The data showed that RTA 402 had an excellent safety profile and provided a clinical benefit in a significant percentage of patients. Investigators also confirmed that the drug was active against its biological targets, NF-kappa B and STAT3. These pro-inflammatory transcription factors promote tumor growth, angiogenesis, invasion, metastasis, and resistance to therapy. Overall, RTA 402 showed a profile of disease control and tumor reduction comparable or superior to any recently approved targeted therapy in a Phase I patient population and a markedly superior side effect profile.
As is standard in the development of cancer drugs, this first-in-man study was conducted in patients with advanced cancers without other treatment options. In this difficult patient population, RTA 402 provided a clinical benefit of tumor regression or stable disease to almost half of the evaluable patients in the study. Two patients, one with mantle cell lymphoma and one with thyroid cancer, experienced ‘objective responses’ in which their tumors disappeared or shrank significantly following treatment.
Additionally, several patients with refractory, melanoma or renal cell carcinoma saw their tumors stop growing for more than six months. The activity seen with RTA 402 in this study compares favorably with Phase I studies of recently approved, targeted anticancer agents. Additional data from the study suggest that RTA 402 treatment may promote an anti-tumor immune response. Current research by Reata and its collaborators is focused on characterizing these effects in detail.
RTA 402 was exceptionally well tolerated, with a significantly better side effect profile than recently approved agents. With this safety profile, the drug is an excellent candidate for use in combination therapy with other types of cancer therapies, particularly first-line regimens.