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 R&D, 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 I clinical trials in myeloid leukemias and Genta plans to pursue further clinical development of LR3001 in patients with both hematologic cancers and solid tumors.
LR3001 targets an oncogene known as c-myb, 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.
Potential clinical targets for LR3001 include chronic myelocytic leukemia (CML), malignant melanoma, neuroblastoma, and cancers of the breast, pancreas and colon.