Amylyx Pharmaceuticals Announces Interim Data From Phase 2 HELIOS Clinical Trial Of AMX0035 In Adults With Wolfram Syndrome
Amylyx Pharmaceuticals Announces Interim Data From Phase 2 HELIOS Clinical Trial Of AMX0035 In Adults With Wolfram Syndrome
- Interim analysis, including eight participants assessed at Week 24, demonstrated improvements in pancreatic function and glycemic control, as measured by C-peptide and other markers of glucose metabolism, rather than worsening typically expected with disease progression
- 包括在第24周評估的八名參與者的中期分析表明，根據C肽和其他葡萄糖代謝標誌物的測量，胰腺功能和血糖控制有所改善，而不是通常隨着疾病進展而惡化
- All eight participants met prespecified responder criteria demonstrating either improvement or stabilization of disease according to both the Patient Reported Global Impression of Change (PGIC) and the Clinician Reported Global Impression of Change (CGIC) scales
- 根據患者報告的全球變化印象（PGIC）和臨床醫生報告的全球變化印象（CGIC）量表，所有八名參與者均符合預先規定的反應者標準，表明疾病有所改善或穩定
- Majority of participants reported some improvement in vision
- 大多數參與者表示視力有所改善
- AMX0035 was generally well-tolerated in all participants
- 所有參與者對 AMX0035 的耐受性普遍良好
- Interim data to be presented during a webcast today at 1:30 p.m. Eastern Time
-中期數據將在東部時間今天下午 1:30 的網絡直播中公佈