The Food and Drug Administration (FDA) has granted Fast Track designation to TT-00420 for the treatment of patients with cholangiocarcinoma (CCA) who have no standard treatment options. The FDA previously granted Orphan Drug designation to TT-00420 for this indication.
TT-00420 is a spectrum selective multikinase inhibitor (Aurora kinases A and B, Janus kinases, fibroblast growth factor receptor [FGFR], vascular endothelial growth factor). The designation is based on results from a phase 1 study (ClinicalTrials.gov Identifier: NCT03654547) that evaluated TT-00420 at different dose levels in 9 CCA patients.
Findings showed that 2 patients achieved partial response and 5 achieved stable disease. Among evaluable patients, there were 5 who had FGFR2 fusion or rearrangement and were resistant to treatment with FGFR inhibitors; 1 patients achieved partial response with progression free survival of about 10 months, while the other 4 achieved stable disease. Stable disease and shrinkage of the target lesion was also reported in 1 patient without FGFR alterations.
TransThera is currently investigating TT-00420 in adults with advanced/metastatic and surgically unresectable CCA with: 1) FGFR 2 fusions who failed prior FGFR inhibitor treatment; 2) FGFR2 fusions who responded on prior FGFR inhibitor treatment; 3) with other FGFR alterations; or 4) whose tumors do not contain a detectable FGFR alteration in a phase 2 study (ClinicalTrials.gov Identifier: NCT04919642).
The efficacy and safety of TT-00420 is also being evaluated in adults with advanced solid tumors, including CCA, in a phase 1/2 study (ClinicalTrials.gov Identifier: NCT04742959).
“Receiving Fast Track Designation is an important milestone for the development of TT-00420.” said Dr Frank Wu, CEO of TransThera, “We have been and will continue to actively work with FDA, expediting the clinical development of TT-00420 in the CCA field.”
The FDA’s Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to therapies that are expected to have an impact on factors such as survival and daily functioning.
- TransThera receives Fast Track designation from FDA for its core product TT-00420 to treat cholangiocarcinoma. News release. TransThera Sciences (Nanjing) Inc. Accessed November 3, 2021. https://www.prnewswire.com/news-releases/transthera-receives-fast-track-designation-from-fda-for-its-core-product-tt-00420-to-treat-cholangiocarcinoma-301415291.html
- Piha-Paul SA, Xu B, Janku F, et al. Phase I study of TT-00420, a multiple kinase inhibitor, as a single agent in advanced solid tumors. J Clin Oncol. Published online May 28, 2021. doi: 10.1200/JCO.2021.39.15_suppl.3090.