Neuroscience biotech companies

The 12 neuroscience companies we cover, with cash and runway from their latest SEC filings, trial milestones from ClinicalTrials.gov, and FDA decisions from their own 8-Ks. Companies are tagged by the areas their pipelines target.

Companies

CompanyMost advanced programCash & investmentsRunwayProfile
VRTXVertex PharmaceuticalsTrikafta · cystic fibrosisApproved$13.64B2026-06-30—maintained
IONSIonis PharmaceuticalsTryngolza · familial chylomicronemia syndromeApproved$2.70B2026-06-30~71 moarchived
PRAXPraxis Precision MedicinesPRAX-562 · Developmental and epileptic encephalopathies (DEEs), including SCN2A and SCN8A-related epilepsyPhase 2$1.37B2026-06-30~50 momaintained
NBIXNeurocrine BiosciencesValbenazine · Chorea Associated with Huntington DiseaseApproved$482M2026-06-30—archived
ABBVAbbVieSkyrizi · psoriasis, inflammatory bowel disease, and related immunology usesApprovedno filing parsed—archived
ACADACADIA PharmaceuticalsNuplazid · Parkinson disease psychosisApprovedno filing parsed—archived
ARGXargenx SEVyvgart · Generalized Myasthenia GravisApprovedno filing parsed—archived
BIIBBiogenLeqembi · Alzheimer diseaseApprovedno filing parsed—archived
DNLIDenali TherapeuticsDNL310 · Hunter syndromePhase 2no filing parsed—archived
MNMDMindMedMM120 (lysergide D-tartrate ODT) · Generalized anxiety disorder (GAD)Phase 3no filing parsed—ticker changed
PRTAProthenaBirtamimab · AL amyloidosisPhase 3 · stoppedno filing parsed—archived
SAVACassava SciencesSimufilam · Alzheimer diseasePhase 3 · stoppedno filing parsed—archived

Runway is cash divided by quarterly operating burn and is not shown for companies funded by their own revenue. Figures and their dates are on each company page, linked to the filing.

Upcoming trial milestones

  • 2026-12 · IONS · Donidalorsen OLE: Donidalorsen in Hereditary angioedema prophylaxis — primary completion ClinicalTrials.gov
  • 2027-01-25 · VRTX · Suzetrigine DPN open-label extension: Suzetrigine in Diabetic peripheral neuropathic pain — primary completion ClinicalTrials.gov
  • 2027-03 · DNLI · BIIB122 LRRK2-PD: BIIB122 in LRRK2-associated Parkinson disease — primary completion ClinicalTrials.gov
  • 2027-04-06 · VRTX · ELX/TEZ/IVA non-F508del: Elexacaftor/tezacaftor/ivacaftor in Cystic fibrosis, non-F508del genotypes — primary completion ClinicalTrials.gov
  • 2027-05-31 · VRTX · Suzetrigine DPN Phase 3: Suzetrigine in Pain associated with diabetic peripheral neuropathy — primary completion ClinicalTrials.gov
  • 2027-08 · ACAD · ACP-211 MDD: ACP-211 in Major depressive disorder with inadequate response — primary completion ClinicalTrials.gov
  • 2027-12 · DNLI · COMPASS: Tividenofusp alfa in Mucopolysaccharidosis type II (Hunter syndrome) — primary completion ClinicalTrials.gov
  • 2028-01 · ACAD · ADVANCE-2 master protocol: ACP-204 in Alzheimer's disease psychosis — primary completion ClinicalTrials.gov
  • 2028-03 · ABBV · Venetoclax ramp-up CLL: Venetoclax in Previously untreated chronic lymphocytic leukaemia — primary completion ClinicalTrials.gov
  • 2028-06-23 · BIIB · Salanersen SMA 15-60y: Salanersen (BIIB115) in Spinal muscular atrophy, ages 15-60 — primary completion ClinicalTrials.gov
  • 2028-08 · DNLI · DNL126: DNL126 in Mucopolysaccharidosis type IIIA (Sanfilippo A) — primary completion ClinicalTrials.gov
  • 2028-11-28 · BIIB · Salanersen presymptomatic infants: Salanersen (BIIB115) in Presymptomatic spinal muscular atrophy — primary completion ClinicalTrials.gov
  • 2029-06 · IONS · Donidalorsen paediatric: Donidalorsen in Hereditary angioedema, ages 2 to <12 — primary completion ClinicalTrials.gov
  • 2029-07-27 · BIIB · Salanersen post-onasemnogene: Salanersen (BIIB115) in SMA after onasemnogene abeparvovec — primary completion ClinicalTrials.gov
  • 2030-08 · IONS · Olezarsen paediatric FCS: Olezarsen in Familial chylomicronemia syndrome — primary completion ClinicalTrials.gov

A registry milestone is the date data collection is scheduled to end, not the date results are released.

FDA decisions

None of these companies has an FDA decision on our PDUFA calendar, which lists only dates a company has stated in a filing.

Educational information, not investment advice. See where each field comes from.