Statements on Takeda Clinical Trial (TAK-883-3001)

From the Desk of Miriam O’Day, President & CEO of the Alpha-1 Foundation:

“It is with extreme disappointment that the Alpha-1 Foundation received the news that the Takeda Clinical Trial will not proceed forward at this time. The Foundation has every confidence that Takeda will continue their commitment to the Alpha-1 community. The regulatory authorities did not exhibit flexibility when approaching this clinical trial design with the sponsor. The Alpha-1 Foundation still has much work to do to advance the opinions of the Alpha-1 experts in the field into the agencies drug review process. Last fall when we came to agreement during the public workshop the Foundation was confident that we were ensuring that our population with so many unmet needs would benefit from flexible design and the use of new endpoints. We have the patient at the center of everything that we do. Advancing the timely development of new and next generation therapies continues to be a priority. Alphas still have many unmet needs and we will continue to advocate on their behalf. We are confident that Takeda also is dedicated to the advancement of care and research in Alpha-1 antitrypsin deficiency,” stated Miriam O’Day, President & CEO of the Alpha-1 Foundation.

Takeda’s Statement Regarding the TAK-883-3001 Clinical Trial

Takeda has made the difficult decision to withdraw the TAK-883-3001 study of its Alpha-1 Proteinase Inhibitor (A1PI) 120 mg/kg and 60 mg/kg evaluating changes in lung density in participants with A1PI deficiency and Chronic Obstructive Pulmonary Disease-Emphysema. While we have worked collaboratively with the Alpha-1 research and patient communities to develop the design for this trial through the lens of clinical care experiences and patient needs, ultimately we could not reach agreement on a protocol that would satisfy regulatory requirements while also addressing the intent of the study.

While we are disappointed that the trial will not proceed, Takeda remains unwavering in our commitment to the Alpha-1 community. We look forward to collaborating with community leaders to identify and pursue new opportunities to collect clinically meaningful data on our Alpha-1 portfolio that may inform and advance care for this population.