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Amyotrophic Lateral Sclerosis - KOL Insight

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¼¼°èÀÇ ±ÙÀ§Ã༺Ãø»ö°æÈ­Áõ(ALS) ½ÃÀå¿¡ ´ëÇØ Á¶»çÇßÀ¸¸ç, ½ÃÀåÀÇ °³¿ä¿Í Sanofi RilutekÀÌ ´ã´çÇÏ´Â ±âÃÊÀû ¿ªÇÒºÎÅÍ SOD1-ALS¿¡ ´ëÇÑ Biogen/IonisÀÇ QalsodyÀÇ À¯¸ÁÇÑ °¡´É¼º, FUS º¯ÀÌÇü ALS¿¡ ´ëÇÑ IonisÀÇ ulefnersen¸¦ µÑ·¯½Ñ ³«°ü·Ð±îÁö ALS Ä¡·áÀÇ ¹Ì·¡¸¦ Çü¼ºÇÏ´Â Àü·«Àû µ¿ÇâÀ» »ìÆ캾´Ï´Ù. ¶ÇÇÑ ÁøÈ­ÇÏ´Â ALS Ä¡·á Àü¸ÁÀ» »ó¼¼È÷ ºÐ¼®ÇÏ°í, ALS Ä¡·áÀÇ Áøº¸¸¦ À§ÇÑ ÁÖ¿ä ±âȸ¿Í °úÁ¦¸¦ °­Á¶ÇÕ´Ï´Ù.

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  • ±Û·çŸ¹Î»ê ¼ö¿ëü ÀÛ¿ëÁ¦
    • Rilutek(riluzole; Sanofi)
  • ÁöÁú °ú»êÈ­¹° 15-HPETE ÀúÇØÁ¦
    • Radicava(edaravone; Mitsubishi Tanabe Pharma)
  • ¾ÈƼ¼¾½º ¿Ã¸®°í´ºÅ¬·¹¿ÀƼµå ¿ä¹ý
    • Qalsody(tofersen; Biogen/Ionis)

ÆÄÀÌÇÁ¶óÀÎ ¾àÁ¦

  • ¾ÈƼ¼¾½º ¿Ã¸®°í´ºÅ¬·¹¿ÀƼµå ¿ä¹ý
    • Ulefnersen(ION363; Ionis)
  • Ƽ·Î½ÅÅ°³ª¾ÆÁ¦ ÀúÇØÁ¦
    • Masitinib(AB Science)
  • ³ª³ë°áÁ¤ ±Ý
    • CNM-Au8(Clene Nanomedicine)
  • Áٱ⼼Æ÷ Ä¡·á
    • NurOwn(BrainStorm)
  • Æ÷½ºÆ÷µð¿¡½ºÅ׶óÁ¦ ÀúÇØÁ¦
    • Ibudilast(MediciNova)
  • eIF2B È°¼ºÈ­ÀÎÀÚ
    • DNL343(Denali Therapeutics)
  • ½Ã±×¸¶ 1 ¼ö¿ëü ÀÛ¿ëÁ¦
    • Pridopidine(Prilenia Therapeutics)
  • ÀúºÐÀÚ·® ÀÌ´ç·ù
    • Trehalose(SLS-005; Seelos Therapeutics)
  • ciprofloxacin°ú celecoxibÀÇ °íÁ¤¿ë·® ¹èÇÕÁ¦
    • PrimeC(NeuroSense Therapeutics)
  • LINE-1 ¿ªÀü»ç È¿¼Ò ÀúÇØÁ¦
    • Censavudine(TPN-101; Transposon Therapeutics)
  • PIKfyve Å°³ª¾ÆÁ¦ ÀúÇØÁ¦
    • AIT-101(OrphAI Therapeutics)
  • C1q ÀúÇØÁ¦
    • ANX005(Annexon)

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KOL º¸°í¼­

KSA 24.08.08

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

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