PureTech Health plc (NASDAQ:PRTC, LSE: PRTC))))) ("PureTech" or the "Company"), a clinical-stage biotherapeutics company dedicated to discovering, developing and commercializing highly differentiated medicines for devastating diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for its wholly owned clinical therapeutic candidate, LYT-200, for the treatment of pancreatic cancer.
LYT-200 is a fully human IgG4 monoclonal antibody (mAb) targeting a foundational immunosuppressive protein, galectin-9, for the potential treatment of solid tumors, including pancreatic ductal adenocarcinoma (PDAC), colorectal cancer (CRC) and cholangiocarcinoma (CCA), that are difficult to treat and have poor survival rates. LYT-200 is currently being evaluated in the first stage of an adaptive Phase 1/2 trial, with topline results from the Phase 1 portion expected in the first half of 2022 to allow for continued dose escalation as a maximum tolerated dose has not yet been reached.
"The FDA's decision to grant orphan drug designation for LYT-200 reflects its potential as a novel anti-cancer therapy designed to block multiple immunosuppressive pathways in the tumor microenvironment," said Julie Krop, M.D., Chief Medical Officer at PureTech. "Too many pancreatic cancer patients do not respond to existing immunotherapy agents and other standard of care regimens. We are looking forward to advancing LYT-200 through the clinic in hopes of meeting this substantial need."
The FDA grants orphan drug designation to novel drug and biologic products for the treatment, diagnosis or prevention of conditions affecting fewer than 200,000 persons in the U.S. Orphan drug designation qualifies PureTech for incentives under the Orphan Drug Act, including tax credits for some clinical trials and eligibility for seven years of market exclusivity in the U.S., if the drug is approved.