아이오니스: 질가너센 1-3상 톱라인, 알렉산더병에서 긍정적 결과

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.

핵심 사실

제출: 2025년 9월 22일 AM 11:05
이벤트: 탑라인 결과
방향: 긍정적
기업: IONS아이오니스
자산: zilganersen
출처: SEC 8-K
신뢰도: 출처 확인
캘린더: FDA 심사 결정 (PDUFA) · 2026년 9월 22일 · 긍정적
연령 범위: 1.5-53세
연구 주차: 61주
참가자: 54명
10MWT p 값: 0.0412
10MWT 평균 차이: 33.3%

출처 발췌

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

SEC 8-K