Cornerstone Pharmaceuticals, a cancer metabolism-based therapeutics company, has been granted orphan-drug designation by FDA for CPI-613, the company's altered energy metabolism directed (AEMD) drug candidate for treating myelodysplastic syndrome (MDS).
Cornerstone Pharmaceuticals previously received orphan-drug designation for the use of CPI-613 in the treatment of acute myeloid leukemia and pancreatic carcinoma. CPI-613 induces cancer-specific inhibition of pyruvate dehydrogenase and alpha ketoglutarate dehydrogenase, key mitochondrial enzymes involved in cancer-cell metabolism. CPI-613 is currently being evaluated in Phase I, I/II, and Phase II trials in hematologic malignancies and solid tumors.
Source: Cornerstone Pharmaceuticals