RX5902 is an oral anti-tumor and immune modulating drug targeting the phosphorylated form of p68 (also known as DDX5), a member of the DEAD box family of RNA helicases. Phosphorylated p68 may play a vital role in cell proliferation and tumor/cancer progression by affecting elements in the Wnt Canonical pathway.
This study is conducted as a phase 1b/2a with a 2-stage design. In stage 2 of the phase 2a study, eligible subjects (aged ≥ 18 years) are those diagnosed with triple negative breast cancer, refractory intolerant or ineligible to receive approved standard therapies. There is no limit on the number of prior therapies. Subjects need to have measurable or evaluable disease per RECIST ver 1.1, ECOG performance of 0 or 1 and normal organ function. Use of potent inhibitors or inducers of CYP3A4/3A5, within 14 days of planned study treatment, are excluded. Eligible subjects, who have been fasting, will receive oral RX-5902 at 250 mg administered 5 times per week followed by 2 days off for 4 weeks in each cycle. Treatment will continue for 6 or more cycles, or until confirmed disease progression, unacceptable toxicity, withdrawal of consent, or decision to discontinue. Primary endpoint is progression free survival (by RECIST v1.1); secondary end points include safety, overall response rate, time to progression and duration of response (by RECIST v1.1), duration of response. adverse events will be graded per NCI CTCAE v4.0. Response will be assessed every 8 weeks. Exploratory endpoints include biochemical levels of drug targets. Approximately 40 evaluable subjects will be enrolled in stage 2 of the phase 2.
Clinical trial identification
Legal entity responsible for the study
Rexahn Pharmaceuticals Inc.
Rexahn Pharmaceuticals, Inc.
C. Peterson, E. Benaim: Employee: Rexahn Pharmaceuticals. All other authors have declared no conflicts of interest.