沃斯堡Nacuity三期试验完成首名患者给药：NPI-001评估延缓Usher综合征相关视网膜色素变性，试验编号NCT07710196
得克萨斯州塔兰特县沃斯堡市消息：总部位于沃斯堡的临床阶段生物制药公司Nacuity Pharmaceuticals 10月28日宣布，其在研药物NPI-001的全球确证性三期临床试验SLO-RP Preserve已完成首名患者给药。该药物用于治疗与Usher综合征相关的视网膜色素变性，位于达拉斯的西南视网膜基金会遗传性视网膜变性诊所及其他美国和澳大利亚试验点正在积极招募更多患者。
与Usher综合征相关的视网膜色素变性是一种罕见的进行性遗传性视网膜疾病，会导致不可逆的视力丧失，目前尚无获批的能够延缓感光细胞退化的疗法。这项为期24个月的随机双盲试验计划招募最多80名成年参与者，参与者将每天两次口服NPI-001或安慰剂，并在约两年内完成九次临床访视，以评估该药物能否通过减少感光细胞丢失来延缓疾病进展。试验编号为NCT07710196。
公司首席临床官Jami Kern表示，二期试验的积极结果增强了团队推进这一重要临床项目的动力。
来源：GSM·美国
Fort Worth-Based Nacuity Doses First Patient in Phase 3 Trial of NPI-001 for Usher Syndrome-Associated Retinitis Pigmentosa (NCT07710196)
FORT WORTH, Tarrant County, Texas — Nacuity Pharmaceuticals, a clinical-stage biopharmaceutical company headquartered in Fort Worth, announced on October 28 that the first patient has been dosed in SLO-RP Preserve, its global confirmatory Phase 3 trial of NPI-001 for the treatment of retinitis pigmentosa associated with Usher syndrome. The Retina Foundation of the Southwest's inherited retinal degeneration clinic in Dallas and other sites in the United States and Australia are actively enrolling additional patients.
Retinitis pigmentosa associated with Usher syndrome is a rare, progressive inherited retinal disease that causes irreversible vision loss, and there is currently no approved therapy that slows photoreceptor degeneration. The 24-month randomized, double-blind trial plans to enroll up to 80 adult participants, who will take NPI-001 or placebo orally twice daily and complete nine clinic visits over about two years, to evaluate whether the drug can slow disease progression by reducing photoreceptor cell loss. The trial is registered as NCT07710196.
Chief Clinical Officer Jami Kern said the positive results from the Phase 2 trial strengthened the team's motivation to advance this important clinical program.
Source: GSM News
来源：GSM·美国（原文链接）
图片来源：Nacuity Pharmaceuticals 公司 logo，取自该公司官网
