In November 2016, the European Medicines Agency’s (EMAs) Committee for Orphan Medicinal Products (COMP) granted Orphan Drug Designation for BP1001 (prexigebersen) for the treatment of Acute Myeloid Leukemia (AML).
BP1001 is a novel liposome-incorporated antisense oligodeoxynucleotide targeted against the Growth factor receptor-bound protein 2 (Grb2) mRNA. BP1001 binds to and blocks Grb2 mRNA thus preventing Grb2 expression. Grb2 is involved in growth signalling pathways and can contribute to increased cell proliferation in cancer cells1.
BP1001 is currently being explored in combination with Low Dose Ara C (LDAC) in a phase 2 study (NCT02781883) for previously untreated elderly AML patients who are ineligible for intensive induction therapy. BP1001 is also be investigated in phase 1 study (NCT01159028) in adult patients with recurrent AML.