Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
branaplam · 1 trial · 1 indication
A DLT is defined as an adverse event or abnormal laboratory value assessed as unrelated to disease, disease progression, inter-current illness, or concomitant therapies that occurs within the first 14 days of treatment with LMI070 and meets any of the criteria for blood and lymphatic system disorders, gastrointestinal disorders, investigations and other toxicities considered clinically significant.
TEAEs are defined as adverse events starting on or after the first dose of study treatment that were absent pre-treatment, or events present prior to the first dose but increased in severity after the first dose. Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post last treatment.
| Arm | Type | Description |
|---|---|---|
| branaplam | EXPERIMENTAL | branaplam Treatment |
| Name | Type | Description |
|---|---|---|
| branaplam | DRUG | - |
Inclusion Criteria: Common for both Parts 1 and 2: * Type 1 SMA, diagnosed clinically, with symptom onset \<6 months of age and genetic confirmation of mutations in both alleles of the SMN1 gene, and with SMN2 copy number of 2. * Best supportive care in place and stable for at least 14 days before...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Biogen Inc. | BIIB | 13 | PHASE3 | Nusinersen |
| Novartis AG Sponsored ADR | NVS | 5 | PHASE3 | onasemnogene abeparvovec |
| Biohaven Ltd. | BHVN | 1 | PHASE3 | taldefgrobep alfa |
| Scholar Rock Holding Corp. | SRRK | 2 | PHASE3 | Apitegromab |
| argenx SE Sponsored ADR | ARGX | 1 | PHASE2 | ARGX-119 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
Branaplam is an investigational small molecule being developed for the treatment of Spinal Muscular Atrophy (SMA), a genetic neuromuscular disorder. It is currently in clinical development and has not been approved by regulatory authorities.
Branaplam is being developed by Novartis AG, a multinational pharmaceutical company listed on the New York Stock Exchange under the ticker symbol NVS. The drug is also known by the code name LMI070.
Branaplam is in Phase 1 clinical development. A Phase 1 study has been completed, and the drug remains investigational. It is not approved for any use and is still being evaluated for safety and tolerability.
Branaplam has been studied in one completed Phase 1 clinical trial, identified as NCT02268552. This open-label, uncontrolled study enrolled 40 participants with Type 1 Spinal Muscular Atrophy across multiple countries including Belgium, Bulgaria, Denmark, Germany, Italy, Poland, and Russia.
Yes, branaplam is also known as LMI070. The clinical trial NCT02268552, which investigated branaplam in Type 1 Spinal Muscular Atrophy, used the name LMI070 in its official title.