AML is a hematologic malignancy characterized by the accumulation of clonal myeloid progenitor cells ("blasts") in the blood or bone marrow. Malignant hematopoietic stem and myeloid progenitor cells proliferate uncontrollably and disrupt normal hematopoiesis, resulting in impairments in the ability of these myeloid progenitor cells to differentiate. Many patients who achieve a remission and complete consolidation therapy still have a guarded prognosis. This is driven by the fact that over 50% of patients will experience a relapse, and most of them will die from AML within a year. There is no consensus standard treatment for relapsed or refractory disease, but most fit patients are treated with a high dose cytarabine (HiDAC) based regimen.
Devimistat is a first-in-class drug developed based on Rafael's Altered Metabolism Directed (AMD) platform. Devimistat targets the altered regulation of metabolic processes specific to cancer cells. It is highly specific, simultaneously attacking multiple targets, minimally toxic and has broad spectrum activity across a wide variety of cancers. Devimistat is currently being evaluated in 7 trials as a single agent, as well as in combination with standard drug therapies for hematological malignancies and solid tumors. To date, over 300 subjects have received one or more doses of devimistat with encouraging response rates and durations of response in several tumor types. In elderly patients with relapsed or refractory AML, devimistat at the biologically effective dose in combination with high dose cytarabine and mitoxantrone exhibited 52% CR+CRi and 12.4 months median overall survival. These results are substantially higher than standard therapy. Devimistat showed a very good safety profile both as single agent and in combination with other standard-of-care drugs. Devimistat has been granted orphan drug designation by the U.S. FDA for Pancreatic Cancer, AML, MDS, Peripheral T-cell Lymphoma and Burkitt's Lymphoma, and has been granted orphan drug designation by the EMA for Pancreatic Cancer and AML.
Sanjeev Luther, President and Chief Executive Officer of Rafael Pharmaceuticals, commented, "Our motto, 'To Save A Life Is To Save A Universe,' reflects our commitment to developing potential treatments for patients with significant unmet clinical needs. We are glad that the EMA has granted Orphan Designation to devimistat for the treatment of patients with Relapsed or Refractory Acute Myeloid Leukemia (AML)."
Dr. Timothy Pardee, Chief Medical Officer of Rafael Pharmaceuticals, said, "Elderly patients with relapsed or refractory AML have so few options. Obtaining orphan drug designation from the EMA is very encouraging and is an important step in the development of devimistat for patients who suffer from this terrible disease."
About Rafael Pharmaceuticals, Inc.
Rafael Pharmaceuticals, Inc. is a 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. Rafael's primary objective is to develop innovative, highly selective, well tolerated and highly effective anti-cancer agents by selectively targeting altered metabolism in cancer cells. Rafael's first-in-class clinical lead compound, devimistat, is being evaluated in multiple Phase I, I/II, and III clinical studies. Devimistat has been granted orphan drug designation for the treatment of pancreatic cancer, acute myeloid leukemia (AML), peripheral T-cell lymphoma (PTCL), Burkitt lymphoma and myelodysplastic syndromes (MDS) by the US FDA. The EMA has granted devimistat orphan drug designation for pancreatic cancer and AML. The Company's investors include Rafael Holdings, Inc. (NYSE American: RFL). For more information, visit http://www.rafaelpharma.com/.
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