Novartis Provided an Update on its Phase 1/2 Study in SMA Type 1

15 January 2020

Like Roche’s risdiplam (RG7916), branaplam (LMI070) is a small molecule drug that increases the amount of SMN protein made by the SMN2 gene.

The study is progressing well, with a total of 29 infants receiving the study treatment.

Infants who were enrolled into Part 1 of the study have been receiving branaplam for between approximately 4 and 5 years.

Part 2 of the study has achieved its recruitment target. Participants in Part 2 have been on study treatment for between 7 and 19 months.

Part 3 of the study is assessing the long-term safety, tolerability and effectiveness of extended treatment with branaplam. Infants participating in Parts 1 and 2 who have received branaplam for at least 52 weeks are eligible for this extension study.

Plans for 2020

Novartis anticipates being able to share the 12-month data from Part 2 of the study in the second half of 2020. This data will help define branaplam’s potential for the treatment of SMA and inform future development options.

Further information

Novartis’ community statement 

About branaplam