• Originator Plexxikon
• Developer Array BioPharma; AstraZeneca; Barbara Ann Karmanos Cancer Institute; Columbia University; Daiichi Sankyo Company; Memorial Sloan-Kettering Cancer Center; Merck & Co; National Cancer Institute (USA); Plexxikon; The Christie NHS Foundation Trust; University of California at San Francisco
• Class 2 ring heterocyclic compounds; Antidementias; Antineoplastics; Fluorine compounds; Pyridines; Pyrroles; Small molecules
• Mechanism of Action Fms-like tyrosine kinase 3 inhibitors; Macrophage colony-stimulating factor receptor antagonists; Proto-oncogene protein c-kit inhibitors

• Orphan Drug Status

  Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  Yes - Giant cell tumour of tendon sheath; Pigmented villonodular synovitis
• New Molecular Entity Yes

Highest Development Phases

• Marketed Giant cell tumour of tendon sheath
• Phase II Acral lentiginous melanoma; Glioblastoma; Malignant melanoma; Prostate cancer
• Phase I/II Acute myeloid leukaemia; Breast cancer; Gastrointestinal stromal tumours; Leukaemia; Sarcoma; Solid tumours
• No development reported Colorectal cancer; Pancreatic cancer
• Discontinued Alzheimer's disease; Hodgkin's disease; Neurological disorders; Rheumatoid arthritis

Most Recent Events

• 14 Oct 2024 Daiichi Sankyo completes a phase-I/II clinical trial in Malignant melanoma (Inoperable/Unresectable, First-line therapy, Late-stage disease, Metastatic disease) in South Korea (PO) after January 2017 (NCT02975700)
• 01 Oct 2024 Plexxikon in collaboration with Columbia University terminates phase I/II trial in Sarcoma and Solid tumours (Malignant peripheral nerve sheath tumours) (Inoperable/Unresectable, Late-stage disease, Second-line therapy or greater, Combination therapy) in USA (PO) due to poor enrollment (NCT02584647)
• 31 May 2024 Efficacy and adverse events data from phase-I/II trial in Solid tumours presented at the 60th Annual Meeting of the American Society of Clinical Oncology (ASCO-2024)