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US FDA grants orphan drug status to Rafael Pharma's CPI-613 to treat PTCL

Monday, August 20, 2018, 18:00 Hrs  [IST]

Rafael Pharmaceuticals, Inc., a leader in the growing field of cancer metabolism-based therapeutics, announced that the US 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 Peripheral T-Cell Lymphoma (PTCL).

T-Cell Lymphomas account for 10-15 per cent of lymphoid malignancies and display significant heterogeneity. T-Cell Lymphomas have a worse prognosis than most B-Cell Lymphomas. There is no standard treatment for relapsed or refractory disease, and median progression free and overall survival rates have been reported as 3.7 and 6.5 months respectively. There is clear unmet clinical need for relapsed or refractory T-Cell Lymphoma treatments.

CPI-613 is a novel lipoic acid analogue with an anticancer activity that inhibits multiple enzyme targets within the tricarboxylic acid cycle. This drug is currently under investigation in combination with bendamustine in patients with relapsed or refractory T-Cell Lymphoma. 10 patients were dosed to date and 7 were evaluated for efficacy. The interim results of CPI-613 in combination with bendamustine in patients with T-Cell Lymphoma exhibited a very good signal of efficacy with an 86 per cent Objective Response Rate (43 per cent Complete Response and 43 per cent Partial Response). The results from this study were first presented at the American Society of Hematology (ASH) Annual Meeting in 2016.

Zanetta Lamar, MD, the principal investigator of this trial at Wake Forest Baptist commented: “This study of CPI-613 in combination with bendamustine in patients with relapsed or refractory T-Cell Lymphoma showed a good safety profile and encouraging efficacy. The orphan drug designation in this case provides hope to the poor-risk patient population, and inspires us to continue our tireless effort to bring these patients a legitimate treatment option.”

Sanjeev Luther, Rafael Pharmaceutical’s president and chief executive officer, said, “Rafael is now the only oncology company in the US with five orphan drug designations. These orphan drug designations include: Acute Myeloid Leukemia, Myelodysplastic Syndrome, Pancreatic Cancer, Burkitt’s Lymphoma, and now Peripheral T-cell Lymphoma, another patient population with severe unmet clinical need.”

The primary objective of the current study is to determine the Maximum Tolerated Dose (MTD) and safety profile of CPI-613 when used in combination with bendamustine to treat patients with relapsed or refractory T-Cell Lymphoma, or classic Hodgkin Lymphoma. Rafael recently announced the activation of a second clinical trial site for this study at the Abramson Cancer Center of the University of Pennsylvania.

Rafael Pharmaceuticals, Inc. is a privately held clinical-stage, metabolic oncology therapeutics company committed to the development and commercialization of therapies that exploit the metabolic differences between normal cells and cancer cells.


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