In the United States, Orphan Drug designation is granted to drugs or biologics designed to treat, prevent, or diagnose rare conditions. “Rare” conditions refer to those affecting under 200,000 Americans. This designation was designed to help spur drug development in the rare disease space. As a result, it also comes with benefits and incentives such as fee waivers, tax credits, and the potential for seven years of market exclusivity after the drug is approved. In a late February 2023 news release, late clinical-stage oncology company Aravive, Inc. shared that its experimental therapy batiraxcept received Orphan Drug designation for pancreatic ductal adenocarcinoma cancer, a subtype of pancreatic cancer.
Currently, pancreatic cancer comes with a poor prognosis. The five-year survival rate recently rose from 11% to 12%. Although the slightly upward trend is good to see, more effective therapies are urgently needed to further improve outcomes.
What is Batiraxcept?
So what is batiraxcept? According to Aravive, batiraxcept is:
an ultra-high affinity decoy protein that binds to GAS6 and thereby prevents AXL signaling to inhibit metastasis and tumor growth and restore sensitivity to anti-cancer agents.
Currently, researchers are evaluating batiraxcept in a Phase 1b/2 clinical study in conjunction with gemcitabine and nab-paclitaxel. The research team recently initiated the dose escalation phase of the study; more data will be available, hopefully, by later this year.
Outside of pancreatic cancer, Aravive is exploring batiraxcept as a potential therapeutic option in other oncological indications. The drug has also received Orphan Drug designation in Europe for platinum-resistant ovarian cancer, as well as Fast Track designation in the United States for clear cell renal carcinoma and platinum-resistant ovarian cancer.
About Pancreatic Cancer
As the name suggests, pancreatic cancer forms in the pancreas. This organ sits behind the lower part of the stomach. Normally, it releases enzymes to aid in food digestion, as well as hormones that aid in blood sugar management. There are multiple subtypes of pancreatic cancer, such as pancreatic adenocarcinoma and Islet cell tumors. While pancreatic adenocarcinoma is the most common form, Islet cell tumors are believed easier to treat. Many times, people with pancreatic cancer are asymptomatic until the cancer has progressed. This is unfortunate as the cancer is easiest to treat in early stages. Risk factors include smoking, diabetes, pancreatitis, a family history of pancreatic cancer or certain genetic syndromes, obesity, older age, and being male.
When symptoms do appear, they may include:
- Newly onset or worsening diabetes
- Upper abdominal pain that radiates to the back
- Bowel obstructions
- Unintentional weight loss
- Appetite loss
- Jaundice (yellowing of the skin, eyes, and mucous membranes)
- Blood clots
- Dark urine and pale stools
- Itchy skin
Current treatment options may include supportive care, surgery, chemotherapy, radiation, targeted therapy, or other therapies being explored in clinical studies.