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.