BRANAPLAM
Branaplam is a small molecule pharmaceutical. It is currently being investigated in clinical studies.
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Clinical
Clinical Trials
3 clinical trials
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Indications Phases 4
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Indications Phases 3
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Indications Phases 2
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Muscular atrophy | D009133 | — | — | 1 | 1 | — | — | — | 1 |
| Spinal muscular atrophy | D009134 | EFO_0003823 | G12.1 | 1 | 1 | — | — | — | 1 |
| Atrophy | D001284 | — | — | 1 | 1 | — | — | — | 1 |
| Huntington disease | D006816 | Orphanet_399 | G10 | — | 1 | — | — | — | 1 |
Indications Phases 1
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Healthy volunteers/patients | — | — | — | 1 | — | — | — | — | 1 |
Indications Without Phase
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Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| Drug common name | BRANAPLAM |
| INN | branaplam |
| Description | Branaplam (development codes LMI070 and NVS-SM1) is a pyridazine derivative that is being studied as an experimental drug. It was originally developed by Novartis to treat spinal muscular atrophy (SMA); since 2020 it was being developed to treat Huntington's disease (HD) but the trial ended in 2023 with harmful side effects.
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| Classification | Small molecule |
| Drug class | survival motor neurin SMN1 and SMN2 splicing modulators |
| Image (chem structure or protein) |  |
| Structure (InChI/SMILES or Protein Sequence) | CC1(C)CC(Oc2ccc(-c3ccc(-c4cn[nH]c4)cc3O)nn2)CC(C)(C)N1 |
Identifiers
| PDB | — |
| CAS-ID | — |
| RxCUI | — |
| ChEMBL ID | CHEMBL4290141 |
| ChEBI ID | — |
| PubChem CID | 135565042 |
| DrugBank | — |
| UNII ID | P12R69543A (ChemIDplus, GSRS) |
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Safety
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