Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Alpha-1 HC · 1 trial · 1 indication
adverse event frequency
| Arm | Type | Description |
|---|---|---|
| Alpha-1 HC 100 mg | EXPERIMENTAL | 100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks. |
| Alpha-1 HC 200 mg | EXPERIMENTAL | 200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks. |
| Placebo | PLACEBO_COMPARATOR | Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate). |
| Name | Type | Description |
|---|---|---|
| Alpha-1 HC 100 mg | BIOLOGICAL | Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma. Alpha-1 HC 100 mg inhaled once daily for 21 days for a total of 21 inhaled treatments. |
| Placebo | BIOLOGICAL | Phosphate Buffer Saline with Polysorbate (placebo) composed of the same elements listed for Alpha-1 HC, minus the 50 mg/mL of Alpha-1 HC. Placebo inhaled once daily for 21 days for a total of 21 inhaled treatments. |
| Alpha-1 HC 200 mg | BIOLOGICAL | Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma. Alpha-1 HC 200 mg inhaled once daily for 21 days for a total of 21 inhaled treatments. |
Inclusion Criteria: * Age 18 years or older. * Documentation of CF diagnosis. * Have a pre-bronchodilator FEV1 ≥ 40% of predicted at Visit 1 and have a Visit 2 pre-investigational product FEV1 that is ≥ 40% of predicted and within ± 15% of the Visit 1 result. * Deemed by the Investigator to be a su...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | VX-121/TEZ/D-IVA |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719 |
| 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 |
Alpha-1 HC is an investigational inhaled therapy being studied for the treatment of cystic fibrosis. It is a monoclonal antibody developed by Grifols, S.A. that is currently in Phase 2 clinical development. The drug is administered via inhalation to target respiratory complications associated with the disease.
Alpha-1 HC is a monoclonal antibody designed to target alpha-1 proteinase inhibitor, also known as alpha-1 antitrypsin. By delivering this inhibitor directly to the lungs through inhalation, the therapy aims to address the underlying protease-antiprotease imbalance that contributes to lung damage in cystic fibrosis patients.
Alpha-1 HC is being developed by Grifols, S.A., a global healthcare company traded on the NASDAQ under the ticker symbol GRFS. The company is conducting clinical research on this inhaled therapy for cystic fibrosis, with the drug currently in Phase 2 development.
Alpha-1 HC is in Phase 2 clinical development for cystic fibrosis. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still undergoing clinical trials to evaluate its safety and efficacy. The most recent trial for this therapy has been completed.
Alpha-1 HC has been studied in one completed Phase 2 clinical trial, identified as NCT01684410. This randomized, double-blind, placebo-controlled study evaluated the safety and tolerability of inhaled Alpha-1 HC in 30 adult patients with cystic fibrosis in the United States. The trial enrolled participants aged 18 years and older.
Alpha-1 HC is a specific formulation of alpha-1 proteinase inhibitor, also known as alpha-1 antitrypsin. It is produced using a hydrophobic chromatography process and is being developed as an inhaled therapy for cystic fibrosis. This distinguishes it from other forms of alpha-1 antitrypsin replacement therapies.