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