肌肉萎缩性脊髓侧索硬化症市场:KOL的洞察

本报告提供全球肌肉萎缩性脊髓侧索硬化症市场相关调查，市场概要，以及已通过核准治疗方法，开发平台趋势，未来预测等资讯。

目录

摘要整理

现在及未来的治疗流程

调查目的

已通过核准

• 麸胺酸受体拮抗剂
  • Rilutek
• Lipoperoxide 15-HPETE抑制剂
  • Radicava

预註册

• 组蛋白去乙酰酶及磷酸转移酶抑制剂
  • AMX0035

后期开发平台

• 反义寡核苷酸疗法
• 干细胞治疗
• 蛋白酪氨酸激酶抑制剂
• 肌钙蛋白兴奋剂
• 黄金奈米结晶
• 磷酸二酯酶抑制剂

中期开发平台

• 补体C3抑制剂
• 巨噬细胞细胞聚落刺激因子受体抑制剂
• 基因治疗
• 巨噬细胞活性化稳压器
• Cox-2及异构转化酶II抑制剂

ALS治疗的未来趋势与机会

附录

KOL报告书

What developments could impact the dominant position of Sanofi's Rilutek in ALS? How will the availability of an oral formulation of Mitsubishi Tanabe's Radicava impact uptake? What clinical advantages underpin experts' favourable opinion of Amylyx' pipeline AMX0035 sodium phenylbutyrate + taurursodiol fixed-dose combination? US and European KOLs critically assess the prospects of launched and pipeline therapies.

In addition to the full report, licensed users have access to the Report Highlights slidedeck and the following KOL Bulletins via the Attachments area.

• 13 Jun 22 KOL Bulletin: Views on FDA's decision to extend the review period for Amylyx's ALS drug AMX0035

Key brands covered in this report:

Key questions answered:

• Do KOLs believe Astellas/Cytokinetics' reldesemtiv has the potential to improve outcomes in ALS?

• Why do experts express a lack of enthusiasm for the potential of stem cell therapy approaches such as Corestem's NeuroNata-R and BrainStorm's NurOwn?

• In which ALS subpopulation do KOLs see Ionis' pipeline FUS-targeted antisense therapy ION363 delivering value?

• Despite the failure of the Phase III VALOR study, experts still view the targeted mechanism of Biogen/Ionis' tofersen in SOD1 mutated ALS favourably - why?

• What factors underpin the experts' positive view of MediciNova's PDE4 inhibitor ibudilast's role in reducing neuroinflammation?

• How do KOLs assess the potential of Clene Nanomedicine's novel nanocrystalline gold agent CNM-Au8 in ALS?

Companies mentioned:

Novartis, Sanofi, AB Science, Bristol Myers Squibb, Biogen, Astellas, Sobi, Cytokinetics, Ionis, Mitsubishi Tanabe, Amylyx, Corestem, BrainStorm, Clene Nanomedicine, MediciNova, Apellis, Helixmith, Neuvivo, NeuroSense.

Table of Contents

Executive summary

Current and future treatment algorithm

Research objectives

Approved

• Glutamate receptor antagonist
  • Rilutek (riluzole; Sanofi)
• Lipoperoxide 15-HPETE inhibitor
  • Radicava (edaravone; Mitsubishi Tanabe)

Preregistered

• Histone deacetylase and phosphotransferase inhibitor
  • AMX0035 (sodium phenylbutyrate/tauroursodeoxycholic acid; Amylyx)

Late-stage pipeline

• Antisense oligonucleotide therapies
  • Tofersen (BIIB067; Biogen/Ionis)
  • ION363 (Ionis)
• Stem cell therapies
  • NeuroNata-R (lenzumestrocel; Corestem); NurOwn (MSC-NTF cells; BrainStorm)
• Tyrosine kinase inhibitors
  • Masitinib (AB Science)
• Troponin stimulant
  • Reldesemtiv (Astellas/Cytokinetics)
• Nanocrystalline gold
  • CNM-Au8 (Clene Nanomedicine)
• Phosphodiesterase inhibitor
  • Ibudilast (MN-166; MediciNova)

Mid-stage pipeline

• Complement C3 inhibitor
  • Aspaveli/Empaveli (pegcetacoplan/APL-2; Apellis/Sobi)
• Macrophage colony stimulating factor receptor inhibitor
  • Sotuletinib (BLZ945; Novartis/Bristol Myers Squibb)
• Gene therapy
  • Engensis (VM202/donaperminogene seltoplasmid; Helixmith)
• Macrophage activation regulator
  • NP001 (Neuvivo)
• Cox-2 and topoisomerase II inhibitor
  • PrimeC (celecoxib/ciprofloxacin; NeuroSense)

Future trends and opportunities in ALS treatment

• Key insights summary
  • KOLs expect an expansion of genetic targets for ALS subgroups and cocktail drug strategies for sporadic ALS patients in the future

Appendix

• KOL details
  • KOLs from North America
  • KOLs from Europe

KOL Bulletins