SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), presented positive 2-year data from the ongoing RAINBOWFISH study at the 29th World Muscle Society (WMS) Congress, October 8-12, 2024, assessing the efficacy and safety of Evrysdi® (risdiplam) in children with SMA who were treated pre-symptomatically as infants before 6 weeks of age (n=23).

加利福尼亚州南旧金山市——（商业新闻短讯）——罗氏集团（SIX:RO，ROG；OTCQX:RHHBY）成员基因泰克（Genentech）在2024年10月8日至12日举行的第29届世界肌肉协会（WMS）大会上提供了正在进行的虹鳟鱼研究的2年积极数据，评估了Evrysdi®（利培林）对6周龄之前接受症状前治疗的SMA儿童（n=23）的疗效和安全性。

The study found the majority of children achieved key motor milestones, were able to swallow and feed orally, and demonstrated cognitive skills typical of children without SMA, with none requiring permanent ventilation..

该研究发现，大多数儿童达到了关键的运动里程碑，能够吞咽和进食，并且表现出没有SMA的儿童典型的认知技能，没有一个需要永久通气。。

“In children with SMA, motor neuron degeneration starts before the onset of symptoms, so time is of the essence if we hope to preserve muscle function,” said Laurent Servais, M.D., Ph.D., Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre. “It’s heartening to see that through early intervention with Evrysdi these children have achieved important milestones like sitting, standing and walking that would typically be unattainable without treatment.”.

MDUK牛津神经肌肉中心儿科神经肌肉疾病教授Laurent Servais医学博士说：“对于SMA患儿，运动神经元变性在症状出现之前就开始了，因此如果我们希望保持肌肉功能，时间至关重要。”。“令人鼓舞的是，通过Evrysdi的早期干预，这些孩子已经取得了重要的里程碑，如坐着，站立和行走，如果不进行治疗通常是无法实现的。”。

All of the children treated with Evrysdi who had three or more SMN2 copies (n=18), achieved standing and walking (100%) milestones as assessed by Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) and Hammersmith Infant Neurological Examination, Module 2 (HINE-2), with most achieving these milestones within World Health Organization (WHO) windows of typical child development.

根据Bayley婴幼儿发育量表第三版（BSID-III）和Hammersmith婴儿神经系统检查模块2（HINE-2）的评估，所有接受Evrysdi治疗的儿童都有三个或更多SMN2拷贝（n=18），达到了站立和行走（100%）的里程碑，其中大多数在世界卫生组织（who）典型儿童发育窗口内达到了这些里程碑。

Among the children with two SMN2 copies (n=5), all could sit (100%) and most could stand and walk (60%) independently after 2 years of treatment. After 2 years of treatment, all children were able to swallow and feed orally and none required permanent ventilation. Natural history studies indicate that without disease-modifying treatment, children with Type 1 SMA would not be able to reach such milestones, nor typically live past the age of 2..

在有两个SMN2拷贝（n=5）的儿童中，经过2年的治疗，他们都可以坐着（100%），大多数可以独立站立和行走（60%）。经过2年的治疗，所有儿童都能够吞咽和口服，无需永久通气。自然史研究表明，如果不进行疾病缓解治疗，患有1型SMA的儿童将无法达到这样的里程碑，通常也无法活到2岁以上。。

After 2 years of Evrysdi treatment, children in the study showed cognitive skills typical of children without SMA, as assessed by the BSID-III Cognitive Scale. This study was the first clinical trial in SMA to assess cognition as an exploratory endpoint using a standardized scale.

经过2年的Evrysdi治疗，研究中的儿童表现出BSID-III认知量表评估的无SMA儿童典型的认知技能。这项研究是SMA首次使用标准化量表评估认知作为探索终点的临床试验。

“These 2-year findings confirm the potential of early intervention with Evrysdi to meaningfully improve the lives of children with SMA,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “Working in tandem with newborn screening programs, Evrysdi is the only non-invasive SMA treatment that can be administered during a child’s first hours of life.”.

Genentech首席医疗官兼全球产品开发主管Levi Garraway医学博士说：“这些为期两年的研究结果证实了Evrysdi早期干预的潜力，可以有意义地改善SMA儿童的生活。”。“Evrysdi与新生儿筛查计划一起工作，是唯一可以在孩子出生后的头几个小时内进行的非侵入性SMA治疗。”。

To assess outcomes of early treatment initiation before the onset of symptoms, children in the study started treatment with Evrysdi before 6 weeks of age (median age of first dose was 25 days). The study analyzed outcomes against the number of copies of the SMN2 gene each child had. Generally, fewer SMN2 copy numbers are associated with more severe SMA..

为了评估症状发作前早期治疗开始的结果，研究中的儿童在6周龄之前开始使用Evrysdi治疗（第一剂的中位年龄为25天）。该研究根据每个孩子的SMN2基因拷贝数分析了结果。通常，较少的SMN2拷贝数与更严重的SMA相关。。

There were no deaths or adverse events (AEs) leading to withdrawal or treatment discontinuation. The most common AEs were teething, gastroenteritis, diarrhea, eczema and pyrexia. The AEs observed in the year-2 analysis are generally consistent with those AEs seen in other Evrysdi trials in SMA. AEs were more reflective of age than underlying SMA.

没有死亡或不良事件（AE）导致停药或停止治疗。最常见的AE是出牙，胃肠炎，腹泻，湿疹和发热。在第二年分析中观察到的AE通常与SMA中其他Evrysdi试验中观察到的AE一致。AE比潜在的SMA更能反映年龄。

The majority of AEs were not considered treatment-related and resolved over time..

大多数AE不被认为与治疗有关，并且随着时间的推移而得到解决。。

Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.

作为与SMA基金会和PTC Therapeutics合作的一部分，Genentech领导Evrysdi的临床开发。

About Evrysdi® (risdiplam)

关于Evrysdi® （里斯迪普兰）

Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home in liquid form either by feeding tube or by mouth.

Evrysdi是一种存活运动神经元2（SMN2）剪接修饰剂，旨在治疗由5q染色体突变引起的SMA，该突变导致存活运动神经元（SMN）蛋白缺乏。Evrysdi每天在家中通过喂食管或口腔以液体形式给药。

Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system (CNS) and peripheral tissues, as demonstrated in animal models. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and core motor functions, such as swallowing, speaking and breathing..

Evrysdi旨在通过增加和维持中枢神经系统（CNS）和外周组织中SMN蛋白的产生来治疗SMA，如动物模型所示。SMN蛋白遍布全身，对于维持健康的运动神经元和核心运动功能（如吞咽，说话和呼吸）至关重要。。

Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S. Food and Drug Administration in 2017. In 2021, Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research Award for Drug Discovery.

Evrysdi于2018年被欧洲药品管理局（EMA）授予最佳称号，2017年被美国食品和药物管理局授予孤儿药称号。2021年，Evrysdi被英国药理学学会授予年度药物发现奖，以及药物研究学会授予药物发现奖。

Evrysdi is currently approved in more than 100 countries, and the dossier is under review in a further 12 countries. A new risdiplam room-temperature stable tablet is currently under review by regulators..

Evrysdi目前已在100多个国家获得批准，另外12个国家正在审查该档案。监管机构目前正在审查一种新的利培林室温稳定片剂。。

Evrysdi is currently being, or has been, evaluated in numerous global multicenter trials in people with SMA:

Evrysdi目前正在或已经在许多针对SMA患者的全球多中心试验中进行评估：

FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Infants were approximately 5.5 months of age (median) at the time of enrollment and of the 58 infants that completed the first year of treatment, 52 entered the open-label extension study. The study met its primary endpoint and has concluded after 5 years of follow up..

FIREFISH（NCT02913482）–一项针对1型SMA婴儿的开放标签、两部分关键临床试验。入组时婴儿约为5.5个月大（中位数），在完成第一年治疗的58名婴儿中，有52名进入了开放标签扩展研究。该研究达到了其主要终点，并在5年的随访后得出结论。。

SUNFISH (NCT02908685) – a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. The study met its primary endpoint.

SUNFISH（NCT02908685）–一项针对2-25岁2型或3型SMA患者的两部分、双盲、安慰剂对照关键研究。该研究达到了其主要终点。

JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi. The study has completed recruitment (n=174)..

JEWELFISH（NCT03032172）-一项开放标签探索性试验，旨在评估6个月至60岁的SMA患者的安全性，耐受性，药代动力学和药效学，这些患者在接受Evrysdi之前至少90天接受了其他研究或批准的SMA治疗。该研究已完成招聘（n=174）。。

RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicenter study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in babies (n=26), from birth to 6 weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study met its primary endpoint..

RAINBOWFISH（NCT03779334）-一项开放标签，单臂，多中心研究，调查Evrysdi在婴儿（n=26）中的疗效，安全性，药代动力学和药效学，从出生到6周龄（第一次服用），遗传诊断的SMA尚未出现症状。该研究达到了其主要终点。。

MANATEE (NCT05115110) – a Phase II/III clinical study to evaluate the safety and efficacy of GYM329 (RG6237), an anti-myostatin molecule targeting muscle growth, in combination with Evrysdi for the treatment of SMA in patients 2-10 years of age. The FDA Office of Orphan Products Development granted GYM329 Orphan Drug Designation for the treatment of patients with SMA in December 2021.

MANATEE（NCT05115110）–一项II/III期临床研究，旨在评估针对肌肉生长的抗肌肉生长抑制素分子GYM329（RG6237）与Evrysdi联合治疗2-10岁患者SMA的安全性和有效性。2021年12月，FDA孤儿产品开发办公室批准GYM329孤儿药用于治疗SMA患者。

The study is currently recruiting..

该研究目前正在招募。。

HINALEA 1 (NCT05861986) and HINALEA 2 (NCT05861999) – Phase IV clinical studies to evaluate the effectiveness and safety of Evrysdi in patients under 2 years of age at enrollment, who received onasemnogene abeparvovec gene therapy either pre-symptomatically or post-symptomatically, following a genetically confirmed diagnosis of 5q–autosomal recessive SMA.

HINALEA 1（NCT05861986）和HINALEA 2（NCT05861999）-IV期临床研究，用于评估Evrysdi在2岁以下入选患者中的有效性和安全性，这些患者在基因确诊为5q常染色体隐性SMA后，在症状前或症状后接受了onasemogene abeparvovec基因治疗。

The studies are currently recruiting..

目前正在招募研究人员。。

About SMA

关于SMA

SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement.

SMA是一种严重的进行性神经肌肉疾病，可能致命。它影响大约10000名婴儿中的一名，是导致婴儿死亡的主要遗传原因。SMA是由存活运动神经元1（SMN1）基因突变引起的，该突变导致SMN蛋白缺乏。这种蛋白质遍布全身，对控制肌肉和运动的神经功能至关重要。

Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost..

没有它，神经细胞就无法正常工作，随着时间的推移会导致肌肉无力。根据SMA的类型，个人的体力和行走、进食或呼吸的能力可能会显着降低或丧失。。

What is Evrysdi?

什么是Evrysdi？

Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults.

Evrysdi是一种用于治疗儿童和成人脊髓性肌萎缩症（SMA）的处方药。

Important Safety Information

重要安全信息

Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:

服用Evrysdi之前，请告知您的医疗保健提供者您的所有医疗状况，包括您是否：

are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine

怀孕或计划怀孕，因为Evrysdi可能会伤害您未出生的婴儿。服用此药前，请咨询您的医疗保健提供者

are a woman who can become pregnant:

是可以怀孕的女性：

Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy

在您开始使用Evrysdi治疗之前，您的医疗保健提供者可能会对您进行妊娠测试

Talk to your healthcare provider about birth control methods that may be right for you. Use birth control while on treatment and for at least 1 month after stopping Evrysdi

与你的医疗保健提供者谈谈可能适合你的节育方法。在治疗期间和停止Evrysdi后至少1个月内使用节育

Pregnancy Registry. There is a pregnancy registry for women who take Evrysdi during pregnancy. The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving Evrysdi, tell your healthcare provider right away.

怀孕登记处。怀孕期间服用Evrysdi的女性有一个怀孕登记处。本登记册的目的是收集有关孕妇及其婴儿健康的信息。。

Talk to your healthcare provider about registering with the Evrysdi Pregnancy Registry. Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting http://www.evrysdipregnancyregistry.com.

与您的医疗保健提供者讨论如何在Evrysdi妊娠登记处注册。您的医疗保健提供者可以为您注册此注册表，您也可以拨打1-833-760-1098或访问http://www.evrysdipregnancyregistry.com.

are an adult male. Evrysdi may affect a man’s ability to have children (fertility). Ask a healthcare provider for advice before taking this medicine

是成年男性。Evrysdi可能会影响男性的生育能力（生育能力）。服用此药前请咨询医疗保健提供者

are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby

正在母乳喂养或计划母乳喂养。目前尚不清楚Evrysdi是否会进入母乳并可能伤害您的宝宝

Tell your healthcare provider about all the medicines you take

告诉你的医疗保健提供者你服用的所有药物

You should receive Evrysdi from the pharmacy as a liquid. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement

你应该从药房收到Evrysdi作为液体。如果瓶中的药物是粉末，不要使用。请联系药剂师更换

Avoid getting Evrysdi on your skin or in your eyes. If Evrysdi gets on your skin, wash the area with soap and water. If Evrysdi gets in your eyes, rinse your eyes with water

避免让Evrysdi沾到皮肤或眼睛上。如果Evrysdi沾到你的皮肤上，用肥皂和水清洗。如果Evrysdi进入你的眼睛，用水冲洗你的眼睛

The most common side effects of Evrysdi include:

Evrysdi最常见的副作用包括：

For later-onset SMA:

对于迟发性SMA：

fever

发烧

diarrhea

腹泻

rash

皮疹

For infantile-onset SMA:

fever

发烧

diarrhea

腹泻

rash

皮疹

runny nose, sneezing, and sore throat (upper respiratory infection)

流鼻涕、打喷嚏和喉咙痛（上呼吸道感染）

lung infection (lower respiratory infection)

肺部感染（下呼吸道感染）

constipation

便秘

vomiting

呕吐

cough

咳嗽

These are not all of the possible side effects of Evrysdi. For more information on the risk and benefits profile of Evrysdi, ask your healthcare provider or pharmacist.

这些并不是Evrysdi所有可能的副作用。。

You may report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555.

您可以通过1-800-FDA-1088或http://www.fda.gov/medwatch.您也可以致电1-888-835-2555向Genentech报告副作用。

Please see full Prescribing Information for additional Important Safety Information.

有关其他重要安全信息，请参阅完整的处方信息。

For more information, go to https://www.evrysdi.com/.

有关更多信息，请访问https://www.evrysdi.com/.

About Genentech in Neuroscience

关于神经科学中的基因泰克

Neuroscience is a major focus of research and development at Genentech. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases.

神经科学是基因泰克研究和开发的主要重点。。

Genentech and Roche are investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today..

基因泰克（Genentech）和罗氏（Roche）正在研究十几种治疗神经系统疾病的药物，包括多发性硬化症、脊髓性肌萎缩症、视神经脊髓炎谱系障碍、阿尔茨海默氏病、亨廷顿舞蹈病、帕金森氏病和杜兴氏肌营养不良症。与我们的合作伙伴一起，我们致力于突破科学理解的界限，以解决当今神经科学中一些最困难的挑战。。

About Genentech

关于基因泰克

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California.

基因泰克成立于40多年前，是一家领先的生物技术公司，负责发现、开发、制造和商业化药物，用于治疗严重威胁生命的患者。该公司是罗氏集团的成员，总部位于加利福尼亚州南旧金山。

For additional information about the company, please visit http://www.gene.com..

有关该公司的更多信息，请访问http://www.gene.com..

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本版本中使用或提及的所有商标均受法律保护。