EO-3001 Program
Edison Oncology is planning a 20-patient bridging study of EO-3001 to validate pharmacokinetics (PK) and safety in H2 2025, followed by a 90-patient pivotal registration study in 1H 2026 for patients with ARID1A-mutated ovarian clear cell carcinoma.
First -in-Class “Targeted Therapy for ARID1A Mutated Cancers”
EO-3001 is a small-molecule anticancer drug in clinical development for solid tumors harboring an ARID1A mutation. The initial target indication will be Ovarian Clear Cell Carcinoma (OCCC), an aggressive subtype of epithelial ovarian cancer with poor prognosis and limited treatment options.
Scientific Rationale
- ARID1A encodes a key subunit of the SWI/SNF chromatin remodeling complex (BAF250a) and ARID1A mutation disrupts the SWI/SNF complex rendering the cell dependent on OXPHOS for energy production.
- Loss of ARID1A function also leads to epigenetic dysregulation, defective DNA repair, and immune evasion, contributing to tumor progression and resistance to therapy.
Mechanism of Action EO-3001
- EO-3001 is a mitochondrial-targeting small molecule.
- It exploits OXPHOS dependency in ARID1A-deficient cells by inhibiting mitochondrial proteins critical for oxidative phosphorylation. EO-3001 induces a cellular energy crisis and apoptosis, selectively in ARID1A-mutant cells.
Clinical Development History
- EO-3001 was previously studied in multiple clinical trials involving more than 1,000 patients.
- Safety Profile: EO-3001 was well tolerated with no single-agent dose-limiting toxicity observed.
- Efficacy Signal: Response to treatment (CR or PR) was observed in multiple solid tumors.
Preclinical Profile
- EO-3001 Demonstrated high selectivity for ARID1A mutant ovarian cancer cells at low nanomolar concentrations.
- Spares wild-type cells, reducing potential toxicity.
Market Opportunity
ARID1A mutations are observed in up to 10% of all solid tumors.
OCCC – Unmet Need
- OCCC accounts for ~10% of epithelial ovarian cancers in Western populations and up to 35% in patients of East Asian descent.
- These tumors are less responsive to platinum-based chemotherapy and radiation.
- ~1,000–1,800 new U.S. ovarian cancer cases annually may involve ARID1A mutations.
- As of April 2025, NCCN guidelines do not provide specific treatment recommendations for ARID1Amutant OCCC, highlighting a significant unmet need.
Development Status (as of April 2025)
- GMP manufacturing of EO-3001 drug product is complete.
- IND-enabling toxicity/finding study completed.
- Bridging study (20 patients) to validate PK and safety planned for H2-2025.
- Pivotal registration study (90 patients) in ARID1A-mutant OCCC to start in 2026.
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