Developed by Ellodi, APT-1011 is a novel tablet which delivers treatment directly to the esophageal mucosa, offering a more targeted treatment option. The therapy is currently being evaluated in Phase 3 clinical trials. In prior studies, APT-1011 showed promise for reducing eosinophil (a type of white blood cell) counts within the esophagus. In addition to Fast Track designation, APT-1011 received Orphan Drug designation within the United States and Europe.

The FDA’s Fast Track designation expedites the development and review of treatment options for patients with rare or serious conditions, or patients with unmet medical needs. According to the FDA:

Any drug being developed to treat or prevent a condition with no current therapy obviously is directed at an unmet need. If there are available therapies, a fast track drug must show some advantage over available therapy, such as:

• Showing superior effectiveness, effect on serious outcomes or improved effect on serious outcomes; avoiding serious side effects of an available therapy; improving the diagnosis of a serious condition where early diagnosis results in an improved outcome; [or] decreasing a clinical significant toxicity of an available therapy that is common and causes discontinuation of treatment.

In this case, the FDA’s decision hinged on the fact that EoE has few effective or available treatment options. Thus, APT-1011 could greatly improve the quality of life (QOL) for patients with this condition. The FDA also used data from the Phase 2b FLUTE trial. Ultimately, this trial showed that APT-1011 offered symptom improvement and maintenance over a period of time.

Eosinophilic Esophagitis (EoE)

Eosinophilic esophagitis (EoE) is a chronic allergic inflammatory and digestive system disorder that affects the esophagus. Normally, eosinophils, a type of white blood cell, play a role in immune response and fighting infections. EoE is characterized by a high number of eosinophils in the esophagus, which is a hallmark of allergic conditions. Normally, there are no eosinophils in the esophagus at all. Generally, EoE is thought to be caused by environmental allergens or immune sensitivity to certain foods. However, over-expression of the eotaxin-3 gene may also cause EoE. Symptoms include:

• Difficulty swallowing
• Nausea and vomiting
• Stomach pain
• Poor appetite and/or appetite loss
• Food becoming stuck in the throat
• Unintended weight loss
• Malnutrition
• Esophageal fibrosis
• Failure to thrive (in infants)
• Asthma
• Eczema
• Other allergies