Q4 2021 Results
Participants
Company overview
Pharmaceuticals
Oncology
Financial review
Conclusion
Appendix
Financial performance
Innovation: Pipeline overview
Innovation: Clinical trials
CRM
IHD
Neuroscience
Ophthalmology
Respiratory & Allergy
Oncology: Solid Tumors
Hematology
Biosimilars
Global Health
Abbreviations
LMI070 - mRNA splicing modulator
Study
Indication
Phase
Patients
Primary Outcome
Measures
Arms Intervention
NCT05111249 VIBRANT-HD (CLMI070C12203)
Huntington's disease
Phase 2
75
1. Reduction (%) of mHTT protein in cerebrospinal fluid (CSF)
2. Number of treatment emergent adverse events and serious adverse events
Arm 1: Experimental; Branaplam 56 mg oral solution once weekly
Arm 2: Experimental; Branaplam 112 mg oral solution once weekly
Arm 3: Experimental; (C) Branaplam 154 mg oral solution once weekly, OR (X)
Branaplam 84 mg oral solution once weekly OR (Y) Branaplam 28 mg oral solution
once weekly
Arm 4: Placebo; Matching placebo oral solution once weekly
Participants with early manifest Huntington's Disease
Target Patients
Read-out Milestone(s)
Publication
2025
TBD
97 Investor Relations | Q4 2021 Results
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