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Majority of Children With Spinal Muscular Atrophy (SMA) Treated With Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate

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Genentech presented positive 2-year data from the ongoing RAINBOWFISH study at the 29th World Muscle Society Congress, evaluating Evrysdi® (risdiplam) in children with Spinal Muscular Atrophy (SMA) treated pre-symptomatically before 6 weeks of age. The study found that the majority of children achieved key motor milestones, were able to swallow and feed orally, and demonstrated typical cognitive skills, with none requiring permanent ventilation.

All children with three or more SMN2 copies achieved standing and walking milestones, while all children with two SMN2 copies could sit and most could stand and walk independently after 2 years of treatment. The study also assessed cognition as an exploratory endpoint, showing cognitive skills typical of children without SMA.

Evrysdi is the only non-invasive SMA therapy approved in over 100 countries, with more than 16,000 people treated globally. The treatment was well-tolerated, with no deaths or adverse events leading to withdrawal or treatment discontinuation.

Genentech ha presentato dati positivi a 2 anni dallo studio in corso RAINBOWFISH durante il 29° Congresso Mondiale della Società dei Muscoli, valutando Evrysdi® (risdiplam) nei bambini con Atrofia Muscolare Spinale (SMA) trattati in modo pre-sintomatico prima delle 6 settimane di vita. Lo studio ha rivelato che la maggior parte dei bambini ha raggiunto importanti traguardi motori, è stata in grado di ingoiare e nutrirsi per via orale e ha dimostrato abilità cognitive tipiche, senza che nessuno di loro necessitasse di ventilazione permanente.

Tutti i bambini con tre o più copie di SMN2 hanno raggiunto traguardi di stazione eretta e camminata, mentre tutti i bambini con due copie di SMN2 potevano sedersi e la maggior parte di loro poteva stare in piedi e camminare in modo indipendente dopo 2 anni di trattamento. Lo studio ha anche valutato la cognizione come obiettivo esplorativo, mostrando abilità cognitive tipiche di bambini senza SMA.

Evrysdi è l'unica terapia non invasiva per la SMA approvata in oltre 100 paesi, con più di 16.000 persone trattate a livello globale. Il trattamento è risultato ben tollerato, senza decessi o eventi avversi che abbiano portato a interruzione o sospensione del trattamento.

Genentech presentó datos positivos de 2 años del estudio en curso RAINBOWFISH en el 29° Congreso Mundial de la Sociedad del Músculo, evaluando Evrysdi® (risdiplam) en niños con Atrofia Muscular Espinal (AME) tratados de manera pre-sintomática antes de las 6 semanas de edad. El estudio descubrió que la mayoría de los niños alcanzaron hitos motores clave, pudieron tragar y alimentarse por vía oral, y mostraron habilidades cognitivas típicas, sin que ninguno requiriera ventilación permanente.

Todos los niños con tres o más copias de SMN2 alcanzaron hitos de estar de pie y caminar, mientras que todos los niños con dos copias de SMN2 podían sentarse y la mayoría podía estar de pie y caminar de manera independiente después de 2 años de tratamiento. El estudio también evaluó la cognición como un objetivo exploratorio, mostrando habilidades cognitivas típicas de niños sin AME.

Evrysdi es la única terapia no invasiva para la AME aprobada en más de 100 países, con más de 16,000 personas tratadas a nivel global. El tratamiento fue bien tolerado, sin muertes ni eventos adversos que llevaran a la interrupción o discontinuación del tratamiento.

젠앤텍은 제29회 세계 근육 학회에서 진행 중인 RAINBOWFISH 연구의 2년 긍정적 데이터를 발표하며, Evrysdi® (리스디플람)을 6주 전 증상이 없는 아동에게 척수 근위축증(SMA) 치료에 대해 평가했습니다. 연구에 따르면 대부분의 아동이 주요 운동 발달 이정표를 달성하였고, 삼키기 및 경구 섭식이 가능했으며, 전형적인 인지 능력을 보여주었으며, 영구적인 기계환기에 필요한 아동은 없었습니다.

세 개 이상의 SMN2 복사본이 있는 모든 아동이 서기 및 걷기 이정표를 달성한 반면, 두 개의 SMN2 복사본이 있는 모든 아동은 앉을 수 있었고, 대부분은 2년 치료 후 독립적으로 서고 걸을 수 있었습니다. 이 연구는 탐색적 목표로서 인지 평가도 포함하며, SMA가 없는 아동과 같은 전형적인 인지 능력을 나타냈습니다.

Evrysdi는 100개 이상의 국가에서 승인된 유일한 비침습적 SMA 치료제이며, 전 세계적으로 16,000명 이상의 환자가 치료받았습니다. 치료는 잘 견디며, 중단이나 치료 중단으로 이어지는 사망이나 부작용이 없었습니다.

Genentech a présenté des données positives sur 2 ans de l'étude en cours RAINBOWFISH lors du 29ème Congrès Mondial de la Société des Muscles, évaluant Evrysdi® (risdiplam) chez des enfants atteints d'Atrophie Musculaire Spinale (AMS) traités pré-symptomatiquement avant 6 semaines d'âge. L'étude a révélé que la majorité des enfants avait atteint des jalons moteurs clés, était capable d'avaler et de s'alimenter par voie orale, et démontrait des compétences cognitives typiques, aucun d'entre eux n'ayant besoin de ventilation permanente.

Tous les enfants avec trois copies ou plus de SMN2 ont atteint des jalons de station debout et de marche, tandis que tous les enfants avec deux copies de SMN2 pouvaient s'asseoir et la plupart pouvaient se lever et marcher de manière autonome après 2 ans de traitement. L'étude a également évalué la cognition comme un objectif exploratoire, montrant des compétences cognitives typiques des enfants sans AMS.

Evrysdi est la seule thérapie non invasive pour l'AMS approuvée dans plus de 100 pays, avec plus de 16 000 personnes traitées dans le monde. Le traitement a été bien toléré, sans décès ni événements indésirables ayant conduit à un retrait ou une interruption du traitement.

Genentech stellte auf dem 29. Weltkongress der Muskulaturgesellschaft positive 2-Jahres-Daten aus der laufenden RAINBOWFISH-Studie vor, die Evrysdi® (risdiplam) bei Kindern mit spinaler Muskelatrophie (SMA) evaluiert, die prä-symptomatisch vor dem 6. Lebensmonat behandelt wurden. Die Studie zeigte, dass die Mehrheit der Kinder wichtige motorische Meilensteine erreichte, eigenständig schlucken und oral essen konnte und typische kognitive Fähigkeiten aufwies, bei denen niemand auf eine dauerhafte Beatmung angewiesen war.

Alle Kinder mit drei oder mehr Kopien von SMN2 erreichten die Ziele Stehen und Laufen, während alle Kinder mit zwei SMN2-Kopien sitzen konnten und die meisten nach 2 Jahren Behandlung eigenständig stehen und gehen konnten. Die Studie bewertete auch die Kognition als explorativen Endpunkt und zeigte kognitive Fähigkeiten, die bei Kindern ohne SMA typisch sind.

Evrysdi ist die einzige nicht-invasive SMA-Therapie, die in über 100 Ländern zugelassen ist, mit mehr als 16.000 weltweit behandelten Personen. Die Behandlung wurde gut vertragen, ohne Todesfälle oder unerwünschte Ereignisse, die zu einem Abbruch oder einer Unterbrechung der Therapie führten.

Positive
  • Majority of children treated with Evrysdi achieved key motor milestones
  • All children were able to swallow and feed orally
  • No children required permanent ventilation
  • 100% of children with 3+ SMN2 copies achieved standing and walking milestones
  • 100% of children with 2 SMN2 copies could sit, 60% could stand and walk independently
  • Children showed cognitive skills typical of those without SMA
  • Evrysdi is approved in over 100 countries with 16,000+ patients treated globally
  • No deaths or adverse events leading to treatment discontinuation
Negative
  • None.

– Positive data confirm Evrysdi efficacy and safety in children first treated pre-symptomatically before 6 weeks of age, with most achieving motor milestones similar to children without SMA –

– All children were able to swallow and feed orally, with none requiring permanent ventilation –

– Evrysdi is the only non-invasive SMA therapy and is approved in over 100 countries, with more than 16,000 people with SMA treated globally –

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). 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.

“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.”

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. 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.

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.

“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.”

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.

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

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

About Evrysdi® (risdiplam)

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

  • 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.
  • 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.
  • 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).
  • 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.
  • 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. The study is currently recruiting.
  • 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. The studies are currently recruiting.

About 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. 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.

What is Evrysdi?

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

Important Safety Information

  • Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you:
    • are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine
    • are a woman who can become pregnant:
      • Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy
      • 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
      • 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. 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
    • 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
    • are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby
  • Tell your healthcare provider about all the medicines you take
  • 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
  • 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
  • The most common side effects of Evrysdi include:
    • For later-onset SMA:
      • fever
      • diarrhea
      • rash
    • For infantile-onset SMA:
      • fever
      • diarrhea
      • rash
      • runny nose, sneezing, and sore throat (upper respiratory infection)
      • lung infection (lower respiratory infection)
      • constipation
      • vomiting
      • cough

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.

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.

Please see full Prescribing Information for additional Important Safety Information.

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

About Genentech in Neuroscience

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.

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.

About Genentech

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

All trademarks used or mentioned in this release are protected by law.

Media Contact:

Adam Pryor, (650) 467-6800

Advocacy Contact:

Alana Paull, (925) 528-1014

Investor Contacts:

Loren Kalm, (650) 225-3217

Bruno Eschli, +41 61 68 75284

Source: Genentech

FAQ

What were the key findings of the RAINBOWFISH study for Evrysdi (RHHBY) in SMA treatment?

The 2-year RAINBOWFISH study showed that the majority of children with SMA treated pre-symptomatically with Evrysdi before 6 weeks of age achieved key motor milestones, could swallow and feed orally, and demonstrated typical cognitive skills. All children with 3+ SMN2 copies achieved standing and walking milestones, while all with 2 SMN2 copies could sit and 60% could stand and walk independently.

How does Evrysdi (RHHBY) compare to other SMA treatments in terms of administration?

Evrysdi is the only non-invasive SMA therapy that can be administered during a child's first hours of life. It is approved in over 100 countries and has been used to treat more than 16,000 people with SMA globally.

What were the safety results for Evrysdi (RHHBY) in the RAINBOWFISH study?

The 2-year RAINBOWFISH study showed that Evrysdi was well-tolerated. There were no deaths or adverse events leading to withdrawal or treatment discontinuation. The most common adverse events were teething, gastroenteritis, diarrhea, eczema, and pyrexia, which were generally consistent with other Evrysdi trials and more reflective of age than underlying SMA.

How did Evrysdi (RHHBY) affect cognitive development in children with SMA?

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

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