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...
Top 6 of 7 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | Alyftrek, Trikafta, Ivacaftor, VX-581, 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 |
| Roivant Sciences Ltd. | ROIV | 1 | PHASE1 | SP-101 |
Alpha-1 HC is an inhaled formulation of alpha1-proteinase inhibitor (human) that is being studied as a treatment for cystic fibrosis. It is a monoclonal antibody developed by Grifols, S.A., which trades under the ticker GRFS. The drug is also known as Alpha-1 HC 100 mg.
Alpha-1 HC is being evaluated for use in cystic fibrosis, a respiratory condition. It was tested in a Phase 2 trial of 30 adults with cystic fibrosis in the United States. The study assessed safety and tolerability of the inhaled formulation rather than confirming efficacy.
Alpha-1 HC is developed by Grifols, S.A., the Spanish plasma-derived medicines company whose shares trade under the ticker GRFS. Grifols produced the inhaled alpha1-proteinase inhibitor (human) using a hydrophobic chromatography process, which is what the HC in the drug name refers to.
Alpha-1 HC is in Phase 2 development. It is investigational and has not been approved for any indication. The single Phase 2 trial of Alpha-1 HC has been completed, and no active trials of the drug are currently listed.
Alpha-1 HC was studied in one completed Phase 2 trial, NCT01684410, titled Safety and Tolerability Trial of Inhaled Alpha1-Proteinase Inhibitor (Human), Hydrophobic Chromatography Process (Alpha-1 HC) in Subjects With Cystic Fibrosis. The trial enrolled 30 adults, was randomized, double blind, and placebo controlled, and ran in the United States.