In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to prevent, diagnose, or treat rare conditions. These are conditions affecting fewer than 200,000 Americans. Along with this designation comes a set of incentives, designed to help spur drug developers to create more therapies for rare conditions. Incentives include fee waivers, tax credits, and seven years of market exclusivity upon approval. According to a news release from biopharmaceutical company Antengene Corporation Limited (“Antengene”), the FDA recently granted Orphan Drug designation to ATG-101 for pancreatic cancer. 

ATG-101 is a novel PD-L1/4-1BB bispecific antibody. OncLive explains that ATG-101:

dually targets PD-L1 and 4-1BB [and] activates 4-1BB-positive T cells and exhausted T cells in a PD-L1-crosslinking dependent manner by which ‘cold’ tumors are rendered ‘hot.’ With this mechanism of action, the therapy is hypothesized to treat tumors without eliciting on-target, off-tumor liver toxicity.

Preclinical studies found that ATG-101 was safe, effective, and well-tolerated in animal models. Additionally, the treatment increased anti-tumor activity in these models, even in those with treatment-resistant tumors. 

Currently, no therapies of this kind are approved for pancreatic cancer throughout the world. 

What is Pancreatic Cancer?

Pancreatic cancer is a cancer which forms in the pancreas, an organ which aids in food digestion and blood sugar management (through releasing enzymes and hormones). A majority of pancreatic cancers are adenocarcinomas; these form in exocrine cells that line pancreatic ducts. There are also pancreatic neuroendocrine tumors, a rare form which occurs in neuroendocrine cells. 

Risk factors for developing pancreatic cancer include having a family history of this cancer, smoking cigarettes, obesity, being male, being older in age (55+), type 2 diabetes, and chronic pancreatitis. Unfortunately, pancreatic cancer is difficult to detect in early stages. As a result, the prognosis is often poor. This cancer is one of the leading causes of death within the United States. Symptoms can (but do not always) include:

• Newly onset or worsening diabetes
• Jaundice (yellowing of the skin, eyes, and mucous membranes)
• Appetite loss
• Blood clots
• Nausea and vomiting
• Bloating or a feeling of fullness
• Unintended weight loss
• Abdominal pain that radiates to the back
• Fatigue
• Bowel obstructions
• Dark urine or light-colored stool