In a previous Phase 2 study, olesoxime had a favorable safety profile. Although the primary endpoint was not met, analyses suggested that olesoxime might help in the maintenance of motor function in patients with Types 2/3 SMA. This open-label extension study (OLEOS) further characterizes the safety, tolerability and efficacy of olesoxime over longer therapy durations. In OLEOS, no new safety risks were identified. Compared to matched natural history data, patients treated with olesoxime demonstrated small, non-significant changes in motor function over 52 weeks. Motor function scores were stable for 52 weeks but declined over the remainder of the study. The greatest decline in motor function was seen in patients ≤15 years old, and those with Type 2 SMA had faster motor function decline versus those with Type 3 SMA. Previous treatment with olesoxime in the Phase 2 study was not protective of motor function in OLEOS. Respiratory outcomes were stable in patients with Type 3 SMA >15 years old but declined in patients with Type 2 SMA and in patients with Type 3 SMA ≤15 years old. Overall, with no stabilization of functional measures observed over 130 weeks, OLEOS did not support significant benefit of olesoxime in patients with SMA.

中文翻译：

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在 OLEOS 试验中对使用 olesoxime 治疗的 2 型和不能行走的 3 型脊髓性肌萎缩症 (SMA) 患者进行长期随访

在之前的 2 期研究中，olesoxime 具有良好的安全性。尽管未达到主要终点，但分析表明，olesoxime 可能有助于维持 2/3 型 SMA 患者的运动功能。这项开放标签扩展研究 (OLEOS) 进一步表征了 olesoxime 在较长治疗时间内的安全性、耐受性和有效性。在 OLEOS 中，没有发现新的安全风险。与匹配的自然病程数据相比，接受 olesoxime 治疗的患者在 52 周内表现出小的、非显着的运动功能变化。运动功能评分在 52 周内保持稳定，但在研究的剩余时间里有所下降。≤ 15 岁的患者运动功能下降最大，2 型 SMA 患者的运动功能下降比 3 型 SMA 更快。在第 2 阶段研究中使用 olesoxime 的先前治疗不能保护 OLEOS 的运动功能。3 型 SMA > 15 岁患者的呼吸结果稳定，但 2 型 SMA 患者和 3 型 SMA ≤ 15 岁患者的呼吸结果下降。总体而言，由于在 130 周内没有观察到功能测量的稳定性，因此 OLEOS 不支持 SMA 患者使用奥来肟的显着益处。