Novartis’ small molecule Branaplam, also known as LMI070, is an orally available drug which aims to correct the splicing of SMN2, the “back-up” gene, thereby increasing the amount of SMN protein made.
Following a pause brought on by safety concerns, Novartis has now resumed the clinical development of LMI070 (branaplam) to treat spinal muscular atrophy (SMA) through its ongoing Phase 1/2 clinical trial.
Sites as at October 2017:
This trial is an open-label study (all patients will receive branaplam) where different doses are being tested in patients with type 1 SMA who are younger than 6 months of age. You can find more information about the ongoing clinical trial available on the Novartis clinical trials website. Enrollment will start as soon as the research team obtains approvals by health authorities and the ethics committee.