkinson’s disease, with a focus on LRRK2 mutation carriers.

This next step in OPM-201’s development follows the reacquisition of all rights to the program from its collaboration partner Servier in December 2024. OPM partners with expert collaborators on opportunities and assets delivered by its innovative technologies outside of oncology. These partnerships have enabled OPM to reach the current stage of clinical development for its asset OPM-201.

Building on the positive results in the Phase 1 healthy volunteers’ study, OPM continues the development of OPM-201, a novel small molecule inhibitor of LRRK2 kinase (Leucine-Rich Repeat Kinase 2), a target under active investigation in Parkinson’s disease research.

In Phase 1 studies in healthy volunteers, OPM-201 demonstrated target engagement and an acceptable safety profile at evaluated doses. LRRK2 biology continues to be studied in both genetically defined and broader Parkinson’s disease populations as part of ongoing efforts to understand therapeutic strategies targeting this pathway.

Preclinical studies demonstrated biochemical and cellular selectivity consistent with continued investigation of this pathway. Noteworthy is complete selectivity versus LRRK1, a closely related off target with potential risk for bone related disorders. Participation in LITE is further strengthening OPM’s preclinical package through collaborative evaluation of LRRK2-targeted approaches and associated biomarker strategies.

OPM intends to continue development of OPM-201 in Phase 1b and Phase 2 clinical studies beginning in 2027, with continued engagement within the LITE consortium as appropriate.

The current grant supports the CMC work related to the preparation of Drug Product (drug tablets to be used in the studies) for the Phase 1b and the start of Phase 2 studies, starting from the available 67 kgs of GMP Drug Substance (the pure OPM-201 chemical material).

In addition, OPM-201 material will be prepared for a long-term toxicology study required for Phase 2 and Phase 3 further development. This study will be executed following regulatory approval and is also part of the current award program.

The total duration of the grant program is 24 months, with the preparation of the Phase 1b drug tablets scheduled to finish early 2027.

“This grant from the Michael J Fox Foundation, through its Therapeutic Pipeline funding program, 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 disease. The LITE advisory team has evaluated our available data in high levels of detail, including Phase 1 data in healthy volunteers, and their Dundee experimental team has validated the important properties of the compound: potency and selectivity, both in protein binding and cellular assays,” said Dr. Jan Hoflack, co-founder and Chief Scientific Officer of OPM. “They have also helped us to optimize and prioritize plans for further development of OPM-201 in people with Parkinson’s disease. Following completion of the CMC and toxicology work supported by this grant, OPM intends to advance OPM-201 into Phase 1b and Phase 2 clinical studies. OPM will remain engaged with the LITE consortium for collaborative scientific exchange as development progresses. We look forward to driving our program in this partnership at high speed to bring a new disease modifying treatment towards people with Parkinson’s disease that are in urgent need.”

Philippe Genne, co-founder, President and Chief Executive Officer of OPM said: “We are particularly proud to have been selected by the MJFF, specifically by its LITE expert group specializing in new therapeutic approaches targeting LRRK2 kinase. This recognition is very important and has resulted in the award of a $6.92 million grant. This partnership will enable us to conduct proof-of-concept studies of OPM-201 in people with Parkinson’s disease soon with the support of a world-leading partner. We will also have the opportunity to discuss the further development of the molecule based on the results. This award also represents a major validation of the potential of our Nanocyclix® technology for new generation kinase inhibitors to address major diseases with high unmet need for the benefit of large populations of patients. We will continue to focus on our Nanocyclix®, Oncosniper® and Promethe® technology platforms to bring major innovation in pharmaceutical research. Despite the current difficult financial climate, I am very pleased to see that the quality of our assets enables us to attract the interest of the best experts in the field. We have high hopes that our second asset, OPM-101 for immune-oncology and inflammatory diseases will also attract the interest of potential partners.”

“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, Senior Vice President of Discovery and Translational Research at MJFF. “Through our funding and collaborative models like LITE, we aim to give partners the opportunity to rigorously evaluate their approaches with guidance from leading experts in the field.”

About Parkinson’s disease

Parkinson's disease is a progressive neurodegenerative disorder that affects 1% of the population over the age of 60. This disease, present in nearly 12 million people worldwide in 2021, 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 largely symptomatic, aiming to increase dopamine levels close to the remaining dopaminergic neurons.

About OPM-201

This program began in 2011 and was developed in multiple partnerships with expert companies in neurological diseases. From 2017, Oncodesign pursued the LRRK2 drug discovery program internally for 2 years, which led to the collaboration with Servier, starting in 2019. This collaboration led to the identification of a drug candidate in 2021, with all preclinical and CMC development steps completed in a short timeframe and with convincing results. A Phase 1 clinical trial in healthy volunteers 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 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 protocol for the Phase 1b/2a oncology trial submitted at the end of March 2025 and accepted in 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,
- PROMETHE® for the design and selection of radiolabeled biological molecules for systemic radiotherapy.
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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