AP26113 is an investigational oral inhibitor of anaplastic lymphoma kinase (ALK). AP26113 was internally discovered by ARIAD scientists, and targets unique genetic features of cancer cells.
ALK was first identified as a chromosomal rearrangement in anaplastic large-cell lymphoma (ALCL). Genetic studies indicate that abnormal expression of ALK is a key driver of certain types of non-small cell lung cancer (NSCLC) and neuroblastomas, as well as ALCL. Since ALK is generally not expressed in normal adult tissues, it represents a highly promising molecular target for cancer therapy.
AP26113 has received a Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with ALK+ metastatic NSCLC whose tumors are resistant to crizotinib. This designation is based on results from the ongoing Phase 1/2 trial that show anti-tumor activity of AP26113 in patients with ALK+ NSCLC, including patients with active brain metastases. A Breakthrough Therapy designation is intended to expedite the development and FDA review of drugs for serious or life-threatening conditions.