Ulefnersen
✓ Phase 3 complete
Ionis · Otsuka
RNA therapy for FUS-ALS
Positive phase 3 in 73 patients: better function and survival than placebo. FDA filing under discussion.
Result: Sep 2026
Neurodegenerative · motor neurons
ALS
Amyotrophic lateral sclerosis: progressive loss of movement. The biggest advances are RNA therapies for genetic forms, which have changed the course for some patients.
- Cure
- None yet
- Current treatment
- Riluzole and edaravone; tofersen for SOD1-ALS
- Who has it
- About 2 new cases per 100,000 people a year
- Updated
6candidates on the board
Phase 3 completefurthest phase
2updates in 2026
What changed
The latest developments, newest first.
ProgressUlefnersen works in FUS-ALS
First controlled study against the genetic cause of FUS-ALS, which strikes young people and moves fast.
ProgressPridopidine phase 3 begins
500 participants with early, fast-progressing disease.
Treatments in development
Each column is a step on the road to the pharmacy. Colors show the type of treatment.
- Oral drug (4)
- Cell, gene or RNA therapy (2)
Phase 3confirmation · 6
CNM-Au8
Clene
Gold nanocrystals
Phase 2 showed fewer deaths. Company seeks accelerated approval on a biomarker.
Filing in preparation
Pridopidina
Prilenia · Ferrer
S1R activator
Slowed decline 32% in a fast-progressing subgroup. 500-person phase 3 began Mar 2026.
Result: 2028
Tofersen (ATLAS)
Biogen
RNA therapy for SOD1
Already approved. Now testing whether it prevents disease in gene carriers before symptoms.
Ends: 2027
Masitinibe
AB Science
Tyrosine kinase inhibitor
Phase 3 cleared, waiting for funding to start.
Not started
PrimeC
NeuroSense
Ciprofloxacin + celecoxib
Combines two existing drugs. Filing in Canada; US phase 3 awaits funding.
Under review (Canada)
Left behind
Candidates that looked promising and were stopped. Most treatments tested never reach the market.
Relyvrio
2024 · Withdrawn from market after failing phase 3.
Frequently asked questions
Is there a cure for ALS?
None yet. Current treatment: Riluzole and edaravone; tofersen for SOD1-ALS.
Which ALS treatments are being tested?
Fasevia tracks 6 candidates: Ulefnersen (Phase 3 complete); CNM-Au8 (Phase 3); Pridopidina (Phase 3); Tofersen (ATLAS) (Phase 3); Masitinibe (Phase 3); PrimeC (Phase 3).
What is the most advanced ALS treatment in development?
The furthest along today is Ulefnersen (Ionis · Otsuka), in phase 3 complete. Positive phase 3 in 73 patients: better function and survival than placebo. FDA filing under discussion.
When could a new ALS treatment arrive?
A drug has to complete phase 3 and be reviewed by health agencies such as the FDA or EMA, which usually takes several years. The leading candidate today is in phase 3 complete.