Tofersen SOD1-ALS Ph3/OLE + AP-101 Ph2 + PrimeC + Pridopidine Ph3 + Silence FUS ASO + GoALS [developing]
Key Questions
What is the status of Biogen's tofersen for ALS?
Tofersen for SOD1-ALS is in Phase 3 and open-label extension, monitoring neurofilament light chain (NfL) levels as a biomarker.
What is pridopidine's Phase 3 trial?
Pridopidine is in the Phase 3 PREVAiLS trial, aiming to slow ALS progression as part of emerging therapies.
What is AP-101 and its trial status?
AP-101 is in Phase 2 for ALS, alongside combination therapy PrimeC and the GoALS study evaluating multiple approaches.
How many ALS cases are there in the US?
Approximately 35,000 people in the US are living with ALS, a neurodegenerative disease with no cure, highlighting needs for experimental treatments.
What is the update on RADICAVA?
Shionogi completed acquisition of all rights to oral RADICAVA (edaravone), an approved ALS therapy to slow disease progression.
Pridopidine Ph3 PREVAiLS; Tofersen NfL; AP-101 Ph2; PrimeC; Silence ALS custom FUS ASO presymp EMG reversal symptom-free 3yrs URGenT $15M; GoALS; RADICAVA oral; ~35k US cases, patient stories highlight experimental needs.