Leap Therapeutics Inc. (NASDAQ:LPTX) has announced that it has received Orphan Drug Designation from The FDA for DKN-01 in the treatment of gastroesophageal and gastric junction cancer.
Leap receives Orphan Drug Designation for DKN-01
DKN-01 is a monoclonal antibody that binds to and helps in blocking the Dickkopf-1 protein (DKK) activity as a Wnt/Beta-catenin modulator signaling. It is a signaling pathway that is mostly implicated in suppression g the immune system and tumorigenesis. DKK1 plays a vital role in the signaling of tumor cells and the mediation of immune-suppression in the tumor microenvironment. The mechanism of action is through enhancement of myeloid-derived suppressor cells activity as well as downregulation of tumor cells NK ligands.
The company’s CEO and President Douglas Onsi indicated that the Orphan Drug Designation award for DKN-1 is a huge milestone for the company as well as the treatment of gastroesophageal junction and gastric cancer. Onsi indicated that the move signifies the importance of DKN-01 as a new treatment alternative to the indications. He added that they are optimistic that DKN-01 can be a potential new therapy in this area of unmet medical need.
Leap to benefit from the Orphan Drug Designation
The Orphan Drug Designation is granted by the FDA, which gives orphan status supporting the development of drugs for patients populations that are underserved or rare disorders. These are diseases or conditions affecting less than 200,000 people across the US. With the designation, Leap will have some benefits that include FDA application fees exemption, market exclusivity once it receives regulatory approval as well as tax credits for clinical trials.
Leap is evaluating DKN-01 in Phase 1/2 and second phase clinical trials for gynecologic, gastroesophageal, prostate, and hepatobiliary cancers. Already sire initiation activities have commenced for Leap’s combination DKN-01 study with BeiGene Ltd.’s tislelizumab PD-1 anti-antibody in gastroesophageal or gastric junction cancer. Leap is in a partnership with Beigenee for the development of DKN-01 in Asia, New Zealand, and Australia. The company expects to dose the first patients in the third quarter of 2020.