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FDA Grants Orphan Drug Designation to TT-00420 for Treatment of Cholangiocarcinoma

Nichole Tucker
Published Online:4:00 PM, Fri November 8, 2019
TT-00420, an investigational spectrum-selective multiple kinase inhibitor received an orphan drug designation from the FDA for the treatment of cholangiocarcinoma, TransThera Biosciences Co. Ltd announced in a press release.
The safety of TT-00420 is being assessed in the ongoing first-in-human phase Ib/II study in patients with triple-negative breast cancer (TNBC) or other advanced solid tumors (NCT03654547), which is searching for the maximum tolerated dose of the drug in these patients.
Patients between the ages of 18 and 75 with locally advanced or metastatic solid tumors or locally advanced or metastatic TNBC or selected advanced tumors are eligible to enroll in the study if they have an ECOG performance status of 0 or 1 and adequate organ function. Individuals with some unresolved conditions like impaired cardiac function, central nervous system tumors, and mood disorders were excluded from the trial. Patients who have undergone major surgery or had wide-field radiotherapy were also listed among the exclusion criteria.

For treatment, patients are divided into 3 cohorts, consisting of the dose-escalation cohort and 2 dose-expansion cohorts. In the dose-escalation cohort from phase Ib, either 1 mg, 5 mg, or 20 mg of TT-00420 is being administered once daily for a period of 28 days. In the phase II dose-expansion phase, which includes the TNBC cohort and the parallel basket selected advanced solid tumors cohort, a TT-00420 capsule of 1 mg, 5 mg, or 20 mg will be administered once daily for 28 days. The dose-expansion cohorts are expected to start in 2020.
The dose-expansion cohorts are measuring the secondary outcomes, which include dose recommendations for dose expansion, optimal biological dose, number of participants with abnormal laboratory values, number of participants with treatment-emergent adverse events, and peak plasma concentration. The study is also evaluating efficacy in terms of the objective response rate, disease control rate, duration of response, and progression-free survival rate.

The potential introduction of TT-00420 into the treatment paradigm of cholangiocarcinoma, a disease for which recurrence is common, serves an unmet need. Currently, the options for patients with cholangiocarcinoma are chemotherapy and surgery.
“Cholangiocarcinoma lacks effective therapies and remains a huge unmet medical need around the world. TT-00420 has demonstrated great potential in multiple experiments. We believe that today's orphan drug designation will help accelerate the development of this potential product in the clinical trials and bring meaningful benefit to cholangiocarcinoma patients,” said Frank Wu, founder and CEO, TransThera Biosciences Co. Ltd, in a press release. 
TT-00420 is already indicated for the treatment of breast cancer in China. TransThera Biosciences Co. Ltd hopes that the ongoing clinical trials will further develop the drug in the United States.
“We are very excited about this news, which expands the horizon of our lead product in development. In addition to triple-negative breast cancer, cholangiocarcinoma is identified by our scientists to be the second indication for TT-00420,” Wu said in the press release.
TransThera Biosciences Lead Product TT-00420 Granted Orphan Drug Designation from FDA to Treat Cholangiocarcinoma [press release]. Nanjing, China: TransThera Biosciences Co. Ltd; November 7, 2019. https://bit.ly/2CqE4aV. Accessed November 8, 2019.

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