Elevidys-treated boys aged 4-7 years with Duchenne showed an increase on the North Star Ambulatory Assessment (NSAA), a measure of motor function, compared to placebo at 52 weeks but the primary endpoint was not metFor all key pre-specified secondary functional endpoints, time to rise and 10-metre walk test across age groups, clinically meaningful and statistically significant treatment benefits were observedNo new safety signals observed, reinforcing the favourable and manageable safety profile observed with Elevidys to dateFurther evaluation of data is ongoing and Roche will discuss the path forward with health authorities Basel, 30 October 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today topline results from the global, randomised, double-blind Phase 3 EMBARK study of Elevidys™ (delandistrogene moxeparvovec) in ambulatory boys (those who can walk) with Duchenne muscular dystrophy aged 4-7 years.

使用Duchenne治疗的4-7岁Elevidys治疗男孩在52周时与安慰剂相比，North Star动态评估（NSAA）（一种运动功能的测量）有所增加，但主要终点并未满足所有关键的预先指定的次要功能终点，上升时间和各年龄组的10米步行测试，观察到具有临床意义和统计学意义的治疗益处没有观察到新的安全性信号，加强了Elevidys观察到的有利和可管理的安全性，迄今为止正在进一步评估数据，罗氏将与巴塞尔卫生当局讨论前进的道路，2023年10月30日-罗氏（六：RO，ROG；OTCQX:RHHBY）今天宣布全球，随机，双盲3期EMBARK Elevidys研究的顶级成果™ （delandistrogene moxeparvovec）在4-7岁的杜兴氏肌营养不良症的非卧床男孩（可以走路的男孩）中。

In the study, Elevidys-treated patients improved 2.6 points on their NSAA total score 52 weeks after treatment, compared to 1.9 points in placebo-treated patients (0.65; n=125; P=0.24). In all pre-specified, timed functional key secondary endpoints, time to rise from floor and 10 metre walk test, clinically meaningful and statistically significant improvements were observed.

在该研究中，Elevidys治疗的患者在治疗后52周的NSAA总分改善了2.6分，而安慰剂治疗的患者为1.9分（0.65；n=125；P=0.24）。在所有预先指定的定时功能关键次要终点，从地板上升的时间和10米步行测试，观察到临床意义和统计学显着的改善。

Both endpoints are prognostic factors for disease progression and loss of ability to walk. Additionally, a clinically meaningful and statistically significant improvement was also observed for the pre-specified secondary endpoint stride velocity 95th centile. This novel digital endpoint, qualified by the European Medicines Agency (EMA), measures speed of walking via a wearable device (Syde®).

两个终点都是疾病进展和行走能力丧失的预后因素。此外，还观察到预先指定的次要终点步幅速度第95百分位具有临床意义和统计学显着改善。这个由欧洲药品管理局（EMA）认证的新型数字终点测量通过可穿戴设备（Syde®）的步行速度。

The time to ascend 4-steps secondary endpoint also demonstrated consistent treatment benefit in favour of.

上升4步次要终点的时间也显示出一致的治疗益处。

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