Grifols Completes Cohort 1 in Clinical Study of Alpha-1 15%, Evaluating First-in-Human Subcutaneous Dosing Option for Patients with Alpha1-Antitrypsin Deficiency

• Study designed to demonstrate impact of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% (Alpha-1 15%), an alpha₁ antitrypsin treatment, compared with Liquid Alpha1-Proteinase Inhibitor (Human) intravenous
  
  
• This first in-human subcutaneous approach to treating alpha₁-antitrypsin deficiency, if proven successful in clinical trials, could give patients the convenience and flexibility to administer their medication from home
  

BARCELONA, Spain, Nov. 15, 2023 (GLOBE NEWSWIRE) -- Grifols (MCE: GRF, MCE: GRF.P NASDAQ: GRFS), one of the world’s leading producers of plasma-derived medicines, today 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) alpha₁ antitrypsin (AAT) treatment being compared to Liquid Alpha1-Proteinase Inhibitor (Human) intravenous (IV).

Alpha₁-antitrypsin deficiency (also known as alpha-1) is an underdiagnosed¹ 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.

“A subcutaneous option would be a first for alpha-1 patients, providing more freedom when it comes to managing their AAT deficiency by allowing patients to administer their medication from the comfort of their own home. We are pleased to announce this milestone during Alpha-1 Awareness Month, contributing to raising further visibility about this rare disease,” said Jörg Schüttrumpf, Chief Scientific Innovation Officer, Grifols. “We look forward to moving this study into Cohort 2. Grifols has a strong commitment to the alpha-1 community and continues to innovate to find additional treatment options for patients living with this disease, in addition to more convenient alpha-1 testing alternatives such as AlphaID At Home, our recently launched direct-to-consumer genetic health risk service.”