Overview
Karyopharm Therapeutics is a biopharmaceutical company focusing on discovery and development of novel drugs for the treatment of cancer, inflammation and diseases related to cell proliferation. Karyopharm Therapeutics is developing small molecule drugs that will modulate the activity of critical pathways in cancer, inflammation, and cell proliferation by targeting the nuclear pore complex machinery, which controls the import and export of key regulatory and tumor suppressor proteins between the nucleus and cytoplasm.
Karyopharm Therapeutic's first target, Crm1 is the main exporter of key tumor suppressor (TSP) and growth regulatory (GRP) proteins such as p53, p21, FOXO, pRB, and NFкB/IкB. Crm1 overexpression has been found to be correlated with poor prognosis in ovarian, cervical, pancreatic, and liver cancers, as well as osteosarcoma and gliomas. Karyopharm has undertaken genomic analyses that have identified genetic alterations of Crm1 in specific cancers. This in-depth validation provides a clear path to clinical targets. The company has used its unique in silico technology platform, developed by our cofounder and CSO, Dr. Sharon Shacham, to identify novel small molecules and to optimize them into clinical candidates. Our novel Selective Inhibitors of Nuclear Export (SINEs) are being characterized for submission of an Investigational New Drug (IND) application for human clinical trial evaluation.
Karyopharm believes that our SINEs can provide novel, well tolerated therapies for cancer, preneoplastic conditions, and other diseases of pathologic proliferation including inflammation and viral diseases.