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 *...
Top 5 of 6 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | Alyftrek, Trikafta, Ivacaftor, VX-522, VX-272 |
| 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 |
It is being developed for cystic fibrosis, a genetic disease that affects the lungs and other organs. Kamada Ltd. studied an inhaled formulation of this plasma-derived Alpha-1 Antitrypsin in cystic fibrosis patients. The program reached Phase 2 clinical development, and the completed study enrolled 21 participants.
Kamada Ltd., which trades under the ticker KMDA, is the developer of aerosolized, human, plasma-derived Alpha-1 Antitrypsin. The company advanced the inhaled cystic fibrosis program into Phase 2 testing. The candidate is a plasma-derived Alpha-1 Antitrypsin delivered as an aerosol to the lungs.
It is in Phase 2 development for cystic fibrosis. The Phase 2 study has been completed, and no trials of this candidate are currently active. Because it has not been approved, aerosolized, human, plasma-derived Alpha-1 Antitrypsin remains an investigational therapy rather than an available treatment for cystic fibrosis.
The Phase 2 trial NCT00499837 evaluated the safety and efficacy of inhaled Alpha-1 Antitrypsin in cystic fibrosis patients. It was a randomized, double-blind, placebo-controlled study that enrolled 21 participants in Israel, including patients aged 5 years and older. The trial has been completed.
NCT00499837 was a randomized, double-blind, placebo-controlled Phase 2 study of inhaled Alpha-1 Antitrypsin in cystic fibrosis. It enrolled 21 participants of any sex, aged 5 years and older, at sites in Israel. Participants were not selected based on a biomarker. The study has been completed.