Branaplam, LMI070 – (1562338-42-4)

Branaplam (development codes LMI070 and NVS-SM1) is a pyridazine derivative being studied as an experimental drug. Originally developed by Novartis to treat spinal muscular atrophy (SMA), it increases functional survival motor neuron (SMN) protein by modifying the splicing pattern of the SMN2 gene.

From 2020, it was investigated for Huntington’s disease but was discontinued in 2023 due to toxicity concerns. Clinical trials for SMA type 1 in children began in 2014 but were halted in 2021.

In 2020, Novartis reported that Branaplam reduced huntingtin protein levels, a key therapeutic target in Huntington’s disease. The FDA granted it orphan drug status for Huntington’s in 2021, but dosing was paused in 2022, and the study was discontinued due to safety concerns. Branaplam is also used in biomedical research as an Xon inducer for gene expression control.

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Sources:

https://en.wikipedia.org/wiki/Branaplam
https://pubchem.ncbi.nlm.nih.gov/compound/135565042
https://www.medchemexpress.com/LMI070.html
https://www.medkoo.com/products/10548
https://pubmed.ncbi.nlm.nih.gov/30407821/