Grifols announced that it has completed Cohort 1 of its Phase 1/2 study (NCT04722887) evaluating Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% (Alpha-1 15%), a subcutaneous (SC) Alpha(1) antitrypsin (AAT) treatment being compared to Liquid Alpha1-Proteinase inhibitor (Human) intravenous (IV). Alpha(1) -antitrypsin deficiency (also known as alpha-1) is an underdiagnosed(1) genetic disorder that occurs when a patient has low levels of AAT, a protective protein that safeguards the lungs. Augmentation therapy with IV AAT is the standard medical treatment option for patients with severe AAT deficiency and emphysema.

If proven successful in clinical trials, a SC option could provide alpha-1 patients the ability to independently administer AAT therapy from home, allowing for greater convenience and flexibility. In this multi-center, single-dose and repeat-dose study over eight weeks, Cohort 1 has been completed and demonstrated no safety issues with Alpha-1 15% that would prevent the study from moving forward into Cohort 2.