PTC Therapeutics, Inc. provided  several positive updates on the vatiquinone Friedreich ataxia (FA) program.

PTC Therapeutics，Inc.对vatiquinone-Friedreich共济失调（FA）计划提供了一些积极的更新。

The pre-specified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression. In addition, PTC recently aligned with FDA on key aspects of the planned NDA submission for vatiquinone. 'The results of the extension studies provide further evidence of the potential benefit of vatiquinone in slowing disease progression,' said Matthew B.

满足了两项不同FA长期延长研究的预先指定的终点，具有高度统计学意义的证据表明持久治疗对疾病进展有益。此外，PTC最近就计划提交的梵蒂醌NDA的关键方面与FDA达成了一致。”扩展研究的结果进一步证明了瓦替醌在减缓疾病进展方面的潜在益处。

Klein, M.D., Chief Executive Officer of PTC Therapeutics. 'In addition, the strong safety profile of vatiquinone positions it to be a potentially meaningful therapy for all Friedreich ataxia patients, particularly children and adolescents for whom there are no approved therapies. We look forward to submitting the NDA by the end of the year.'.

医学博士克莱恩（Klein）是PTC Therapeutics的首席执行官此外，梵蒂醌的强大安全性使其成为所有Friedreich共济失调患者的潜在有意义的治疗方法，特别是没有批准治疗的儿童和青少年。我们期待在年底前提交NDA。”。

Analysis of the MOVE-FA long-term extension study demonstrated that 144 weeks of vatiquinone treatment resulted in a 3.7-point benefit (p<0.0001, N=70) on the modified Friedreich Ataxia Rating Scale (mFARS) relative to a matched natural history cohort from the FACOMS (Friedreich Ataxia Clinical Outcome Measures) disease registry.

对MOVE-FA长期扩展研究的分析表明，相对于FACOMS（Friedreich共济失调临床结果测量）疾病登记处的匹配自然史队列，改良Friedreich共济失调评定量表（mFARS）的144周伐他醌治疗可产生3.7分的益处（p<0.0001，N=70）。

This treatment difference on the primary endpoint represents a clinically meaningful 50% slowing in disease progression over 3 years. These results confirm that the slowing of disease progression recorded in the 72-week placebo-controlled MOVE-FA trial are maintained over 144 weeks of treatment. In addition, vatiquinone continued to be safe and well tolerated without any treatment-related serious adverse events reported..

主要终点的这种治疗差异代表了3年内疾病进展减缓50%的临床意义。这些结果证实，在72周的安慰剂对照MOVE-FA试验中记录的疾病进展减缓在144周的治疗中得以维持。此外，梵蒂醌仍然安全且耐受性良好，未报告任何与治疗相关的严重不良事件。。

In addition, PTC analyzed long-term open-label data from an earlier study of vatiquinone in adults with FA. Following 24-months of treatment with vatiquinone, subjects had a 4.8-point benefit on the mFARS relative to a matched natural history population (p<0.0001, N=41).

此外，PTC分析了早期对FA患者进行的一项瓦替醌研究的长期开放标签数据。使用瓦替醌治疗24个月后，相对于匹配的自然史人群，受试者的mFARS获益4.8分（p<0.0001，N=41）。

'MOVE-FA was a well-conducted international clinical trial in children and adults with Friedreich's ataxia. Both this trial data and the open-label extension data are compelling with positive results in clinical endpoints that are meaningful to the FA community,' said Jennifer Farmer, Chief Executive Officer of the Friedreich's Ataxia Research Alliance (FARA).

“MOVE-FA是一项针对弗里德里希共济失调儿童和成人的国际临床试验，效果良好。弗里德里希共济失调研究联盟（FARA）首席执行官詹妮弗·法默（JenniferFarmer）说，这项试验数据和开放标签扩展数据都令人信服，在临床终点方面取得了积极的结果，这对FA社区有意义。

'We are also encouraged that vatiquinone treatment continues to be safe and well-tolerated. Given the high unmet need, especially in the pediatric population, we are excited that PTC Therapeutics is submitting an NDA.'.

。鉴于未满足的需求很高，特别是在儿科人群中，我们很高兴PTC Therapeutics正在提交NDA。”。

PTC plans to submit the vatiquinone NDA in December 2024. The NDA will include results from the placebo-controlled portion of the MOVE-FA study in which there was significant benefit recorded on the Upright Stability Subscale of the mFARS. This subscale is the most relevant and sensitive mFARS component for pediatric and young adults.

PTC计划于2024年12月提交梵蒂醌NDA。NDA将包括MOVE-FA研究中安慰剂对照部分的结果，其中mFARS的直立稳定性分量表记录了显着的益处。该分量表是儿科和年轻人最相关和最敏感的mFARS分量表。

The NDA will also include confirmatory evidence from the two long-term treatment analyses discussed above, as well mechanistic data demonstrating treatment effect on biomarkers of disease pathology..

NDA还将包括上述两项长期治疗分析的确证证据，以及证明对疾病病理生物标志物的治疗效果的机制数据。。

Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich ataxia. Inhibition of 15-LO helps to alleviate the consequences of mitochondrial dysfunction and oxidative stress, ultimately preventing ferroptosis and aiding neuronal survival.

梵蒂醌是一种小分子，是15-脂氧合酶（15-LO）的一流选择性抑制剂，15-脂氧合酶是弗里德里希共济失调中能量和氧化应激途径的关键调节剂。抑制15-LO有助于减轻线粒体功能障碍和氧化应激的后果，最终预防铁浓化并帮助神经元存活。

Vatiquinone has been evaluated in a number of clinical studies, many focused on pediatric patients, and has demonstrated an impact on mortality risk and a number of neurological and neuromuscular disease symptoms..

Vatiquinone已经在许多临床研究中进行了评估，许多研究都集中在儿科患者身上，并且已经证明对死亡风险以及许多神经和神经肌肉疾病症状有影响。。

Condition: Friedreichs Ataxia

病情：弗里德赖希共济失调

Type: drug

类型：药物