Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Aerosolized, human, plasma-derived Alpha-1 Antitrypsin · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| 80 mg/kg AAT inhaled | EXPERIMENTAL | 80 mg/kg AAT inhaled |
| Placebo inhaled | PLACEBO_COMPARATOR | Placebo inhaled |
| Name | Type | Description |
|---|---|---|
| Aerosolized, human, plasma-derived Alpha-1 Antitrypsin | DRUG | - |
Inclusion Criteria: * Diagnosis of CF by clinical symptoms and positive sweat test or disease inducing mutation. * Age \>5 yrs * Proven ability to perform reproducible PFTs * FEV1 \>25% predicted * Steady disease state for 3 months and no decrease in lung function exceeding 10% during that period *...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | VX-121/TEZ/D-IVA |
| BiomX Inc. | PHGE | 1 | PHASE2 | BX004 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |