肌萎缩侧索硬化症市场：KOL 洞察

本报告调查了全球肌萎缩侧索硬化症（ALS）市场，并概述了该市场，以及Sanofi的Rilutek以及Biogen/Ionis的Qalsody对 SOD1-ALS 所发挥的基础作用。围绕着Ionis 的ulefnersen 对FUS 突变ALS 的乐观态度，揭示了塑造ALS 治疗未来的策略趋势。也对不断发展的 ALS 治疗格局进行深入分析，以确定推动 ALS 治疗的关键机会和挑战。

目录

执行摘要

目前与未来的治疗演算法

研究目的

核准的药品

• 麸胺酸受体促效剂
  • Rilutek (riluzole; Sanofi)
• 脂质过氧化物15-HPETE抑制剂
  • Radicava (edaravone; Mitsubishi Tanabe Pharma)
• 反义寡核苷酸疗法
  • Qalsody (tofersen; Biogen/Ionis)

在研药物

• 反义寡核苷酸疗法
  • Ulefnersen (ION363; Ionis)
• 酪胺酸激酶抑制剂
  • Masitinib (AB Science)
• Nanocrystalline gold
  • CNM-Au8 (Clene Nanomedicine)
• 干细胞治疗
  • NurOwn (BrainStorm)
• 磷酸二酯酶抑制剂
  • Ibudilast (MediciNova)
• eIF2B 活化剂
  • DNL343 (Denali Therapeutics)
• Sigma 1 受体促效剂
  • Pridopidine (Prilenia Therapeutics)
• 低分子量双醣
  • Trehalose (SLS-005; Seelos Therapeutics)
• ciprofloxacin和celecoxib的固定剂量复方製剂
  • PrimeC (NeuroSense Therapeutics)
• LINE-1逆转录酶抑制剂
  • Censavudine (TPN-101; Transposon Therapeutics)
• PIKfyve 激酶抑制剂
  • AIT-101 (OrphAI Therapeutics)
• C1q 抑制剂
  • ANX005 (Annexon)

未来治疗趋势

• 主要见解摘要

附录

KOL报表

From the foundational role of Sanofi's Rilutek, the promising potential of Biogen/Ionis' Qalsody for SOD1-ALS, to the optimism surrounding Ionis' ulefnersen for FUS-mutated ALS, uncover the strategic trends shaping the future of ALS treatment. This report provides an in-depth analysis of the evolving therapeutic landscape, highlighting key opportunities and challenges in advancing ALS care. Explore the strategic implications for drug development, market dynamics, and patient care strategies.

Table of Contents

Executive summary

Current and future treatment algorithm

Research objectives

Approved drugs

• Glutamate receptor agonist
  • Rilutek (riluzole; Sanofi)
• Lipoperoxide 15-HPETE inhibitor
  • Radicava (edaravone; Mitsubishi Tanabe Pharma)
• Antisense oligonucleotide therapy
  • Qalsody (tofersen; Biogen/Ionis)

Pipeline drugs

• Antisense oligonucleotide therapy
  • Ulefnersen (ION363; Ionis)
• Tyrosine kinase inhibitor
  • Masitinib (AB Science)
• Nanocrystalline gold
  • CNM-Au8 (Clene Nanomedicine)
• Stem cell therapy
  • NurOwn (BrainStorm)
• Phosphodiesterase inhibitor
  • Ibudilast (MediciNova)
• eIF2B activator
  • DNL343 (Denali Therapeutics)
• Sigma-1 receptor agonist
  • Pridopidine (Prilenia Therapeutics)
• Low molecular weight disaccharide
  • Trehalose (SLS-005; Seelos Therapeutics)
• Fixed-dose combination of ciprofloxacin and celecoxib
  • PrimeC (NeuroSense Therapeutics)
• LINE-1 reverse transcriptase inhibitor
  • Censavudine (TPN-101; Transposon Therapeutics)
• PIKfyve kinase inhibitor
  • AIT-101 (OrphAI Therapeutics)
• C1q inhibitor
  • ANX005 (Annexon)

Future treatment trends

• Key insights summary

Appendix

• KOL details
  • KOLs from the USA
  • KOLs from Europe

KOL Bulletins