
UB784
TargetsVMAT2 and Sigma-1 Receptor
IndicationsTardive dyskinesia; Huntington's disease
ClinicalCN - II
TherapeuticsNeurology; Rare Diseases
ModalitiesSmall Molecule
Exclusive Summary
- First-in-class dual-target mechanism (VMAT2 inhibitor & Sigma-1 Receptor agonist) that combines rapid symptom control with long-term pathological modification for Tardive Dyskinesia (TD).
- Best-in-class efficacy potential with preliminary Phase II data showing 44% of patients achieving ≥50% AIMS score reduction (vs 28% for current SOC), plus persistent effect post-discontinuation.
- Overcomes critical SOC limitations: Drug exposure is completely void of CYP2D6 polymorphism impacts, avoiding life-threatening extrapyramidal and psychiatric off-target side effects.
- Immense commercial and out-licensing value: Targets a highly lucrative $3.9 billion SOC market with global IP coverage (PCT filed, patents granted in JP/CN, US Notice of Allowance issued).
Asset Materials

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