肌肉萎縮性脊髓側索硬化症市場:KOL的洞察

本報告提供全球肌肉萎縮性脊髓側索硬化症市場相關調查，市場概要，以及已通過核准治療方法，開發平台趨勢，未來預測等資訊。

目錄

摘要整理

現在及未來的治療流程

調查目的

已通過核准

• 麩胺酸受體拮抗劑
  • Rilutek
• Lipoperoxide 15-HPETE抑制劑
  • Radicava

預註冊

• 組蛋白去乙醯酶及磷酸轉移酶抑制劑
  • AMX0035

後期開發平台

• 反義寡核苷酸療法
• 幹細胞治療
• 蛋白酪氨酸激酶抑制劑
• 肌鈣蛋白興奮劑
• 黃金奈米結晶
• 磷酸二酯酶抑制劑

中期開發平台

• 補體C3抑制劑
• 巨噬細胞細胞聚落刺激因子受體抑制劑
• 基因治療
• 巨噬細胞活性化穩壓器
• Cox-2及異構轉化酶II抑制劑

ALS治療的未來趨勢與機會

附錄

KOL報告書

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