Cornerstone Pharmaceuticals has received the US Food and Drug Administration (FDA) orphan drug designation for its altered energy metabolism directed (AEMD) drug candidate 'CPI-613' for treatment of myelodysplastic syndrome (MDS).
MDS is a type of cancer that is caused when the blood-forming cells in the bone marrow are damaged, leading to low numbers of one or more type of blood cells.
According to the company, currently there is no cure for patients suffering from MDS and in about one-third of the cases, the disease progresses to a growing cancer of bone marrow cells called acute myeloid leukemia (AML).
CPI-613, which was developed using Cornerstone's proprietary AEMD drug platform, is currently being assessed in Phase I, I/II and Phase II trials in hematologic malignancies and solid tumors.
The AEMD platform disrupts biochemical alterations in the conversion of glucose to energy that occur in many types of tumor cells.
As a result of the disruption, tumor mitochondrial metabolism gets disabled starving tumor cells of energy and biosynthetic intermediates, thereby causing cell death.
Cornerstone president and chief operating officer Robert Rodriguez said every year about 15,000 people are diagnosed with MDS in the US.
"Treatment options for MDS patients are limited, especially for patients with relapsed or refractory disease, and are often associated with myelosuppressive side effects," Rodriguez added.
"We are encouraged by the lack of myelosuppression, good tolerability and signals of activity that were observed in MDS patients in the Phase I trial.
"We look forward to expanding our experience with CPI-613 as a monotherapy in the treatment of relapsed / refractory MDS patients in the recently launched Phase II MDS clinical trial as well as the possibility of establishing CPI-613 as an alternative treatment option for this rare and devastating disease."
Apart from MDS, the company had already received orphan drug designation for the use of CPI-613 in the treatments of AML and pancreatic carcinoma.