INDEPENDENT RESEARCH MONITOR
One view of the full tracked field, including negative and inactive programmes.
One view of the full tracked field, including negative and inactive programmes.
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Phase 3 platform
Metformin + R/S alpha-lipoic acid
Active • Stage 1 analysis in progress
Evidence level: Human Phase 3 platform • Stage 1 efficacy decision pending
Next substantive checkpoint: Go/no-go decision for the initial arms around October 2026
Phase 2
Metformin • progressive MS
Active, not recruiting
Evidence level: Human Phase 2 • results pending
Next substantive checkpoint: Completion of follow-up and results after the primary study completion
Phase 2
Metformin • ages 55–75
Recruiting • NCT06463743
Evidence level: Human Phase 2 • results pending
Next substantive checkpoint: Safety plus MRS/MRI, myelin-water-fraction and neuroprotection or repair readouts
Phase 2
CXCR7 / ACKR3 antagonist • progressive MS
Recruiting
Evidence level: Human Phase 2 • results pending
Next substantive checkpoint: MRI, electrophysiological and biochemical remyelination endpoints at 48 weeks
Phase 1/2
Clemastine • chronic demyelination
Recruiting
Evidence level: Human Phase 1/2 • results pending
Next substantive checkpoint: Multiparametric MRI for changes in myelin • estimated completion 2027
Phase 2
Clemastine • acute optic neuritis
Recruiting
Evidence level: Human Phase 2 • results pending
Next substantive checkpoint: VEP and MRI remyelination evidence • estimated completion 2028
Phase 1/2
Clemastine • acute demyelinating lesions
Not yet recruiting • scheduled start 15 Sep 2026
Evidence level: Human Phase 1/2 • no results
Next substantive checkpoint: Recruitment opening and multiparametric MRI readouts for myelin repair
Phase 1/2
Ifenprodil • remyelination
Temporarily halted in CTIS
Evidence level: Human Phase 1/2 • temporarily halted
Next substantive checkpoint: Confirmation of restart or a new official status update
Pilot • device study
Vagus nerve stimulation • RRMS
Recruiting
Evidence level: Human pilot/device study • efficacy exploratory
Next substantive checkpoint: Safety and exploratory remyelination after the 48-week blinded period
Phase 2
Testosterone • RRMS
Ongoing / recruiting
Evidence level: Human Phase 2 • results pending
Next substantive checkpoint: MRI endpoints for remyelination and neuroprotection
Phase 1 • healthy volunteers
GPR17 antagonist
First-in-human programme authorised
Evidence level: Healthy-volunteer development • no MS efficacy data
Next substantive checkpoint: Safety, tolerability and PK before any efficacy study in people with MS
Phase 2 • planned
PAD2 inhibitor • progressive MS
FDA IND cleared • public trial registration not located
Evidence level: Human development milestone • no MS efficacy data
Next substantive checkpoint: Public Phase 2 registration, sites and recruitment opening
Translational / Phase 1 preparation
Transdermal low-dose theophylline • HDAC2 activation
ForTra-funded GMP/patch optimisation • Phase 1 in healthy volunteers planned
Evidence level: No human MS efficacy data • preclinical mechanism + formulation development
Next substantive checkpoint: Optimised patch validation, GMP manufacture and first-in-human safety/PK; public trial registration
Preclinical / clinical-development candidate
Histamine H3 receptor antagonist
Peer-reviewed multi-model validation • no registered MS trial
Evidence level: Human oligodendroglia assays + mouse models • no patient efficacy data
Next substantive checkpoint: Drug-development/formulation work and formal clinical-trial registration
Preclinical / translational
DITPA • MCT8-independent thyroid-hormone analogue
Active Monash programme through 2028 • NeuOrphan describes it as preclinical
Evidence level: Preclinical only • no proven clinical benefit in MS
Next substantive checkpoint: Peer-reviewed confirmation, regulatory package and public registration of an MS human trial
Lead optimisation
Small-molecule OPC differentiation lead
Peer-reviewed preclinical efficacy in human OPCs in vitro and mouse demyelination
Evidence level: Preclinical only • no proven clinical benefit in MS
Next substantive checkpoint: Medicinal-chemistry optimisation, PK/toxicology and selection of a clinical candidate
Development candidate / preclinical
Fixed-dose binary small-molecule combination
Candidate selected from synergistic remyelination-inducing drug combinations
Evidence level: Preclinical only • no proven clinical benefit in MS
Next substantive checkpoint: Pre-IND development and formal clinical-trial registration
Preclinical / licensing
Novel small-molecule mGluR5 agonists
Human oligodendrocyte activity + animal-model remyelination; patent pending
Evidence level: Preclinical only • no proven clinical benefit in MS
Next substantive checkpoint: Lead selection, drug-development optimisation and IND-enabling work
Preclinical cell therapy
Directly induced neural stem-cell transplantation
Peer-reviewed proof-of-concept in chronic demyelination models
Evidence level: Preclinical only • no proven clinical benefit in MS
Next substantive checkpoint: Delivery, dosing and safety work needed before MS clinical translation
Preclinical cell therapy
Engineered human oligodendrocyte progenitor cells
Enhanced migration and remyelination in rodent chronic-lesion models
Evidence level: Preclinical only • no proven clinical benefit in MS
Next substantive checkpoint: Manufacturing, long-term safety and translational studies before human testing
Phase 2a • completed
Metformin + clemastine
Positive biological VEP signal
Evidence level: Human Phase 2a • biological VEP signal; no short-term disability benefit shown
What comes next: Full peer-reviewed results and a larger confirmatory study
Phase 2 • completed
Bazedoxifene acetate
Primary and secondary remyelination endpoints not met
Evidence level: Human Phase 2 • null remyelination efficacy endpoints
Meaning: Generally well tolerated, but without a statistically significant efficacy signal
Phase 3 • terminated
Clemastine • INO in MS
Terminated after a futility no-go analysis
Evidence level: Human Phase 3 • terminated after futility analysis amid slow recruitment
Meaning: The decision followed slow recruitment and futility analysis; it is not by itself definitive proof that clemastine is ineffective
Phase 2 • completed
M1R antagonist
Efficacy endpoints not met
Evidence level: Human Phase 2 • efficacy endpoints not met
Meaning: Further development in MS remains uncertain
Early Phase 1 • completed
Metformin • PPMS/SPMS
Completed • results not yet published
Evidence level: Human early Phase 1 • completed; public results pending
What comes next: Public efficacy and safety readout from NCT05349474