Drugs / Branaplam
Branaplam
also known as
BRANAPLAM HYDROCHLORIDE · LMI-070 HYDROCHLORIDE · LMI070 · NVP-LMI070-AAA · NVP-LMI070-NX · Nvs-sm1
Small molecule targets HTT via inhibition
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT05330286 | Apr → Aug 2022 | — | Novartis Pharmaceuticals | Terminated | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT02268552 | Apr 2015 → Dec 2022 | spinal muscular atrophy | Novartis Pharmaceuticals | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT05111249 | Dec 2021 → Oct 2023 | Huntington disease | Novartis Pharmaceuticals | Terminated | No outcome recorded |
News releases announcing trial results or a regulatory action · 2
| Date | Issuer | Release |
|---|---|---|
| 2020-10-21 | Novartis Pharmaceuticals | Regulatory Novartis receives US Food and Drug Administration (FDA) Orphan Drug Designation for branaplam (LMI070) in Huntington’s disease (HD) novartis.com ↗
Novartis today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for branaplam (LMI070) in Huntington’s disease (HD). |
| 2019-04-30 | Novartis Pharmaceuticals | Results New data at AAN reinforce Novartis commitment to transforming the lives of people of all ages who live with neurological conditions novartis.com ↗
New data supporting the potential use of serum neurofilament light chain as biomarker for SMA Type 1 disease activity and therapy response under branaplam (LMI070), an oral, once-weekly RNA splicing modulator. |
All press releases naming this drug 2 releases
| Date | Issuer | Release |
|---|---|---|
| 2020-10-21 | Novartis Pharmaceuticals | Novartis receives US Food and Drug Administration (FDA) Orphan Drug Designation for branaplam (LMI070) in Huntington’s disease (HD) novartis.com ↗ |
| 2019-04-30 | Novartis Pharmaceuticals | New data at AAN reinforce Novartis commitment to transforming the lives of people of all ages who live with neurological conditions novartis.com ↗ |
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 5 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Branaplam | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05111249 ↗ |
| Known as | BRANAPLAM HYDROCHLORIDE | ChEMBL registry synonym — accepted as the source's own label CHEMBL4290141 ↗ |
| Known as | LMI-070 HYDROCHLORIDE | ChEMBL registry synonym — accepted as the source's own label CHEMBL4290141 ↗ |
| Known as | LMI070 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05330286 ↗ |
| Known as | NVP-LMI070-AAA | ChEMBL registry synonym — accepted as the source's own label CHEMBL4290141 ↗ |
| Known as | NVP-LMI070-NX | ChEMBL registry synonym — accepted as the source's own label CHEMBL4290141 ↗ |
| Known as | Nvs-sm1 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4290141 ↗ |
| Action | Inhibit | “branaplam lowers mHTT protein levels” PMID 35241644 ↗ Mar 2022 |
| Modality | Small molecule | “the orally available, brain penetrant small molecule branaplam” PMID 35241644 ↗ Mar 2022 |
| Route | Oral | “dosed orally for SMA” PMID 35241644 ↗ Mar 2022 |
| Target | HTT | “By promoting inclusion of a pseudoexon in the primary transcript, branaplam lowers mHTT protein levels in HD patient cells, in an HD mouse model and in blood samples from...” PMID 35241644 ↗ Mar 2022 |