TARRYTOWN, N.Y., December 19, 2024 – Sapience Therapeutics, Inc., a clinical-stage biotechnology company focused on the discovery and development of peptide therapeutics to address oncogenic and immune dysregulation that drive cancer, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ST316 for the treatment of familial adenomatous polyposis (FAP).FAP is a pre-malignant rare genetic condition that causes the formation of hundreds to thousands of polyps throughout the colon, typically starting in adolescence.

纽约州塔里敦，2024年12月19日–Sapience Therapeutics，Inc.，一家临床阶段生物技术公司，专注于发现和开发肽疗法，以解决致癌和免疫失调导致癌症的问题，今天宣布美国食品和药物管理局（FDA）已授予ST316孤儿药名称（ODD），用于治疗家族性腺瘤性息肉病（FAP）。FAP是一种恶变前罕见的遗传病，通常在青春期开始在整个结肠中形成数百至数千个息肉。

With no approved therapies for FAP, without surgical intervention it will invariably lead to the development of colorectal cancer (CRC) by the age of 40. Development of FAP is considered an initiation event in the development of CRC, which is expected to be diagnosed in 150,000 patients in the United States next year.ST316 is a first-in-class antagonist of β-catenin and its co-activator, BCL9, currently in a Phase 2 study for the treatment of CRC.

由于没有批准的FAP治疗方法，如果不进行手术干预，它将不可避免地导致40岁时结直肠癌（CRC）的发展。。ST316是β-连环蛋白及其共激活剂BCL9的一流拮抗剂，目前正在进行CRC治疗的2期研究。

ST316 is designed to selectively shut down the Wnt/β-catenin signaling pathway, which is a key driver of FAP and more than 80% of CRCs, underscoring the importance of this pathway for therapeutic intervention.“We are thrilled that the FDA has granted Orphan Drug Designation to ST316 for the treatment of FAP.

。“我们很高兴FDA批准ST316用于治疗FAP的孤儿药。

This award is an important regulatory milestone that advances our approach to targeting genetic alterations in the Wnt/β-catenin signaling pathway, which are drivers of both pre-malignant and malignant disease,” commented Dr. Abi Vainstein-Haras, Sapience’s Chief Medical Officer. “Other than high morbidity surgery and intensive colonoscopy surveillance, patients with FAP have no available treatment options to prevent their disease from progressing to CRC, the.

。“除了高发病率的手术和强化的结肠镜检查外，FAP患者没有可用的治疗选择来防止他们的疾病进展为CRC。