Cornerstone Gets Orphan Drug Status for MDS Treatment

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Cornerstone Pharmaceuticals, Inc. is announcing that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CPI-613, the company's lead Altered Energy Metabolism Directed (AEMD) drug candidate for the treatment of myelodysplastic syndrome (MDS).

MDS represents a number of cancers that develop when the blood-forming cells in the bone marrow are damaged. In about one out of every three cases of MDS, the disease transforms from a manageable one into acute myeloid leukemia (AML), a highly aggressive cancer.

Currently there is no cure for MDS.

Expanding treatment options

Said Robert Rodriguez, Cornerstone's President and Chief Operating Officer:

Approximately 15,000 people are diagnosed with MDS in the United States each year. Treatment options for MDS patients are limited, especially for patients with relapsed or refractory disease, and are often associated with myelosuppressive side effects. 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.

According to the company, CPI-613 "induces cancer-specific inhibition of pyruvate dehydrogenase (PDH) and alpha ketoglutarate dehydrogenase (KGDH), key mitochondrial enzymes involved in cancer cell metabolism. Disruption of PDH and KGDH function leads to catastrophic disruption of tumor mitochondrial metabolism. As a result, tumor cells are starved of energy and biosynthetic intermediates, culminating in cell death."

The molecule is currently being evaluated in Phase I, I/II and Phase II trials in hematologic malignancies and solid tumors.

Source: Cornerstone

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