OD status for C-Myb antisense drug in CML
Genta's LR3001, an antisense compound directed against a gene known as c-myb, has received Orphan Drug designation from the FDA for the treatment of chronic myelocytic leukaemia (CML).
Genta's LR3001, an antisense compound directed against a gene known as c-myb, has received Orphan Drug designation from the FDA for the treatment of chronic myelocytic leukaemia (CML).
Designation as an Orphan Drug, which is intended to facilitate product development, provides eligibility for a seven- year period of market exclusivity after approval, grants and tax credits for research and development, and reduced filing fees for marketing applications.
Genta obtained rights to LR3001 from Temple University in December 2004. The drug has been tested in two Phase 1 clinical trials in patients with myeloid leukaemias. Genta plans to pursue further clinical development of LR3001 in patients with both haematologic cancers and solid tumours.
About LR3001
LR3001 is part of Genta's DNA/RNA Medicines programme. This programme uses drugs that are based on chemical modifications of DNA or RNA to selectively knock out the function of genes that may be involved in cancer cause or progression. LR3001 targets an oncogene known as cmyb, which is a protein that directly binds to cellular DNA. C-myb is believed to regulate the expression of other genes that are involved in the growth and differentiation of primitive cells, including Bcl-2, Bcl-XL, c-myc, cyclin A1, cyclin D1, cdc2, and cox-2. Over-expression of cmyb blocks differentiation, promotes proliferation, and decreases apoptosis. Potential clinical targets for LR3001 include CML, malignant melanoma, neuroblastoma, and cancers of the breast, pancreas and colon.