Branaplam (LMI070, NVS-101) is an experimental compound being developed by Novartis Pharma as a treatment for SMA. It is a small-molecule drug which modifies alternative splicing of the SMN2 gene, bringing about increased levels of SMN protein. Branaplam is taken orally, usually in a liquid form once a week.
Since late 2015, branaplam has been undergoing a clinical trial in SMA type 1 children. The trial was opened in Belgium, Denmark, Germany, and Italy and recruited 13 babies aged up to 7 months. In May 2016, severe adverse effects were observed in animals being fed the drug and Novartis decided to reduce the dose 10-fold. Following this, most children rapidly deteriorated and two passed away. The target dose was restored in July 2016 upon demands of clinicians conducting the study. However, it then closed for recruitment of new patients for over a year.
The trial re-opened for recruitment in September 2017. As of September 2018, trial sites in the following countries accept babies in the trial: Belgium, Czechia, Denmark, Italy, Poland and Russia. More sites may be added in the future. The company communicated that it intends to test branaplam also in other SMA types.