In a news release from early February 2022, clinical-stage biotechnology company Eureka Therapeutics, Inc. (“Eureka”) shared that two of its therapies – ET140203 and ECT204 – received Orphan Drug designation for the treatment of hepatocellular carcinoma (HCC). Both ET140203 and ECT204 and considered ARTEMIS T cells, a type of therapy developed using Eureka’s proprietary ARTEMIS cell receptor and E-Alpha antibody discovery platforms.

About ET140203 and ECT204

To begin, what are ET140203 and ECT204? These investigational therapies were developed by Eureka for patients with HCC. In ET140203, T cells (a type of lymphocyte, or white blood cell, that plays an important role in immune response) are collected directly from the patient. After being specially engineered to target antibodies present on HCC-related cells, these engineered cells are infused back into the body. Ultimately, this helps the treatment to identify and attack cancer cells. Currently, researchers are evaluating ET140203 within the Phase 1/2 ARYA-1 clinical trial and the Phase 1/2 ARYA-2 clinical trial. The latter focuses more on pediatric patients. 

ECT204, on the other hand, specifically targets GPC3. GPC3 is an antigen which is expressed or found on over 70% of cells associated with hepatocellular carcinoma. It is currently being evaluated within the Phase 1/2 ARYA-3 trial. 

Recently, the FDA granted Orphan Drug designation to both of these therapies. Orphan Drug designation is granted to drugs or biologics intending to treat, prevent, or diagnose rare diseases or conditions; these are defined as those affecting under 200,000 Americans. As a result of this designation, Eureka also earns a variety of benefits. These include fee waivers, tax credits, increased regulatory assistance, and 7 years market exclusivity upon approval. 

What is Hepatocellular Carcinoma (HCC)?

Although rare, hepatocellular carcinoma (HCC) is the most common form of primary liver cancer. Primary liver cancer means the cancer formed in the liver, as opposed to secondary liver cancer, which spreads from other organs. While doctors are not exactly sure what causes HCC, there are a number of associated risk factors. These include obesity, diabetes, pre-existing liver conditions, hepatitis B or C, and heavy alcohol use. In early HCC stages, many patients may be asymptomatic. However, as the cancer grows and/or spreads, symptoms can include:

• Appetite loss
• Unintended weight loss
• Weakness, fatigue, or general malaise
• Nausea and vomiting
• Abdominal bloating or inflammation
• Upper abdominal pain, lumps, or heaviness
• Dark urine and pale, chalky bowel movements
• Fever
• Jaundice (yellowing of the skin, eyes, and mucous membranes)