share_log

New Data From the Phase 3 NEURO-TTRansform Study Evaluating WAINUA (Eplontersen) to Be Presented at the 2024 International Symposium on Amyloidosis (ISA)

New Data From the Phase 3 NEURO-TTRansform Study Evaluating WAINUA (Eplontersen) to Be Presented at the 2024 International Symposium on Amyloidosis (ISA)

評估WAINUA(Eplontersen)的3期神經轉化研究的新數據將在2024年澱粉樣變國際研討會(ISA)上公佈
PR Newswire ·  05/23 19:00

– Results across NEURO-TTRansform subgroups show consistent benefit in neuropathy impairment and improved quality of life, regardless of patient segmentation –

— 無論患者分類如何,Neuro-TTransform 亞組的結果均顯示,在神經病損傷和生活質量改善方面均有持續的益處 —

CARLSBAD, Calif., May 23, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will present new subgroup analyses from Ionis and AstraZeneca's Phase 3 NEURO-TTRansform study of WAINUA (eplontersen) at the 2024 International Symposium on Amyloidosis (ISA) in Rochester, Minnesota, May 26-30. WAINUA was approved by the U.S. Food and Drug Administration (FDA) in December 2023 for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN.

加利福尼亞州卡爾斯巴德,2024年5月23日 /PRNewswire/ — 愛奧尼斯製藥公司(納斯達克股票代碼:IONS)今天宣佈,將在5月26日至30日在明尼蘇達州羅切斯特舉行的2024年澱粉樣變國際研討會(ISA)上介紹愛奧尼斯和阿斯利康對WAINUA(eplontersen)的3期Neuro-tTransform研究的新亞組分析。WAINUA 於 2023 年 12 月獲得美國食品藥品監督管理局 (FDA) 的批准,用於治療成人遺傳性轉甲狀腺素介導澱粉樣變的多發性神經病,通常稱爲 Hattr-PN 或 Attrv-PN。

Notable presentations include:

值得注意的演講包括:

  • Neuropathy Impairment and Nutritional Status with Eplontersen in Patients with Hereditary Transthyretin-Mediated Amyloidosis
    • Abstract ID 174: May 29, 2024, 10:00-10:30 am ET (poster presentation 1) and 2:45-3:45 pm ET (poster presentation 2)
    • Presenting Author: Jonas Wixner
  • Eplontersen for Hereditary Transthyretin Amyloidosis with Polyneuropathy: An Exploratory Analysis in Patients with the V30M TTR Variant and Early-Onset or Late-Onset Disease
    • Abstract ID 230: May 29, 2024, 10:00-10:30 am ET (poster presentation 1) and 2:45-3:45 pm ET (poster presentation 2)
    • Presenting Author: Julian D. Gillmore
  • Eplontersen for Hereditary Transthyretin Amyloidosis with Polyneuropathy: An Exploratory Analysis of Treatment Effect in Male and Female Patients
    • Abstract ID 202: May 29, 2024, 10:00-10:30 am ET (poster presentation 1) and 2:45-3:45 pm ET (poster presentation 2)
    • Presenting Author: Márcia Waddington Cruz
  • 遺傳性轉甲狀腺素介導的澱粉樣變性患者Eplontersen的神經病損傷和營養狀況
    • 摘要 ID 174:美國東部時間 2024 年 5 月 29 日上午 10:00-10:30(海報演示 1)和美國東部時間下午 2:45-3:45(海報演示 2)
    • 主講作者:喬納斯·威克斯納
  • Eplontersen 治療遺傳性轉甲狀腺素澱粉樣變性伴多發性神經病:對V30M患者的探索性分析 TTR 變異和早發或晚發疾病
    • 摘要 ID 230:美國東部時間 2024 年 5 月 29 日上午 10:00-10:30(海報演示 1)和美國東部時間下午 2:45-3:45(海報演示 2)
    • 主講作者:朱利安·吉爾摩爾
  • Eplontersen治療遺傳性轉甲狀腺素澱粉樣變性多發性神經病:對男性和女性患者治療效果的探索性分析
    • 摘要 ID 202:美國東部時間 2024 年 5 月 29 日上午 10:00-10:30(海報演示 1)和美國東部時間下午 2:45-3:45(海報演示 2)
    • 主講作者:瑪西婭·沃丁頓·克魯茲

As part of a global development and commercialization agreement, AstraZeneca and Ionis are commercializing WAINUA for the treatment of ATTRv-PN in the U.S. and are seeking regulatory approval in Europe and other parts of the world.

作爲全球開發和商業化協議的一部分,阿斯利康和愛奧尼斯正在美國將用於治療Attrv-PN的WAINUA商業化,並正在歐洲和世界其他地區尋求監管部門的批准。

Eplontersen is currently being evaluated in the Phase 3 CARDIO-TTRansform study for adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a systemic, progressive and fatal condition that typically leads to progressive heart failure and often death within three-to-five years from disease onset. The CARDIO-TTRansform Phase 3 study is fully enrolled with more than 1,400 patients – making it the largest study in this patient population to date.

Eplontersen目前正在接受針對轉甲狀腺素介導澱粉樣蛋白心肌病(ATTR-CM)成人的3期Cardio-TTransform研究的評估,該病是一種全身性、進行性和致命性疾病,通常會導致進行性心力衰竭,通常在發病後的三到五年內死亡。Cardio-TTransform的3期研究已全部入組,涉及1400多名患者,這是迄今爲止該患者群體中規模最大的研究。

About WAINUA (eplontersen)
WAINUA (eplontersen) is a LIgand-Conjugated Antisense (LICA) medicine designed to inhibit the production of transthyretin, or TTR protein. WAINUA has been approved in the U.S. for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults (also referred to as ATTRv-PN). Please see full Prescribing Information.

關於 WAINUA(eplontersen)
WAINUA(eplontersen)是一個 gand-C強迫的 一個ntisense(LICA)藥物旨在抑制轉甲狀腺素或TTR蛋白的產生。WAINUA已在美國獲准用於治療成人遺傳性轉甲狀腺素介導的澱粉樣變性多發性神經病(也稱爲Attrv-PN)。請查看完整的處方信息。

INDICATION for WAINUA (eplontersen)
WAINUA injection for subcutaneous use 45 mg is indicated for treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

WAINUA(eplontersen)的適應症
WAINUA 皮下注射液 45 mg 適用於治療成人遺傳性轉甲狀腺素介導澱粉樣變的多發性神經病。

IMPORTANT SAFETY INFORMATION for WAINUA (eplontersen)

WAINUA(eplontersen)的重要安全信息

WARNINGS AND PRECAUTIONS
Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.

警告和注意事項
血清維生素 A 水平降低,推薦補充劑 WAINUA 導致血清維生素 A 水平降低。補充維生素 A 的每日推薦攝入量。如果出現提示維生素 A 缺乏的眼部症狀,請將患者轉診給眼科醫生。

ADVERSE REACTIONS

不良反應

Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%).

最常見的不良反應(在接受Wainua治療的患者中≥9%)是維生素A減少(15%)和嘔吐(9%)。

Please see link to U.S. Full Prescribing Information for WAINUA.

請參閱 WAINUA 的美國完整處方信息鏈接。

About the NEURO-TTRansform study

關於 Neuro-transform 研究

NEURO-TTRansform is a global, open-label, randomized trial evaluating the efficacy and safety of eplontersen in patients with ATTRv-PN at week 35, week 66 and week 85. The final analysis comparing eplontersen to an external placebo group was completed at week 66. All patients were then followed on treatment until week 85 and evaluated four weeks after the last dose in an end-of-trial assessment. Following treatment and the end-of-trial assessments, patients were eligible to enter an open-label extension study to continue receiving eplontersen once every four weeks or enter a 20-week post-treatment evaluation period.

Neuro-ttransform是一項全球性、開放標籤、隨機試驗,評估了eplontersen在第35周、第66周和第85周對Attrv-PN患者的療效和安全性。將eplontersen與外部安慰劑組進行比較的最終分析於第66周完成。然後,對所有患者進行了隨訪治療直至第85周,並在試驗結束評估中對最後一次給藥四周後進行了評估。在治療和試驗結束評估之後,患者有資格參加一項開放標籤的延期研究,繼續每四周接受一次eplontersen治療,或者進入爲期20周的治療後評估期。

About Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (ATTRv-PN)

關於遺傳性轉甲狀腺素介導的澱粉樣多發性神經病 (Attrv-PN)

ATTRv-PN is caused by the accumulation of misfolded mutated TTR protein in the peripheral nerves. Patients with ATTRv-PN experience ongoing debilitating nerve damage throughout their body resulting in the progressive loss of motor functions, such as walking. These patients also accumulate TTR in other major organs, which progressively compromises their function. The damage from misfolded TTR protein accumulation leads to disability within five years of diagnosis and is generally fatal within a decade.

Attrv-PN 是由周圍神經中錯誤摺疊的突變 TTR 蛋白積聚引起的。Attrv-PN 患者全身持續遭受使人衰弱的神經損傷,導致行走等運動功能逐漸喪失。這些患者還會在其他主要器官中積累 TTR,這會逐漸損害他們的功能。錯誤摺疊的 TTR 蛋白積累造成的損害會在診斷後的五年內導致殘疾,通常在十年內死亡。

About Ionis Pharmaceuticals, Inc.

關於 Ionis Pharmicals, Inc

For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionispharma.com and follow us on X (Twitter) and LinkedIn.

三十年來,愛奧尼斯發明了爲嚴重疾病患者帶來更美好未來的藥物。愛奧尼斯目前有五種上市藥物,並在神經病學、心臟病學和其他患者需求旺盛的領域擁有領先的產品線。作爲RNA靶向藥物的先驅,Ionis除了推進基因編輯的新方法外,還繼續推動RNA療法的創新。對疾病生物學和行業領先技術的深刻理解推動了我們的工作,以及爲患者提供改變生活的進步的熱情和緊迫性。要了解有關 Ionis 的更多信息,請訪問 IonisPharma.com 並在 X(推特)和 LinkedIn 上關注我們。

Forward-looking Statements

前瞻性陳述

This press release includes forward-looking statements regarding eplontersen, Ionis' business, and the therapeutic and commercial potential of Ionis' commercial medicines, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions, or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended Dec. 31, 2023, and most recent Form 10-Q, which are on file with the SEC. Copies of these and other documents are available at .

本新聞稿包括有關eplontersen、Ionis業務以及Ionis商用藥物、其他正在開發的藥物和技術的治療和商業潛力的前瞻性陳述。任何描述愛奧尼斯目標、預期、財務或其他預測、意圖或信念的陳述均爲前瞻性陳述,應被視爲風險陳述。此類聲明存在某些風險和不確定性,包括但不限於與我們的商業產品和正在研發的藥物相關的風險和不確定性,尤其是發現、開發和商業化可用作人類療法的安全有效藥物的過程以及圍繞此類藥物開展業務所固有的風險和不確定性。愛奧尼斯的前瞻性陳述還涉及假設,如果這些假設從未實現或被證明是正確的,則可能導致其結果與此類前瞻性陳述所表達或暗示的結果存在重大差異。儘管愛奧尼斯的前瞻性陳述反映了其管理層的真誠判斷,但這些陳述僅基於愛奧尼斯目前已知的事實和因素。除非法律要求,否則我們沒有義務出於任何原因更新任何前瞻性陳述。因此,提醒您不要依賴這些前瞻性陳述。愛奧尼斯截至2023年12月31日止年度的10-K表年度報告以及向美國證券交易委員會提交的最新10-Q表進一步詳細描述了與愛奧尼斯計劃有關的這些風險和其他風險。這些文件和其他文件的副本可在以下網址獲得。

Ionis Pharmaceuticals is a registered trademark of Ionis Pharmaceuticals, Inc. WAINUATM is a registered trademark of the AstraZeneca group of companies.

Ionis Pharmaceuticals 是 Ionis 製藥公司的註冊商標。WAINUATM 是阿斯利康集團公司的註冊商標。

Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph.D. - [email protected] - 760-603-2331; Ionis Pharmaceuticals Media Contact: Daria Binder - [email protected] - 760-603-4679

愛奧尼斯製藥投資者聯繫方式: D. Wade Walke,博士-[email protected]-760-603-2331; 愛奧尼斯製藥媒體聯繫人: Daria Binder-[email protected]-760-603-4679

SOURCE Ionis Pharmaceuticals, Inc.

來源 Ionis 製藥公司

譯文內容由第三人軟體翻譯。


以上內容僅用作資訊或教育之目的,不構成與富途相關的任何投資建議。富途竭力但無法保證上述全部內容的真實性、準確性和原創性。
    搶先評論