Table 1.
Table reporting the different smallmolecules currentlyin FDA clinical trials for ALS treatment (updated 13 April 2022).
| Molecule | Target/Mechanism | Developer | Clinical Phase |
|---|---|---|---|
| Ibudilast | Macrophage migration inhibitory factor inhibitor | MediciNova | Phase II/III |
| Prosetin | Mitogen-activated protein kinase inhibitor | ProJenX | Phase I |
| Sotuletinib | Macrophage colony-stimulating factor receptor antagonist | Novartis | Phase II |
| EPI 589 | NAD(P)H dehydrogenase modulator | PTC Therapeutics | Phase II |
| DNL 343 | Eukaryoticinitiationfactor2b stimulant | Denali Therapeutics Inc | Phase I |
| Celecoxib/ciprofloxacin | Cyclo-oxygenase 2 inhibitors/DNA gyrase inhibitors | NeuroSense Therapeutics | Phase I |
| Fingolimod | Apoptosis stimulant and immunosuppressant | ALS Therapy Development Institute | Phase II |
| Trehalose | Autophagy stimulant and protein aggregation inhibitor | Massachusetts General Hospital | Phase II/III |
| Sodium cromoglicate | Glial cell modulator and mast cell stabilizer | AZTherapies | Phase II |
| Dexpramipexole | Antioxidant and apoptosis inhibitor | Knopp Biosciences | Phase II |
| Masitinib | Tyrosine kinase inhibitor | AB Science | Phase III |
| NP 001 | Macrophage modulator | Neuvivo | Phase II |
| Fasudil | Rho-associated kinase inhibitor and vasodilatator | Woolsey Pharmaceuticals | Phase II |
| Levosimendan | Calcium-sensitising phosphodiesterase inhibitor and potassium channel agonist | Orion | Phase III |
| Apilimoddimesylate | Interleukin 12 inhibitor and interleukin 23 inhibitor | AI Therapeutics | Phase II |
| Verdiperstat | Peroxidase inhibitor | Biohaven Pharmaceuticals | Phase II/III |
| Pridopidine | Sigma-1 receptor agonist | Massachusetts General Hospital, Prilenia Therapeutics | Phase II/III |
| Triheptanoin | Triglyceride replacement agent | Ultragenyx Pharmaceutical | Phase I/II |
| Reldesemtiv | Troponin stimulant | Cytokinetics | Phase III |
| BIIB 100 | Exportin-1 protein inhibitor | Biogen | Phase I |
| AGX 201 | Histamine receptor modulator | AgoneX Biopharmaceuticals | Phase I |
| Ranolazine extended release | Sodium channel antagonist | Gilead Sciences | Phase II |
| GDC 0134 | Mitogen-activated protein kinase 12 inhibitor | Genentech | Phase I |
| NPT520 34 | Phosphatidylinositol 3 kinase modulator | Neuropore Therapies | Phase I |