October 22, 2025- The Alpha-1 Foundation (A1F) is proud to share the news that Sanofi’s efdoralprin alfa has met all primary and key secondary endpoints in phase 2 study when dosed every three weeks or every four weeks in adults with alpha-1 antitrypsin deficiency (Alpha-1). Efdoralprin alfa (SAR447537, formerly known as INBRX-101) is an investigational recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein.
Efdoralprin alfa was previously granted fast track and orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of AATD emphysema. Recombinant forms of AAT protein are made by combining genetic material from two different sources. A restorative recombinant therapeutic option requires less frequent dosing, independence from blood and plasma donations resulting in improved efficacy and quality of life for Alpha-1 patients.
A1F’s venture philanthropy program, the Alpha-1 Project (TAP) enabled direct investment into promising treatments providing healthcare stakeholders opportunities to partner with biotech and pharmaceutical companies, like Inhibrx, it’s Alpha-1 asset was one of TAP’s early investments, acquired by and moved forward by Sanofi. This is an example of TAP’s venture philanthropy investment that has had a positive and hopeful return. Like venture philanthropy models created by similar cure-focused non-profits and private foundations, TAP committed to accelerating progress through partnership investment, an essential need in rare conditions as limited incidence rates often require enticement to the field. Accelerating the discovery, development and commercialization of cures, treatments and drug therapies has always been at the core of A1F’s mission and remains a top priority.
“The rapid progress made in emerging therapies in Alpha-1 is the result of Alpha-1 Foundation funding. A1F support laid the groundwork that made it possible to identify new approaches. We are excited for the Alpha-1 community for this news and look forward to Sanofi’s continued success in this clinical trial,” stated Jon Hagstrom, A1F Board of Directors Chair.
The success of any clinical trial is dependent on the involvement of patients. A1F is proud of the dedication of the Alpha-1 Research Registry participants which fostered expediated and positive recruitment in this phase 2 clinical trial. The Alpha-1 Research Registry advances innovative Alpha-1 research, including investigations into improved treatments and a cure for the condition.
“The Alpha-1 Research Registry is a powerful tool to connect patients with clinical trials that they may qualify for. We are so proud of the dedicated Alpha-1 patient community for their efforts in getting this trial to the finish line. It is through the robust and outstanding community of researchers, physicians, A1F staff, and patients working together that meaningful impact on studies can be made,” said Dr. Jeanine D’Armiento, A1F Medical Liaison and Principal Investigator of the Alpha-1 Research Registry.
A1F is committed to working with Sanofi to continue progress on this clinical trial. Patient involvement remains the heart of these efforts. Participation in the Alpha-1 Research Registry supports the discovery of new therapies and progress towards ad cure for Alpha-1. Together, these initiatives are paving the way for breakthroughs in the fight against Alpha-1.
“This is an exciting moment for the Alpha-1 community. John W. Walsh always referred to himself the “Impatient Patient” and the Alpha-1 Foundation is still committed to the urgency that our founder created three decades ago. 30 years marks a significant milestone and an opportunity to reflect on what we have accomplished and the work that remains to be done. We are committed to the Alpha-1 patient on this journey together, “exclaimed Scott Santarella, A1F President and CEO.
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About the Alpha-1 Foundation
The Alpha-1 Foundation, founded in 1995 and located in Coral Gables, FL is a 501(C)(3) charity committed to finding a cure for Alpha-1 Antitrypsin Deficiency (Alpha-1) and to improving the lives of people affected by the condition worldwide. A1F has invested over $100 million to support Alpha-1 research and programs at 130 institutions in North America, the Middle East and Australia.
Contact: Jeanne Kushner
Senior Director of Communications & Policy
877-228-7321
