Beijing, China August 4, 2026, Beijing Pyrotech Therapeutics ("Pyrotech" or "the Company") today announced that China's National Medical Products Administration (NMPA) has granted Clinical Trial Authorization (CTA) for PTT-121, the Company's first-in-class small-molecule pyroptosis inhibitor, for the treatment of sepsis and septic shock. Pyrotech will initiate clinical studies in China in the coming months.
PTT-121 is the world's first selective Gasdermin D (GSDMD) inhibitor to receive clinical authorization, a milestone that overturns the long-held view of GSDMD as an "undruggable" target and marks a landmark advance in the development of pyroptosis-targeted therapeutics.
A New Therapeutic Target for Pyroptosis
Pyroptosis is a form of programmed, inflammatory cell death executed by the Gasdermin family of proteins. In 2015, a research team led by Dr. Feng Shao at the National Institute of Biological Sciences (NIBS), Beijing, published a landmark paper in Nature identifying GSDMD as the direct executioner of pyroptosis. The discovery redefined the molecular mechanism of the pathway and opened an entirely new avenue for developing therapies against inflammatory diseases.
PTT-121's initial indication, sepsis, is a life-threatening organ dysfunction caused by a dysregulated host response to infection. Disease progression is often extremely rapid, with patients developing systemic cytokine storm, septic shock, and multi-organ failure within hours, making sepsis one of the most critical conditions managed in intensive care units (ICUs). A growing body of clinical evidence points to GSDMD-mediated pyroptosis as a central driver of both the onset and progression of sepsis.
Overcoming an "Undruggable" Target
GSDMD is a pore-forming membrane protein with no enzymatic activity and no conventional small-molecule binding pocket properties that have led to its widespread classification as undruggable, with no prior precedent for successful drug development against this protein class.
Building on foundational discoveries from Dr. Feng Shao's laboratory together with its proprietary drug discovery platform, Pyrotech has developed a highly selective, highly potent, intravenously administered small-molecule GSDMD inhibitor. In preclinical studies, PTT-121 demonstrated robust efficacy across multiple sepsis models effectively suppressing cytokine storm and significantly reducing mortality in animal studies.
From Fundamental Discovery to Clinical Translation
The CTA for PTT-121 marks the successful translation of groundbreaking, China-originated discoveries in innate immunity and pyroptosis biology into a clinical-stage therapy with direct application in critically ill ICU patients. The program has the potential to offer an entirely new treatment strategy for sepsis, addressing a major unmet medical need worldwide.
Over the coming years, Pyrotech plans to accelerate clinical development of this first-in-class GSDMD inhibitor, with the goal of completing Phase II proof-of-concept studies in sepsis and related indications. In parallel, the Company's next-generation oral GSDMD inhibitor, designed for chronic inflammatory diseases, is expected to enter clinical development in 2027.
Continued progress in the GSDMD inhibitor program is expected to bring transformative advances to the treatment of pyroptosis-associated diseases and stands as a landmark example of China's ability to translate pioneering basic-science discoveries into innovative medicines through close integration of academia and industry.
