Clinical milestone validates the company's platform for drugging 'undruggable' targets

临床里程碑验证了公司针对“不可成药”靶点进行药物开发的平台

BEIJING

北京

,

，

Sept. 1, 2026

2026年9月1日

/PRNewswire/ -- Pyrotech Therapeutics ('Pyrotech' or the 'Company'), a clinical-stage biopharmaceutical company pioneering innate immune modulation and pyroptosis-targeted therapies,

/PRNewswire/ -- Pyrotech Therapeutics（“Pyrotech”或“公司”）是一家处于临床阶段的生物制药公司，致力于开创先天免疫调节和靶向细胞焦亡的疗法，

announced

宣布

that the first healthy volunteer has been dosed in the Phase 1 clinical study of PTT-121,

PTT-121 的 I 期临床试验中，首位健康志愿者已接受给药，

its novel gasdermin D (GSDMD) inhibitor

其新型气体死亡蛋白D（GSDMD）抑制剂

. This marks a pivotal milestone for PTT-121 as the world's first selective GSDMD inhibitor to advance into human clinical testing, breaking the previous characterization of GSDMD as an 'undruggable' target.

这标志着PTT-121的一个重要里程碑，作为全球首个进入人体临床试验的选择性GSDMD抑制剂，打破了此前将GSDMD视为“不可成药”靶点的认知。

PTT-121 is being developed initially for the treatment of septic shock, a life-threatening condition driven by dysregulated inflammatory responses and widespread pyroptotic cell death. The compound was granted clinical trial approval by China's National Medical Products Administration (NMPA) on August 4, 2026, and is now being evaluated in a first-in-human study at Peking University Third Hospital..

PTT-121 最初是为治疗脓毒性休克而开发的，这是一种由失调的炎症反应和广泛的细胞焦亡导致的危及生命的状况。该化合物于 2026 年 8 月 4 日获得中国国家药品监督管理局（NMPA）的临床试验批准，目前在北京大学第三医院进行首次人体研究评估。

Dr. Feng Shao, co-founder of Pyrotech Therapeutics, said,

Pyrotech Therapeutics 联合创始人冯少博士表示，

'

'

The

这

first-in-human dosing of PTT-121 represents the culmination of years of relentless scientific pursuit by our team to translate groundbreaking basic research into a transformative therapy for patients. GSDMD has long been regarded as the holy grail of pyroptosis drug development — a target of extraordinary therapeutic potential that defied conventional medicinal chemistry approaches.

PTT-121的首次人体给药标志着我们团队多年来不懈科学追求的巅峰，旨在将突破性的基础研究转化为为患者带来变革性疗法。GSDMD长期以来被视为细胞焦亡药物开发的“圣杯”——一个具有非凡治疗潜力却挑战传统药物化学方法的靶点。

By achieving this milestone, we are not only validating our proprietary drug discovery platform but also opening an entirely new therapeutic paradigm for septic shock and a broad spectrum of inflammatory diseases.'.

通过实现这一里程碑，我们不仅验证了我们的专有药物发现平台，还为脓毒性休克和广泛的炎症性疾病开辟了一种全新的治疗范式。

The Phase 1 study is a single-center, randomized, double-blind, placebo-controlled study designed to evaluate the safety, tolerability and pharmacokinetics of intravenously administered PTT-121 following single and multiple ascending doses (SAD/MAD) in healthy Chinese adults. The study plans to enroll approximately 84 healthy volunteers aged 18 to 55 years.

这项1期研究是一项单中心、随机、双盲、安慰剂对照研究，旨在评估健康中国成年人在单次和多次递增剂量（SAD/MAD）下静脉注射PTT-121的安全性、耐受性和药代动力学。该研究计划招募约84名年龄在18至55岁之间的健康志愿者。

The primary endpoint is the incidence of treatment-emergent adverse events (TEAEs); secondary endpoints include comprehensive pharmacokinetic and biomarker assessments..

主要终点为治疗期出现的不良事件（TEAEs）的发生率；次要终点包括全面的药代动力学和生物标志物评估。

Dr. Tianjing Deng, Chief Executive Officer and co-founder of Pyrotech Therapeutics, said,

Pyrotech Therapeutics 首席执行官兼联合创始人邓天敬博士表示，

'Septic shock remains one of the most devastating and poorly managed conditions in modern medicine, claiming millions of lives worldwide each year despite decades of research. The current standard of care offers only supportive management, with no approved therapy that directly targets the underlying inflammatory cascade.

脓毒性休克仍然是现代医学中最具破坏性且管理不善的疾病之一，尽管经过数十年的研究，每年仍导致全球数百万人死亡。目前的护理标准仅提供支持性治疗，尚无直接针对潜在炎症级联反应的获批疗法。

PTT-121's unique mechanism — directly inhibiting GSDMD pore formation, blocking the release of pro-inflammatory cytokines and subsequent pyroptotic cell death — represents a fundamentally different approach. We are eager to generate the first human data and build on the compelling preclinical evidence that has positioned PTT-121 as a potential best-in-class therapeutic.'.

PTT-121的独特机制——直接抑制GSDMD孔道形成，阻断促炎细胞因子的释放及随后的细胞焦亡——代表了一种根本不同的治疗策略。我们热切期待获得首批人体数据，并在有力的临床前证据基础上进一步推进，这些证据已将PTT-121定位为潜在的同类最佳疗法。

About GSDMD and Pyroptosis

关于GSDMD与细胞焦亡

Pyroptosis is a highly pro-inflammatory form of programmed cell death mediated by the gasdermin family of proteins. GSDMD serves as the central executioner of both the canonical and non-canonical inflammasome/pyroptosis pathway: upon activation by inflammatory caspases, GSDMD is cleaved to release its N-terminal domain, which oligomerizes and forms pores in cell membranes.

细胞焦亡是一种由gasdermin蛋白家族介导的高度促炎性的程序性细胞死亡形式。GSDMD作为经典和非经典炎症小体/细胞焦亡通路的核心执行者：在被炎症性caspase激活后，GSDMD被切割并释放其N端结构域，该结构域寡聚化并在细胞膜上形成孔洞。

These pores can produce cellular energy failure, the release of inflammatory cytokines such as IL-1β and IL-18, and inflammatory cell death. Excessive and uncontrolled GSDMD-mediated pyroptosis is a key driver of tissue damage, organ failure and mortality in septic shock, as well as a growing list of autoimmune, neurodegenerative and inflammatory disorders..

这些孔道可导致细胞能量衰竭、炎症细胞因子（如IL-1β和IL-18）的释放，以及炎症性细胞死亡。过度且不受控制的GSDMD介导的焦亡是脓毒性休克中组织损伤、器官衰竭和死亡的关键驱动因素，也在越来越多的自身免疫性疾病、神经退行性疾病和炎症性疾病中发挥重要作用。

Despite its well-validated role in many diseases, GSDMD has been considered 'undruggable' due to the challenges of selectively targeting its pore-forming activity without disrupting essential cellular functions. PTT-121 overcomes this barrier through a novel mechanism of action, demonstrating high selectivity and potency against GSDMD in preclinical studies.

尽管GSDMD在许多疾病中的作用已得到充分验证，但由于难以在不破坏关键细胞功能的情况下选择性靶向其成孔活性，它一直被视为“不可成药”靶点。PTT-121通过一种新颖的作用机制克服了这一障碍，在临床前研究中展现出对GSDMD的高度选择性和强效抑制作用。

In addition to PTT-121, Pyrotech has developed an orally bioavailable GSDMD inhibitor with potential application to many chronic inflammatory diseases that result from excessive secretion of IL-1β/IL-18, such as gout and hidradenitis suppurativa. These GSDMD inhibitors work downstream of NLRP3 and other inflammasomes, so may have widespread clinical utility..

除PTT-121外，Pyrotech还开发了一种具有口服生物利用度的GSDMD抑制剂，该抑制剂在多种由IL-1β/IL-18过度分泌引起的慢性炎症性疾病（如痛风和化脓性汗腺炎）中具有潜在应用价值。这些GSDMD抑制剂作用于NLRP3及其他炎症小体的下游，因此可能具有广泛的临床应用前景。

About PTT-121

关于PTT-121

PTT-121 is a first-in-class, intravenously administered inhibitor of GSDMD, discovered and developed internally by Pyrotech Therapeutics

PTT-121 是一种首创的静脉注射用 GSDMD 抑制剂，由 Pyrotech Therapeutics 内部发现并开发。

. It is the first selective GSDMD inhibitor to receive regulatory approval for clinical evaluation. Repurposed compounds (e.g., disulfiram) that may inhibit GSDMD are poorly selective and have different molecular targets. PTT-121's initial clinical indication is septic shock, with potential expansion into a broad range of inflammatory and autoimmune conditions..

这是首个获得监管机构批准进入临床评估的选择性GSDMD抑制剂。可能抑制GSDMD的再利用药物（例如双硫仑）选择性较差，且作用分子靶点不同。PTT-121的初始临床适应症为脓毒性休克，未来有望扩展至广泛的炎症性和自身免疫性疾病领域。

About Pyrotech Therapeutics

关于 Pyrotech Therapeutics

Pyrotech Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing revolutionary therapies for inflammation and cancer by precisely modulating innate immunity and the pyroptosis pathway. Founded in 2020 based on breakthrough scientific discoveries from Dr. Shao's lab, Pyrotech has built a technology platform that enables the selective modulation of key proteins in the innate immune and pyroptosis pathways..

Pyrotech Therapeutics 是一家临床阶段的生物制药公司，致力于通过精确调控先天免疫和细胞焦亡通路，开发针对炎症和癌症的革命性疗法。Pyrotech 基于邵博士实验室的突破性科学发现于 2020 年创立，已构建起一个能够选择性调控先天免疫和细胞焦亡通路中关键蛋白质的技术平台。

The Company's pipeline comprises more than ten first-in-class programs targeting novel mechanisms; these span autoimmune diseases, oncology, neurodegenerative disorders and septic shock. Six programs have advanced into clinical development, with several drug candidates soon entering Phase 2 studies..

该公司的产品管线包含十余个针对全新作用机制的首创类项目，涵盖自身免疫性疾病、肿瘤学、神经退行性疾病和脓毒性休克等领域。六个项目已进入临床开发阶段，多个候选药物即将进入二期临床试验。

For more information, please visit

更多信息，请访问

www.pyrotech.com

www.pyrotech.com

.

。

Forward-Looking Statements

前瞻性陈述

This press release contains forward-looking statements regarding Pyrotech Therapeutics' business, including statements regarding the clinical development and therapeutic potential of PTT-121, the expected timeline and outcomes of clinical trials, and the Company's strategic plans. These statements are based on current expectations and assumptions and are subject to risks and uncertainties that could cause actual results to differ materially.

本新闻稿包含有关Pyrotech Therapeutics业务的前瞻性陈述，包括关于PTT-121的临床开发和治疗潜力、临床试验的预期时间表和结果以及公司战略计划的陈述。这些陈述基于当前的预期和假设，并受到风险和不确定性的影响，可能导致实际结果与预期存在重大差异。

Factors that could cause actual results to differ include, but are not limited to, risks associated with the clinical development process, including the possibility that clinical trials may not demonstrate safety or efficacy, delays in regulatory approvals, the availability of financing, and competition from other therapeutic approaches.

可能导致实际结果出现差异的因素包括但不限于与临床开发过程相关的风险，包括临床试验可能无法证明安全性或有效性、监管审批延迟、融资的可获得性以及来自其他治疗方法的竞争。

Pyrotech undertakes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise, except as required by law..

Pyrotech 不承担更新或修订任何前瞻性陈述的义务，无论是因为新信息、未来事件还是其他原因，除非法律另有要求。

SOURCE Pyrotech Therapeutics

来源：Pyrotech Therapeutics

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