Amyotrophic Lateral Sclerosis

Description

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.

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)