肌萎縮側索硬化症市場：KOL 洞察

本報告調查了全球肌萎縮側索硬化症（ALS）市場，並概述了該市場，以及Sanofi的Rilutek以及Biogen/Ionis的Qalsody對 SOD1-ALS 所發揮的基礎作用。圍繞著Ionis 的ulefnersen 對FUS 突變ALS 的樂觀態度，揭示了塑造ALS 治療未來的策略趨勢。也對不斷發展的 ALS 治療格局進行深入分析，以確定推動 ALS 治療的關鍵機會和挑戰。

目錄

執行摘要

目前與未來的治療演算法

研究目的

核准的藥品

• 麩胺酸受體促效劑
  • Rilutek (riluzole; Sanofi)
• 脂質過氧化物15-HPETE抑制劑
  • Radicava (edaravone; Mitsubishi Tanabe Pharma)
• 反義寡核苷酸療法
  • Qalsody (tofersen; Biogen/Ionis)

在研藥物

• 反義寡核苷酸療法
  • Ulefnersen (ION363; Ionis)
• 酪胺酸激酶抑制劑
  • Masitinib (AB Science)
• Nanocrystalline gold
  • CNM-Au8 (Clene Nanomedicine)
• 幹細胞治療
  • NurOwn (BrainStorm)
• 磷酸二酯酶抑制劑
  • Ibudilast (MediciNova)
• eIF2B 活化劑
  • DNL343 (Denali Therapeutics)
• Sigma 1 受體促效劑
  • Pridopidine (Prilenia Therapeutics)
• 低分子量雙醣
  • Trehalose (SLS-005; Seelos Therapeutics)
• ciprofloxacin和celecoxib的固定劑量複方製劑
  • PrimeC (NeuroSense Therapeutics)
• LINE-1逆轉錄酶抑制劑
  • Censavudine (TPN-101; Transposon Therapeutics)
• PIKfyve 激酶抑制劑
  • AIT-101 (OrphAI Therapeutics)
• C1q 抑制劑
  • ANX005 (Annexon)

未來治療趨勢

• 主要見解摘要

附錄

KOL報表

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