Grifols , a global healthcare company and leading manufacturer of plasma-derived medicines,  announced it has finished recruiting the second cohort of its Phase 1/2 study (NCT04722887) evaluating the safety and tolerability of two different doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% (Alpha-1 15%) as a subcutaneous (SC) option for the treatment of alpha₁-antitrypsin (AAT) deficiency, compared to Liquid Alpha1-Proteinase Inhibitor (Human) intravenous (IV).

Cohort 2 patients will receive a 180 mg/kg dose of SC Alpha-1 15%, after treatment of cohort 1 patients with 72 mg/kg of SC Alpha-1 15% showed a good safety profile. The last patient, last visit for cohort 2 of this multi-center, single dose followed by repeat-dose study over eight weeks is expected in late summer 2025.

AAT deficiency is an underdiagnosed 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. A SC option could provide AAT patients the ability to independently administer their treatments from home, enabling greater convenience and flexibility.

“We are excited about the possibility of providing alpha-1 patients with a subcutaneous alternative, giving them more freedom when managing their AAT condition,” said Dr. Jörg Schüttrumpf, Grifols Chief Scientific Innovation Officer. “Our commitment to the Alpha-1 community remains unwavering and we continue applying our expertise in plasma science and plasma proteins to innovate new treatment options for patients.”

Grifols’ longstanding commitment to the Alpha-1 community also includes the diagnostic test AlphaID™, designed to detect the most prevalent variants associated with alpha₁-antitrypsin deficiency, also known as genetic chronic obstructive pulmonary disorder, a serious respiratory ailment. In 2023, Grifols launched its AlphaID™ At Home service, enabling U.S. adults to screen for the genetic risk of AAT deficiency without prescription from a healthcare professional. Since then, approximately 70,000 tests have been ordered.

“The Alpha-1 Foundation applauds Grifols for their continued commitment to the Alpha-1 patient community,” said Scott Santarella, President & CEO, Alpha-1 Foundation. “Their dedication to providing patients with innovative new treatment options could provide them with flexibility and convenience in managing their weekly vital treatments for AATD. Our thanks to Grifols for this outstanding investment. It is crucial for our community to have continued access to this lifesaving plasma therapy.”