What is Crenolanib?
Crenolanib is a small molecule investigational drug candidate that is being evaluated for safety and efficacy in Phase 3 clinical trials in acute myeloid leukemia (AML) and gastrointestinal stromal tumor (GIST). Crenolanib is a potent small molecule inhibitor of wild-type and mutant forms of FLT3 (FMS-like Tyrosine Kinase 3) and PDGFRα/β (Platelet-Derived Growth Factor Receptor).
FLT3 and PDGFRα/β are receptor tyrosine kinases (RTK) which play important roles in signaling cell growth and other processes. Dysregulation of RTK signaling is associated with a variety of diseases and cancers.
The lead indication for crenolanib is FLT3 mutated AML, an aggressive and deadly disease with limited targeted therapy options. FLT3 mutations are the most common mutations in AML, occurring in 25 – 33% of patients and are associated with increased rates of relapse and decreased survival.
We believe crenolanib has the potential to become a significant therapeutic advance for the treatment of FLT3-mutated AML, and that we have optimized the design of our enrolling global Phase 3 pivotal clinical trials to improve our chances to generate compelling efficacy and safety data that could establish crenolanib as a targeted treatment option for newly diagnosed and relapsed / refractory (R/R) FLT3-mutated AML.
Data from our Phase 1 and Phase 2 clinical trials of crenolanib with intensive chemotherapy have demonstrated encouraging rates of durable remissions in patients with newly diagnosed and as relapsed / refractory FLT3-mutated AML.
Additionally, crenolanib monotherapy has demonstrated encouraging activity in PDGFRA mutant GIST and is enrolling patients to a global Phase 3 study. Crenolanib is also being evaluated in phase I-II trials in advanced glioma as a monotherapy and in combination with CYRAMZA (ramucirumab) for advanced gastric cancer.
Clinical Trial Pipeline
|Indication||Patient subset||Phase I||Phase II||Phase III|
|AML||Relapsed/Refractory (2L & 3L)||NCT03250338
|Newly Diagnosed (1L)||NCT03258931|
|Solid Tumor Pipeline|
|GIST||PDGFRa D842V mutated||NCT02847429|