PTT-121

PTT-121 is a first-in-class small molecule inhibitor of Gasdermin D (GSDMD) developed by Pyrotech Therapeutics (Beijing Yanming Biological Technology). GSDMD is a key executioner of pyroptosis, a form of programmed inflammatory cell death. Upon activation by inflammatory caspases, GSDMD undergoes cleavage, and its N-terminal domain oligomerizes to form large pores in the plasma membrane. These pores facilitate the release of pro-inflammatory cytokines, such as IL-1β and IL-18, and damage-associated molecular patterns (DAMPs), which drive systemic inflammation. By inhibiting GSDMD pore formation, PTT-121 aims to block the inflammatory cascade at its source, offering a novel therapeutic strategy for severe inflammatory conditions including sepsis and septic shock. The drug is currently undergoing Phase I clinical evaluation to assess its safety, tolerability, and pharmacokinetics.