Adcentrx Therapeutics has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for its investigational treatment ADRX‑0405.

Adcentrx Therapeutics 公司的在研药物 ADRX‑0405 已获得美国食品药品监督管理局 (FDA) 的孤儿药资格认定。

ADRX-0405 is a next-generation ADC targeting six-transmembrane epithelial antigen of the prostate 1 (STEAP1), a protein commonly overexpressed in prostate and some other cancers. Normal healthy tissues express the protein at limited levels, allowing for more targeted treatment. The therapy consists of a humanised IgG1 antibody linked to a novel topoisomerase inhibitor payload.

ADRX-0405 是一种下一代 ADC，靶向前列腺六次跨膜上皮抗原 1（STEAP1），这种蛋白在前列腺癌和某些其他癌症中常过度表达。正常健康组织中该蛋白的表达水平有限，从而允许更精准的治疗。该疗法由一种人源化 IgG1 抗体与一种新型拓扑异构酶抑制剂载荷连接而成。

This linkage is enabled through Adcentrx’s proprietary i-Conjugation® technology, which uses a cleavable linker and stable conjugation method to improve drug delivery..

这种连接通过Adcentrx专有的i-Conjugation®技术实现，该技术使用可切割的连接子和稳定的偶联方法来改善药物递送。

The designation applies to the treatment of gastric cancer, a rare and often late-diagnosed disease.

该指定适用于治疗胃癌，这是一种罕见且常常晚期才被诊断出的疾病。

ADRX-0405 is a STEAP1-targeting ADC currently under evaluation in the Phase 1a portion of an ongoing Phase 1a/b clinical trial (NCT06710379). The trial includes patients with advanced solid tumours such as metastatic castration-resistant prostate cancer, non-small cell lung cancer, and gastric cancer.

ADRX-0405是一种靶向STEAP1的抗体药物偶联物（ADC），目前正处于Ia/b期临床试验（NCT06710379）的Ia阶段评估中。该试验包括晚期实体瘤患者，如转移性去势抵抗性前列腺癌、非小细胞肺癌和胃癌。

Although STEAP1 is commonly linked to prostate cancer, the target is also present in notable levels in gastric cancer tissues, making it a viable focus for clinical development..

尽管STEAP1通常与前列腺癌相关，但在胃癌组织中该靶点也有显著水平的存在，这使其成为临床开发的可行焦点。

The FDA’s orphan drug designation is granted to therapies intended for the treatment of rare diseases—defined in the United States as those affecting fewer than 200,000 people. Gastric cancer meets this criterion, with the American Cancer Society estimating approximately 30,300 new cases expected in 2025.

美国 FDA 的孤儿药认定是授予用于治疗罕见疾病的疗法，在美国，罕见疾病被定义为患者少于 20 万人的疾病。胃癌符合这一标准，美国癌症协会估计 2025 年预计新增病例约为 30,300 例。

The designation offers various incentives to developers, including eligibility for research grants, tax credits for clinical trials, exemption from certain FDA fees, and potential seven-year market exclusivity upon regulatory approval..

该指定为开发者提供了各种激励措施，包括有资格获得研究资助、临床试验的税收抵免、某些FDA费用的豁免，以及在监管批准后可能获得七年的市场独占权。

The ADC is designed with a high drug-antibody ratio (DAR 8), allowing for enhanced delivery of the therapeutic payload to tumour cells. Preclinical studies have shown encouraging results, including favourable pharmacokinetics, safety, and efficacy across various tumour models. ADRX-0405 is now undergoing clinical evaluation to assess its safety, tolerability, and anti-tumour activity in multiple cancer types, including gastric cancer..

该ADC设计具有较高的药物抗体比率（DAR 8），能够增强治疗载荷向肿瘤细胞的递送。 临床前研究显示出令人鼓舞的结果，包括在多种肿瘤模型中具有良好药代动力学、安全性和有效性。 ADRX-0405目前正在进行临床评估，以测试其在包括胃癌在内的多种癌症类型中的安全性、耐受性和抗肿瘤活性。

This development marks a step forward in expanding treatment options for patients with rare and difficult-to-treat cancers.

这一进展标志着在扩大罕见且难以治疗的癌症患者的治疗选择方面迈出了一步。