Biomarker-driven therapeutics for cancer
Apeiron Therapeutics is a clinical-stage biopharmaceutical company. We use structure-based, AI-guided drug design to develop small molecule therapies for genetically defined cancers.
9p21.3 MTAP and CDKN2A co-deletion
Our approach
- Biomarker-driven precision oncology
- Each program is developed for tumors with a defined genetic alteration, such as MTAP deletion or chromosomal instability. The same biomarker is used to select patients.
- Synthetic lethality
- The loss of one gene can make a tumor cell dependent on a second. Inhibiting that second target affects tumor cells selectively, while cells that retain the first gene are less affected.
- Structure-based, AI-guided design
- We combine protein structure with AI-guided molecular modeling to design potent, selective small molecules.
Lead program: GTA182
GTA182 is an oral, brain-penetrant, second-generation MTA-cooperative PRMT5 inhibitor for MTAP-deleted solid tumors. MTAP is deleted in approximately 10–15% of solid tumors. GTA182 is in a Phase 1/2 trial as monotherapy and in combination with furmonertinib or chemotherapy. First-in-human results were presented at the ESMO Asia Congress in December 2025.
| Population | Measure | Result (95% CI) | Percent of patients |
|---|---|---|---|
| All MTAP-deleted tumors (n=30) | Objective response rate | 30.0% (14.7–49.4%) | |
| Disease control rate | 83.3% (65.3–94.4%) | ||
| MTAP-deleted NSCLC (n=14) | Objective response rate | 57.1% (28.9–82.3%) | |
| Disease control rate | 85.7% (57.2–98.2%) |
Preliminary Phase 1 data. 41 patients were treated at 20–450 mg once daily; 30 at active doses were evaluable for efficacy. Grade ≥3 treatment-related adverse events occurred in 26.8% of patients. GTA182 is an investigational drug. Its safety and efficacy have not been established, and it has not been approved by any regulatory authority.
Pipeline
Pipeline details| Program | Indication and biomarker | Hit identificationLead optimizationIND-enablingPhase 1/2 |
|---|---|---|
| GTA182PRMT5 inhibitor, second generation | MonotherapyMTAP-deleted advanced solid tumors, including CNS malignancies | Phase 1/2, enrolling in China |
| Combination with furmonertinibMTAP-deleted, EGFR-TKI-refractory non-small cell lung cancer | Phase 1/2 |
|
| Combination with chemotherapyMTAP-deleted pancreatic cancer, second line | Phase 1/2 |
|
| Program 2103KIF18A inhibitor | Advanced solid tumors with chromosome instability | Lead optimization, in research collaboration with the University of Vermont |
| Program 2201MAPK pathway inhibitor | Advanced solid tumors, biomarker enabled | Lead optimization |
| Program 2301PRMT5 inhibitor, third generation | MTAP-deleted tumors resistant or refractory to second-generation PRMT5 inhibitors | Lead optimization |
| REC-617CDK7 inhibitor | Advanced solid tumors | Phase 1/2, globally licensed to Recursion |
About Apeiron
Apeiron Therapeutics was founded in 2019 and is based in the San Francisco Bay Area and Shanghai. The GTA182 IND was cleared by China’s NMPA in October 2024, and first-in-human data were presented at the ESMO Asia Congress in December 2025.
Latest news
All news- GTA182Apeiron Therapeutics Presents First-in-Human Data from Phase 1 Clinical Trial of GTA182 in MTAP-Deleted Advanced Non-Small Cell Lung Cancer at the ESMO Asia Congress 2025
- GTA182Apeiron Therapeutics to Present FiH Phase 1 Data on PRMT5 Inhibitor GTA182 at ESMO Asia 2025
- GTA182Apeiron Therapeutics Presents Preclinical Data from its Oncology Program on PRMT5 Inhibitor GTA182 at AACR 2025 Annual Meeting