Phase 1: Early Preclinical Work

Study Initiation:

Theranib initiated efficacy studies on VE3, its first-in-class ALDH1A3 inhibitor, targeting cancers with elevated ALDH1A3 expression.
The focus was primarily on breast cancer, where ALDH1A3 plays a pivotal role in tumor progression and metastasis.
Researchers utilized cutting-edge laboratory models to mimic tumor microenvironments, ensuring relevance to clinical conditions.

In Vitro Studies:

Conducted cell-based assays to evaluate VE3’s mechanism of action and potency in inhibiting ALDH1A3 enzyme activity.
Observed marked reductions in cancer cell proliferation and metastatic behavior, providing initial proof of VE3’s therapeutic potential.
Collected data on the impact of ALDH1A3 inhibition on gene expression pathways related to chemotherapy resistance and metastasis.

Dose Optimization:

Systematic testing of multiple doses to identify the therapeutic window with maximum efficacy and minimal toxicity.
Focused on achieving nanomolar potency, distinguishing VE3 from other ALDH inhibitors with inferior activity.
Utilized pharmacodynamic profiling to determine the relationship between dose and biological response, laying the groundwork for future in vivo studies.

Significant Tumor Inhibition in Cell-Based Experiments:
- Demonstrated VE3’s ability to selectively inhibit ALDH1A3-positive cancer cells without affecting healthy cells, ensuring its specificity.
Pharmacodynamic Data Collection:
- Generated critical data on the kinetics of ALDH1A3 inhibition, tumor growth suppression, and biomarkers for treatment response.
Foundational Insights for Animal Studies:
- Built a robust dataset to support the transition to in vivo efficacy studies, with clear evidence of VE3’s anti-tumor effects in vitro.
Strengthened Patent Claims:
- Reinforced the intellectual property surrounding VE3 by substantiating its unique mechanism and therapeutic potential with strong experimental data.