INDEPENDENT RESEARCH MONITOR

Preclinical and translational research

Selected work with a defined mechanism or credible development path. No clinical benefit is implied.

Preclinical and translational research

Selected work with a defined mechanism or credible development path. No clinical benefit is implied.

Late preclinicalAnnounced / plannedNo efficacy data yet

Translational / Phase 1 preparation

Low-dose theophylline patch

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

PreclinicalPreclinical programmePositive signal

Preclinical / clinical-development candidate

Bavisant / BRAVEinMS

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

PreclinicalPreclinical programmePositive signal

Preclinical / translational

Petratos / DITPA programme

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

PreclinicalPreclinical programmePositive signal

Lead optimisation

CN045

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

Late preclinicalPreclinical programmeNo efficacy data yet

Development candidate / preclinical

Scripps / Lairson combination

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

PreclinicalPreclinical programmeNo efficacy data yet

Preclinical / licensing

Rutgers mGluR5 agonists

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

PreclinicalPreclinical programmePositive signal

Preclinical cell therapy

Cambridge directly induced NSCs

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

PreclinicalPreclinical programmePositive signal

Preclinical cell therapy

CRISPR-edited human OPCs

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