EP0042 for AML Earns Orphan Drug Designation


The Orphan Drug Act was passed with a goal in mind: to incentivize the development of therapeutics for orphan drugs. These drugs are used for patients with rare conditions, or those affecting fewer than 200,000 people within the United States. Now, the Orphan Drug designation comes with a variety of benefits for drug developers such as fee waivers, tax credits, and seven years of market exclusivity upon approval. According to an article published by OncLive, the U.S. Food and Drug Administration (FDA) recently granted Orphan Drug designation to EP0042, a treatment for relapsed or refractory acute myeloid leukemia (AML). 

What is EP0042?

The National Cancer Institute (NCI) explains that EP0042 is:

an orally bioavailable inhibitor of both the serine/threonine protein kinase Aurora kinase and FMS-related tyrosine kinase 3 (FLT3; STK1; CD135; FLK2) with potential antineoplastic activity. Aurora kinase and FLT3 are overexpressed in a variety of cancers and play key roles in tumor cell proliferation.

This orally administered treatment provides another therapeutic option for those whose AML is FLT inhibitor-resistant. These patients, who often have FLT3 gene mutations, have worse outcomes and a higher risk of disease relapse. Thus, a more effective therapeutic option is urgently needed. 

Preclinical data, as well as data from a Phase 1/2a study, contributed to the Orphan Drug designation being granted. Originally, preclinical data highlighted how EP0042 overcame FLT3 inhibitor resistance. Later, in the early stages of the Phase 1/2a study, participants received 20-50mg EP0042 as a monotherapy or in conjunction with the current standards-of-care. The research found that EP0042 was safe and well-tolerated. Common side effects included dizziness, coordination issues, fatigue, diarrhea, febrile neutropenia, and peripheral edema. Additional research is now being performed to identify the maximum tolerated dose of EP0042 which may be used in further clinical studies. 

Acute Myeloid Leukemia (AML): The Basics

Normally, our body makes healthy blood cells in the bone marrow—the spongy tissue inside bones. In people with acute myeloid leukemia (AML), a blood and bone marrow cancer, the bone marrow develops abnormal platelets, red blood cells, or myeloblasts (a type of white blood cell). These abnormal cells crowd healthy blood cells out of the bone marrow, causing health difficulties. Doctors know that AML results from DNA damage to developing cells, but the exact cause of the damage is unknown. Potential risk factors include chemotherapy drugs, radiation, smoking cigarettes, being male, bleeding and genetic disorders, and certain chemical exposures. AML most often affects people aged 65 or older.

Symptoms related to this cancer may include fever, bone pain, pallor (extremely pale skin), shortness of breath, lethargy and/or fatigue, frequent or recurrent infections, easy bruising and bleeding, frequent nosebleeds or gum bleeding, night sweats, and unintentional weight loss. 

Treatment typically begins with remission induction therapy, followed by consolidation therapy. Therapies within these phases can include all-trans retinoic acid, arsenic trioxide, chemotherapy, stem cell transplantation, and investigational treatments. 

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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