प्राथमिक स्रोत रिकॉर्ड
निर्णायकटॉपलाइन परिणाम
Ionis: Alexander disease में zilganersen के फेज 1-3 अध्ययन का टॉपलाइन परिणाम सकारात्मक
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.
मुख्य तथ्य
- फाइल किया गया
- 22 सित॰ 2025, 11:05 am
- घटना
- टॉपलाइन परिणाम
- दिशा
- सकारात्मक
- कंपनी
IONSIonis- संपत्ति
- zilganersen
- स्रोत
- SEC 8-K
- विश्वसनीयता
- स्रोत सहित
- कैलेंडर
- FDA फ़ैसला (PDUFA) · 22 सित॰ 2026 · सकारात्मक
- आयु सीमा
- 1.5-53 वर्ष
- अध्ययन सप्ताह
- 61
- प्रतिभागी
- 54
- 10MWT p मान
- 0.0412
- 10MWT माध्य अंतर
- 33.3%
स्रोत का अंश
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