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 | 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 |
Taldefgrobep Alfa is an investigational drug being studied for the treatment of Spinal Muscular Atrophy (SMA) and Obesity. It is currently in clinical development for these conditions, with trials evaluating its efficacy and safety in patients with SMA and in adults with overweight and obesity.
Taldefgrobep Alfa is being developed by Biohaven Ltd., a biopharmaceutical company traded under the ticker symbol BHVN. The company is conducting clinical trials to evaluate the drug's safety and efficacy for Spinal Muscular Atrophy and Obesity.
Taldefgrobep Alfa is in Phase 2 and Phase 3 clinical trials. A Phase 3 study is evaluating the drug in participants with Spinal Muscular Atrophy, while a Phase 2 study is assessing it in adults with overweight and obesity. The drug is investigational and not yet approved.
Taldefgrobep Alfa is being studied in two clinical trials. NCT05337553 is a Phase 3, placebo-controlled study in 269 participants with Spinal Muscular Atrophy across multiple countries. NCT07281495 is a Phase 2, placebo-controlled study in 150 adults with overweight and obesity in the United States.
Taldefgrobep Alfa is not FDA approved. It is an investigational drug currently in clinical development. It has received Orphan Drug and Fast Track designations from the FDA, which are intended to expedite the development and review of drugs for serious conditions.