BDC-1001 Granted Orphan Drug Status for Gastric Cancer


Historically, it has been challenging to mobilize drug development and research within the rare disease sphere. Given the smaller community sizes, as well as a general lack of profitability, some companies have stayed away from this sphere. However, it is necessary to engage in research and development to improve the lives for people with rare diseases. The Orphan Drug designation was created to stimulate drug development. Granted to drugs or biologics intended for rare conditions (affecting fewer than 200,000 people nationwide), Orphan Drug designation comes with benefits such as tax credits, fee waivers, increased regulatory and developmental assistance, and 7 years of market exclusivity upon the drug’s approval. 

In a news release on Benzinga, clinical-stage biopharmaceutical company Bolt Biotherapeutics shared that its therapy BDC-1001 was granted Orphan Drug designation for the treatment of gastric cancer. This indication covers multiple types and subtypes of gastric cancer, such as gastroesophageal junction carcinoma, a rare form of esophageal cancer. 

Bolt Biotherapeutics developed BDC-1001 using its unique and proprietary Boltbody™ Immune-Stimulating Antibody Conjugate (ISAC) platform. This platform prompts the reprogramming of the tumor microenvironment to improve the anti-cancer immune response, more effectively fighting tumors. BDC-1001 is an immune-stimulating antibody conjugate that consists of a HER2-targeting biosimilar trastuzumab with a proprietary TLR7/8 agonist. As Bolt Biotherapeutics explains

BDC-1001 stimulates anti-tumor activity with a three-pronged approach: direct tumor cell killing by trastuzumab-mediated mechanisms, localized phagocytosis, and elimination of HER2-expressing tumor cells by activated myeloid APCs, and durable immunity manifested by T cells reactive to tumor-associated antigens or neoantigens.

Outside of gastric cancer, BDC-1001 is also being explored as a potential therapeutic option for colorectal cancer, breast cancer, and endometrial cancer. 

Learn About Gastric Cancer

Also known as stomach cancer, gastric cancer first forms in the cells that line the stomach. The stomach has three tissue layers: mucosal (inner), muscularis (middle), and serosal (outer). Gastric cancer begins in the mucosal layer before spreading outwards. There are multiple types of gastric cancer, such as adenocarcinoma, lymphoma, squamous cell carcinoma, leiomyosarcoma, carcinoid tumors, and stromal tumors. 

While gastric cancer can occur in people of varying ages, it is most commonly seen in individuals older than 65. Men are also more likely to develop this cancer than women. Additional risk factors include a high sodium but low fruit/vegetable diet, gastrointestinal reflux disease, H. pylori infection, obesity, smoking, or having a family history of stomach cancer. 

People affected by this cancer may not show symptoms until the cancer has progressed. When symptoms appear, these may include:

  • Jaundice (yellowing of the skin, eyes, and mucous membranes)
  • Feeling full or bloated after eating, even if only eating small portions
  • Stomach pain 
  • Chronic and severe indigestion
  • Mild nausea
  • Appetite loss
  • Dysphagia (difficulty swallowing)
  • Ascites (the accumulation of fluid in the abdomen) 
  • Unintentional weight loss
  • Heartburn 
  • Bloody stool 
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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