Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a clinical-stage biopharmaceutical company focused on addressing key mechanisms of tumor drug resistance, today announced positive, preliminary, top-line data from the ongoing dose escalation part of the Phase 1 clinical study with DCC-3014. DCC-3014 is the Company’s investigational small molecule switch control inhibitor of CSF1R and is used in patients with advanced malignancies. In addition, the Company announced a plan to expand the Phase 1 study to evaluate DCC-3014 in patients diagnosed with Tenosynovial Giant Cell Tumors (TGCT). A review of further data from this Phase 1 study will be presented at a medical meeting in 2019.
The Phase 1 study was designed to evaluate the safety, pharmacokinetics and pharmacodynamics of multiple doses of DCC-3014 in up to 55 patients.
- As of the data cut-off date of November 9, 2018, increasing doses of DCC-3014 were assessed in five dose cohorts across 24 patients with advanced solid tumor malignancies. This included one dose cohort that received 10 mg once daily (QD) and four dose cohorts that followed a schedule of once or twice-weekly maintenance dosing preceded by a five-day loading dose regimen at doses of up to 30 mg per dose. In addition, four patients are currently enrolled in a dose cohort that will receive 40 mg twice weekly preceded by a five-day loading regimen.
- Preliminary pharmacokinetic (PK) analysis showed dose-proportional exposure for DCC-3014 that we believe supports twice-weekly maintenance dosing preceded by a five-day loading dose regimen.
- Biomarker data demonstrated strong target engagement of CSF1R, including material reductions in CSF1R positive macrophages in the blood that we believe constitutes mechanistic proof-of-concept (mPoC) for DCC-3014.
- DCC-3014 was generally well tolerated in patients enrolled in the dose cohorts that received twice-weekly maintenance doses of DCC-3014 preceded by a five-day loading regimen at doses of up to 30 mg, which is summarized below:
- Treatment emergent adverse events (TEAEs) were mostly Grade 1 or 2;
- No Grade 3 or 4 DCC-3014 related TEAEs in ≥10% of patients;
- No dose-limiting toxicities; and
- A maximum tolerated dose has yet to be established.
“We are very pleased with the progress made to date in the Phase 1 clinical study with DCC-3014, our selective, and potent small molecule inhibitor of CSF1R,” said Michael D. Taylor, Ph.D., President and Chief Executive Officer of Deciphera. “Based on the preliminary tolerability, PK, and mechanistic proof-of-concept data observed to date, we believe DCC-3014 is well suited for further development. While we will continue to enroll further patients in this Phase 1 study, we are planning to expand this study to enroll patients with tenosynovial giant cell tumors and to explore combinations with other therapies in the first half of 2019.”