筋萎縮性側索硬化症市場：KOLの洞察

当レポートでは、世界の筋萎縮性側索硬化症市場について調査し、市場の概要とともに、承認済み治療法、パイプライン動向、将来の見通しなどを提供しています。

目次

エグゼクティブサマリー

現在および将来の治療アルゴリズム

調査目的

承認済み

• グルタミン酸受容体拮抗薬
  • リルテック
• リポペルオキシド15-HPETE阻害剤
  • ラディカヴァ

事前登録

• ヒストンデアセチラーゼおよびホスホトランスフェラーゼ阻害剤
  • AMX0035

後期パイプライン

• アンチセンスオリゴヌクレオチド療法
• 幹細胞治療
• チロシンキナーゼ阻害剤
• トロポニン覚醒剤
• 金ナノ結晶
• ホスホジエステラーゼ阻害剤

中期パイプライン

• 補体C3阻害剤
• マクロファージコロニー刺激因子受容体阻害剤
• 遺伝子治療
• マクロファージ活性化レギュレーター
• Cox-2およびトポイソメラーゼII阻害剤

ALS治療の将来の動向と機会

付録

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