Marina Biotech Receives Orphan Drug Designation for CEQ508

Strategic Insights -

Marina Biotech, Inc., a leading RNAi-based drug discovery and development company, announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company's drug candidate CEQ508 for the treatment of Familial Adenomatous Polyposis (FAP). This designation provides Marina Biotech with significant regulatory and financial incentives to rapidly move CEQ508 to market, including seven years of marketing exclusivity and the opportunity to apply for grant funding from the U.S. government.

Key Takeaways:

  • CEQ508 is the first drug in a novel class of therapeutic agents utilizing the transkingdom RNA interference (tkRNAi) platform and the first orally administered RNAi-based therapeutic.
  • CEQ508 targets the mRNA of beta-catenin, which is known to be dysregulated in classical FAP, and reduces the levels of beta-catenin protein in the epithelial cells of the small and large intestine.
  • The orphan drug designation entitles Marina Biotech to seven years of marketing exclusivity for CEQ508 for the treatment of FAP upon regulatory approval.
  • The U.S. Orphan Drug Act of 1983 was created to promote the development of new drug therapies for the treatment of diseases that affect fewer than 200,000 individuals in the United States.
  • CEQ508 is being developed as an orally administered treatment and will be administered daily in an oral suspension for 28 consecutive days.
  • The trial plan calls for a stable-dose phase in which additional patients will receive the highest safe dose after completion of the dose escalation phase.
  • Approximately 1 in 10,000 persons worldwide have FAP, a hereditary condition caused by mutations in the Adenomatous Polyposis Coli (APC) gene.
  • FAP patients are at an increased risk of adenocarcinoma and other gastrointestinal cancers, particularly colon cancer, due to the formation of numerous polyps throughout the large intestine.

Statistics:

  • 1 in 10,000 persons worldwide have Familial Adenomatous Polyposis (FAP) (Source: Marina Biotech, Inc.).
  • 95% of individuals with FAP develop polyps by age 35 (Source: Marina Biotech, Inc.).
  • 100% of FAP patients are at risk of adenocarcinoma and other gastrointestinal cancers due to the formation of numerous polyps (Source: Marina Biotech, Inc.).
  • CEQ508 is the first orally administered RNAi-based therapeutic for the treatment of FAP (Source: Marina Biotech, Inc.).
  • 7 years of marketing exclusivity for CEQ508 upon regulatory approval (Source: U.S. Orphan Drug Act of 1983).

Sources:

  • Marina Biotech, Inc.
  • U.S. Food and Drug Administration (FDA)
  • U.S. Orphan Drug Act of 1983