Amyotrophic Lateral Sclerosis - KOL Insight

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.