Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
taldefgrobep alfa · 2 trials · 5 indications
Change in MFM-32 total score from baseline to Week 48. Scores range from 0-3 on each item. The scores from the 32 items are summed and transformed to a 0-100 scale, with higher scores reflecting higher levels of functional abilities.
| Arm | Type | Description |
|---|---|---|
| taldefgrobep alfa | EXPERIMENTAL | taldefgrobep alfa - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase. taldefgrobep alfa/taldefgrobep alfa - Extension Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for Open label Extension (OLE) phase. |
| Placebo | PLACEBO_COMPARATOR | Placebo - Double-blind (DB) Phase: Participants receive weight based 35 mg/50 mg weekly subcutaneous injection for 48-week DB phase. Placebo/taldefgrobep alfa - Extension Phase: Participants who receive placebo during DB phase, receive weight based 35 mg/50 mg weekly subcutaneous taldefgrobep alfa injection for OLE phase. |
| Taldefgrobep Alpha once weekly | EXPERIMENTAL | - |
| Placebo once weekly | PLACEBO_COMPARATOR | - |
| Taldefgrobep Alpha once every 4 weeks | EXPERIMENTAL | - |
| Placebo once every 4 weeks | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| taldefgrobep alfa | DRUG | DB Phase: 35 mg/50 mg weekly subcutaneous injection |
| Placebo | DRUG | DB Phase: matching placebo 35 mg/50 mg weekly subcutaneous injection |
Key Inclusion Criteria: * Spinal Muscular Atrophy confirmed by genetic diagnosis of 5q-autosomal recessive SMA as well as SMN2 copy number * Ambulant or Non-Ambulant * Treated with an SMA disease-modifying therapy and anticipated to remain on that same treatment regimen and dose throughout the tria...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Biogen Inc. | BIIB | 14 | 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 |
| Jin Medical International Ltd. Class A | ZJYL | 1 | - | Undisclosed |