一次情報の記録
決定級トップライン結果
アイオニス:ザイルガナーセンの第1-3相試験、アレクサンダー病でトップライン良好
Zilganersen 50 mg met the primary endpoint of gait speed stabilization (10MWT) with a 33.3% mean difference vs control (p=0.0412) at week 61. The company plans NDA submission in Q1 2026, marking the first disease-modifying therapy to show benefit in this rare neurological condition.
主要事実
- 提出
- 2025年9月22日 11:05
- イベント
- トップライン結果
- 方向
- ポジティブ
- アセット
- zilganersen
- 出典
- SEC 8-K
- 信頼度
- 出典あり
- カレンダー
- FDA 審査決定 (PDUFA) · 2026年9月22日 · ポジティブ
- 年齢範囲
- 1.5-53 歳
- 試験週
- 61
- 参加者
- 54 名
- 10MWT p 値
- 0.0412
- 10MWT 平均差
- 33.3%
出典の抜粋
EX-99.1 2 ef20055880_ex99-1.htm EXHIBIT 99.1 Exhibit 99.1 Ionis announces positive topline results from pivotal study of zilganersen in Alexander disease – First and only investigational medicine to demonstrate a clinically meaningful and disease-modifying impact on this rare, often fatal neurological condition – – NDA submission planned in Q1 2026 – CARLSBAD, Calif., Sept. 22, 2025 – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments. Zilgane rsen 50 mg demonstrated statistically significant and clinically meaningful stabilization on the primary endpoi