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Rafael Holdings rallies as devimistat clinches orphan drug status in Europe

The drug is for relapsed cases of acute myeloid leukemia in older patients as well as metastatic pancreatic cancer
cancerous cells
Devimistat is currently being evaluated in seven trials as a single agent, as well as in combination with other therapies for blood malignancies and solid tumors

Rafael Pharmaceuticals announced Thursday that the European Medicines Agency has granted orphan drug status to its drug devimistat for the treatment of older patients with relapsed cases of acute myeloid leukemia as well as metastatic pancreatic cancer.

Devimistat is a drug developed based on Rafael’s Altered Metabolism Directed (AMD) platform. The drug is capable of attacking multiple targets and can fight a variety of cancers. The drug is currently being evaluated in seven trials on its own, as well as in combination with standard drug therapies for blood malignancies and solid tumors.

Investors were impressed by the results, sending shares of parent company Rafael Holdings Inc (NYSEAMERICAN:RFL) up nearly 14% to $10.37 in morning trade on Thursday.

READ: Rafael Holdings focuses on ‘very strong’ balance sheet after posting fiscal 3Q results

Elderly patients (60 or older) with relapsed or refractory acute myeloid leukemia who took devimistat in combination with high-dose cytarabine and mitoxantrone saw a median overall survival rate of 12.4 months, according to Rafael.

Acute myeloid leukemia is a blood malignancy characterized by the accumulation of clonal myeloid progenitor cells in the blood or bone marrow.  Malignant hematopoietic stem and myeloid progenitor cells proliferate uncontrollably and disrupt normal hematopoiesis, the process by which blood cells are formed. 

Many patients with this type of leukemia who see their cancer enter remission still have a guarded prognosis as relapses of the disease are common.

“Elderly patients with relapsed or refractory acute myeloid leukemia have so few options,” noted Timothy Pardee, Rafael’s chief medical officer, in a statement.

Pancreatic cancer in focus

In pancreatic cancer, meanwhile, devimistat in combination with the therapy Folfirinox showed patients reported an objective response rate of 61% and median overall survival of 19.9 months.

Pancreatic cancer is a deadly disease, with a mortality rate of more than 95% within five years. Chemotherapy is currently the most sought-after treatment option for metastatic pancreatic cancer.

“We are glad that devimistat has received orphan designation to treat this dreaded disease,” said Sanjeev Luther, Rafael Pharma’s CEO.

Rafael is a clinical-stage oncology therapeutics company that focuses on the development and commercialization of therapies that exploit the metabolic differences between normal cells and cancer cells. 

Devimistat has already been granted orphan drug designation for the treatment of pancreatic cancer, acute myeloid leukemia (AML), peripheral T-cell lymphoma (PTCL) as well as Burkitt lymphoma and myelodysplastic syndromes (MDS) by the US Food and Drug Administration.

Contact Ellen Kelleher at [email protected]

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