INDEPENDENT RESEARCH MONITOR
Human and translational programmes explicitly connected to myelin repair. Biomarkers remain biomarkers, not proof of clinical benefit.
Human and translational programmes explicitly connected to myelin repair. Biomarkers remain biomarkers, not proof of clinical benefit.
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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